{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,16]],"date-time":"2026-10-16T16:21:44Z","timestamp":1792167704525,"version":"4.4.0"},"reference-count":285,"publisher":"MDPI AG","issue":"11","license":[{"start":{"date-parts":[[2025,10,30]],"date-time":"2025-10-30T00:00:00Z","timestamp":1761782400000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"DOI":"10.13039\/501100001809","name":"National Natural Science Foundation of China","doi-asserted-by":"crossref","award":["82571246"],"award-info":[{"award-number":["82571246"]}],"id":[{"id":"10.13039\/501100001809","id-type":"DOI","asserted-by":"crossref"}]},{"name":"Shanghai \u201cRising Stars of Medical Talents\u201d Youth Development Program","award":["SHWSRS(2025)_071"],"award-info":[{"award-number":["SHWSRS(2025)_071"]}]}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Pharmaceutics"],"abstract":"<jats:p>Inherited retinal diseases (IRDs) are a clinically and genetically heterogeneous spectrum of disorders that lead to progressive and irreversible vision loss. Gene therapy is the most promising emerging treatment for IRDs. While gene augmentation strategies have demonstrated clinical benefit and results within the first approved ocular gene therapy, their application is restricted by adeno-associated virus (AAV) packaging capacity and limited efficacy for dominant mutations. Recent breakthroughs in precision genome editing, particularly base editing (BE) and prime editing (PE), have provided alternatives capable of directly correcting pathogenic variants. BE enables targeted single-nucleotide conversions, whereas PE further allows for precise insertions and deletions, both circumventing the double-strand DNA cleavage or repair processes typically induced by conventional CRISPR\u2013Cas editing systems, thereby offering advantages in post-mitotic retinal cells. Preclinical investigations across murine and non-human primate models have demonstrated the feasibility, molecular accuracy, and preliminary safety profiles of these platforms in targeting IRD-associated mutations. However, critical challenges remain before clinical application can be realized, including limited editing efficiency in photoreceptors, interspecies variability in therapeutic response, potential risks of off-target effects, and barriers in large-scale vector manufacturing. Moreover, the delivery of genome editors to the outer retina remains suboptimal, prompting intensive efforts in capsid engineering and the development of non-viral delivery systems. This review synthesizes the current progress in BE and PE optimization, highlights innovations in delivery platforms that encompass viral and emerging non-viral systems and summarizes the major barriers to clinical translation. We further discuss AI-driven strategies for the rational design of BE\/PE systems, thereby outlining their future potential and perspectives in the treatment of IRDs.<\/jats:p>","DOI":"10.3390\/pharmaceutics17111405","type":"journal-article","created":{"date-parts":[[2025,10,31]],"date-time":"2025-10-31T01:23:59Z","timestamp":1761873839000},"page":"1405","update-policy":"https:\/\/doi.org\/10.3390\/mdpi_crossmark_policy","source":"Crossref","is-referenced-by-count":8,"title":["Base and Prime Editing for Inherited Retinal Diseases: Delivery Platforms, Safety, Efficacy, and Translational Perspectives"],"prefix":"10.3390","volume":"17","author":[{"ORCID":"https:\/\/orcid.org\/0009-0006-8154-1192","authenticated-orcid":false,"given":"Haoliang","family":"Zhang","sequence":"first","affiliation":[{"name":"Department of Ophthalmology, Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai 200080, China"},{"name":"National Clinical Research Center for Ophthalmic Diseases, Shanghai 200080, China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yuxuan","family":"Li","sequence":"additional","affiliation":[{"name":"School of Medicine, Shanghai Jiao Tong University, Shanghai 200025, China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0009-0009-4750-6117","authenticated-orcid":false,"given":"Jiajie","family":"Li","sequence":"additional","affiliation":[{"name":"School of Medicine, Shanghai Jiao Tong University, Shanghai 200025, China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Xiaosa","family":"Li","sequence":"additional","affiliation":[{"name":"Department of Ophthalmology, Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai 200080, China"},{"name":"National Clinical Research Center for Ophthalmic Diseases, Shanghai 200080, China"},{"name":"Shanghai Engineering Center for Visual Science and Photomedicine, Shanghai 200080, China"},{"name":"Shanghai Key Laboratory of Fundus Diseases, Shanghai 200080, China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-2544-1254","authenticated-orcid":false,"given":"Tong","family":"Li","sequence":"additional","affiliation":[{"name":"Department of Ophthalmology, Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai 200080, China"},{"name":"National Clinical Research Center for Ophthalmic Diseases, Shanghai 200080, China"},{"name":"Shanghai Engineering Center for Visual Science and Photomedicine, Shanghai 200080, China"},{"name":"Shanghai Gene Therapy Clinical Research Center, Shanghai 200080, China"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"1968","published-online":{"date-parts":[[2025,10,30]]},"reference":[{"key":"ref_1","unstructured":"(2025, October 14). RetNet: Diseases Table. Available online: https:\/\/retnet.org\/disease."},{"key":"ref_2","doi-asserted-by":"crossref","unstructured":"Ben-Yosef, T. (2022). Inherited Retinal Diseases. Int. J. Mol. Sci., 23.","DOI":"10.3390\/ijms232113467"},{"key":"ref_3","doi-asserted-by":"crossref","unstructured":"Geiger, A.B., Kennedy, J.G., Staker, L.G., Wensel, T.G., Casson, R.J., and Thomas, P.Q. (2025). Shining light on CRISPR\/Cas9 therapeutics for inherited retinal diseases. Prog. Retin. Eye Res., 107.","DOI":"10.1016\/j.preteyeres.2025.101376"},{"key":"ref_4","doi-asserted-by":"crossref","unstructured":"Chen, X., Xu, N., Li, J., Zhao, M., and Huang, L. (2023). Stem cell therapy for inherited retinal diseases: A systematic review and meta-analysis. Stem Cell Res. Ther., 14.","DOI":"10.1186\/s13287-023-03526-x"},{"key":"ref_5","doi-asserted-by":"crossref","first-page":"199","DOI":"10.1097\/ICU.0000000000000558","article-title":"Retinal neuroprotection: Current strategies and future directions","volume":"30","author":"Wubben","year":"2019","journal-title":"Curr. Opin. Ophthalmol."},{"key":"ref_6","doi-asserted-by":"crossref","unstructured":"Zhou, R., and Caspi, R.R. (2010). Ocular immune privilege. F1000 Biol. Rep., 2.","DOI":"10.3410\/B2-3"},{"key":"ref_7","unstructured":"The IRD Gene Therapy Pipeline (2025, October 14). Retina Today. Available online: https:\/\/retinatoday.com\/articles\/2024-july-aug\/the-ird-gene-therapy-pipeline."},{"key":"ref_8","unstructured":"(2025, October 14). A Patient Registry Study for Patients Treated with Voretigene Neparvovec in US. Available online: https:\/\/www.medthority.com\/clinical-trials\/luxturna\/a-patient-registry-study-for-patients-treated-with-voretigene-neparvovec-in-us\/."},{"key":"ref_9","unstructured":"(2025, October 14). Real World Data from the Treatment by Voretigene Neparvovec (Luxturna) of LCA2-RPE65 Patients Across 24 Countries\u2014Euretina Brief. 10 October 2024. Available online: https:\/\/brief.euretina.org\/clinical\/real-world-data-from-the-treatment-by-voretigene-neparvovec-luxturna-of-lca2-rpe65-patients-across-24-countries."},{"key":"ref_10","unstructured":"Fischer, M.D., Maier, R., Suhner, A., Stiehl, D., Fasser, C., and Leroy, B.P. (2022). PERCEIVE study report: Real-world safety and effectiveness of voretigene neparvovec. Investig. Ophthalmol. Vis. Sci., 63."},{"key":"ref_11","doi-asserted-by":"crossref","first-page":"1255","DOI":"10.1016\/S0142-9612(02)00507-0","article-title":"Mesenchymal stem cells, MG63 and HEK293 transfection using chitosan-DNA nanoparticles","volume":"24","author":"Corsi","year":"2003","journal-title":"Biomaterials"},{"key":"ref_12","doi-asserted-by":"crossref","first-page":"142","DOI":"10.1016\/j.ajo.2019.11.012","article-title":"CEP290 Mutation Spectrum and Delineation of the Associated Phenotype in a Large German Cohort: A Monocentric Study","volume":"211","author":"Feldhaus","year":"2020","journal-title":"Am. J. Ophthalmol."},{"key":"ref_13","doi-asserted-by":"crossref","unstructured":"Ewert, K.K., Scodeller, P., Sim\u00f3n-Gracia, L., Steffes, V.M., Wonder, E.A., Teesalu, T., and Safinya, C.R. (2021). Cationic Liposomes as Vectors for Nucleic Acid and Hydrophobic Drug Therapeutics. Pharmaceutics, 13.","DOI":"10.3390\/pharmaceutics13091365"},{"key":"ref_14","doi-asserted-by":"crossref","unstructured":"Ghosh, R., and Tabrizi, S.J. (2017). Gene suppression approaches to neurodegeneration. Alzheimers Res. Ther., 9.","DOI":"10.1186\/s13195-017-0307-1"},{"key":"ref_15","doi-asserted-by":"crossref","unstructured":"Germain, N.D., Chung, W.K., and Sarmiere, P.D. (2023). RNA interference (RNAi)-based therapeutics for treatment of rare neurologic diseases. Mol. Asp. Med., 91.","DOI":"10.1016\/j.mam.2022.101148"},{"key":"ref_16","doi-asserted-by":"crossref","first-page":"4623","DOI":"10.1113\/JP282076","article-title":"Optogenetic approaches to therapy for inherited retinal degenerations","volume":"600","author":"Moore","year":"2022","journal-title":"J. Physiol."},{"key":"ref_17","doi-asserted-by":"crossref","first-page":"2281","DOI":"10.1038\/nprot.2013.143","article-title":"Genome engineering using the CRISPR-Cas9 system","volume":"8","author":"Ran","year":"2013","journal-title":"Nat. Protoc."},{"key":"ref_18","doi-asserted-by":"crossref","first-page":"229","DOI":"10.1038\/s41591-018-0327-9","article-title":"Development of a gene-editing approach to restore vision loss in Leber congenital amaurosis type 10","volume":"25","author":"Maeder","year":"2019","journal-title":"Nat. Med."},{"key":"ref_19","doi-asserted-by":"crossref","first-page":"1972","DOI":"10.1056\/NEJMoa2309915","article-title":"Gene Editing for CEP290-Associated Retinal Degeneration","volume":"390","author":"Pierce","year":"2024","journal-title":"N. Engl. J. Med."},{"key":"ref_20","doi-asserted-by":"crossref","first-page":"497","DOI":"10.1016\/j.molcel.2012.07.029","article-title":"Playing the end game: DNA double-strand break repair pathway choice","volume":"47","author":"Chapman","year":"2012","journal-title":"Mol. Cell"},{"key":"ref_21","doi-asserted-by":"crossref","first-page":"121","DOI":"10.1038\/nm.3793","article-title":"Therapeutic genome editing: Prospects and challenges","volume":"21","author":"Cox","year":"2015","journal-title":"Nat. Med."},{"key":"ref_22","doi-asserted-by":"crossref","first-page":"765","DOI":"10.1038\/nbt.4192","article-title":"Repair of double-strand breaks induced by CRISPR-Cas9 leads to large deletions and complex rearrangements","volume":"36","author":"Kosicki","year":"2018","journal-title":"Nat. Biotechnol."},{"key":"ref_23","doi-asserted-by":"crossref","first-page":"523","DOI":"10.1016\/j.omtn.2020.06.019","article-title":"Large-Fragment Deletions Induced by Cas9 Cleavage while Not in the BEs System","volume":"21","author":"Song","year":"2020","journal-title":"Mol. Ther. Nucleic Acids"},{"key":"ref_24","doi-asserted-by":"crossref","first-page":"895","DOI":"10.1038\/s41588-021-00838-7","article-title":"Chromothripsis as an on-target consequence of CRISPR-Cas9 genome editing","volume":"53","author":"Leibowitz","year":"2021","journal-title":"Nat. Genet."},{"key":"ref_25","doi-asserted-by":"crossref","unstructured":"Alanis-Lobato, G., Zohren, J., McCarthy, A., Fogarty, N.M.E., Kubikova, N., Hardman, E., Greco, M., Wells, D., Turner, J.M.A., and Niakan, K.K. (2021). Frequent loss of heterozygosity in CRISPR-Cas9-edited early human embryos. Proc. Natl. Acad. Sci. USA, 118.","DOI":"10.1073\/pnas.2004832117"},{"key":"ref_26","doi-asserted-by":"crossref","first-page":"420","DOI":"10.1038\/nature17946","article-title":"Programmable editing of a target base in genomic DNA without double-stranded DNA cleavage","volume":"533","author":"Komor","year":"2016","journal-title":"Nature"},{"key":"ref_27","doi-asserted-by":"crossref","unstructured":"da Costa, B.L., Levi, S.R., Eulau, E., Tsai, Y.-T., and Quinn, P.M.J. (2021). Prime Editing for Inherited Retinal Diseases. Front. Genome Ed., 3.","DOI":"10.3389\/fgeed.2021.775330"},{"key":"ref_28","doi-asserted-by":"crossref","first-page":"149","DOI":"10.1038\/s41586-019-1711-4","article-title":"Search-and-replace genome editing without double-strand breaks or donor DNA","volume":"576","author":"Anzalone","year":"2019","journal-title":"Nature"},{"key":"ref_29","doi-asserted-by":"crossref","first-page":"3278","DOI":"10.1016\/j.scib.2023.11.015","article-title":"Prime editing: Current advances and therapeutic opportunities in human diseases","volume":"68","author":"Fu","year":"2023","journal-title":"Sci. Bull."},{"key":"ref_30","unstructured":"Abbaszadeh, A., and Shahlai, A. (2025). Artificial Intelligence for CRISPR Guide RNA Design: Explainable Models and Off-Target Safety. arXiv."},{"key":"ref_31","doi-asserted-by":"crossref","first-page":"464","DOI":"10.1038\/nature24644","article-title":"Programmable base editing of A\u2022T to G\u2022C in genomic DNA without DNA cleavage","volume":"551","author":"Gaudelli","year":"2017","journal-title":"Nature"},{"key":"ref_32","doi-asserted-by":"crossref","first-page":"824","DOI":"10.1038\/s41587-020-0561-9","article-title":"Genome editing with CRISPR\u2013Cas nucleases, base editors, transposases and prime editors","volume":"38","author":"Anzalone","year":"2020","journal-title":"Nat. Biotechnol."},{"key":"ref_33","doi-asserted-by":"crossref","first-page":"161","DOI":"10.1038\/s41576-022-00541-1","article-title":"Prime editing for precise and highly versatile genome manipulation","volume":"24","author":"Chen","year":"2023","journal-title":"Nat. Rev. Genet."},{"key":"ref_34","doi-asserted-by":"crossref","unstructured":"Nishida, K., Arazoe, T., Yachie, N., Banno, S., Kakimoto, M., Tabata, M., Mochizuki, M., Miyabe, A., Araki, M., and Hara, K.Y. (2016). Targeted nucleotide editing using hybrid prokaryotic and vertebrate adaptive immune systems. Science, 353.","DOI":"10.1126\/science.aaf8729"},{"key":"ref_35","doi-asserted-by":"crossref","first-page":"35","DOI":"10.1038\/s41587-020-0592-2","article-title":"Glycosylase base editors enable C-to-A and C-to-G base changes","volume":"39","author":"Zhao","year":"2021","journal-title":"Nat. Biotechnol."},{"key":"ref_36","doi-asserted-by":"crossref","first-page":"41","DOI":"10.1038\/s41587-020-0609-x","article-title":"CRISPR C-to-G base editors for inducing targeted DNA transversions in human cells","volume":"39","author":"Kurt","year":"2021","journal-title":"Nat. Biotechnol."},{"key":"ref_37","doi-asserted-by":"crossref","first-page":"875","DOI":"10.1038\/s41587-019-0393-7","article-title":"Targeted, random mutagenesis of plant genes with dual cytosine and adenine base editors","volume":"38","author":"Li","year":"2020","journal-title":"Nat. Biotechnol."},{"key":"ref_38","doi-asserted-by":"crossref","unstructured":"Xie, J., Huang, X., Wang, X., Gou, S., Liang, Y., Chen, F., Li, N., Ouyang, Z., Zhang, Q., and Ge, W. (2020). ACBE, a new base editor for simultaneous C-to-T and A-to-G substitutions in mammalian systems. BMC Biol., 18.","DOI":"10.1186\/s12915-020-00866-5"},{"key":"ref_39","doi-asserted-by":"crossref","first-page":"5384","DOI":"10.1093\/nar\/gkac353","article-title":"AGBE: A dual deaminase-mediated base editor by fusing CGBE with ABE for creating a saturated mutant population with multiple editing patterns","volume":"50","author":"Liang","year":"2022","journal-title":"Nucleic Acids Res."},{"key":"ref_40","doi-asserted-by":"crossref","first-page":"1019","DOI":"10.1126\/science.aaq0180","article-title":"RNA editing with CRISPR-Cas13","volume":"358","author":"Cox","year":"2017","journal-title":"Science"},{"key":"ref_41","doi-asserted-by":"crossref","first-page":"631","DOI":"10.1038\/s41586-020-2477-4","article-title":"A bacterial cytidine deaminase toxin enables CRISPR-free mitochondrial base editing","volume":"583","author":"Mok","year":"2020","journal-title":"Nature"},{"key":"ref_42","doi-asserted-by":"crossref","first-page":"1764","DOI":"10.1016\/j.cell.2022.03.039","article-title":"Targeted A-to-G base editing in human mitochondrial DNA with programmable deaminases","volume":"185","author":"Cho","year":"2022","journal-title":"Cell"},{"key":"ref_43","doi-asserted-by":"crossref","first-page":"3107","DOI":"10.1016\/j.ymthe.2021.09.002","article-title":"In vivo somatic cell base editing and prime editing","volume":"29","author":"Newby","year":"2021","journal-title":"Mol. Ther."},{"key":"ref_44","doi-asserted-by":"crossref","first-page":"620","DOI":"10.1038\/s41587-020-0414-6","article-title":"Evaluation and minimization of Cas9-independent off-target DNA editing by cytosine base editors","volume":"38","author":"Doman","year":"2020","journal-title":"Nat. Biotechnol."},{"key":"ref_45","doi-asserted-by":"crossref","first-page":"770","DOI":"10.1038\/s41576-018-0059-1","article-title":"Base editing: Precision chemistry on the genome and transcriptome of living cells","volume":"19","author":"Rees","year":"2018","journal-title":"Nat. Rev. Genet."},{"key":"ref_46","doi-asserted-by":"crossref","first-page":"1041","DOI":"10.1038\/s41587-019-0236-6","article-title":"CRISPR DNA base editors with reduced RNA off-target and self-editing activities","volume":"37","author":"Zhou","year":"2019","journal-title":"Nat. Biotechnol."},{"key":"ref_47","doi-asserted-by":"crossref","first-page":"843","DOI":"10.1038\/nbt.4172","article-title":"Improving cytidine and adenine base editors by expression optimization and ancestral reconstruction","volume":"36","author":"Koblan","year":"2018","journal-title":"Nat. Biotechnol."},{"key":"ref_48","doi-asserted-by":"crossref","first-page":"408","DOI":"10.1016\/j.cj.2020.01.003","article-title":"Developing high-efficiency base editors by combining optimized synergistic core components with new types of nuclear localization signal peptide","volume":"8","author":"Wang","year":"2020","journal-title":"Crop J."},{"key":"ref_49","doi-asserted-by":"crossref","unstructured":"Hu, J., Guo, M., Gao, Q., Jia, H., He, M., Wang, Z., Guo, L., Liu, G., Gao, Q., and Zhao, K.T. (2025). QBEmax is a sequence-permuted and internally protected base editor. Nat. Biotechnol., 1\u20137.","DOI":"10.1038\/s41587-025-02641-9"},{"key":"ref_50","doi-asserted-by":"crossref","first-page":"2256","DOI":"10.1016\/j.cell.2023.03.034","article-title":"Prediction of efficiencies for diverse prime editing systems in multiple cell types","volume":"186","author":"Yu","year":"2023","journal-title":"Cell"},{"key":"ref_51","doi-asserted-by":"crossref","unstructured":"Kantor, A., McClements, M.E., and MacLaren, R.E. (2020). CRISPR-Cas9 DNA Base-Editing and Prime-Editing. Int. J. Mol. Sci., 21.","DOI":"10.3390\/ijms21176240"},{"key":"ref_52","doi-asserted-by":"crossref","first-page":"5635","DOI":"10.1016\/j.cell.2021.09.018","article-title":"Enhanced prime editing systems by manipulating cellular determinants of editing outcomes","volume":"184","author":"Chen","year":"2021","journal-title":"Cell"},{"key":"ref_53","doi-asserted-by":"crossref","first-page":"731","DOI":"10.1038\/s41587-021-01133-w","article-title":"Programmable deletion, replacement, integration and inversion of large DNA sequences with twin prime editing","volume":"40","author":"Anzalone","year":"2022","journal-title":"Nat. Biotechnol."},{"key":"ref_54","doi-asserted-by":"crossref","first-page":"10785","DOI":"10.1093\/nar\/gkab792","article-title":"Optimized nickase- and nuclease-based prime editing in human and mouse cells","volume":"49","author":"Adikusuma","year":"2021","journal-title":"Nucleic Acids Res."},{"key":"ref_55","doi-asserted-by":"crossref","first-page":"3983","DOI":"10.1016\/j.cell.2023.07.039","article-title":"Phage-assisted evolution and protein engineering yield compact, efficient prime editors","volume":"186","author":"Doman","year":"2023","journal-title":"Cell"},{"key":"ref_56","doi-asserted-by":"crossref","unstructured":"Chauhan, V.P., Sharp, P.A., and Langer, R. (2025). Engineered prime editors with minimal genomic errors. Nature, 1\u201326.","DOI":"10.1101\/2024.08.02.606370"},{"key":"ref_57","doi-asserted-by":"crossref","unstructured":"Liang, R., Wang, S., Cai, Y., Li, Z., Li, K.M., Wei, J., Sun, C., Zhu, H., Chen, K., and Gao, C. (2025). Circular RNA-mediated inverse prime editing in human cells. Nat. Commun., 16.","DOI":"10.1038\/s41467-025-59120-7"},{"key":"ref_58","doi-asserted-by":"crossref","first-page":"22","DOI":"10.1038\/s41551-024-01227-1","article-title":"Efficient site-specific integration of large genes in mammalian cells via continuously evolved recombinases and prime editing","volume":"9","author":"Pandey","year":"2025","journal-title":"Nat. Biomed. Eng."},{"key":"ref_59","doi-asserted-by":"crossref","unstructured":"Hew, B.E., Gupta, S., Sato, R., Waller, D.F., Stoytchev, I., Short, J.E., Sharek, L., Tran, C.T., Badran, A.H., and Owens, J.B. (2024). Directed evolution of hyperactive integrases for site specific insertion of transgenes. Nucleic Acids Res., 52.","DOI":"10.1101\/2024.06.10.598370"},{"key":"ref_60","doi-asserted-by":"crossref","unstructured":"Liu, J., Zhang, J., Zhao, T., Zhao, M., Su, M., Chen, Y., Huang, Z., Wang, Y., Zhong, C., and Hu, Z. (2025). SunTag-PE: A modular prime editing system enables versatile and efficient genome editing. Commun. Biol., 8.","DOI":"10.1038\/s42003-025-07893-4"},{"key":"ref_61","doi-asserted-by":"crossref","unstructured":"Kaukonen, M., McClements, M.E., and MacLaren, R.E. (2022). CRISPR DNA Base Editing Strategies for Treating Retinitis Pigmentosa Caused by Mutations in Rhodopsin. Genes, 13.","DOI":"10.3390\/genes13081327"},{"key":"ref_62","doi-asserted-by":"crossref","first-page":"331","DOI":"10.1016\/j.ymthe.2016.12.006","article-title":"CRISPR\/Cas9-Mediated Genome Editing as a Therapeutic Approach for Leber Congenital Amaurosis 10","volume":"25","author":"Ruan","year":"2017","journal-title":"Mol. Ther."},{"key":"ref_63","doi-asserted-by":"crossref","first-page":"403","DOI":"10.1126\/science.aad5143","article-title":"In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy","volume":"351","author":"Nelson","year":"2016","journal-title":"Science"},{"key":"ref_64","doi-asserted-by":"crossref","first-page":"596","DOI":"10.1016\/j.omtn.2023.02.011","article-title":"In vivo base editing rescues photoreceptors in a mouse model of retinitis pigmentosa","volume":"31","author":"Su","year":"2023","journal-title":"Mol. Ther. Nucleic Acids"},{"key":"ref_65","doi-asserted-by":"crossref","unstructured":"Wu, Y., Wan, X., Zhao, D., Chen, X., Wang, Y., Tang, X., Li, J., Li, S., Sun, X., and Bi, C. (2023). AAV-mediated base-editing therapy ameliorates the disease phenotypes in a mouse model of retinitis pigmentosa. Nat. Commun., 14.","DOI":"10.1038\/s41467-023-40655-6"},{"key":"ref_66","doi-asserted-by":"crossref","first-page":"883","DOI":"10.1038\/s41587-020-0453-z","article-title":"Phage-assisted evolution of an adenine base editor with improved Cas domain compatibility and activity","volume":"38","author":"Richter","year":"2020","journal-title":"Nat. Biotechnol."},{"key":"ref_67","doi-asserted-by":"crossref","first-page":"16","DOI":"10.1016\/j.omtn.2022.11.021","article-title":"Visual function restoration in a mouse model of Leber congenital amaurosis via therapeutic base editing","volume":"31","author":"Jo","year":"2023","journal-title":"Mol. Ther. Nucleic Acids"},{"key":"ref_68","doi-asserted-by":"crossref","unstructured":"Choi, E.H., Suh, S., Foik, A.T., Leinonen, H., Newby, G.A., Gao, X.D., Banskota, S., Hoang, T., Du, S.W., and Dong, Z. (2022). In vivo base editing rescues cone photoreceptors in a mouse model of early-onset inherited retinal degeneration. Nat. Commun., 13.","DOI":"10.1038\/s41467-022-29490-3"},{"key":"ref_69","doi-asserted-by":"crossref","unstructured":"Fry, L.E., Major, L., Salman, A., McDermott, L.A., Yang, J., King, A.J., McClements, M.E., and MacLaren, R.E. (2025). Comparison of CRISPR-Cas13b RNA base editing approaches for USH2A-associated inherited retinal degeneration. Commun. Biol., 8.","DOI":"10.1038\/s42003-025-07557-3"},{"key":"ref_70","doi-asserted-by":"crossref","first-page":"7103","DOI":"10.1073\/pnas.95.12.7103","article-title":"Disease sequence from mutant rhodopsin allele to rod and cone photoreceptor degeneration in man","volume":"95","author":"Cideciyan","year":"1998","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"ref_71","doi-asserted-by":"crossref","unstructured":"Fu, Y., He, X., Ma, L., Gao, X.D., Liu, P., Shi, H., Chai, P., Ge, S., Jia, R., and Liu, D.R. (2025). In vivo prime editing rescues photoreceptor degeneration in nonsense mutant retinitis pigmentosa. Nat. Commun., 16.","DOI":"10.1038\/s41467-025-57628-6"},{"key":"ref_72","unstructured":"(2025, October 16). Prime Medicine Presents First In Vivo Proof-of-Concept Prime Editing Data Demonstrating Ability of Prime Editors to Treat Ophthalmological Diseases\u2014Prime Medicine, Inc.. Available online: https:\/\/investors.primemedicine.com\/news-releases\/news-release-details\/prime-medicine-presents-first-vivo-proof-concept-prime-editing\/."},{"key":"ref_73","doi-asserted-by":"crossref","first-page":"97","DOI":"10.1038\/s41551-019-0501-5","article-title":"Cytosine and adenine base editing of the brain, liver, retina, heart and skeletal muscle of mice via adeno-associated viruses","volume":"4","author":"Levy","year":"2020","journal-title":"Nat. Biomed. Eng."},{"key":"ref_74","doi-asserted-by":"crossref","unstructured":"Du, S.W., Newby, G.A., Salom, D., Gao, F., Menezes, C.R., Suh, S., Choi, E.H., Chen, P.Z., Liu, D.R., and Palczewski, K. (2024). In vivo photoreceptor base editing ameliorates rhodopsin-E150K autosomal-recessive retinitis pigmentosa in mice. Proc. Natl. Acad. Sci. USA, 121.","DOI":"10.1073\/pnas.2416827121"},{"key":"ref_75","doi-asserted-by":"crossref","first-page":"887","DOI":"10.1016\/j.jgg.2024.12.012","article-title":"In vivo adenine base editing ameliorates Rho-associated autosomal dominant retinitis pigmentosa","volume":"52","author":"Hu","year":"2025","journal-title":"J. Genet. Genom."},{"key":"ref_76","doi-asserted-by":"crossref","first-page":"283","DOI":"10.1016\/j.ymthe.2021.07.011","article-title":"Dual-AAV delivering split prime editor system for in vivo genome editing","volume":"30","author":"Zhi","year":"2022","journal-title":"Mol. Ther."},{"key":"ref_77","doi-asserted-by":"crossref","unstructured":"Qin, H., Zhang, W., Zhang, S., Feng, Y., Xu, W., Qi, J., Zhang, Q., Xu, C., Liu, S., and Zhang, J. (2023). Vision rescue via unconstrained in vivo prime editing in degenerating neural retinas. J. Exp. Med., 220.","DOI":"10.1084\/jem.20220776"},{"key":"ref_78","doi-asserted-by":"crossref","unstructured":"She, K., Liu, Y., Zhao, Q., Jin, X., Yang, Y., Su, J., Li, R., Song, L., Xiao, J., and Yao, S. (2023). Dual-AAV split prime editor corrects the mutation and phenotype in mice with inherited retinal degeneration. Signal Transduct. Target. Ther., 8.","DOI":"10.1038\/s41392-022-01234-1"},{"key":"ref_79","doi-asserted-by":"crossref","first-page":"136","DOI":"10.1016\/j.cell.2020.03.023","article-title":"CRISPR-Based Therapeutic Genome Editing: Strategies and In Vivo Delivery by AAV Vectors","volume":"181","author":"Wang","year":"2020","journal-title":"Cell"},{"key":"ref_80","doi-asserted-by":"crossref","unstructured":"Ferrari, S., Vavassori, V., Canarutto, D., Jacob, A., Castiello, M.C., Javed, A.O., and Genovese, P. (2021). Gene Editing of Hematopoietic Stem Cells: Hopes and Hurdles Toward Clinical Translation. Front. Genome Ed., 3.","DOI":"10.3389\/fgeed.2021.618378"},{"key":"ref_81","doi-asserted-by":"crossref","first-page":"101","DOI":"10.1038\/s41573-020-0090-8","article-title":"Engineering precision nanoparticles for drug delivery","volume":"20","author":"Mitchell","year":"2021","journal-title":"Nat. Rev. Drug Discov."},{"key":"ref_82","doi-asserted-by":"crossref","unstructured":"Staring, J., Raaben, M., and Brummelkamp, T.R. (2018). Viral escape from endosomes and host detection at a glance. J. Cell Sci., 131.","DOI":"10.1242\/jcs.216259"},{"key":"ref_83","doi-asserted-by":"crossref","unstructured":"Li, A., Mitsunobu, H., Yoshioka, S., Suzuki, T., Kondo, A., and Nishida, K. (2022). Cytosine base editing systems with minimized off-target effect and molecular size. Nat. Commun., 13.","DOI":"10.1038\/s41467-022-32157-8"},{"key":"ref_84","doi-asserted-by":"crossref","unstructured":"Chen, F., Alphonse, M., and Liu, Q. (2020). Strategies for nonviral nanoparticle-based delivery of CRISPR\/Cas9 therapeutics. Wiley Interdiscip. Rev. Nanomed. Nanobiotechnol., 12.","DOI":"10.1002\/wnan.1609"},{"key":"ref_85","doi-asserted-by":"crossref","first-page":"59","DOI":"10.1007\/s40484-014-0030-x","article-title":"Target specificity of the CRISPR-Cas9 system","volume":"2","author":"Wu","year":"2014","journal-title":"Quant. Biol."},{"key":"ref_86","doi-asserted-by":"crossref","first-page":"1179","DOI":"10.1038\/nbt.4005","article-title":"Structure-guided chemical modification of guide RNA enables potent non-viral in vivo genome editing","volume":"35","author":"Yin","year":"2017","journal-title":"Nat. Biotechnol."},{"key":"ref_87","doi-asserted-by":"crossref","first-page":"44","DOI":"10.1016\/j.jbiotec.2015.04.024","article-title":"Rapid and highly efficient mammalian cell engineering via Cas9 protein transfection","volume":"208","author":"Liang","year":"2015","journal-title":"J. Biotechnol."},{"key":"ref_88","doi-asserted-by":"crossref","first-page":"1166","DOI":"10.1039\/D1BM01658J","article-title":"Non-viral delivery of the CRISPR\/Cas system: DNA versus RNA versus RNP","volume":"10","author":"Lin","year":"2022","journal-title":"Biomater. Sci."},{"key":"ref_89","doi-asserted-by":"crossref","unstructured":"Ginn, S.L., Amaya, A.K., Alexander, I.E., Edelstein, M., and Abedi, M.R. (2018). Gene therapy clinical trials worldwide to 2017: An update. J. Gene Med., 20.","DOI":"10.1002\/jgm.3015"},{"key":"ref_90","doi-asserted-by":"crossref","unstructured":"Jony, M.J., Joshi, A., Dash, A., and Shukla, S. (2025). Non-Viral Delivery Systems to Transport Nucleic Acids for Inherited Retinal Disorders. Pharmaceuticals, 18.","DOI":"10.3390\/ph18010087"},{"key":"ref_91","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1016\/j.jconrel.2023.07.028","article-title":"Selective drug delivery to the retinal cells: Biological barriers and avenues","volume":"361","author":"Ramsay","year":"2023","journal-title":"J. Control. Release"},{"key":"ref_92","doi-asserted-by":"crossref","first-page":"2806","DOI":"10.1016\/j.cell.2022.03.045","article-title":"Therapeutic in vivo delivery of gene editing agents","volume":"185","author":"Raguram","year":"2022","journal-title":"Cell"},{"key":"ref_93","doi-asserted-by":"crossref","first-page":"728","DOI":"10.1039\/D2CS00106C","article-title":"Polymersome-based protein drug delivery\u2014Quo vadis?","volume":"52","author":"Gouveia","year":"2023","journal-title":"Chem. Soc. Rev."},{"key":"ref_94","doi-asserted-by":"crossref","first-page":"549","DOI":"10.1038\/s41434-021-00232-2","article-title":"Cas9-directed immune tolerance in humans-a model to evaluate regulatory T cells in gene therapy?","volume":"28","author":"Wagner","year":"2021","journal-title":"Gene Ther."},{"key":"ref_95","doi-asserted-by":"crossref","first-page":"1183","DOI":"10.2217\/fvl.13.112","article-title":"Adeno-associated virus structural biology as a tool in vector development","volume":"8","author":"Drouin","year":"2013","journal-title":"Future Virol."},{"key":"ref_96","doi-asserted-by":"crossref","first-page":"358","DOI":"10.1038\/s41573-019-0012-9","article-title":"Adeno-associated virus vector as a platform for gene therapy delivery","volume":"18","author":"Wang","year":"2019","journal-title":"Nat. Rev. Drug Discov."},{"key":"ref_97","doi-asserted-by":"crossref","first-page":"317","DOI":"10.1007\/s40259-017-0234-5","article-title":"Adeno-Associated Virus (AAV) as a Vector for Gene Therapy","volume":"31","author":"Naso","year":"2017","journal-title":"BioDrugs"},{"key":"ref_98","doi-asserted-by":"crossref","first-page":"80","DOI":"10.1038\/mt.2009.255","article-title":"Effect of genome size on AAV vector packaging","volume":"18","author":"Wu","year":"2010","journal-title":"Mol. Ther."},{"key":"ref_99","doi-asserted-by":"crossref","first-page":"641","DOI":"10.1038\/nrd.2018.110","article-title":"Gene therapy for neurological disorders: Progress and prospects","volume":"17","author":"Deverman","year":"2018","journal-title":"Nat. Rev. Drug Discov."},{"key":"ref_100","doi-asserted-by":"crossref","first-page":"106","DOI":"10.1038\/s41593-021-00969-4","article-title":"AAV capsid variants with brain-wide transgene expression and decreased liver targeting after intravenous delivery in mouse and marmoset","volume":"25","author":"Goertsen","year":"2022","journal-title":"Nat. Neurosci."},{"key":"ref_101","doi-asserted-by":"crossref","unstructured":"Byrne, L.C., Day, T.P., Visel, M., Strazzeri, J.A., Fortuny, C., Dalkara, D., Merigan, W.H., Schaffer, D.V., and Flannery, J.G. (2020). In vivo-directed evolution of adeno-associated virus in the primate retina. JCI Insight, 5.","DOI":"10.1172\/jci.insight.135112"},{"key":"ref_102","doi-asserted-by":"crossref","unstructured":"Dalkara, D., Byrne, L.C., Klimczak, R.R., Visel, M., Yin, L., Merigan, W.H., Flannery, J.G., and Schaffer, D.V. (2013). In vivo-directed evolution of a new adeno-associated virus for therapeutic outer retinal gene delivery from the vitreous. Sci. Transl. Med., 5.","DOI":"10.1126\/scitranslmed.3005708"},{"key":"ref_103","doi-asserted-by":"crossref","first-page":"204","DOI":"10.1038\/nbt.3440","article-title":"Cre-dependent selection yields AAV variants for widespread gene transfer to the adult brain","volume":"34","author":"Deverman","year":"2016","journal-title":"Nat. Biotechnol."},{"key":"ref_104","doi-asserted-by":"crossref","first-page":"121","DOI":"10.1146\/annurev-vision-102016-061413","article-title":"Vectors and Gene Delivery to the Retina","volume":"3","author":"Planul","year":"2017","journal-title":"Annu. Rev. Vis. Sci."},{"key":"ref_105","doi-asserted-by":"crossref","first-page":"2240","DOI":"10.1056\/NEJMoa0802315","article-title":"Safety and efficacy of gene transfer for Leber\u2019s congenital amaurosis","volume":"358","author":"Maguire","year":"2008","journal-title":"N. Engl. J. Med."},{"key":"ref_106","doi-asserted-by":"crossref","first-page":"236","DOI":"10.1016\/j.omtm.2022.03.016","article-title":"AAV-PHP.eB transduces both the inner and outer retina with high efficacy in mice","volume":"25","author":"Palfi","year":"2022","journal-title":"Mol. Ther. Methods Clin. Dev."},{"key":"ref_107","doi-asserted-by":"crossref","first-page":"3441","DOI":"10.1016\/j.ymthe.2023.10.001","article-title":"Intravitreal injection of a rationally designed AAV capsid library in non-human primate identifies variants with enhanced retinal transduction and neutralizing antibody evasion","volume":"31","author":"Kellish","year":"2023","journal-title":"Mol. Ther."},{"key":"ref_108","doi-asserted-by":"crossref","first-page":"3470","DOI":"10.1167\/iovs.16-19316","article-title":"AAV-Mediated CRISPR\/Cas Gene Editing of Retinal Cells In Vivo","volume":"57","author":"Hung","year":"2016","journal-title":"Investig. Ophthalmol. Vis. Sci."},{"key":"ref_109","doi-asserted-by":"crossref","unstructured":"Xiao, Y., Muhuri, M., Li, S., Qin, W., Xu, G., Luo, L., Li, J., Letizia, A.J., Wang, S.K., and Chan, Y.K. (2019). Circumventing cellular immunity by miR142-mediated regulation sufficiently supports rAAV-delivered OVA expression without activating humoral immunity. JCI Insight, 5.","DOI":"10.1172\/jci.insight.99052"},{"key":"ref_110","doi-asserted-by":"crossref","first-page":"111","DOI":"10.1016\/j.omtm.2018.11.009","article-title":"A Self-Deleting AAV-CRISPR System for In Vivo Genome Editing","volume":"12","author":"Li","year":"2019","journal-title":"Mol. Ther. Methods Clin. Dev."},{"key":"ref_111","doi-asserted-by":"crossref","unstructured":"Hoffmann, M.D., Aschenbrenner, S., Grosse, S., Rapti, K., Domenger, C., Fakhiri, J., Mastel, M., B\u00f6rner, K., Eils, R., and Grimm, D. (2019). Cell-specific CRISPR-Cas9 activation by microRNA-dependent expression of anti-CRISPR proteins. Nucleic Acids Res., 47.","DOI":"10.1101\/480384"},{"key":"ref_112","doi-asserted-by":"crossref","first-page":"1319","DOI":"10.1038\/s41591-024-03466-w","article-title":"In vivo base editing extends lifespan of a humanized mouse model of prion disease","volume":"31","author":"An","year":"2025","journal-title":"Nat. Med."},{"key":"ref_113","doi-asserted-by":"crossref","first-page":"671","DOI":"10.1016\/S0022-5223(03)00082-5","article-title":"Direct comparison of efficiency and stability of gene transfer into the mammalian heart using adeno-associated virus versus adenovirus vectors","volume":"126","author":"Chu","year":"2003","journal-title":"J. Thorac. Cardiovasc. Surg."},{"key":"ref_114","doi-asserted-by":"crossref","first-page":"709","DOI":"10.1016\/j.ymthe.2020.01.001","article-title":"Immune Responses to Viral Gene Therapy Vectors","volume":"28","author":"Shirley","year":"2020","journal-title":"Mol. Ther."},{"key":"ref_115","doi-asserted-by":"crossref","first-page":"249","DOI":"10.1038\/s41591-018-0326-x","article-title":"Identification of preexisting adaptive immunity to Cas9 proteins in humans","volume":"25","author":"Charlesworth","year":"2019","journal-title":"Nat. Med."},{"key":"ref_116","doi-asserted-by":"crossref","unstructured":"Hanlon, K.S., Kleinstiver, B.P., Garcia, S.P., Zaborowski, M.P., Volak, A., Spirig, S.E., Muller, A., Sousa, A.A., Tsai, S.Q., and Bengtsson, N.E. (2019). High levels of AAV vector integration into CRISPR-induced DNA breaks. Nat. Commun., 10.","DOI":"10.1038\/s41467-019-12449-2"},{"key":"ref_117","first-page":"73","article-title":"Random integration of AAV plasmid into the mouse genome following sub-retinal delivery","volume":"63","author":"Farkas","year":"2022","journal-title":"Investig. Ophthalmol. Vis. Sci."},{"key":"ref_118","doi-asserted-by":"crossref","first-page":"1232","DOI":"10.1038\/s41587-023-01974-7","article-title":"Integrated vector genomes may contribute to long-term expression in primate liver after AAV administration","volume":"42","author":"Greig","year":"2024","journal-title":"Nat. Biotechnol."},{"key":"ref_119","doi-asserted-by":"crossref","first-page":"291","DOI":"10.1038\/s41586-021-03770-2","article-title":"Regulated control of gene therapies by drug-induced splicing","volume":"596","author":"Monteys","year":"2021","journal-title":"Nature"},{"key":"ref_120","doi-asserted-by":"crossref","unstructured":"Sun, Y., Chen, Q., Cheng, Y., Wang, X., Deng, Z., Zhou, F., and Sun, Y. (2024). Design and Engineering of Light-Induced Base Editors Facilitating Genome Editing with Enhanced Fidelity. Adv. Sci., 11.","DOI":"10.1002\/advs.202305311"},{"key":"ref_121","doi-asserted-by":"crossref","first-page":"2443","DOI":"10.1016\/j.ymthe.2022.04.010","article-title":"Eliminating predictable DNA off-target effects of cytosine base editor by using dual guiders including sgRNA and TALE","volume":"30","author":"Zhou","year":"2022","journal-title":"Mol. Ther."},{"key":"ref_122","doi-asserted-by":"crossref","first-page":"47","DOI":"10.1089\/hum.2019.220","article-title":"Can Adeno-Associated Viral Vectors Deliver Effectively Large Genes?","volume":"31","author":"Tornabene","year":"2020","journal-title":"Hum. Gene Ther."},{"key":"ref_123","doi-asserted-by":"crossref","first-page":"R204","DOI":"10.1016\/j.cub.2017.01.016","article-title":"Inteins","volume":"27","author":"Lennon","year":"2017","journal-title":"Curr. Biol."},{"key":"ref_124","doi-asserted-by":"crossref","first-page":"263","DOI":"10.1093\/protein\/gzu028","article-title":"Nature\u2019s recipe for splitting inteins","volume":"27","author":"Aranko","year":"2014","journal-title":"Protein Eng. Des. Sel."},{"key":"ref_125","doi-asserted-by":"crossref","first-page":"896","DOI":"10.1089\/hum.2004.15.896","article-title":"Trans-splicing adeno-associated viral vector-mediated gene therapy is limited by the accumulation of spliced mRNA but not by dual vector coinfection efficiency","volume":"15","author":"Xu","year":"2004","journal-title":"Hum. Gene Ther."},{"key":"ref_126","doi-asserted-by":"crossref","first-page":"383","DOI":"10.1006\/mthe.2001.0456","article-title":"Expanding AAV packaging capacity with trans-splicing or overlapping vectors: A quantitative comparison","volume":"4","author":"Duan","year":"2001","journal-title":"Mol. Ther."},{"key":"ref_127","doi-asserted-by":"crossref","first-page":"1343","DOI":"10.1016\/j.ymthe.2022.01.005","article-title":"A flexible split prime editor using truncated reverse transcriptase improves dual-AAV delivery in mouse liver","volume":"30","author":"Zheng","year":"2022","journal-title":"Mol. Ther."},{"key":"ref_128","doi-asserted-by":"crossref","unstructured":"B\u00f6ck, D., Rothgangl, T., Villiger, L., Schmidheini, L., Matsushita, M., Mathis, N., Ioannidi, E., Rimann, N., Grisch-Chan, H.M., and Kreutzer, S. (2022). In vivo prime editing of a metabolic liver disease in mice. Sci. Transl. Med., 14.","DOI":"10.1126\/scitranslmed.abl9238"},{"key":"ref_129","doi-asserted-by":"crossref","unstructured":"Tornabene, P., Trapani, I., Minopoli, R., Centrulo, M., Lupo, M., de Simone, S., Tiberi, P., Dell\u2019Aquila, F., Marrocco, E., and Iodice, C. (2019). Intein-mediated protein trans-splicing expands adeno-associated virus transfer capacity in the retina. Sci. Transl. Med., 11.","DOI":"10.1126\/scitranslmed.aav4523"},{"key":"ref_130","doi-asserted-by":"crossref","first-page":"402","DOI":"10.1038\/s41587-021-01039-7","article-title":"Engineered pegRNAs improve prime editing efficiency","volume":"40","author":"Nelson","year":"2022","journal-title":"Nat. Biotechnol."},{"key":"ref_131","doi-asserted-by":"crossref","first-page":"253","DOI":"10.1038\/s41587-023-01758-z","article-title":"Efficient prime editing in mouse brain, liver and heart with dual AAVs","volume":"42","author":"Davis","year":"2024","journal-title":"Nat. Biotechnol."},{"key":"ref_132","doi-asserted-by":"crossref","first-page":"1355","DOI":"10.1007\/s11427-020-1775-2","article-title":"Efficient and high-fidelity base editor with expanded PAM compatibility for cytidine dinucleotide","volume":"64","author":"Liu","year":"2021","journal-title":"Sci. China Life Sci."},{"key":"ref_133","doi-asserted-by":"crossref","unstructured":"Kim, E., Koo, T., Park, S.W., Kim, D., Kim, K., Cho, H.-Y., Song, D.W., Lee, K.J., Jung, M.H., and Kim, S. (2017). In vivo genome editing with a small Cas9 orthologue derived from Campylobacter jejuni. Nat. Commun., 8.","DOI":"10.1038\/ncomms14500"},{"key":"ref_134","doi-asserted-by":"crossref","unstructured":"Hu, Z., Wang, S., Zhang, C., Gao, N., Li, M., Wang, D., Wang, D., Liu, D., Liu, H., and Ong, S.-G. (2020). A compact Cas9 ortholog from Staphylococcus Auricularis (SauriCas9) expands the DNA targeting scope. PLoS Biol., 18.","DOI":"10.1371\/journal.pbio.3000686"},{"key":"ref_135","doi-asserted-by":"crossref","first-page":"1272","DOI":"10.1038\/s41551-022-00911-4","article-title":"Efficient in vivo base editing via single adeno-associated viruses with size-optimized genomes encoding compact adenine base editors","volume":"6","author":"Davis","year":"2022","journal-title":"Nat. Biomed. Eng."},{"key":"ref_136","doi-asserted-by":"crossref","first-page":"285","DOI":"10.1089\/genbio.2022.0015","article-title":"Adenine Base Editing In Vivo with a Single Adeno-Associated Virus Vector","volume":"1","author":"Zhang","year":"2022","journal-title":"GEN Biotechnol."},{"key":"ref_137","doi-asserted-by":"crossref","unstructured":"Yi, Z., Zhang, X., Wei, X., Li, J., Ren, J., Zhang, X., Zhang, Y., Tang, H., Chang, X., and Yu, Y. (2024). Programmable DNA pyrimidine base editing via engineered uracil-DNA glycosylase. Nat. Commun., 15.","DOI":"10.1038\/s41467-024-50012-w"},{"key":"ref_138","doi-asserted-by":"crossref","unstructured":"Shams, A., Higgins, S.A., Fellmann, C., Laughlin, T.G., Oakes, B.L., Lew, R., Kim, S., Lukarska, M., Arnold, M., and Staahl, B.T. (2021). Comprehensive deletion landscape of CRISPR-Cas9 identifies minimal RNA-guided DNA-binding modules. Nat. Commun., 12.","DOI":"10.1038\/s41467-021-25992-8"},{"key":"ref_139","doi-asserted-by":"crossref","unstructured":"Kabadi, A.M., Ousterout, D.G., Hilton, I.B., and Gersbach, C.A. (2014). Multiplex CRISPR\/Cas9-based genome engineering from a single lentiviral vector. Nucleic Acids Res., 42.","DOI":"10.1093\/nar\/gku749"},{"key":"ref_140","doi-asserted-by":"crossref","first-page":"476","DOI":"10.1038\/gt.2009.22","article-title":"Lentiviral vectors for gene transfer into the spinal cord glial cells","volume":"16","author":"Meunier","year":"2009","journal-title":"Gene Ther."},{"key":"ref_141","doi-asserted-by":"crossref","first-page":"291","DOI":"10.1089\/hgtb.2017.084","article-title":"Pseudotyped Lentiviral Vectors: One Vector, Many Guises","volume":"28","author":"Joglekar","year":"2017","journal-title":"Hum. Gene Ther. Methods"},{"key":"ref_142","doi-asserted-by":"crossref","first-page":"428","DOI":"10.1089\/hum.2013.172","article-title":"Long-term episomal transgene expression from mitotically stable integration-deficient lentiviral vectors","volume":"25","author":"Appelt","year":"2014","journal-title":"Hum. Gene Ther."},{"key":"ref_143","doi-asserted-by":"crossref","first-page":"144","DOI":"10.1038\/s41551-020-00656-y","article-title":"Lentiviral delivery of co-packaged Cas9 mRNA and a Vegfa-targeting guide RNA prevents wet age-related macular degeneration in mice","volume":"5","author":"Ling","year":"2021","journal-title":"Nat. Biomed. Eng."},{"key":"ref_144","doi-asserted-by":"crossref","first-page":"627","DOI":"10.1038\/gt.2016.27","article-title":"Lentivirus pre-packed with Cas9 protein for safer gene editing","volume":"23","author":"Choi","year":"2016","journal-title":"Gene Ther."},{"key":"ref_145","doi-asserted-by":"crossref","first-page":"942","DOI":"10.1038\/sj.gt.3302485","article-title":"Lentiviral vector-mediated gene transfer in adult mouse photoreceptors is impaired by the presence of a physical barrier","volume":"12","author":"Kostic","year":"2005","journal-title":"Gene Ther."},{"key":"ref_146","doi-asserted-by":"crossref","first-page":"169","DOI":"10.1038\/s41551-020-00632-6","article-title":"Restoration of visual function in adult mice with an inherited retinal disease via adenine base editing","volume":"5","author":"Suh","year":"2021","journal-title":"Nat. Biomed. Eng."},{"key":"ref_147","doi-asserted-by":"crossref","first-page":"1529","DOI":"10.1038\/s41375-018-0106-0","article-title":"Clinical use of lentiviral vectors","volume":"32","author":"Milone","year":"2018","journal-title":"Leukemia"},{"key":"ref_148","doi-asserted-by":"crossref","first-page":"43","DOI":"10.1016\/j.gendis.2017.04.001","article-title":"Adenovirus-Mediated Gene Delivery: Potential Applications for Gene and Cell-Based Therapies in the New Era of Personalized Medicine","volume":"4","author":"Lee","year":"2017","journal-title":"Genes Dis."},{"key":"ref_149","doi-asserted-by":"crossref","first-page":"387","DOI":"10.2174\/1566523054546224","article-title":"Altering the tropism of lentiviral vectors through pseudotyping","volume":"5","author":"Cronin","year":"2005","journal-title":"Curr. Gene Ther."},{"key":"ref_150","doi-asserted-by":"crossref","first-page":"432","DOI":"10.1089\/hum.2015.087","article-title":"Adenovirus-Mediated Somatic Genome Editing of Pten by CRISPR\/Cas9 in Mouse Liver in Spite of Cas9-Specific Immune Responses","volume":"26","author":"Wang","year":"2015","journal-title":"Hum. Gene Ther."},{"key":"ref_151","doi-asserted-by":"crossref","first-page":"1410","DOI":"10.1038\/sj.gt.3301251","article-title":"Recombinant adenoviral vectors have adjuvant activity and stimulate T cell responses against tumor cells","volume":"7","author":"Geutskens","year":"2000","journal-title":"Gene Ther."},{"key":"ref_152","doi-asserted-by":"crossref","first-page":"1157","DOI":"10.1089\/hum.2004.15.1157","article-title":"The innate immune response to adenovirus vectors","volume":"15","author":"Muruve","year":"2004","journal-title":"Hum. Gene Ther."},{"key":"ref_153","first-page":"335","article-title":"Adenovirus vectors: Biology, design, and production","volume":"273","author":"Imperiale","year":"2004","journal-title":"Curr. Top. Microbiol. Immunol."},{"key":"ref_154","doi-asserted-by":"crossref","first-page":"19","DOI":"10.1634\/stemcells.18-1-19","article-title":"Latest developments in gene transfer technology: Achievements, perspectives, and controversies over therapeutic applications","volume":"18","author":"Romano","year":"2000","journal-title":"Stem Cells"},{"key":"ref_155","doi-asserted-by":"crossref","first-page":"541","DOI":"10.1038\/nrg3763","article-title":"Non-viral vectors for gene-based therapy","volume":"15","author":"Yin","year":"2014","journal-title":"Nat. Rev. Genet."},{"key":"ref_156","doi-asserted-by":"crossref","first-page":"108","DOI":"10.1016\/j.preteyeres.2014.08.001","article-title":"Vector platforms for gene therapy of inherited retinopathies","volume":"43","author":"Trapani","year":"2014","journal-title":"Prog. Retin. Eye Res."},{"key":"ref_157","doi-asserted-by":"crossref","first-page":"1669","DOI":"10.1021\/acs.molpharmaceut.1c00916","article-title":"Lipid-Nanoparticle-Based Delivery of CRISPR\/Cas9 Genome-Editing Components","volume":"19","author":"Kazemian","year":"2022","journal-title":"Mol. Pharm."},{"key":"ref_158","doi-asserted-by":"crossref","first-page":"447","DOI":"10.1007\/s11095-005-9445-4","article-title":"The nuclear pore complex: The gateway to successful nonviral gene delivery","volume":"23","author":"Mastrobattista","year":"2006","journal-title":"Pharm. Res."},{"key":"ref_159","doi-asserted-by":"crossref","first-page":"136","DOI":"10.1016\/j.molmed.2023.11.009","article-title":"Toward low-cost gene therapy: mRNA-based therapeutics for treatment of inherited retinal diseases","volume":"30","author":"Antas","year":"2024","journal-title":"Trends Mol. Med."},{"key":"ref_160","doi-asserted-by":"crossref","first-page":"1149","DOI":"10.1586\/erv.10.115","article-title":"Virus-like particles in vaccine development","volume":"9","author":"Mellado","year":"2010","journal-title":"Expert. Rev. Vaccines"},{"key":"ref_161","doi-asserted-by":"crossref","first-page":"1561","DOI":"10.1021\/acsptsci.3c00185","article-title":"Looking Back, Moving Forward: Lipid Nanoparticles as a Promising Frontier in Gene Delivery","volume":"6","author":"Pozzi","year":"2023","journal-title":"ACS Pharmacol. Transl. Sci."},{"key":"ref_162","doi-asserted-by":"crossref","unstructured":"Xu, S., Hu, Z., Song, F., Xu, Y., and Han, X. (2025). Lipid nanoparticles: Composition, formulation, and application. Mol. Ther. Methods Clin. Dev., 33.","DOI":"10.1016\/j.omtm.2025.101463"},{"key":"ref_163","doi-asserted-by":"crossref","first-page":"265","DOI":"10.1038\/s41576-021-00439-4","article-title":"Drug delivery systems for RNA therapeutics","volume":"23","author":"Paunovska","year":"2022","journal-title":"Nat. Rev. Genet."},{"key":"ref_164","doi-asserted-by":"crossref","first-page":"403","DOI":"10.1056\/NEJMoa2035389","article-title":"Efficacy and Safety of the mRNA-1273 SARS-CoV-2 Vaccine","volume":"384","author":"Baden","year":"2021","journal-title":"N. Engl. J. Med."},{"key":"ref_165","doi-asserted-by":"crossref","first-page":"870","DOI":"10.1038\/nbt.3298","article-title":"Visualizing lipid-formulated siRNA release from endosomes and target gene knockdown","volume":"33","author":"Wittrup","year":"2015","journal-title":"Nat. Biotechnol."},{"key":"ref_166","doi-asserted-by":"crossref","first-page":"1570","DOI":"10.1038\/mt.2013.124","article-title":"Biodegradable lipids enabling rapidly eliminated lipid nanoparticles for systemic delivery of RNAi therapeutics","volume":"21","author":"Maier","year":"2013","journal-title":"Mol. Ther."},{"key":"ref_167","doi-asserted-by":"crossref","unstructured":"Jang, H.-K., Jo, D.H., Lee, S.-N., Cho, C.S., Jeong, Y.K., Jung, Y., Yu, J., Kim, J.H., Woo, J.-S., and Bae, S. (2021). High-purity production and precise editing of DNA base editing ribonucleoproteins. Sci. Adv., 7.","DOI":"10.1126\/sciadv.abg2661"},{"key":"ref_168","doi-asserted-by":"crossref","first-page":"42","DOI":"10.1016\/j.omtn.2017.02.005","article-title":"Targeted Multifunctional Lipid ECO Plasmid DNA Nanoparticles as Efficient Non-viral Gene Therapy for Leber\u2019s Congenital Amaurosis","volume":"7","author":"Sun","year":"2017","journal-title":"Mol. Ther. Nucleic Acids"},{"key":"ref_169","doi-asserted-by":"crossref","first-page":"4289","DOI":"10.1039\/D0BM01947J","article-title":"Illuminating endosomal escape of polymorphic lipid nanoparticles that boost mRNA delivery","volume":"9","author":"Herrera","year":"2021","journal-title":"Biomater. Sci."},{"key":"ref_170","doi-asserted-by":"crossref","unstructured":"Herrera-Barrera, M., Gautam, M., Lokras, A., Vlasova, K., Foged, C., and Sahay, G. (2023). Lipid Nanoparticle-Enabled Intracellular Delivery of Prime Editors. AAPS J., 25.","DOI":"10.1208\/s12248-023-00833-2"},{"key":"ref_171","doi-asserted-by":"crossref","unstructured":"Herrera-Barrera, M., Ryals, R.C., Gautam, M., Jozic, A., Landry, M., Korzun, T., Gupta, M., Acosta, C., Stoddard, J., and Reynaga, R. (2023). Peptide-guided lipid nanoparticles deliver mRNA to the neural retina of rodents and nonhuman primates. Sci. Adv., 9.","DOI":"10.1126\/sciadv.add4623"},{"key":"ref_172","doi-asserted-by":"crossref","first-page":"244","DOI":"10.1016\/j.jconrel.2023.05.033","article-title":"BDNF gene delivery to the retina by cell adhesion peptide-conjugated gemini nanoplexes in vivo","volume":"359","author":"Narsineni","year":"2023","journal-title":"J. Control. Release"},{"key":"ref_173","doi-asserted-by":"crossref","first-page":"5257","DOI":"10.1021\/nl502275s","article-title":"Nanoparticle-assisted targeted delivery of eye-specific genes to eyes significantly improves the vision of blind mice in vivo","volume":"14","author":"Rajala","year":"2014","journal-title":"Nano Lett."},{"key":"ref_174","doi-asserted-by":"crossref","unstructured":"Bisht, R., Mandal, A., Jaiswal, J.K., and Rupenthal, I.D. (2018). Nanocarrier mediated retinal drug delivery: Overcoming ocular barriers to treat posterior eye diseases. Wiley Interdiscip. Rev. Nanomed. Nanobiotechnol., 10.","DOI":"10.1002\/wnan.1473"},{"key":"ref_175","doi-asserted-by":"crossref","first-page":"194","DOI":"10.1016\/j.jconrel.2020.04.052","article-title":"A pH-responsive silica-metal-organic framework hybrid nanoparticle for the delivery of hydrophilic drugs, nucleic acids, and CRISPR-Cas9 genome-editing machineries","volume":"324","author":"Wang","year":"2020","journal-title":"J. Control. Release"},{"key":"ref_176","doi-asserted-by":"crossref","first-page":"296","DOI":"10.1016\/j.jconrel.2021.06.030","article-title":"In vivo targeted delivery of nucleic acids and CRISPR genome editors enabled by GSH-responsive silica nanoparticles","volume":"336","author":"Wang","year":"2021","journal-title":"J. Control. Release"},{"key":"ref_177","doi-asserted-by":"crossref","unstructured":"Sioson, V.A., Kim, M., and Joo, J. (2021). Challenges in delivery systems for CRISPR-based genome editing and opportunities of nanomedicine. Biomed. Eng. Lett., 11.","DOI":"10.1007\/s13534-021-00199-4"},{"key":"ref_178","doi-asserted-by":"crossref","unstructured":"Kabra, M., Shahi, P.K., Wang, Y., Sinha, D., Spillane, A., Newby, G.A., Saxena, S., Tong, Y., Chang, Y., and Abdeen, A.A. (2023). Nonviral base editing of KCNJ13 mutation preserves vision in a model of inherited retinal channelopathy. J. Clin. Investig., 133.","DOI":"10.1172\/JCI171356"},{"key":"ref_179","doi-asserted-by":"crossref","unstructured":"Chou, S.-J., Yang, P., Ban, Q., Yang, Y.-P., Wang, M.-L., Chien, C.-S., Chen, S.-J., Sun, N., Zhu, Y., and Liu, H. (2020). Dual Supramolecular Nanoparticle Vectors Enable CRISPR\/Cas9-Mediated Knockin of Retinoschisin 1 Gene-A Potential Nonviral Therapeutic Solution for X-Linked Juvenile Retinoschisis. Adv. Sci., 7.","DOI":"10.1002\/advs.201903432"},{"key":"ref_180","doi-asserted-by":"crossref","first-page":"250","DOI":"10.1016\/j.cell.2021.12.021","article-title":"Engineered virus-like particles for efficient in vivo delivery of therapeutic proteins","volume":"185","author":"Banskota","year":"2022","journal-title":"Cell"},{"key":"ref_181","doi-asserted-by":"crossref","first-page":"69","DOI":"10.1089\/crispr.2020.0095","article-title":"Adenine Base Editor Ribonucleoproteins Delivered by Lentivirus-like Particles Show High On-Target Base Editing and Undetectable RNA Off-Target Activities","volume":"4","author":"Lyu","year":"2021","journal-title":"CRISPR J."},{"key":"ref_182","doi-asserted-by":"crossref","first-page":"10059","DOI":"10.1093\/nar\/gkad676","article-title":"Engineered lentivirus-derived nanoparticles (LVNPs) for delivery of CRISPR\/Cas ribonucleoprotein complexes supporting base editing, prime editing and in vivo gene modification","volume":"51","author":"Haldrup","year":"2023","journal-title":"Nucleic Acids Res."},{"key":"ref_183","doi-asserted-by":"crossref","first-page":"309","DOI":"10.1016\/j.molcel.2004.09.023","article-title":"Retroviral pseudotransduction for targeted cell manipulation","volume":"16","author":"Galla","year":"2004","journal-title":"Mol. Cell"},{"key":"ref_184","doi-asserted-by":"crossref","unstructured":"Prel, A., Caval, V., Gayon, R., Ravassard, P., Duthoit, C., Payen, E., Maouche-Chretien, L., Creneguy, A., Nguyen, T.H., and Martin, N. (2015). Highly efficient in vitro and in vivo delivery of functional RNAs using new versatile MS2-chimeric retrovirus-like particles. Mol. Ther. Methods Clin. Dev., 2.","DOI":"10.1038\/mtm.2015.39"},{"key":"ref_185","doi-asserted-by":"crossref","first-page":"256","DOI":"10.1016\/j.omtn.2018.09.006","article-title":"Transient Retrovirus-Based CRISPR\/Cas9 All-in-One Particles for Efficient, Targeted Gene Knockout","volume":"13","author":"Knopp","year":"2018","journal-title":"Mol. Ther. Nucleic Acids"},{"key":"ref_186","doi-asserted-by":"crossref","unstructured":"Lu, B., Javidi-Parsijani, P., Makani, V., Mehraein-Ghomi, F., Sarhan, W.M., Sun, D., Yoo, K.W., Atala, Z.P., Lyu, P., and Atala, A. (2019). Delivering SaCas9 mRNA by lentivirus-like bionanoparticles for transient expression and efficient genome editing. Nucleic Acids Res., 47.","DOI":"10.1093\/nar\/gkz093"},{"key":"ref_187","doi-asserted-by":"crossref","unstructured":"Wang, W., Bartholomae, C.C., Arens, A., Gabriel, R., Glimm, H., von Kalle, C., and Schmidt, M. (2010). Genome Wide Characterization of Insertion Profiles of Integration Deficient Lentiviral Vectors Using Two-Sided Non-Restrictive and Standard LAM-PCR. Mol. Ther., 18.","DOI":"10.1016\/S1525-0016(16)37788-7"},{"key":"ref_188","doi-asserted-by":"crossref","first-page":"882","DOI":"10.1126\/science.abg6155","article-title":"Mammalian retrovirus-like protein PEG10 packages its own mRNA and can be pseudotyped for mRNA delivery","volume":"373","author":"Segel","year":"2021","journal-title":"Science"},{"key":"ref_189","doi-asserted-by":"crossref","first-page":"57","DOI":"10.1016\/j.omtn.2023.06.004","article-title":"Nanoblades allow high-level genome editing in murine and human organoids","volume":"33","author":"Tiroille","year":"2023","journal-title":"Mol. Ther. Nucleic Acids"},{"key":"ref_190","doi-asserted-by":"crossref","first-page":"259","DOI":"10.1016\/j.virusres.2008.01.012","article-title":"Chimaeric HIV-1 subtype C Gag molecules with large in-frame C-terminal polypeptide fusions form virus-like particles","volume":"133","author":"Halsey","year":"2008","journal-title":"Virus Res."},{"key":"ref_191","doi-asserted-by":"crossref","unstructured":"Gee, P., Lung, M.S.Y., Okuzaki, Y., Sasakawa, N., Iguchi, T., Makita, Y., Hozumi, H., Miura, Y., Yang, L.F., and Iwasaki, M. (2020). Extracellular nanovesicles for packaging of CRISPR-Cas9 protein and sgRNA to induce therapeutic exon skipping. Nat. Commun., 11.","DOI":"10.1038\/s41467-020-14957-y"},{"key":"ref_192","doi-asserted-by":"crossref","first-page":"8178","DOI":"10.1093\/nar\/gkaa561","article-title":"Highly efficient \u2018hit-and-run\u2019 genome editing with unconcentrated lentivectors carrying Vpr.Prot.Cas9 protein produced from RRE-containing transcripts","volume":"48","author":"Indikova","year":"2020","journal-title":"Nucleic Acids Res."},{"key":"ref_193","doi-asserted-by":"crossref","unstructured":"Lyu, P., Javidi-Parsijani, P., Atala, A., and Lu, B. (2019). Delivering Cas9\/sgRNA ribonucleoprotein (RNP) by lentiviral capsid-based bionanoparticles for efficient \u2018hit-and-run\u2019 genome editing. Nucleic Acids Res., 47.","DOI":"10.1093\/nar\/gkz605"},{"key":"ref_194","doi-asserted-by":"crossref","first-page":"1526","DOI":"10.1038\/s41587-023-02078-y","article-title":"Engineered virus-like particles for transient delivery of prime editor ribonucleoprotein complexes in vivo","volume":"42","author":"An","year":"2024","journal-title":"Nat. Biotechnol."},{"key":"ref_195","doi-asserted-by":"crossref","unstructured":"Rodrigues, A., Alves, P.M., Coroadinha, A., Rodrigues, A., Alves, P.M., and Coroadinha, A. (2011). Production of Retroviral and Lentiviral Gene Therapy Vectors: Challenges in the Manufacturing of Lipid Enveloped Virus. Viral Gene Therapy, IntechOpen.","DOI":"10.5772\/18615"},{"key":"ref_196","doi-asserted-by":"crossref","first-page":"R42","DOI":"10.1093\/hmg\/ddv451","article-title":"Progress and challenges in viral vector manufacturing","volume":"25","author":"Wright","year":"2016","journal-title":"Hum. Mol. Genet."},{"key":"ref_197","doi-asserted-by":"crossref","unstructured":"Park, E.Y., and Minkner, R. (2025). A systematic approach for scalable purification of virus-like particles. Protein Expr. Purif., 228.","DOI":"10.1016\/j.pep.2025.106664"},{"key":"ref_198","doi-asserted-by":"crossref","unstructured":"Li, M., Liu, Z., Wang, D., Ye, J., Shi, Z., Pan, C., Zhang, Q., Ju, R., Zheng, Y., and Liu, Y. (2024). Intraocular mRNA delivery with endogenous MmPEG10-based virus-like particles. Exp. Eye Res., 243.","DOI":"10.1016\/j.exer.2024.109899"},{"key":"ref_199","doi-asserted-by":"crossref","unstructured":"Wang, W., Wang, N., Zhao, X., Su, X., and Liu, Z. (2025). Recent advancements in polymer science for retinal diseases: New frontiers in drug delivery systems. APL Bioeng., 9.","DOI":"10.1063\/5.0264382"},{"key":"ref_200","doi-asserted-by":"crossref","unstructured":"Eblimit, A., Makia, M.S., Strayve, D., Crane, R., Conley, S.M., Sinha, T., Acharya, G., Al-Ubaidi, M.R., and Naash, M.I. (2021). Co-Injection of Sulfotyrosine Facilitates Retinal Uptake of Hyaluronic Acid Nanospheres Following Intravitreal Injection. Pharmaceutics, 13.","DOI":"10.3390\/pharmaceutics13091510"},{"key":"ref_201","doi-asserted-by":"crossref","first-page":"81","DOI":"10.1007\/978-1-4939-6518-2_7","article-title":"Cell-Penetrating Peptide-Mediated Delivery of Cas9 Protein and Guide RNA for Genome Editing","volume":"1507","author":"Suresh","year":"2017","journal-title":"Methods Mol. Biol."},{"key":"ref_202","doi-asserted-by":"crossref","first-page":"78","DOI":"10.1016\/j.peptides.2014.04.015","article-title":"Cell penetrating peptides: Efficient vectors for delivery of nanoparticles, nanocarriers, therapeutic and diagnostic molecules","volume":"57","author":"Farkhani","year":"2014","journal-title":"Peptides"},{"key":"ref_203","doi-asserted-by":"crossref","unstructured":"Wei, G., Wang, Y., Huang, X., Hou, H., and Zhou, S. (2018). Peptide-Based Nanocarriers for Cancer Therapy. Small Methods, 2.","DOI":"10.1002\/smtd.201700358"},{"key":"ref_204","doi-asserted-by":"crossref","first-page":"3092","DOI":"10.1007\/s12033-023-00932-7","article-title":"Exosomes for CRISPR-Cas9 Delivery: The Cutting Edge in Genome Editing","volume":"66","author":"Aslan","year":"2024","journal-title":"Mol. Biotechnol."},{"key":"ref_205","doi-asserted-by":"crossref","unstructured":"Wallen, M., Aqil, F., Spencer, W., and Gupta, R.C. (2023). Exosomes as an Emerging Plasmid Delivery Vehicle for Gene Therapy. Pharmaceutics, 15.","DOI":"10.3390\/pharmaceutics15071832"},{"key":"ref_206","doi-asserted-by":"crossref","unstructured":"Iqbal, Z., Rehman, K., Mahmood, A., Shabbir, M., Liang, Y., Duan, L., and Zeng, H. (2024). Exosome for mRNA delivery: Strategies and therapeutic applications. J. Nanobiotechnol., 22.","DOI":"10.1186\/s12951-024-02634-x"},{"key":"ref_207","doi-asserted-by":"crossref","unstructured":"Yang, Q., Li, S., Ou, H., Zhang, Y., Zhu, G., Li, S., and Lei, L. (2024). Exosome-based delivery strategies for tumor therapy: An update on modification, loading, and clinical application. J. Nanobiotechnol., 22.","DOI":"10.1186\/s12951-024-02298-7"},{"key":"ref_208","doi-asserted-by":"crossref","unstructured":"Koh, H.B., Kim, H.J., Kang, S.-W., and Yoo, T.-H. (2023). Exosome-Based Drug Delivery: Translation from Bench to Clinic. Pharmaceutics, 15.","DOI":"10.3390\/pharmaceutics15082042"},{"key":"ref_209","doi-asserted-by":"crossref","first-page":"357","DOI":"10.1038\/s41586-023-05870-7","article-title":"Programmable protein delivery with a bacterial contractile injection system","volume":"616","author":"Kreitz","year":"2023","journal-title":"Nature"},{"key":"ref_210","doi-asserted-by":"crossref","unstructured":"Kreitz, J., Yang, V., Friedrich, M.J., Pham, J., Macrae, R.K., and Zhang, F. (2025). Targeted delivery of diverse biomolecules with engineered bacterial nanosyringes. Nat. Biotechnol., 1\u20135.","DOI":"10.1038\/s41587-025-02774-x"},{"key":"ref_211","doi-asserted-by":"crossref","first-page":"490","DOI":"10.1038\/s41591-024-03422-8","article-title":"High-efficiency base editing in the retina in primates and human tissues","volume":"31","author":"Muller","year":"2025","journal-title":"Nat. Med."},{"key":"ref_212","doi-asserted-by":"crossref","unstructured":"Gautam, M., Jozic, A., Su, G.L.-N., Herrera-Barrera, M., Curtis, A., Arrizabalaga, S., Tschetter, W., Ryals, R.C., and Sahay, G. (2023). Lipid nanoparticles with PEG-variant surface modifications mediate genome editing in the mouse retina. Nat. Commun., 14.","DOI":"10.1038\/s41467-023-42189-3"},{"key":"ref_213","doi-asserted-by":"crossref","unstructured":"Vagni, P., Perlini, L.E., Chenais, N.A.L., Marchetti, T., Parrini, M., Contestabile, A., Cancedda, L., and Ghezzi, D. (2019). Gene Editing Preserves Visual Functions in a Mouse Model of Retinal Degeneration. Front. Neurosci., 13.","DOI":"10.3389\/fnins.2019.00945"},{"key":"ref_214","doi-asserted-by":"crossref","first-page":"1763","DOI":"10.1097\/IAE.0000000000003857","article-title":"Intraoperative bleb behavior in subretinal gene augmentation therapy for inherited retinal diseases","volume":"43","author":"Sisk","year":"2023","journal-title":"Retina"},{"key":"ref_215","doi-asserted-by":"crossref","first-page":"281","DOI":"10.1136\/bjo-2024-326221","article-title":"12-month outcomes after voretigene neparvovec gene therapy in paediatric patients with RPE65-mediated inherited retinal dystrophy","volume":"109","author":"Daruich","year":"2025","journal-title":"Br. J. Ophthalmol."},{"key":"ref_216","doi-asserted-by":"crossref","unstructured":"Wang, T., Yu, T., Liu, Q., Sung, T.-C., and Higuchi, A. (2024). Lipid nanoparticle technology-mediated therapeutic gene manipulation in the eyes. Mol. Ther. Nucleic Acids, 35.","DOI":"10.1016\/j.omtn.2024.102236"},{"key":"ref_217","doi-asserted-by":"crossref","first-page":"162","DOI":"10.1007\/s10354-024-01070-8","article-title":"Intravitreal therapy-success stories and challenges","volume":"175","author":"Egger","year":"2025","journal-title":"Wien. Med. Wochenschr."},{"key":"ref_218","doi-asserted-by":"crossref","first-page":"1403","DOI":"10.1007\/s40265-022-01777-1","article-title":"Suprachoroidal Space Triamcinolone Acetonide: A Review in Uveitic Macular Edema","volume":"82","author":"Fung","year":"2022","journal-title":"Drugs"},{"key":"ref_219","first-page":"191","article-title":"Voltammetrically Determined Differences in Changes Evoked by KC1 Microinjections on Catecholamine Levels in the Reticular Formation and Corpus Striatum of the Rat","volume":"41","author":"Bielik","year":"1992","journal-title":"Physiol. Res."},{"key":"ref_220","doi-asserted-by":"crossref","unstructured":"Scruggs, B.A., Berger, A., Knudsen, T., Kopp, F.N., Hill, M., Trncic, E., Anderson, K., Iezzi, R., and Marmorstein, A.D. (2025). Retinal gene therapy using epiretinal AAV-containing fibrin hydrogel implants. Sci. Adv., 11.","DOI":"10.1126\/sciadv.adv7922"},{"key":"ref_221","doi-asserted-by":"crossref","unstructured":"Liu, Z., Zhang, H., Jia, H., Wang, H., Huang, Z., Tang, Y., Wang, Z., Hu, J., Zhao, X., and Li, T. (2025). The clinical safety landscape for ocular AAV gene therapies: A systematic review and meta-analysis. iScience, 28.","DOI":"10.1016\/j.isci.2025.112265"},{"key":"ref_222","doi-asserted-by":"crossref","unstructured":"Moffit, J.S., Alatsis, K.R., Blanset, D.L., Buss, N., and Rana, P. (2025). Nonclinical strategies and considerations to enable the redosing of gene therapies. Mol. Ther. Methods Clin. Dev., 33.","DOI":"10.1016\/j.omtm.2025.101520"},{"key":"ref_223","doi-asserted-by":"crossref","unstructured":"Liu, Z., Chen, S., Davis, A.E., Lo, C.-H., Wang, Q., Li, T., Ning, K., Zhang, Q., Zhao, J., and Wang, S. (2024). Efficient Rescue of Retinal Degeneration in Pde6a Mice by Engineered Base Editing and Prime Editing. Adv. Sci., 11.","DOI":"10.1002\/advs.202405628"},{"key":"ref_224","doi-asserted-by":"crossref","first-page":"181","DOI":"10.1038\/s41551-021-00788-9","article-title":"Application of prime editing to the correction of mutations and phenotypes in adult mice with liver and eye diseases","volume":"6","author":"Jang","year":"2022","journal-title":"Nat. Biomed. Eng."},{"key":"ref_225","doi-asserted-by":"crossref","unstructured":"Kumar, S., Fry, L.E., Wang, J.H., Martin, K.R., Hewitt, A.W., Chen, F.K., and Liu, G.S. (2023). RNA-targeting strategies as a platform for ocular gene therapy. Prog. Retin. Eye Res., 92.","DOI":"10.1016\/j.preteyeres.2022.101110"},{"key":"ref_226","doi-asserted-by":"crossref","unstructured":"Jo, D.H., Bae, S., Kim, H.H., Kim, J.-S., and Kim, J.H. (2023). In vivo application of base and prime editing to treat inherited retinal diseases. Prog. Retin. Eye Res., 94.","DOI":"10.1016\/j.preteyeres.2022.101132"},{"key":"ref_227","unstructured":"Stefanidakis, M., Maeder, M., Bounoutas, G., Yudkoff, C., Chao, H., Haskett, S., Nguyen, D., Samuelsson, S.J., Giannoukos, G., and Ciulla, D. (2018). Efficient in vivo editing of CEP290 IVS26 by EDIT-101 as a novel therapeutic for treatment of Leber Congenital Amaurosis 10. Investig. Ophthalmol. Vis. Sci., 59."},{"key":"ref_228","doi-asserted-by":"crossref","unstructured":"Pniakowska, Z., Dzie\u017ca, N., Kustosik, N., Przybylak, A., and Jurowski, P. (2025). Genetic Therapies for Retinitis Pigmentosa: Current Breakthroughs and Future Directions. J. Clin. Med., 14.","DOI":"10.3390\/jcm14165661"},{"key":"ref_229","doi-asserted-by":"crossref","first-page":"939","DOI":"10.1016\/j.hrthm.2024.02.001","article-title":"Adeno-associated virus-mediated gene therapy for cardiac tachyarrhythmia: A systematic review and meta-analysis","volume":"21","author":"Mundisugih","year":"2024","journal-title":"Heart Rhythm."},{"key":"ref_230","doi-asserted-by":"crossref","first-page":"1247","DOI":"10.1001\/jamaophthalmol.2019.3278","article-title":"Efficacy and Safety of Retinal Gene Therapy Using Adeno-Associated Virus Vector for Patients with Choroideremia: A Randomized Clinical Trial","volume":"137","author":"Fischer","year":"2019","journal-title":"JAMA Ophthalmol."},{"key":"ref_231","doi-asserted-by":"crossref","unstructured":"Wang, J., Zhang, J., Yu, S., Li, H., Chen, S., Luo, J., Wang, H., Guan, Y., Zhang, H., and Yin, S. (2024). Gene replacement therapy in Bietti crystalline corneoretinal dystrophy: An open-label, single-arm, exploratory trial. Signal Transduct. Target. Ther., 9.","DOI":"10.1038\/s41392-024-01806-3"},{"key":"ref_232","doi-asserted-by":"crossref","unstructured":"Pechnikova, N.A., Poimenidou, M., Iliadis, I., Zafeiriou-Chatziefraimidou, M., Iaremenko, A.V., Yaremenko, T.V., Domvri, K., and Yaremenko, A.V. (2025). Pre-Clinical and Clinical Advances in Gene Therapy of X-Linked Retinitis Pigmentosa: Hope on the Horizon. J. Clin. Med., 14.","DOI":"10.3390\/jcm14030898"},{"key":"ref_233","doi-asserted-by":"crossref","unstructured":"Gehrke, M., Diedrichs-M\u00f6hring, M., Bogedein, J., B\u00fcning, H., Michalakis, S., and Wildner, G. (2022). Immunogenicity of Novel AAV Capsids for Retinal Gene Therapy. Cells, 11.","DOI":"10.3390\/cells11121881"},{"key":"ref_234","doi-asserted-by":"crossref","first-page":"7","DOI":"10.1007\/s40123-022-00602-y","article-title":"X-Linked Retinitis Pigmentosa Gene Therapy: Preclinical Aspects","volume":"12","author":"Mansouri","year":"2023","journal-title":"Ophthalmol. Ther."},{"key":"ref_235","doi-asserted-by":"crossref","first-page":"491","DOI":"10.59249\/HWID7537","article-title":"Ocular Gene Therapy: An Overview of Viral Vectors, Immune Responses, and Future Directions","volume":"97","author":"Banou","year":"2024","journal-title":"Yale J. Biol. Med."},{"key":"ref_236","doi-asserted-by":"crossref","first-page":"270","DOI":"10.1111\/ceo.13917","article-title":"Inherited retinal diseases: Therapeutics, clinical trials and end points\u2014A review","volume":"49","author":"Georgiou","year":"2021","journal-title":"Clin. Exper. Ophthalmol."},{"key":"ref_237","doi-asserted-by":"crossref","unstructured":"Merle, D.A., Hertens, L., Dimopoulos, S., Kohl, S., Van Haute, M., De Baere, E., De Bruyne, M., Janssens, B., R\u00fcther, K., and Huchzermeyer, C. (2025). Short-Term Outcomes of Pediatric Patients with Mild Autosomal Recessive RPE65\u2014Associated Retinal Dystrophy Treated with Voretigene Neparvovec. Trans. Vis. Sci. Technol., 14.","DOI":"10.1167\/tvst.14.8.8"},{"key":"ref_238","doi-asserted-by":"crossref","first-page":"1460","DOI":"10.1016\/j.ophtha.2021.03.031","article-title":"Durability of Voretigene Neparvovec for Biallelic RPE65-Mediated Inherited Retinal Disease","volume":"128","author":"Maguire","year":"2021","journal-title":"Ophthalmology"},{"key":"ref_239","doi-asserted-by":"crossref","first-page":"126","DOI":"10.1001\/jamaophthalmol.2024.5619","article-title":"Safety and Vision Outcomes Following Gene Therapy for Bietti Crystalline Dystrophy: A Nonrandomized Clinical Trial","volume":"143","author":"Chen","year":"2025","journal-title":"JAMA Ophthalmol."},{"key":"ref_240","doi-asserted-by":"crossref","first-page":"275","DOI":"10.1001\/jamaophthalmol.2022.6254","article-title":"Changes in Retinal Sensitivity Associated with Cotoretigene Toliparvovec in X-Linked Retinitis Pigmentosa with RPGR Gene Variations","volume":"141","author":"Liu","year":"2023","journal-title":"JAMA Ophthalmol."},{"key":"ref_241","doi-asserted-by":"crossref","first-page":"1083","DOI":"10.1016\/j.ophtha.2024.02.023","article-title":"Assessment of Visual Function with Cotoretigene Toliparvovec in X-Linked Retinitis Pigmentosa in the Randomized XIRIUS Phase 2\/3 Study","volume":"131","author":"Lam","year":"2024","journal-title":"Ophthalmology"},{"key":"ref_242","doi-asserted-by":"crossref","first-page":"849","DOI":"10.1016\/S0140-6736(17)31868-8","article-title":"Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65 -mediated inherited retinal dystrophy: A randomised, controlled, open-label, phase 3 trial","volume":"390","author":"Russell","year":"2017","journal-title":"Lancet"},{"key":"ref_243","doi-asserted-by":"crossref","first-page":"3","DOI":"10.1007\/s10633-023-09962-7","article-title":"ISCEV and IPS guideline for the full-field stimulus test (FST)","volume":"148","author":"Jolly","year":"2024","journal-title":"Doc. Ophthalmol."},{"key":"ref_244","doi-asserted-by":"crossref","unstructured":"Tuohy, G.P., and Megaw, R. (2021). A Systematic Review and Meta-Analyses of Interventional Clinical Trial Studies for Gene Therapies for the Inherited Retinal Degenerations (IRDs). Biomolecules, 11.","DOI":"10.3390\/biom11050760"},{"key":"ref_245","doi-asserted-by":"crossref","unstructured":"Yamamoto, M., Matsuyama, T., Maeda, T., Takagi, S., Motozawa, N., Sakai, D., Hirami, Y., Maeda, A., Kurimoto, Y., and Takahashi, M. (2023). Detailed Evaluation of Chromatic Pupillometry and Full-Field Stimulus Testing to Assess Ultralow Vision in Retinitis Pigmentosa. Ophthalmol. Sci., 3.","DOI":"10.1016\/j.xops.2023.100328"},{"key":"ref_246","doi-asserted-by":"crossref","unstructured":"Rukmini, A.V., Milea, D., and Gooley, J.J. (2019). Chromatic Pupillometry Methods for Assessing Photoreceptor Health in Retinal and Optic Nerve Diseases. Front. Neurol., 10.","DOI":"10.3389\/fneur.2019.00076"},{"key":"ref_247","doi-asserted-by":"crossref","first-page":"21","DOI":"10.1007\/s40123-023-00862-2","article-title":"Gene Therapy for Inherited Retinal Diseases: From Laboratory Bench to Patient Bedside and Beyond","volume":"13","author":"Brar","year":"2024","journal-title":"Ophthalmol. Ther."},{"key":"ref_248","doi-asserted-by":"crossref","unstructured":"Sobh, M., Lagali, P.S., Ghiasi, M., Montroy, J., Dollin, M., Hurley, B., Leonard, B.C., Dimopoulos, I., Lafreniere, M., and Fergusson, D.A. (2023). Safety and Efficacy of Adeno-Associated Viral Gene Therapy in Patients with Retinal Degeneration: A Systematic Review and Meta-Analysis. Trans. Vis. Sci. Technol., 12.","DOI":"10.1167\/tvst.12.11.24"},{"key":"ref_249","doi-asserted-by":"crossref","first-page":"354","DOI":"10.1038\/s41591-020-0763-1","article-title":"Retinal gene therapy in X-linked retinitis pigmentosa caused by mutations in RPGR: Results at 6 months in a first in human clinical trial","volume":"26","author":"Xue","year":"2020","journal-title":"Nat. Med."},{"key":"ref_250","doi-asserted-by":"crossref","unstructured":"Igoe, J.M., Lam, B.L., and Gregori, N.Z. (2024). Update on Clinical Trial Endpoints in Gene Therapy Trials for Inherited Retinal Diseases. J. Clin. Med., 13.","DOI":"10.3390\/jcm13185512"},{"key":"ref_251","doi-asserted-by":"crossref","first-page":"2069","DOI":"10.1007\/s40123-023-00724-x","article-title":"Development of Novel Patient-Reported Outcome (PRO) and Observer-Reported Outcome (ObsRO) Instruments in Retinitis Pigmentosa (RP) and Leber Congenital Amaurosis (LCA): ViSIO-PRO and ViSIO-ObsRO","volume":"12","author":"Audo","year":"2023","journal-title":"Ophthalmol. Ther."},{"key":"ref_252","doi-asserted-by":"crossref","first-page":"116","DOI":"10.1016\/j.ajo.2022.11.021","article-title":"Construct Validity of Inherited Retinal Disease-Specific Patient-Reported Outcome Measures","volume":"248","author":"Jayasundera","year":"2023","journal-title":"Am. J. Ophthalmol."},{"key":"ref_253","doi-asserted-by":"crossref","unstructured":"Gouveia, N., Karuntu, J., Almushattat, H., Silva, R., Boon, C., and Marques, J.P. (2026). Retinitis Pigmentosa GTPase regulator-Associated Retinal Degeneration: Integrating Patient-Reported Outcomes, Genetic, and Structural Biomarkers. Ophthalmol. Sci., 6.","DOI":"10.1016\/j.xops.2025.100915"},{"key":"ref_254","doi-asserted-by":"crossref","unstructured":"Bucher, K., Rodr\u00edguez-Bocanegra, E., Dauletbekov, D., and Fischer, M.D. (2021). Immune responses to retinal gene therapy using adeno-associated viral vectors\u2014Implications for treatment success and safety. Prog. Retin. Eye Res., 83.","DOI":"10.1016\/j.preteyeres.2020.100915"},{"key":"ref_255","doi-asserted-by":"crossref","first-page":"2504","DOI":"10.1038\/s41433-024-03065-6","article-title":"Voretigene neparvovec for inherited retinal dystrophy due to RPE65 mutations: A scoping review of eligibility and treatment challenges from clinical trials to real practice","volume":"38","author":"Testa","year":"2024","journal-title":"Eye"},{"key":"ref_256","doi-asserted-by":"crossref","unstructured":"Marie, M., Churet, L., Gautron, A.-S., Farjo, R., Mizuyoshi, K., Stevenson, V., Khabou, H., L\u00e9veillard, T., Sahel, J.-A., and Lorget, F. (2025). Preclinical safety and biodistribution of SPVN06, a novel gene- and mutation-independent gene therapy for rod-cone dystrophies. Gene Ther., 1\u201314.","DOI":"10.1038\/s41434-025-00556-3"},{"key":"ref_257","doi-asserted-by":"crossref","unstructured":"Zhao, Q., Wei, L., and Chen, Y. (2025). From bench to bedside: Developing CRISPR\/Cas-based therapy for ocular diseases. Pharmacol. Res., 213.","DOI":"10.1016\/j.phrs.2025.107638"},{"key":"ref_258","doi-asserted-by":"crossref","first-page":"445","DOI":"10.1136\/bjophthalmol-2020-318483","article-title":"Leber congenital amaurosis\/early-onset severe retinal dystrophy: Current management and clinical trials","volume":"106","author":"Georgiou","year":"2022","journal-title":"Br. J. Ophthalmol."},{"key":"ref_259","doi-asserted-by":"crossref","unstructured":"Jia, R., Chen, S., Li, W., Zhang, J., Qu, B., Qiao, J., Meng, X., Yu, S., Liu, X., and Xu, B. (2025). Unravelling CYP4V2: Clinical features, genetic insights, pathogenic mechanisms and therapeutic strategies in Bietti crystalline corneoretinal dystrophy. Prog. Retin. Eye Res., 107.","DOI":"10.1016\/j.preteyeres.2025.101377"},{"key":"ref_260","doi-asserted-by":"crossref","unstructured":"Pierce, E.A., Ashimatey, B.S., Jayasundera, T., Hoyng, C., Lam, B.L., Lorenz, B., Kim, K., Rashid, A., Myers, R., and Pennesi, M.E. (2024). Twelve-month Natural History Study of Centrosomal Protein 290 (CEP290)-associated Inherited Retinal Degeneration. Ophthalmol. Sci., 4.","DOI":"10.1016\/j.xops.2024.100483"},{"key":"ref_261","doi-asserted-by":"crossref","unstructured":"Kharisova, C.B., Kitaeva, K.V., Solovyeva, V.V., Sufianov, A.A., Sufianova, G.Z., Akhmetshin, R.F., Bulgar, S.N., and Rizvanov, A.A. (2025). Looking to the Future of Viral Vectors in Ocular Gene Therapy: Clinical Review. Biomedicines, 13.","DOI":"10.3390\/biomedicines13020365"},{"key":"ref_262","doi-asserted-by":"crossref","first-page":"404","DOI":"10.1016\/j.omtm.2022.10.014","article-title":"The seroprevalence of neutralizing antibodies against the adeno-associated virus capsids in Japanese hemophiliacs","volume":"27","author":"Kashiwakura","year":"2022","journal-title":"Mol. Ther. Methods Clin. Dev."},{"key":"ref_263","doi-asserted-by":"crossref","first-page":"179","DOI":"10.2147\/TACG.S230720","article-title":"Voretigene Neparvovec and Gene Therapy for Leber\u2019s Congenital Amaurosis: Review of Evidence to Date","volume":"13","author":"Padhy","year":"2020","journal-title":"Appl. Clin. Genet."},{"key":"ref_264","doi-asserted-by":"crossref","unstructured":"Shamshad, A., Kang, C., Jenny, L.A., Persad-Paisley, E.M., and Tsang, S.H. (2023). Translatability barriers between preclinical and clinical trials of AAV gene therapy in inherited retinal diseases. Vis. Res., 210.","DOI":"10.1016\/j.visres.2023.108258"},{"key":"ref_265","doi-asserted-by":"crossref","first-page":"1854","DOI":"10.1016\/j.ymthe.2017.05.005","article-title":"Codon-Optimized RPGR Improves Stability and Efficacy of AAV8 Gene Therapy in Two Mouse Models of X-Linked Retinitis Pigmentosa","volume":"25","author":"Fischer","year":"2017","journal-title":"Mol. Ther."},{"key":"ref_266","doi-asserted-by":"crossref","first-page":"33","DOI":"10.1038\/s41433-023-02636-3","article-title":"Full-field stimulus threshold testing: A scoping review of current practice","volume":"38","author":"Shi","year":"2024","journal-title":"Eye"},{"key":"ref_267","first-page":"291","article-title":"Parental Permission and Child Assent in Research on Children","volume":"86","author":"Nelson","year":"2013","journal-title":"Yale J. Biol. Med."},{"key":"ref_268","doi-asserted-by":"crossref","first-page":"743","DOI":"10.1089\/hum.2019.337","article-title":"Dose Range Finding Studies with Two RPGR Transgenes in a Canine Model of X-Linked Retinitis Pigmentosa Treated with Subretinal Gene Therapy","volume":"31","author":"Song","year":"2020","journal-title":"Hum. Gene Ther."},{"key":"ref_269","doi-asserted-by":"crossref","unstructured":"Alipanahi, R., Safari, L., and Khanteymoori, A. (2024). DTMP-prime: A deep transformer-based model for predicting prime editing efficiency and PegRNA activity. Mol. Ther. Nucleic Acids, 35.","DOI":"10.1016\/j.omtn.2024.102370"},{"key":"ref_270","doi-asserted-by":"crossref","unstructured":"Mathis, N., Marquart, K.F., Allam, A., Krauthammer, M., and Schwank, G. (2025). Systematic pegRNA design with PRIDICT2.0 and ePRIDICT for efficient prime editing. Nat. Protoc., 1\u201321.","DOI":"10.1038\/s41596-025-01244-7"},{"key":"ref_271","doi-asserted-by":"crossref","first-page":"W121","DOI":"10.1093\/nar\/gkae304","article-title":"SynDesign: Web-based prime editing guide RNA design and evaluation tool for saturation genome editing","volume":"52","author":"Park","year":"2024","journal-title":"Nucleic Acids Res."},{"key":"ref_272","doi-asserted-by":"crossref","unstructured":"Zhang, W., Petri, K., Ma, J., Lee, H., Tsai, C.-L., Joung, J.K., and Yeh, J.-R.J. (2024). Enhancing CRISPR prime editing by reducing misfolded pegRNA interactions. eLife, 12.","DOI":"10.7554\/eLife.90948.2"},{"key":"ref_273","doi-asserted-by":"crossref","first-page":"3182","DOI":"10.1016\/j.cell.2023.05.041","article-title":"Discovery of deaminase functions by structure-based protein clustering","volume":"186","author":"Huang","year":"2023","journal-title":"Cell"},{"key":"ref_274","doi-asserted-by":"crossref","first-page":"1139","DOI":"10.1126\/science.aaw2900","article-title":"Comprehensive AAV capsid fitness landscape reveals a viral gene and enables machine-guided design","volume":"366","author":"Ogden","year":"2019","journal-title":"Science"},{"key":"ref_275","doi-asserted-by":"crossref","first-page":"276","DOI":"10.1016\/j.omtm.2020.11.017","article-title":"Applying machine learning to predict viral assembly for adeno-associated virus capsid libraries","volume":"20","author":"Marques","year":"2021","journal-title":"Mol. Ther. Methods Clin. Dev."},{"key":"ref_276","doi-asserted-by":"crossref","first-page":"691","DOI":"10.1038\/s41587-020-00793-4","article-title":"Deep diversification of an AAV capsid protein by machine learning","volume":"39","author":"Bryant","year":"2021","journal-title":"Nat. Biotechnol."},{"key":"ref_277","doi-asserted-by":"crossref","first-page":"1687","DOI":"10.1016\/j.ymthe.2024.04.003","article-title":"Fast, accurate ranking of engineered proteins by target-binding propensity using structure modeling","volume":"32","author":"Ding","year":"2024","journal-title":"Mol. Ther."},{"key":"ref_278","doi-asserted-by":"crossref","unstructured":"Eid, F.-E., Chen, A.T., Chan, K.Y., Huang, Q., Zheng, Q., Tobey, I.G., Pacouret, S., Brauer, P.P., Keyes, C., and Powell, M. (2024). Systematic multi-trait AAV capsid engineering for efficient gene delivery. Nat. Commun., 15.","DOI":"10.1038\/s41467-024-50555-y"},{"key":"ref_279","doi-asserted-by":"crossref","first-page":"1261","DOI":"10.1038\/s42256-023-00739-w","article-title":"Design of prime-editing guide RNAs with deep transfer learning","volume":"5","author":"Liu","year":"2023","journal-title":"Nat. Mach. Intell."},{"key":"ref_280","doi-asserted-by":"crossref","unstructured":"Saraswat, P., and Ranjan, R. (2025). Unlocking the potential of CRISPR tools and databases for precision genome editing. Front. Plant Sci., 16.","DOI":"10.3389\/fpls.2025.1563711"},{"key":"ref_281","doi-asserted-by":"crossref","unstructured":"Sanvicente-Garc\u00eda, M., Garc\u00eda-Valiente, A., Jouide, S., Jaraba-Wallace, J., Bautista, E., Escobosa, M., S\u00e1nchez-Mej\u00edas, A., and G\u00fcell, M. (2023). CRISPR-Analytics (CRISPR-A): A platform for precise analytics and simulations for gene editing. PLoS Comput. Biol., 19.","DOI":"10.15252\/rc.2023944536"},{"key":"ref_282","doi-asserted-by":"crossref","first-page":"990","DOI":"10.1101\/gr.200535.115","article-title":"Basset: Learning the regulatory code of the accessible genome with deep convolutional neural networks","volume":"26","author":"Kelley","year":"2016","journal-title":"Genome Res."},{"key":"ref_283","doi-asserted-by":"crossref","unstructured":"Yuan, T., Wu, L., Li, S., Zheng, J., Li, N., Xiao, X., Zhang, H., Fei, T., Xie, L., and Zuo, Z. (2024). Deep learning models incorporating endogenous factors beyond DNA sequences improve the prediction accuracy of base editing outcomes. Cell Discov., 10.","DOI":"10.1038\/s41421-023-00624-1"},{"key":"ref_284","first-page":"42","article-title":"AI-Driven Bioinformatics: Transforming Genomics, Gene Editing, and Personalized Medicine","volume":"5","author":"Pasupuleti","year":"2025","journal-title":"Int. J. Acad. Ind. Res. Innov."},{"key":"ref_285","doi-asserted-by":"crossref","unstructured":"Pushkaran, A.C., and Arabi, A.A. (2024). From understanding diseases to drug design: Can artificial intelligence bridge the gap?. Artif. Intell. Rev., 57.","DOI":"10.1007\/s10462-024-10714-5"}],"container-title":["Pharmaceutics"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/www.mdpi.com\/1999-4923\/17\/11\/1405\/pdf","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2025,11,1]],"date-time":"2025-11-01T05:28:12Z","timestamp":1761974892000},"score":1,"resource":{"primary":{"URL":"https:\/\/www.mdpi.com\/1999-4923\/17\/11\/1405"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2025,10,30]]},"references-count":285,"journal-issue":{"issue":"11","published-online":{"date-parts":[[2025,11]]}},"alternative-id":["pharmaceutics17111405"],"URL":"https:\/\/doi.org\/10.3390\/pharmaceutics17111405","relation":{},"ISSN":["1999-4923"],"issn-type":[{"value":"1999-4923","type":"electronic"}],"subject":[],"published":{"date-parts":[[2025,10,30]]}}}