{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,29]],"date-time":"2026-09-29T20:41:41Z","timestamp":1790714501444,"version":"4.1.0"},"reference-count":192,"publisher":"Elsevier BV","issue":"3","license":[{"start":{"date-parts":[[2025,9,1]],"date-time":"2025-09-01T00:00:00Z","timestamp":1756684800000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2025,9,1]],"date-time":"2025-09-01T00:00:00Z","timestamp":1756684800000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/legal\/tdmrep-license"},{"start":{"date-parts":[[2025,7,31]],"date-time":"2025-07-31T00:00:00Z","timestamp":1753920000000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"funder":[{"DOI":"10.13039\/501100010679","name":"Pasteur Institute of Iran","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100010679","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["cell.com","elsevier.com","sciencedirect.com"],"crossmark-restriction":true},"short-container-title":["Molecular Therapy Nucleic Acids"],"published-print":{"date-parts":[[2025,9]]},"DOI":"10.1016\/j.omtn.2025.102666","type":"journal-article","created":{"date-parts":[[2025,8,5]],"date-time":"2025-08-05T22:37:11Z","timestamp":1754433431000},"page":"102666","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":37,"title":["Advancing gene editing therapeutics: Clinical trials and innovative delivery systems across diverse diseases"],"prefix":"10.1016","volume":"36","author":[{"given":"Mozhghan","family":"Raigani","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Zohre","family":"Eftekhari","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ahmad","family":"Adeli","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-5832-1822","authenticated-orcid":false,"given":"Fatemeh","family":"Kazemi-Lomedasht","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"78","reference":[{"key":"10.1016\/j.omtn.2025.102666_bib1","doi-asserted-by":"crossref","first-page":"5429","DOI":"10.1128\/jb.169.12.5429-5433.1987","article-title":"Nucleotide sequence of the iap gene, responsible for alkaline phosphatase isozyme conversion in Escherichia coli, and identification of the gene product","volume":"169","author":"Ishino","year":"1987","journal-title":"J. Bacteriol."},{"key":"10.1016\/j.omtn.2025.102666_bib2","doi-asserted-by":"crossref","first-page":"181","DOI":"10.1038\/nrmicro1793","article-title":"CRISPR\u2014a widespread system that provides acquired resistance against phages in bacteria and archaea","volume":"6","author":"Sorek","year":"2008","journal-title":"Nat. Rev. Microbiol."},{"key":"10.1016\/j.omtn.2025.102666_bib3","doi-asserted-by":"crossref","first-page":"234","DOI":"10.1016\/j.molcel.2014.03.011","article-title":"CRISPR-Cas systems: prokaryotes upgrade to adaptive immunity","volume":"54","author":"Barrangou","year":"2014","journal-title":"Mol. Cell"},{"key":"10.1016\/j.omtn.2025.102666_bib4","doi-asserted-by":"crossref","first-page":"1565","DOI":"10.1046\/j.1365-2958.2002.02839.x","article-title":"Identification of genes that are associated with DNA repeats in prokaryotes","volume":"43","author":"Jansen","year":"2002","journal-title":"Mol. Microbiol."},{"key":"10.1016\/j.omtn.2025.102666_bib5","doi-asserted-by":"crossref","first-page":"1495","DOI":"10.1111\/j.1365-2958.2010.07073.x","article-title":"Identification and characterization of E. coli CRISPR-cas promoters and their silencing by H-NS","volume":"75","author":"Pul","year":"2010","journal-title":"Mol. Microbiol."},{"key":"10.1016\/j.omtn.2025.102666_bib6","doi-asserted-by":"crossref","first-page":"718","DOI":"10.1007\/s00239-005-0223-z","article-title":"The repetitive DNA elements called CRISPRs and their associated genes: evidence of horizontal transfer among prokaryotes","volume":"62","author":"Godde","year":"2006","journal-title":"J. Mol. Evol."},{"key":"10.1016\/j.omtn.2025.102666_bib7","doi-asserted-by":"crossref","DOI":"10.1126\/science.1258096","article-title":"The new frontier of genome engineering with CRISPR-Cas9","volume":"346","author":"Doudna","year":"2014","journal-title":"Science"},{"key":"10.1016\/j.omtn.2025.102666_bib8","doi-asserted-by":"crossref","first-page":"253","DOI":"10.1631\/jzus.B2100009","article-title":"CRISPR\/Cas: a Nobel Prize award-winning precise genome editing technology for gene therapy and crop improvement","volume":"22","author":"Li","year":"2021","journal-title":"J. Zhejiang Univ. Sci. B"},{"key":"10.1016\/j.omtn.2025.102666_bib9","doi-asserted-by":"crossref","first-page":"108","DOI":"10.1016\/j.gpb.2022.02.006","article-title":"Computational tools and resources for CRISPR\/Cas genome editing","volume":"21","author":"Li","year":"2023","journal-title":"Genom. Proteom. Bioinform."},{"key":"10.1016\/j.omtn.2025.102666_bib10","doi-asserted-by":"crossref","first-page":"31","DOI":"10.1186\/s13048-018-0406-z","article-title":"Role of BMI1 in epithelial ovarian cancer: investigated via the CRISPR\/Cas9 system and RNA sequencing","volume":"11","author":"Zhao","year":"2018","journal-title":"J. Ovarian Res."},{"key":"10.1016\/j.omtn.2025.102666_bib11","doi-asserted-by":"crossref","first-page":"234","DOI":"10.1186\/s12870-020-02385-5","article-title":"Precision genome editing in plants: state-of-the-art in CRISPR\/Cas9-based genome engineering","volume":"20","author":"Wada","year":"2020","journal-title":"BMC Plant Biol."},{"key":"10.1016\/j.omtn.2025.102666_bib12","doi-asserted-by":"crossref","first-page":"963","DOI":"10.1016\/j.jmb.2015.10.014","article-title":"Origins of programmable nucleases for genome engineering","volume":"428","author":"Chandrasegaran","year":"2016","journal-title":"J. Mol. Biol."},{"key":"10.1016\/j.omtn.2025.102666_bib13","doi-asserted-by":"crossref","first-page":"2380","DOI":"10.1002\/jcp.25367","article-title":"Programmable site-specific nucleases for targeted genome engineering in higher eukaryotes","volume":"231","author":"Govindan","year":"2016","journal-title":"J. Cell. Physiol."},{"key":"10.1016\/j.omtn.2025.102666_bib14","doi-asserted-by":"crossref","first-page":"809","DOI":"10.1126\/science.2028256","article-title":"Zinc finger-DNA recognition: crystal structure of a Zif268-DNA complex at 2.1 \u00c5","volume":"252","author":"Pavletich","year":"1991","journal-title":"Science"},{"key":"10.1016\/j.omtn.2025.102666_bib15","doi-asserted-by":"crossref","first-page":"183","DOI":"10.1358\/dof.2012.37.3.1779022","article-title":"Zinc finger nucleases: tailor-made for gene therapy","volume":"37","author":"Chou","year":"2012","journal-title":"Drugs Future"},{"key":"10.1016\/j.omtn.2025.102666_bib16","doi-asserted-by":"crossref","first-page":"10570","DOI":"10.1073\/pnas.95.18.10570","article-title":"Fok I dimerization is required for DNA cleavage","volume":"95","author":"Bitinaite","year":"1998","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"10.1016\/j.omtn.2025.102666_bib17","doi-asserted-by":"crossref","first-page":"5560","DOI":"10.1093\/nar\/gks179","article-title":"Engineered zinc finger nickases induce homology-directed repair with reduced mutagenic effects","volume":"40","author":"Ramirez","year":"2012","journal-title":"Nucleic Acids Res."},{"key":"10.1016\/j.omtn.2025.102666_bib18","doi-asserted-by":"crossref","first-page":"773","DOI":"10.1534\/genetics.111.131433","article-title":"Genome engineering with zinc-finger nucleases","volume":"188","author":"Carroll","year":"2011","journal-title":"Genetics"},{"key":"10.1016\/j.omtn.2025.102666_bib19","doi-asserted-by":"crossref","first-page":"1262","DOI":"10.1016\/j.cell.2014.05.010","article-title":"Development and applications of CRISPR-Cas9 for genome engineering","volume":"157","author":"Hsu","year":"2014","journal-title":"Cell"},{"key":"10.1016\/j.omtn.2025.102666_bib20","doi-asserted-by":"crossref","first-page":"3819","DOI":"10.1007\/s00018-015-1956-5","article-title":"Recent developments and clinical studies utilizing engineered zinc finger nuclease technology","volume":"72","author":"Jo","year":"2015","journal-title":"Cell. Mol. Life Sci."},{"key":"10.1016\/j.omtn.2025.102666_bib21","doi-asserted-by":"crossref","first-page":"S33","DOI":"10.1016\/S1525-0016(16)33682-6","article-title":"77. Clinical Scale Zinc Finger Nuclease (ZFN)-Driven Gene-Editing of PD-1 in Tumor Infiltrating Lymphocytes (TIL) for the Potential Treatment of Metastatic Melanoma","volume":"23","author":"Beane","year":"2015","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtn.2025.102666_bib22","series-title":"Characteristics of Gene Editing Clinical Trials from Public Trial Registries","author":"Zlatin","year":"2020"},{"key":"10.1016\/j.omtn.2025.102666_bib23","doi-asserted-by":"crossref","first-page":"33","DOI":"10.18609\/cgti.2017.005","article-title":"Recent advances in the use of ZFN-mediated gene editing for human gene therapy","volume":"3","author":"Chandrasegaran","year":"2017","journal-title":"Cell Gene Ther. Insights"},{"key":"10.1016\/j.omtn.2025.102666_bib24","doi-asserted-by":"crossref","first-page":"245","DOI":"10.1089\/hum.2012.172","article-title":"Efficient clinical scale gene modification via zinc finger nuclease\u2013targeted disruption of the HIV co-receptor CCR5","volume":"24","author":"Maier","year":"2013","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.omtn.2025.102666_bib25","doi-asserted-by":"crossref","first-page":"e198","DOI":"10.1038\/mtna.2014.52","article-title":"CCR5 gene editing of resting CD4+ T cells by transient ZFN expression from HIV envelope pseudotyped nonintegrating lentivirus confers HIV-1 resistance in humanized mice","volume":"3","author":"Yi","year":"2014","journal-title":"Mol. Ther. Nucleic Acids"},{"key":"10.1016\/j.omtn.2025.102666_bib26","doi-asserted-by":"crossref","first-page":"16067","DOI":"10.1038\/mtm.2016.67","article-title":"Preclinical development and qualification of ZFN-mediated CCR5 disruption in human hematopoietic stem\/progenitor cells","volume":"3","author":"DiGiusto","year":"2016","journal-title":"Mol. Ther. Methods Clin. Dev."},{"key":"10.1016\/j.omtn.2025.102666_bib27","doi-asserted-by":"crossref","first-page":"839","DOI":"10.1038\/nbt.1663","article-title":"Human hematopoietic stem\/progenitor cells modified by zinc-finger nucleases targeted to CCR5 control HIV-1 in vivo","volume":"28","author":"Holt","year":"2010","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.omtn.2025.102666_bib28","doi-asserted-by":"crossref","first-page":"1755","DOI":"10.1093\/jac\/dku072","article-title":"Gene editing using a zinc-finger nuclease mimicking the CCR5\u039432 mutation induces resistance to CCR5-using HIV-1","volume":"69","author":"Badia","year":"2014","journal-title":"J. Antimicrob. Chemother."},{"key":"10.1016\/j.omtn.2025.102666_bib29","doi-asserted-by":"crossref","first-page":"3587","DOI":"10.1016\/j.ymthe.2022.10.010","article-title":"First-in-human in vivo genome editing via AAV-zinc-finger nucleases for mucopolysaccharidosis I\/II and hemophilia B","volume":"30","author":"Harmatz","year":"2022","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtn.2025.102666_bib30","doi-asserted-by":"crossref","first-page":"3283","DOI":"10.1182\/blood-2013-04-497354","article-title":"Robust ZFN-mediated genome editing in adult hemophilic mice","volume":"122","author":"Anguela","year":"2013","journal-title":"Blood"},{"key":"10.1016\/j.omtn.2025.102666_bib31","series-title":"Robust Factor IX Expression Following ZFN-Mediated Genome Editing in an Adult Mouse Model of Hemophilia B","author":"Anguela","year":"2011"},{"key":"10.1016\/j.omtn.2025.102666_bib32","doi-asserted-by":"crossref","first-page":"636","DOI":"10.1038\/nrg2842","article-title":"Genome editing with engineered zinc finger nucleases","volume":"11","author":"Urnov","year":"2010","journal-title":"Nat. Rev. Genet."},{"key":"10.1016\/j.omtn.2025.102666_bib33","doi-asserted-by":"crossref","first-page":"397","DOI":"10.1016\/j.tibtech.2013.04.004","article-title":"ZFN, TALEN, and CRISPR\/Cas-based methods for genome engineering","volume":"31","author":"Gaj","year":"2013","journal-title":"Trends Biotechnol."},{"key":"10.1016\/j.omtn.2025.102666_bib34","doi-asserted-by":"crossref","first-page":"143","DOI":"10.1038\/nbt.1755","article-title":"A TALE nuclease architecture for efficient genome editing","volume":"29","author":"Miller","year":"2011","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.omtn.2025.102666_bib35","doi-asserted-by":"crossref","first-page":"49","DOI":"10.1038\/nrm3486","article-title":"TALENs: a widely applicable technology for targeted genome editing","volume":"14","author":"Joung","year":"2013","journal-title":"Nat. Rev. Mol. Cell Biol."},{"key":"10.1016\/j.omtn.2025.102666_bib36","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":"10.1016\/j.omtn.2025.102666_bib37","doi-asserted-by":"crossref","DOI":"10.3389\/fgeed.2021.618406","article-title":"Base and prime editing technologies for blood disorders","volume":"3","author":"Antoniou","year":"2021","journal-title":"Front. Genome Ed."},{"key":"10.1016\/j.omtn.2025.102666_bib38","doi-asserted-by":"crossref","first-page":"483","DOI":"10.1042\/ETLS20190088","article-title":"Base editors: modular tools for the introduction of point mutations in living cells","volume":"3","author":"Evanoff","year":"2019","journal-title":"Emerg. Top. Life Sci."},{"key":"10.1016\/j.omtn.2025.102666_bib39","doi-asserted-by":"crossref","first-page":"75","DOI":"10.1515\/mr-2022-0044","article-title":"Precise genome editing with base editors","volume":"3","author":"Liu","year":"2023","journal-title":"Med. Rev."},{"key":"10.1016\/j.omtn.2025.102666_bib40","doi-asserted-by":"crossref","first-page":"270","DOI":"10.1016\/j.jcyt.2022.11.013","article-title":"CRISPR-Cas9 base editors and their current role in human therapeutics","volume":"25","author":"Lahr","year":"2023","journal-title":"Cytotherapy"},{"key":"10.1016\/j.omtn.2025.102666_bib41","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":"10.1016\/j.omtn.2025.102666_bib42","doi-asserted-by":"crossref","first-page":"939","DOI":"10.1038\/s41591-018-0050-6","article-title":"p53 inhibits CRISPR\u2013Cas9 engineering in human pluripotent stem cells","volume":"24","author":"Ihry","year":"2018","journal-title":"Nat. Med."},{"key":"10.1016\/j.omtn.2025.102666_bib43","doi-asserted-by":"crossref","first-page":"289","DOI":"10.1126\/science.aav9973","article-title":"Cytosine base editor generates substantial off-target single-nucleotide variants in mouse embryos","volume":"364","author":"Zuo","year":"2019","journal-title":"Science"},{"key":"10.1016\/j.omtn.2025.102666_bib44","doi-asserted-by":"crossref","first-page":"536","DOI":"10.1038\/nbt.4148","article-title":"Adenine base editing in mouse embryos and an adult mouse model of Duchenne muscular dystrophy","volume":"36","author":"Ryu","year":"2018","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.omtn.2025.102666_bib45","doi-asserted-by":"crossref","first-page":"535","DOI":"10.1038\/s41591-020-0790-y","article-title":"Therapeutic base editing of human hematopoietic stem cells","volume":"26","author":"Zeng","year":"2020","journal-title":"Nat. Med."},{"key":"10.1016\/j.omtn.2025.102666_bib46","doi-asserted-by":"crossref","first-page":"295","DOI":"10.1038\/s41586-021-03609-w","article-title":"Base editing of haematopoietic stem cells rescues sickle cell disease in mice","volume":"595","author":"Newby","year":"2021","journal-title":"Nature"},{"key":"10.1016\/j.omtn.2025.102666_bib47","doi-asserted-by":"crossref","first-page":"1830","DOI":"10.1038\/s41467-022-29490-3","article-title":"In vivo base editing rescues cone photoreceptors in a mouse model of early-onset inherited retinal degeneration","volume":"13","author":"Choi","year":"2022","journal-title":"Nat. Commun."},{"key":"10.1016\/j.omtn.2025.102666_bib48","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":"10.1016\/j.omtn.2025.102666_bib49","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":"10.1016\/j.omtn.2025.102666_bib50","doi-asserted-by":"crossref","first-page":"797","DOI":"10.1038\/nbt0817-797d","article-title":"Erratum: genome-wide target specificities of CRISPR RNA-guided programmable deaminases","volume":"35","author":"Kim","year":"2017","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.omtn.2025.102666_bib51","doi-asserted-by":"crossref","first-page":"475","DOI":"10.1038\/nbt.3852","article-title":"Genome-wide target specificities of CRISPR RNA-guided programmable deaminases","volume":"35","author":"Kim","year":"2017","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.omtn.2025.102666_bib52","doi-asserted-by":"crossref","first-page":"433","DOI":"10.1038\/s41586-019-1161-z","article-title":"Transcriptome-wide off-target RNA editing induced by CRISPR-guided DNA base editors","volume":"569","author":"Gr\u00fcnewald","year":"2019","journal-title":"Nature"},{"key":"10.1016\/j.omtn.2025.102666_bib53","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":"10.1016\/j.omtn.2025.102666_bib54","doi-asserted-by":"crossref","first-page":"513","DOI":"10.1073\/pnas.1821893117","article-title":"CRISPR-mediated modeling and functional validation of candidate tumor suppressor genes in small cell lung cancer","volume":"117","author":"Ng","year":"2020","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"10.1016\/j.omtn.2025.102666_bib55","doi-asserted-by":"crossref","first-page":"836","DOI":"10.1016\/j.cell.2014.01.027","article-title":"Generation of gene-modified cynomolgus monkey via Cas9\/RNA-mediated gene targeting in one-cell embryos","volume":"156","author":"Niu","year":"2014","journal-title":"Cell"},{"key":"10.1016\/j.omtn.2025.102666_bib56","doi-asserted-by":"crossref","first-page":"759","DOI":"10.1016\/j.cell.2015.09.038","article-title":"Cpf1 is a single RNA-guided endonuclease of a class 2 CRISPR-Cas system","volume":"163","author":"Zetsche","year":"2015","journal-title":"Cell"},{"key":"10.1016\/j.omtn.2025.102666_bib57","doi-asserted-by":"crossref","first-page":"1387","DOI":"10.3389\/fonc.2020.01387","article-title":"CRISPR gene therapy: applications, limitations, and implications for the future","volume":"10","author":"Uddin","year":"2020","journal-title":"Front. Oncol."},{"key":"10.1016\/j.omtn.2025.102666_bib58","doi-asserted-by":"crossref","first-page":"1555","DOI":"10.1021\/acs.accounts.9b00106","article-title":"Delivery Aspects of CRISPR\/Cas for in Vivo Genome Editing","volume":"52","author":"Wilbie","year":"2019","journal-title":"Acc. Chem. Res."},{"key":"10.1016\/j.omtn.2025.102666_bib59","doi-asserted-by":"crossref","first-page":"1234","DOI":"10.1080\/10717544.2018.1474964","article-title":"Delivering CRISPR: a review of the challenges and approaches","volume":"25","author":"Lino","year":"2018","journal-title":"Drug Deliv."},{"key":"10.1016\/j.omtn.2025.102666_bib60","article-title":"Exploring the Potential and Challenges of CRISPR Delivery and Therapeutics for Genetic Disease Treatment","volume":"34","author":"Yang","year":"2024","journal-title":"Adv. Funct. Mater."},{"key":"10.1016\/j.omtn.2025.102666_bib61","doi-asserted-by":"crossref","DOI":"10.1002\/jex2.111","article-title":"CRISPR delivery with extracellular vesicles: Promises and challenges","volume":"2","author":"Berggreen","year":"2023","journal-title":"J. Extracell. Biol."},{"key":"10.1016\/j.omtn.2025.102666_bib62","doi-asserted-by":"crossref","first-page":"217","DOI":"10.1007\/s13534-021-00199-4","article-title":"Challenges in delivery systems for CRISPR-based genome editing and opportunities of nanomedicine","volume":"11","author":"Sioson","year":"2021","journal-title":"Biomed. Eng. Lett."},{"key":"10.1016\/j.omtn.2025.102666_bib63","article-title":"CRISPR\/Cas9 systems: Delivery technologies and biomedical applications","volume":"18","author":"Du","year":"2023","journal-title":"Asian J. Pharm. Sci."},{"key":"10.1016\/j.omtn.2025.102666_bib64","article-title":"Electroporation enables the efficient mRNA delivery into the mouse zygotes and facilitates CRISPR\/Cas9-based genome editing","volume":"5","author":"Hashimoto","year":"2015","journal-title":"Sci. Rep."},{"key":"10.1016\/j.omtn.2025.102666_bib65","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":"10.1016\/j.omtn.2025.102666_bib66","doi-asserted-by":"crossref","first-page":"341","DOI":"10.1038\/nrg2988","article-title":"Therapeutic in vivo gene transfer for genetic disease using AAV: progress and challenges","volume":"12","author":"Mingozzi","year":"2011","journal-title":"Nat. Rev. Genet."},{"key":"10.1016\/j.omtn.2025.102666_bib67","doi-asserted-by":"crossref","first-page":"583","DOI":"10.1128\/CMR.00008-08","article-title":"Gene therapy using adeno-associated virus vectors","volume":"21","author":"Daya","year":"2008","journal-title":"Clin. Microbiol. Rev."},{"key":"10.1016\/j.omtn.2025.102666_bib68","doi-asserted-by":"crossref","first-page":"421","DOI":"10.2174\/1566523213666131125095046","article-title":"Adenovirus vectors for gene therapy, vaccination and cancer gene therapy","volume":"13","author":"Wold","year":"2013","journal-title":"Curr. Gene Ther."},{"key":"10.1016\/j.omtn.2025.102666_bib69","doi-asserted-by":"crossref","first-page":"351","DOI":"10.1038\/nature15818","article-title":"Gene therapy returns to centre stage","volume":"526","author":"Naldini","year":"2015","journal-title":"Nature"},{"key":"10.1016\/j.omtn.2025.102666_bib70","doi-asserted-by":"crossref","first-page":"355","DOI":"10.1007\/s12033-020-00258-8","article-title":"Application of Various Delivery Methods for CRISPR\/dCas9","volume":"62","author":"Liu","year":"2020","journal-title":"Mol. Biotechnol."},{"key":"10.1016\/j.omtn.2025.102666_bib71","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":"10.1016\/j.omtn.2025.102666_bib72","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":"10.1016\/j.omtn.2025.102666_bib73","doi-asserted-by":"crossref","DOI":"10.3390\/biom10060839","article-title":"Recent Advances in CRISPR\/Cas9 Delivery Strategies","volume":"10","author":"Yip","year":"2020","journal-title":"Biomolecules"},{"key":"10.1016\/j.omtn.2025.102666_bib74","doi-asserted-by":"crossref","first-page":"352","DOI":"10.1016\/j.ymthe.2019.12.009","article-title":"Retroviral Insertional Mutagenesis in Humans: Evidence for Four Genetic Mechanisms Promoting Expansion of Cell Clones","volume":"28","author":"Bushman","year":"2020","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtn.2025.102666_bib75","doi-asserted-by":"crossref","DOI":"10.1038\/s41598-017-17180-w","article-title":"CRISPR\/Cas9 delivery with one single adenoviral vector devoid of all viral genes","volume":"7","author":"Ehrke-Schulz","year":"2017","journal-title":"Sci. Rep."},{"key":"10.1016\/j.omtn.2025.102666_bib76","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":"10.1016\/j.omtn.2025.102666_bib77","doi-asserted-by":"crossref","DOI":"10.1016\/j.cellsig.2020.109577","article-title":"CRISPR system: Discovery, development and off-target detection","volume":"70","author":"Chen","year":"2020","journal-title":"Cell. Signal."},{"key":"10.1016\/j.omtn.2025.102666_bib78","doi-asserted-by":"crossref","first-page":"4098","DOI":"10.1038\/s41467-018-06621-3","article-title":"Antigen-selective modulation of AAV immunogenicity with tolerogenic rapamycin nanoparticles enables successful vector re-administration","volume":"9","author":"Meliani","year":"2018","journal-title":"Nat. Commun."},{"key":"10.1016\/j.omtn.2025.102666_bib79","doi-asserted-by":"crossref","DOI":"10.1126\/sciadv.abc9450","article-title":"CRISPR-Cas9 genome editing using targeted lipid nanoparticles for cancer therapy","volume":"6","author":"Rosenblum","year":"2020","journal-title":"Sci. Adv."},{"key":"10.1016\/j.omtn.2025.102666_bib80","doi-asserted-by":"crossref","first-page":"e441","DOI":"10.1038\/am.2017.185","article-title":"Lipid nanoparticle-mediated efficient delivery of CRISPR\/Cas9 for tumor therapy","volume":"9","author":"Zhang","year":"2017","journal-title":"NPG Asia Mater."},{"key":"10.1016\/j.omtn.2025.102666_bib81","doi-asserted-by":"crossref","first-page":"175","DOI":"10.1186\/s12951-024-02427-2","article-title":"Finely tuned ionizable lipid nanoparticles for CRISPR\/Cas9 ribonucleoprotein delivery and gene editing","volume":"22","author":"Im","year":"2024","journal-title":"J. Nanobiotechnol."},{"key":"10.1016\/j.omtn.2025.102666_bib82","doi-asserted-by":"crossref","DOI":"10.1016\/j.ijpharm.2024.123845","article-title":"Development of ionizable lipid nanoparticles and a lyophilized formulation for potent CRISPR-Cas9 delivery and genome editing","volume":"652","author":"Sun","year":"2024","journal-title":"Int. J. Pharm."},{"key":"10.1016\/j.omtn.2025.102666_bib83","doi-asserted-by":"crossref","first-page":"531","DOI":"10.1016\/j.actbio.2024.10.030","article-title":"A biodegradable lipid nanoparticle delivers a Cas9 ribonucleoprotein for efficient and safe in situ genome editing in melanoma","volume":"190","author":"Yang","year":"2024","journal-title":"Acta Biomater."},{"key":"10.1016\/j.omtn.2025.102666_bib84","doi-asserted-by":"crossref","first-page":"7979","DOI":"10.1021\/acsabm.1c00890","article-title":"Polyethylenimine-functionalized carbon dots for delivery of CRISPR\/Cas9 complexes","volume":"4","author":"Hashemzadeh","year":"2021","journal-title":"ACS Appl. Bio Mater."},{"key":"10.1016\/j.omtn.2025.102666_bib85","doi-asserted-by":"crossref","first-page":"4619","DOI":"10.1038\/s41598-020-61465-6","article-title":"Polyethylenimine based magnetic nanoparticles mediated non-viral CRISPR\/Cas9 system for genome editing","volume":"10","author":"Rohiwal","year":"2020","journal-title":"Sci. Rep."},{"key":"10.1016\/j.omtn.2025.102666_bib86","doi-asserted-by":"crossref","first-page":"49","DOI":"10.1016\/j.bbrc.2022.08.076","article-title":"Delivery of Cas9-guided ABE8e into stem cells using poly (l-lysine) polypeptides for correction of the hemophilia-associated FIX missense mutation","volume":"628","author":"Rong","year":"2022","journal-title":"Biochem. Biophys. Res. Commun."},{"key":"10.1016\/j.omtn.2025.102666_bib87","doi-asserted-by":"crossref","DOI":"10.1016\/j.eurpolymj.2024.113408","article-title":"Poly (2-(dimethylamino) ethyl methacrylate)-b-poly (hydroxy propyl methacrylate) nanoparticles for guided delivery of MCL-1 CRISPR-Cas9 plasmid and doxorubicin to non-small cell lung cancer","volume":"219","author":"Vakili-Azghandi","year":"2024","journal-title":"Eur. Polym. J."},{"key":"10.1016\/j.omtn.2025.102666_bib88","doi-asserted-by":"crossref","DOI":"10.1016\/j.nantod.2025.102654","article-title":"Efficient intracellular delivery of CRISPR-Cas9 ribonucleoproteins using dendrimer nanoparticles for robust genomic editing","volume":"61","author":"Liyanage","year":"2025","journal-title":"Nano Today"},{"key":"10.1016\/j.omtn.2025.102666_bib89","doi-asserted-by":"crossref","first-page":"1840","DOI":"10.3390\/pharmaceutics14091840","article-title":"Advances in chitosan-based CRISPR\/Cas9 delivery systems","volume":"14","author":"Caprifico","year":"2022","journal-title":"Pharmaceutics"},{"key":"10.1016\/j.omtn.2025.102666_bib90","doi-asserted-by":"crossref","first-page":"3360","DOI":"10.1002\/cbic.202100359","article-title":"Exosomes as targeted delivery platform of CRISPR\/Cas9 for therapeutic genome editing","volume":"22","author":"Duan","year":"2021","journal-title":"Chembiochem"},{"key":"10.1016\/j.omtn.2025.102666_bib91","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":"10.1016\/j.omtn.2025.102666_bib92","doi-asserted-by":"crossref","first-page":"425","DOI":"10.1007\/s12033-024-01070-4","article-title":"The future of gene therapy: A review of in vivo and ex vivo delivery methods for genome editing-based therapies","volume":"67","author":"Volodina","year":"2025","journal-title":"Mol. Biotechnol."},{"key":"10.1016\/j.omtn.2025.102666_bib93","doi-asserted-by":"crossref","first-page":"464","DOI":"10.1016\/j.ymthe.2020.12.007","article-title":"Current Clinical Applications of In Vivo Gene Therapy with AAVs","volume":"29","author":"Mendell","year":"2021","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtn.2025.102666_bib94","series-title":"Principles of Tissue Engineering","first-page":"493","article-title":"Chapter 28 - Gene therapy","author":"Worgall","year":"2020"},{"key":"10.1016\/j.omtn.2025.102666_bib95","doi-asserted-by":"crossref","first-page":"99","DOI":"10.1016\/bs.pbr.2016.11.003","article-title":"Ex vivo gene therapy for the treatment of neurological disorders","volume":"230","author":"Gowing","year":"2017","journal-title":"Prog. Brain Res."},{"key":"10.1016\/j.omtn.2025.102666_bib96","doi-asserted-by":"crossref","first-page":"415","DOI":"10.1126\/science.1088547","article-title":"LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1","volume":"302","author":"Hacein-Bey-Abina","year":"2003","journal-title":"Science"},{"key":"10.1016\/j.omtn.2025.102666_bib97","doi-asserted-by":"crossref","first-page":"227ra33","DOI":"10.1126\/scitranslmed.3007280","article-title":"Gene therapy for Wiskott-Aldrich syndrome\u2014long-term efficacy and genotoxicity","volume":"6","author":"Braun","year":"2014","journal-title":"Sci. Transl. Med."},{"key":"10.1016\/j.omtn.2025.102666_bib98","doi-asserted-by":"crossref","first-page":"459","DOI":"10.4049\/jimmunol.1701155","article-title":"Chimeric antigen receptor T cell therapy: challenges to bench-to-bedside efficacy","volume":"200","author":"Srivastava","year":"2018","journal-title":"J. Immunol."},{"key":"10.1016\/j.omtn.2025.102666_bib99","doi-asserted-by":"crossref","first-page":"2986","DOI":"10.1016\/j.csbj.2022.06.015","article-title":"Development and clinical translation of ex vivo gene therapy","volume":"20","author":"Wu","year":"2022","journal-title":"Comput. Struct. Biotechnol. J."},{"key":"10.1016\/j.omtn.2025.102666_bib100","doi-asserted-by":"crossref","DOI":"10.1126\/science.aan4672","article-title":"Gene therapy comes of age","volume":"359","author":"Dunbar","year":"2018","journal-title":"Science"},{"key":"10.1016\/j.omtn.2025.102666_bib101","doi-asserted-by":"crossref","first-page":"1507","DOI":"10.1056\/NEJMoa1407222","article-title":"Chimeric antigen receptor T cells for sustained remissions in leukemia","volume":"371","author":"Maude","year":"2014","journal-title":"N. Engl. J. Med."},{"key":"10.1016\/j.omtn.2025.102666_bib102","doi-asserted-by":"crossref","first-page":"843","DOI":"10.3390\/pharmaceutics13060843","article-title":"Mesenchymal stem cells as a gene delivery tool: promise, problems, and prospects","volume":"13","author":"Attia","year":"2021","journal-title":"Pharmaceutics"},{"key":"10.1016\/j.omtn.2025.102666_bib103","doi-asserted-by":"crossref","first-page":"252","DOI":"10.1056\/NEJMoa2031054","article-title":"CRISPR-Cas9 gene editing for sickle cell disease and \u03b2-thalassemia","volume":"384","author":"Frangoul","year":"2021","journal-title":"N. Engl. J. Med."},{"key":"10.1016\/j.omtn.2025.102666_bib104","doi-asserted-by":"crossref","first-page":"205","DOI":"10.1056\/NEJMoa2029392","article-title":"Post-transcriptional genetic silencing of BCL11A to treat sickle cell disease","volume":"384","author":"Esrick","year":"2021","journal-title":"N. Engl. J. Med."},{"key":"10.1016\/j.omtn.2025.102666_bib105","doi-asserted-by":"crossref","first-page":"2052","DOI":"10.1182\/blood-2014-08-532820","article-title":"Rare bleeding disorders: diagnosis and treatment","volume":"125","author":"Palla","year":"2015","journal-title":"Blood"},{"key":"10.1016\/j.omtn.2025.102666_bib106","doi-asserted-by":"crossref","first-page":"207","DOI":"10.1038\/s41434-021-00247-9","article-title":"Genome editing using CRISPR\/Cas9 to treat hereditary hematological disorders","volume":"29","author":"Chen","year":"2022","journal-title":"Gene Ther."},{"key":"10.1016\/j.omtn.2025.102666_bib107","doi-asserted-by":"crossref","DOI":"10.1101\/cshperspect.a011692","article-title":"World distribution, population genetics, and health burden of the hemoglobinopathies","volume":"2","author":"Williams","year":"2012","journal-title":"Cold Spring Harb. Perspect. Med."},{"key":"10.1016\/j.omtn.2025.102666_bib108","doi-asserted-by":"crossref","first-page":"155","DOI":"10.1016\/S0140-6736(17)31822-6","article-title":"Thalassaemia","volume":"391","author":"Taher","year":"2018","journal-title":"Lancet"},{"key":"10.1016\/j.omtn.2025.102666_bib109","doi-asserted-by":"crossref","first-page":"4479","DOI":"10.1038\/s41467-019-12456-3","article-title":"Development of a forward-oriented therapeutic lentiviral vector for hemoglobin disorders","volume":"10","author":"Uchida","year":"2019","journal-title":"Nat. Commun."},{"key":"10.1016\/j.omtn.2025.102666_bib111","doi-asserted-by":"crossref","first-page":"1573","DOI":"10.1038\/s41591-022-01906-z","article-title":"CRISPR\u2013Cas9-mediated gene editing of the BCL11A enhancer for pediatric \u03b20\/\u03b20 transfusion-dependent \u03b2-thalassemia","volume":"28","author":"Fu","year":"2022","journal-title":"Nat. Med."},{"key":"10.1016\/j.omtn.2025.102666_bib112","doi-asserted-by":"crossref","first-page":"1347","DOI":"10.1097\/01.HS9.0000848716.65161.13","article-title":"P1465: INITIAL SAFETY AND EFFICACY STUDY OF RM-001, AUTOLOGOUS HBG1\/2 PROMOTER-MODIFIED CD34+ HEMATOPOIETIC STEM AND PROGENITOR CELLS, IN TRANSFUSION-DEPENDENT \u0392ETA-THALASSEMIA","volume":"6","author":"Wang","year":"2022","journal-title":"HemaSphere"},{"key":"10.1016\/j.omtn.2025.102666_bib113","doi-asserted-by":"crossref","first-page":"e613965e","DOI":"10.1097\/01.HS9.0000968000.61396.5e","article-title":"S272: SAFETY AND EFFICACY OF RM-001 IN PATIENTS WITH TRANSFUSION-DEPENDENT \u0392-THALASSEMIA: EARLY RESULTS FROM THE ONGOING OF AUTOLOGOUS HBG1\/2 PROMOTER-MODIFIED CD34+ HEMATOPOIETIC STEM AND PROGENITOR CELLS","volume":"7","author":"Liu","year":"2023","journal-title":"HemaSphere"},{"key":"10.1016\/j.omtn.2025.102666_bib114","doi-asserted-by":"crossref","DOI":"10.1126\/science.aba7365","article-title":"CRISPR-engineered T cells in patients with refractory cancer","volume":"367","author":"Stadtmauer","year":"2020","journal-title":"Science"},{"key":"10.1016\/j.omtn.2025.102666_bib115","doi-asserted-by":"crossref","first-page":"737","DOI":"10.1038\/s41598-017-00462-8","article-title":"CRISPR\/Cas9-mediated PD-1 disruption enhances anti-tumor efficacy of human chimeric antigen receptor T cells","volume":"7","author":"Rupp","year":"2017","journal-title":"Sci. Rep."},{"key":"10.1016\/j.omtn.2025.102666_bib116","doi-asserted-by":"crossref","first-page":"64","DOI":"10.1056\/NEJMra1706169","article-title":"Chimeric antigen receptor therapy","volume":"379","author":"June","year":"2018","journal-title":"N. Engl. J. Med."},{"key":"10.1016\/j.omtn.2025.102666_bib117","doi-asserted-by":"crossref","first-page":"154","DOI":"10.1038\/cr.2016.142","article-title":"CRISPR-Cas9-mediated multiplex gene editing in CAR-T cells","volume":"27","author":"Liu","year":"2017","journal-title":"Cell Res."},{"key":"10.1016\/j.omtn.2025.102666_bib118","doi-asserted-by":"crossref","first-page":"985","DOI":"10.1084\/jem.20171626","article-title":"Optimized RNP transfection for highly efficient CRISPR\/Cas9-mediated gene knockout in primary T cells","volume":"215","author":"Seki","year":"2018","journal-title":"J. Exp. Med."},{"key":"10.1016\/j.omtn.2025.102666_bib119","doi-asserted-by":"crossref","first-page":"1361","DOI":"10.1126\/science.aar6711","article-title":"CAR T cell immunotherapy for human cancer","volume":"359","author":"June","year":"2018","journal-title":"Science"},{"key":"10.1016\/j.omtn.2025.102666_bib120","doi-asserted-by":"crossref","first-page":"377","DOI":"10.1038\/s41551-018-0235-9","article-title":"Programming CAR-T cells to kill cancer","volume":"2","author":"Labanieh","year":"2018","journal-title":"Nat. Biomed. Eng."},{"key":"10.1016\/j.omtn.2025.102666_bib121","doi-asserted-by":"crossref","first-page":"4617","DOI":"10.1158\/0008-5472.CAN-11-0422","article-title":"In vivo persistence, tumor localization, and antitumor activity of CAR-engineered T cells is enhanced by costimulatory signaling through CD137 (4-1BB)","volume":"71","author":"Song","year":"2011","journal-title":"Cancer Res."},{"key":"10.1016\/j.omtn.2025.102666_bib122","doi-asserted-by":"crossref","DOI":"10.3389\/fmed.2023.1292452","article-title":"Current trends of clinical trials involving CRISPR\/Cas systems","volume":"10","author":"Zhang","year":"2023","journal-title":"Front. Med."},{"key":"10.1016\/j.omtn.2025.102666_bib123","doi-asserted-by":"crossref","first-page":"10303","DOI":"10.1182\/blood-2022-166432","article-title":"CTX110 Allogeneic CRISPR-Cas9-Engineered CAR T Cells in Patients (Pts) with Relapsed or Refractory (R\/R) Large B-Cell Lymphoma (LBCL): Results from the Phase 1 Dose Escalation Carbon Study","volume":"140","author":"McGuirk","year":"2022","journal-title":"Blood"},{"key":"10.1016\/j.omtn.2025.102666_bib124","doi-asserted-by":"crossref","first-page":"ND02","DOI":"10.1158\/1538-7445.AM2023-ND02","article-title":"Abstract ND02: CTX112 and CTX131: Next-generation CRISPR\/Cas9-engineered allogeneic (allo) CAR T cells incorporating novel edits that increase potency and efficacy in the treatment of lymphoid and solid tumors","volume":"83","author":"Terrett","year":"2023","journal-title":"Cancer Res."},{"key":"10.1016\/j.omtn.2025.102666_bib125","doi-asserted-by":"crossref","first-page":"1346","DOI":"10.1097\/01.HS9.0000848712.54842.0a","article-title":"P1464: DECREASING HPK1 EXPRESSION IN CD19 CAR-T CELLS: A NOVEL STRATEGY TO OVERCOME CHALLENGES OF CELL THERAPY FOR ADULT (R\/R) B-ALL","volume":"6","author":"Zhang","year":"2022","journal-title":"HemaSphere"},{"key":"10.1016\/j.omtn.2025.102666_bib126","doi-asserted-by":"crossref","DOI":"10.1126\/scitranslmed.abq3010","article-title":"Phase 1 clinical trial of CRISPR-engineered CAR19 universal T cells for treatment of children with refractory B cell leukemia","volume":"14","author":"Ottaviano","year":"2022","journal-title":"Sci. Transl. Med."},{"key":"10.1016\/j.omtn.2025.102666_bib127","doi-asserted-by":"crossref","first-page":"369","DOI":"10.1038\/s41586-022-05140-y","article-title":"Non-viral, specifically targeted CAR-T cells achieve high safety and efficacy in B-NHL","volume":"609","author":"Zhang","year":"2022","journal-title":"Nature"},{"key":"10.1016\/j.omtn.2025.102666_bib128","doi-asserted-by":"crossref","first-page":"2707","DOI":"10.1016\/j.ymthe.2021.07.001","article-title":"The rational development of CD5-targeting biepitopic CARs with fully human heavy-chain-only antigen recognition domains","volume":"29","author":"Dai","year":"2021","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtn.2025.102666_bib129","doi-asserted-by":"crossref","first-page":"1921","DOI":"10.1182\/blood-2018-99-116443","article-title":"Preclinical Development of CTX120, an Allogeneic CAR-T Cell Targeting Bcma","volume":"132","author":"Dar","year":"2018","journal-title":"Blood"},{"key":"10.1016\/j.omtn.2025.102666_bib130","doi-asserted-by":"crossref","first-page":"163","DOI":"10.1097\/01.HS9.0000843940.96598.e2","article-title":"S262: THE COBALT-LYM STUDY OF CTX130: A PHASE 1 DOSE ESCALATION STUDY OF CD70-TARGETED ALLOGENEIC CRISPR-CAS9\u2013ENGINEERED CAR T CELLS IN PATIENTS WITH RELAPSED\/REFRACTORY (R\/R) T-CELL MALIGNANCIES","volume":"6","author":"Iyer","year":"2022","journal-title":"HemaSphere"},{"key":"10.1016\/j.omtn.2025.102666_bib131","doi-asserted-by":"crossref","first-page":"899","DOI":"10.1056\/NEJMoa2300709","article-title":"Base-Edited CAR7 T Cells for Relapsed T-Cell Acute Lymphoblastic Leukemia","volume":"389","author":"Chiesa","year":"2023","journal-title":"N. Engl. J. Med."},{"key":"10.1016\/j.omtn.2025.102666_bib132","doi-asserted-by":"crossref","first-page":"2188","DOI":"10.1038\/s41423-021-00749-x","article-title":"Phase I study of CAR-T cells with PD-1 and TCR disruption in mesothelin-positive solid tumors","volume":"18","author":"Wang","year":"2021","journal-title":"Cell. Mol. Immunol."},{"key":"10.1016\/j.omtn.2025.102666_bib133","first-page":"A584","article-title":"558 CTX130 allogeneic CRISPR-Cas9\u2013engineered chimeric antigen receptor (CAR) T cells in patients with advanced clear cell renal cell carcinoma: Results from the phase 1 COBALT-RCC study","volume":"10","author":"Pal","year":"2022","journal-title":"J. ImmunoTher. Cancer"},{"key":"10.1016\/j.omtn.2025.102666_bib134","article-title":"Effect of CRISPR\/Cas9-edited PD-1\/PD-L1 on tumor immunity and immunotherapy","volume":"13","author":"Xu","year":"2022","journal-title":"Front. Immunol."},{"key":"10.1016\/j.omtn.2025.102666_bib135","article-title":"CRISPR-Cas9 mediated efficient PD-1 disruption on human primary T cells from cancer patients","volume":"6","author":"Su","year":"2016","journal-title":"Sci. Rep."},{"key":"10.1016\/j.omtn.2025.102666_bib136","doi-asserted-by":"crossref","DOI":"10.1136\/jitc-2023-008174","article-title":"Combining CRISPR-Cas9 and TCR exchange to generate a safe and efficient cord blood-derived T cell product for pediatric relapsed AML","volume":"12","author":"Lo Presti","year":"2024","journal-title":"J. Immunother. Cancer"},{"key":"10.1016\/j.omtn.2025.102666_bib137","article-title":"Genome editing approaches for universal chimeric antigen receptor T cells","volume":"3","author":"Mishra","year":"2024","journal-title":"EJC Paediatr. Oncol."},{"key":"10.1016\/j.omtn.2025.102666_bib138","doi-asserted-by":"crossref","first-page":"346","DOI":"10.3390\/pharmaceutics16030346","article-title":"CRISPR\/Cas9-Mediated Customizing Strategies for Adoptive T-Cell Therapy","volume":"16","author":"Park","year":"2024","journal-title":"Pharmaceutics"},{"key":"10.1016\/j.omtn.2025.102666_bib139","doi-asserted-by":"crossref","first-page":"2255","DOI":"10.1158\/1078-0432.CCR-16-1300","article-title":"Multiplex genome editing to generate universal CAR T cells resistant to PD1 inhibition","volume":"23","author":"Ren","year":"2017","journal-title":"Clin. Cancer Res."},{"key":"10.1016\/j.omtn.2025.102666_bib140","doi-asserted-by":"crossref","first-page":"113","DOI":"10.1038\/nature21405","article-title":"Targeting a CAR to the TRAC locus with CRISPR\/Cas9 enhances tumour rejection","volume":"543","author":"Eyquem","year":"2017","journal-title":"Nature"},{"key":"10.1016\/j.omtn.2025.102666_bib141","doi-asserted-by":"crossref","first-page":"405","DOI":"10.1038\/s41586-018-0326-5","article-title":"Reprogramming human T cell function and specificity with non-viral genome targeting","volume":"559","author":"Roth","year":"2018","journal-title":"Nature"},{"key":"10.1016\/j.omtn.2025.102666_bib142","doi-asserted-by":"crossref","DOI":"10.1098\/rstb.2018.0103","article-title":"Editing the microbiome the CRISPR way","volume":"374","author":"Ramachandran","year":"2019","journal-title":"Philos. Trans. R. Soc. Lond. B Biol. Sci."},{"key":"10.1016\/j.omtn.2025.102666_bib143","doi-asserted-by":"crossref","first-page":"1174","DOI":"10.1016\/j.jiph.2023.05.026","article-title":"Reduction of biofilm formation of Escherichia coli by targeting quorum sensing and adhesion genes using the CRISPR\/Cas9-HDR approach, and its clinical application on urinary catheter","volume":"16","author":"Alshammari","year":"2023","journal-title":"J. Infect. Public Health"},{"key":"10.1016\/j.omtn.2025.102666_bib144","doi-asserted-by":"crossref","first-page":"1146","DOI":"10.1038\/nbt.3043","article-title":"Exploiting CRISPR-Cas nucleases to produce sequence-specific antimicrobials","volume":"32","author":"Bikard","year":"2014","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.omtn.2025.102666_bib145","doi-asserted-by":"crossref","first-page":"1141","DOI":"10.1038\/nbt.3011","article-title":"Sequence-specific antimicrobials using efficiently delivered RNA-guided nucleases","volume":"32","author":"Citorik","year":"2014","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.omtn.2025.102666_bib146","series-title":"Gene Editing in Plants: Crispr-Cas and its Applications","first-page":"139","article-title":"Crispri-mediated gene silencing in biofilm cycle and quorum sensing","author":"Arora","year":"2024"},{"key":"10.1016\/j.omtn.2025.102666_bib147","doi-asserted-by":"crossref","first-page":"691","DOI":"10.1139\/cjm-2019-0100","article-title":"CRISPR-Cas9 knockout of qseB induced asynchrony between motility and biofilm formation in Escherichia coli","volume":"65","author":"Gou","year":"2019","journal-title":"Can. J. Microbiol."},{"key":"10.1016\/j.omtn.2025.102666_bib148","doi-asserted-by":"crossref","first-page":"269","DOI":"10.1038\/nrmicro3432","article-title":"Urinary tract infections: epidemiology, mechanisms of infection and treatment options","volume":"13","author":"Flores-Mireles","year":"2015","journal-title":"Nat. Rev. Microbiol."},{"key":"10.1016\/j.omtn.2025.102666_bib149","doi-asserted-by":"crossref","first-page":"18","DOI":"10.1099\/jmm.0.000406","article-title":"CRISPR-based genome editing of clinically important Escherichia coli SE15 isolated from indwelling urinary catheters of patients","volume":"66","author":"Kang","year":"2017","journal-title":"J. Med. Microbiol."},{"key":"10.1016\/j.omtn.2025.102666_bib150","doi-asserted-by":"crossref","DOI":"10.1128\/spectrum.01602-22","article-title":"Engineered bacteriophages containing anti-CRISPR suppress infection of antibiotic-resistant P. aeruginosa","volume":"10","author":"Qin","year":"2022","journal-title":"Microbiol. Spectr."},{"key":"10.1016\/j.omtn.2025.102666_bib151","doi-asserted-by":"crossref","first-page":"265","DOI":"10.1038\/s41587-023-01759-y","article-title":"Engineered phage with antibacterial CRISPR\u2013Cas selectively reduce E. coli burden in mice","volume":"42","author":"Gencay","year":"2024","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.omtn.2025.102666_bib152","doi-asserted-by":"crossref","first-page":"1319","DOI":"10.1016\/S1473-3099(24)00424-9","article-title":"Safety, pharmacokinetics, and pharmacodynamics of LBP-EC01, a CRISPR-Cas3-enhanced bacteriophage cocktail, in uncomplicated urinary tract infections due to Escherichia coli (ELIMINATE): the randomised, open-label, first part of a two-part phase 2 trial","volume":"24","author":"Kim","year":"2024","journal-title":"Lancet Infect. Dis."},{"key":"10.1016\/j.omtn.2025.102666_bib153","doi-asserted-by":"crossref","first-page":"493","DOI":"10.1056\/NEJMoa2107454","article-title":"CRISPR-Cas9 in vivo gene editing for transthyretin amyloidosis","volume":"385","author":"Gillmore","year":"2021","journal-title":"N. Engl. J. Med."},{"key":"10.1016\/j.omtn.2025.102666_bib154","doi-asserted-by":"crossref","first-page":"949","DOI":"10.1038\/s41587-021-00933-4","article-title":"In vivo adenine base editing of PCSK9 in macaques reduces LDL cholesterol levels","volume":"39","author":"Rothgangl","year":"2021","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.omtn.2025.102666_bib155","doi-asserted-by":"crossref","first-page":"1007","DOI":"10.1056\/NEJMoa1805689","article-title":"Tafamidis treatment for patients with transthyretin amyloid cardiomyopathy","volume":"379","author":"Maurer","year":"2018","journal-title":"N. Engl. J. Med."},{"key":"10.1016\/j.omtn.2025.102666_bib156","doi-asserted-by":"crossref","first-page":"27","DOI":"10.1146\/annurev-biochem-061516-045115","article-title":"Protein misfolding, amyloid formation, and human disease: a summary of progress over the last decade","volume":"86","author":"Chiti","year":"2017","journal-title":"Annu. Rev. Biochem."},{"key":"10.1016\/j.omtn.2025.102666_bib157","doi-asserted-by":"crossref","first-page":"1036","DOI":"10.1136\/jnnp-2014-308724","article-title":"Transthyretin (ATTR) amyloidosis: clinical spectrum, molecular pathogenesis and disease-modifying treatments","volume":"86","author":"Sekijima","year":"2015","journal-title":"J. Neurol. Neurosurg. Psychiatry"},{"key":"10.1016\/j.omtn.2025.102666_bib158","doi-asserted-by":"crossref","first-page":"384","DOI":"10.1038\/nrm3810","article-title":"The amyloid state and its association with protein misfolding diseases","volume":"15","author":"Knowles","year":"2014","journal-title":"Nat. Rev. Mol. Cell Biol."},{"key":"10.1016\/j.omtn.2025.102666_bib159","article-title":"Lessons from the first-in-human in vivo CRISPR\/Cas9 editing of the TTR gene by NTLA-2001 trial in patients with transthyretin amyloidosis with cardiomyopathy","volume":"2023","author":"Kotit","year":"2023","journal-title":"Glob. Cardiol. Sci. Pract."},{"key":"10.1016\/j.omtn.2025.102666_bib160","doi-asserted-by":"crossref","DOI":"10.1126\/scitranslmed.adj6779","article-title":"High-fidelity PAMless base editing of hematopoietic stem cells to treat chronic granulomatous disease","volume":"16","author":"Bzhilyanskaya","year":"2024","journal-title":"Sci. Transl. Med."},{"key":"10.1016\/j.omtn.2025.102666_bib161","doi-asserted-by":"crossref","first-page":"S10","DOI":"10.1016\/j.anai.2022.08.536","article-title":"In vivo CRISPR\/Cas9 editing of KLKB1 in patients with Hereditary Angioedema: A First-in-Human Study","volume":"129","author":"Longhurst","year":"2022","journal-title":"Ann. Allergy Asthma Immunol."},{"key":"10.1016\/j.omtn.2025.102666_bib162","doi-asserted-by":"crossref","first-page":"432","DOI":"10.1056\/NEJMoa2309149","article-title":"CRISPR-Cas9 In Vivo Gene Editing of KLKB1 for Hereditary Angioedema","volume":"390","author":"Longhurst","year":"2024","journal-title":"N. Engl. J. Med."},{"key":"10.1016\/j.omtn.2025.102666_bib163","doi-asserted-by":"crossref","first-page":"429","DOI":"10.1038\/s41586-021-03534-y","article-title":"In vivo CRISPR base editing of PCSK9 durably lowers cholesterol in primates","volume":"593","author":"Musunuru","year":"2021","journal-title":"Nature"},{"key":"10.1016\/j.omtn.2025.102666_bib164","doi-asserted-by":"crossref","DOI":"10.3389\/fcvm.2021.760140","article-title":"Gene therapy for cardiovascular disease: Basic research and clinical prospects","volume":"8","author":"Cao","year":"2021","journal-title":"Front. Cardiovasc. Med."},{"key":"10.1016\/j.omtn.2025.102666_bib165","first-page":"A17013","article-title":"Abstract 17013: CTX320: An Investigational in vivo CRISPR-Based Therapy Efficiently and Durably Reduces Lipoprotein (a) Levels in Non-Human Primates After a Single Dose","volume":"148","author":"Morrow","year":"2023","journal-title":"Circulation"},{"key":"10.1016\/j.omtn.2025.102666_bib166","doi-asserted-by":"crossref","first-page":"89","DOI":"10.1093\/ehjcvp\/pvad103","article-title":"VERVE-101: a promising CRISPR-based gene editing therapy that reduces LDL-C and PCSK9 levels in HeFH patients","volume":"10","author":"Horie","year":"2024","journal-title":"Eur. Heart J. Cardiovasc. Pharmacother."},{"key":"10.1016\/j.omtn.2025.102666_bib167","doi-asserted-by":"crossref","first-page":"753","DOI":"10.1080\/13543784.2024.2369747","article-title":"VERVE-101, a CRISPR base-editing therapy designed to permanently inactivate hepatic PCSK9 and reduce LDL-cholesterol","volume":"33","author":"Hooper","year":"2024","journal-title":"Expert Opin. Investig. Drugs"},{"key":"10.1016\/j.omtn.2025.102666_bib168","article-title":"An investigational in vivo base editing medicine targeting ANGPTL3, VERVE-201, achieves potent and LDLR-independent liver editing in mouse models","volume":"44","author":"Lee","year":"2023","journal-title":"Eur. Heart J."},{"key":"10.1016\/j.omtn.2025.102666_bib169","doi-asserted-by":"crossref","first-page":"901","DOI":"10.1056\/NEJMoa1300662","article-title":"Gene editing of CCR5 in autologous CD4 T cells of persons infected with HIV","volume":"370","author":"Tebas","year":"2014","journal-title":"N. Engl. J. Med."},{"key":"10.1016\/j.omtn.2025.102666_bib170","doi-asserted-by":"crossref","first-page":"328","DOI":"10.1038\/nbt.3471","article-title":"Therapeutic genome editing by combined viral and non-viral delivery of CRISPR system components in vivo","volume":"34","author":"Yin","year":"2016","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.omtn.2025.102666_bib171","doi-asserted-by":"crossref","first-page":"2909","DOI":"10.1016\/j.ymthe.2022.05.013","article-title":"Targeting the hepatitis B cccDNA with a sequence-specific ARCUS nuclease to eliminate hepatitis B virus in vivo","volume":"30","author":"Gorsuch","year":"2022","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtn.2025.102666_bib172","first-page":"S88","article-title":"Preclinical safety data for PBGENE-HBV gene editing program supports advancement to clinical trials as a potentially curative treatment for chronic hepatitis B","volume":"80","author":"Harrison","year":"2024","journal-title":"J. Hepatol."},{"key":"10.1016\/j.omtn.2025.102666_bib173","first-page":"457","article-title":"A Gene Editing Approach to Eliminate Hepatitis B Virus Using ARCUS Meganucleases","volume":"28","author":"Gorsuch","year":"2020","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtn.2025.102666_bib174","doi-asserted-by":"crossref","DOI":"10.3390\/ijms252212346","article-title":"Control of HSV-1 Infection: Directions for the Development of CRISPR\/Cas-Based Therapeutics and Diagnostics","volume":"25","author":"Sosnovtseva","year":"2024","journal-title":"Int. J. Mol. Sci."},{"key":"10.1016\/j.omtn.2025.102666_bib175","doi-asserted-by":"crossref","first-page":"3163","DOI":"10.1016\/j.ymthe.2023.08.021","article-title":"In vivo CRISPR gene editing in patients with herpetic stromal keratitis","volume":"31","author":"Wei","year":"2023","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtn.2025.102666_bib176","doi-asserted-by":"crossref","DOI":"10.1172\/JCI144486","article-title":"CCR5-edited CD4+ T cells augment HIV-specific immunity to enable post-rebound control of HIV replication","volume":"131","author":"Tebas","year":"2021","journal-title":"J. Clin. Investig."},{"key":"10.1016\/j.omtn.2025.102666_bib177","article-title":"Infusion of CCR5 Gene-Edited T Cells Allows Immune Reconstitution, HIV Reservoir Decay, and Long-Term Virological Control","author":"Zeidan","year":"2021","journal-title":"bioRxiv"},{"key":"10.1016\/j.omtn.2025.102666_bib178","doi-asserted-by":"crossref","first-page":"1240","DOI":"10.1056\/NEJMoa1817426","article-title":"CRISPR-Edited Stem Cells in a Patient with HIV and Acute Lymphocytic Leukemia","volume":"381","author":"Xu","year":"2019","journal-title":"N. Engl. J. Med."},{"key":"10.1016\/j.omtn.2025.102666_bib179","article-title":"P008: A first in human, single arm, open label phase 1\/2 study evaluating ECUR-506 in neonatal OTC Deficiency: Initial Observations","volume":"3","author":"Baruteau","year":"2025","journal-title":"Genet. Med. Open"},{"key":"10.1016\/j.omtn.2025.102666_bib180","article-title":"P008: pheEDIT: A phase 1, open-label, dose-escalation safety and efficacy gene editing study evaluating HMI-103 in adults with classical PKU","volume":"1","author":"Shioshita","year":"2023","journal-title":"Genet. Med. Open"},{"key":"10.1016\/j.omtn.2025.102666_bib181","doi-asserted-by":"crossref","first-page":"2235","DOI":"10.1056\/NEJMoa2504747","article-title":"Patient-specific in vivo gene editing to treat a rare genetic disease","volume":"392","author":"Musunuru","year":"2025","journal-title":"N. Engl. J. Med."},{"key":"10.1016\/j.omtn.2025.102666_bib182","doi-asserted-by":"crossref","first-page":"73","DOI":"10.7555\/JBR.32.20170114","article-title":"Angiopoietin-like protein 3 (ANGPTL3) deficiency and familial combined hypolipidemia","volume":"33","author":"Tarugi","year":"2019","journal-title":"J. Biomed. Res."},{"key":"10.1016\/j.omtn.2025.102666_bib183","doi-asserted-by":"crossref","first-page":"104","DOI":"10.1016\/j.ymthe.2024.10.003","article-title":"Efficient and safe in vivo treatment of primary hyperoxaluria type 1 via LNP-CRISPR-Cas9-mediated glycolate oxidase disruption","volume":"33","author":"Jiang","year":"2025","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtn.2025.102666_bib184","doi-asserted-by":"crossref","first-page":"4890","DOI":"10.1016\/j.cell.2024.06.027","article-title":"Allogeneic CD19-targeted CAR-T therapy in patients with severe myositis and systemic sclerosis","volume":"187","author":"Wang","year":"2024","journal-title":"Cell"},{"key":"10.1016\/j.omtn.2025.102666_bib185","doi-asserted-by":"crossref","first-page":"607","DOI":"10.1038\/s41422-025-01128-1","article-title":"Allogeneic anti-CD19 CAR-T cells induce remission in refractory systemic lupus erythematosus","volume":"35","author":"Yang","year":"2025","journal-title":"Cell Res."},{"key":"10.1016\/j.omtn.2025.102666_bib186","doi-asserted-by":"crossref","first-page":"15","DOI":"10.1182\/blood-2020-142423","article-title":"A phase I study of FT819, a first-of-kind, off-the-shelf, iPSC-derived TCR-less CD19 CAR T cell therapy for the treatment of relapsed\/refractory B-cell malignancies","volume":"136","author":"Park","year":"2020","journal-title":"Blood"},{"key":"10.1016\/j.omtn.2025.102666_bib187","doi-asserted-by":"crossref","first-page":"4577","DOI":"10.1182\/blood-2022-167194","article-title":"Interim phase I clinical data of FT819-101, a study of the first-ever, off-the-shelf, iPSC-derived TCR-less CD19 CAR T-cell therapy for patients with relapsed\/refractory B-cell malignancies","volume":"140","author":"Mehta","year":"2022","journal-title":"Blood"},{"key":"10.1016\/j.omtn.2025.102666_bib188","doi-asserted-by":"crossref","first-page":"1189","DOI":"10.1080\/14712598.2024.2422998","article-title":"Recent developments and industry interest in gene therapy for duchenne muscular dystrophy","volume":"24","author":"Moriyama","year":"2024","journal-title":"Expert Opin. Biol. Ther."},{"key":"10.1016\/j.omtn.2025.102666_bib189","doi-asserted-by":"crossref","first-page":"588","DOI":"10.1089\/hum.2023.29244.bfs","article-title":"Case Study Cites Immune Reaction to High Adeno-Associated Virus Dose in Explaining Duchenne Muscular Dystrophy Trial Death","volume":"34","author":"Philippidis","year":"2023","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.omtn.2025.102666_bib190","doi-asserted-by":"crossref","DOI":"10.1016\/j.omtn.2024.102311","article-title":"RNA exon editing: Splicing the way to treat human diseases","volume":"35","author":"Doi","year":"2024","journal-title":"Mol. Ther. Nucleic Acids"},{"key":"10.1016\/j.omtn.2025.102666_bib191","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":"10.1016\/j.omtn.2025.102666_bib192","first-page":"3679","article-title":"Twelve-month Natural History Study of CEP290-associated Retinal Degeneration","volume":"64","author":"Ashimatey","year":"2023","journal-title":"Invest. Ophthalmol. Visual Sci."},{"key":"10.1016\/j.omtn.2025.102666_bib193","first-page":"4357","article-title":"World\u2019s First CRISPR\/RNA-Targeting Therapy (HG202) for Patients with Neovascular Age-Related Macular Degeneration","volume":"65","author":"Luk","year":"2024","journal-title":"Invest. Ophthalmol. Visual Sci."}],"container-title":["Molecular Therapy Nucleic Acids"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S2162253125002203?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S2162253125002203?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2026,4,27]],"date-time":"2026-04-27T20:25:07Z","timestamp":1777321507000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S2162253125002203"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2025,9]]},"references-count":192,"journal-issue":{"issue":"3","published-print":{"date-parts":[[2025,9]]}},"alternative-id":["S2162253125002203"],"URL":"https:\/\/doi.org\/10.1016\/j.omtn.2025.102666","relation":{},"ISSN":["2162-2531"],"issn-type":[{"value":"2162-2531","type":"print"}],"subject":[],"published":{"date-parts":[[2025,9]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"Advancing gene editing therapeutics: Clinical trials and innovative delivery systems across diverse diseases","name":"articletitle","label":"Article Title"},{"value":"Molecular Therapy Nucleic Acids","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.omtn.2025.102666","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"\u00a9 2025 The Author(s). Published by Elsevier Inc. on behalf of The American Society of Gene and Cell Therapy.","name":"copyright","label":"Copyright"}],"article-number":"102666"}}