{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,9]],"date-time":"2026-10-09T02:02:24Z","timestamp":1791511344542,"version":"4.3.4"},"reference-count":76,"publisher":"Elsevier BV","issue":"5","license":[{"start":{"date-parts":[[2023,5,1]],"date-time":"2023-05-01T00:00:00Z","timestamp":1682899200000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2023,5,1]],"date-time":"2023-05-01T00:00:00Z","timestamp":1682899200000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/legal\/tdmrep-license"},{"start":{"date-parts":[[2024,4,18]],"date-time":"2024-04-18T00:00:00Z","timestamp":1713398400000},"content-version":"am","delay-in-days":353,"URL":"http:\/\/www.elsevier.com\/open-access\/userlicense\/1.0\/"},{"start":{"date-parts":[[2023,5,1]],"date-time":"2023-05-01T00:00:00Z","timestamp":1682899200000},"content-version":"stm-asf","delay-in-days":0,"URL":"https:\/\/doi.org\/10.15223\/policy-017"},{"start":{"date-parts":[[2023,5,1]],"date-time":"2023-05-01T00:00:00Z","timestamp":1682899200000},"content-version":"stm-asf","delay-in-days":0,"URL":"https:\/\/doi.org\/10.15223\/policy-037"},{"start":{"date-parts":[[2023,5,1]],"date-time":"2023-05-01T00:00:00Z","timestamp":1682899200000},"content-version":"stm-asf","delay-in-days":0,"URL":"https:\/\/doi.org\/10.15223\/policy-012"},{"start":{"date-parts":[[2023,5,1]],"date-time":"2023-05-01T00:00:00Z","timestamp":1682899200000},"content-version":"stm-asf","delay-in-days":0,"URL":"https:\/\/doi.org\/10.15223\/policy-029"},{"start":{"date-parts":[[2023,5,1]],"date-time":"2023-05-01T00:00:00Z","timestamp":1682899200000},"content-version":"stm-asf","delay-in-days":0,"URL":"https:\/\/doi.org\/10.15223\/policy-004"}],"content-domain":{"domain":["bmn.com","cell.com","clinicalkey.com","clinicalkey.com.au","clinicalkey.es","clinicalkey.fr","clinicalkey.jp","elsevier.com","sciencedirect.com"],"crossmark-restriction":true},"short-container-title":["Trends in Neurosciences"],"published-print":{"date-parts":[[2023,5]]},"DOI":"10.1016\/j.tins.2023.02.005","type":"journal-article","created":{"date-parts":[[2023,3,10]],"date-time":"2023-03-10T08:24:55Z","timestamp":1678436695000},"page":"365-376","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":28,"title":["The potential of gene editing for Huntington\u2019s disease"],"prefix":"10.1016","volume":"46","author":[{"given":"Wenzhen","family":"Duan","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ece","family":"Urani","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mark P.","family":"Mattson","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"78","reference":[{"key":"10.1016\/j.tins.2023.02.005_bb0005","doi-asserted-by":"crossref","first-page":"92","DOI":"10.1016\/j.arr.2018.11.006","article-title":"Targeting the proteostasis network in Huntington's disease","volume":"49","author":"Soares","year":"2019","journal-title":"Ageing Res. Rev."},{"key":"10.1016\/j.tins.2023.02.005_bb0010","doi-asserted-by":"crossref","first-page":"272","DOI":"10.1016\/j.pneurobio.2006.11.003","article-title":"N-methyl-D-aspartate (NMDA) receptor function and excitotoxicity in Huntington\u2019s disease","volume":"81","author":"Fan","year":"2007","journal-title":"Prog. Neurobiol."},{"key":"10.1016\/j.tins.2023.02.005_bb0015","doi-asserted-by":"crossref","first-page":"91","DOI":"10.1016\/bs.pmbts.2020.09.001","article-title":"Applying available knowledge and resources to alleviate familial and sporadic neurodegenerative disorders","volume":"177","author":"Mattson","year":"2021","journal-title":"Prog. Mol. Biol. Transl. Sci."},{"key":"10.1016\/j.tins.2023.02.005_bb0020","doi-asserted-by":"crossref","first-page":"195","DOI":"10.1038\/s41586-021-03191-1","article-title":"The NIH Somatic Cell Genome Editing program","volume":"592","author":"Saha","year":"2021","journal-title":"Nature"},{"key":"10.1016\/j.tins.2023.02.005_bb0025","doi-asserted-by":"crossref","first-page":"442","DOI":"10.1016\/j.cell.2013.06.044","article-title":"CRISPR-mediated modular RNA-guided regulation of transcription in eukaryotes","volume":"154","author":"Gilbert","year":"2013","journal-title":"Cell"},{"key":"10.1016\/j.tins.2023.02.005_bb0030","doi-asserted-by":"crossref","first-page":"495","DOI":"10.1038\/nrm.2017.48","article-title":"Non-homologous DNA end joining and alternative pathways to double-strand break repair","volume":"18","author":"Chang","year":"2017","journal-title":"Nat. Rev. Mol. Cell Biol."},{"key":"10.1016\/j.tins.2023.02.005_bb0035","doi-asserted-by":"crossref","DOI":"10.3389\/fgeed.2022.825236","article-title":"Predictable NHEJ insertion and assessment of HDR editing strategies in plants","volume":"4","author":"Molla","year":"2022","journal-title":"Front. Genome Ed."},{"key":"10.1016\/j.tins.2023.02.005_bb0040","doi-asserted-by":"crossref","first-page":"816","DOI":"10.1126\/science.1225829","article-title":"A programmable dual-RNA-guided DNA endonuclease in adaptive bacterial immunity","volume":"337","author":"Jinek","year":"2012","journal-title":"Science"},{"key":"10.1016\/j.tins.2023.02.005_bb0045","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.tins.2023.02.005_bb0050","doi-asserted-by":"crossref","first-page":"464","DOI":"10.1038\/nature24644","article-title":"Programmable base editing of A*T to G*C in genomic DNA without DNA cleavage","volume":"551","author":"Gaudelli","year":"2017","journal-title":"Nature"},{"key":"10.1016\/j.tins.2023.02.005_bb0055","doi-asserted-by":"crossref","DOI":"10.1126\/science.1247997","article-title":"Structures of Cas9 endonucleases reveal RNA-mediated conformational activation","volume":"343","author":"Jinek","year":"2014","journal-title":"Science"},{"key":"10.1016\/j.tins.2023.02.005_bb0060","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.tins.2023.02.005_bb0065","doi-asserted-by":"crossref","first-page":"26","DOI":"10.1016\/j.molcel.2017.09.029","article-title":"Methods and applications of CRISPR-mediated base editing in eukaryotic genomes","volume":"68","author":"Hess","year":"2017","journal-title":"Mol. Cell"},{"key":"10.1016\/j.tins.2023.02.005_bb0070","doi-asserted-by":"crossref","first-page":"20","DOI":"10.1016\/j.cell.2016.10.044","article-title":"CRISPR-based technologies for the manipulation of eukaryotic genomes","volume":"168","author":"Komor","year":"2017","journal-title":"Cell"},{"key":"10.1016\/j.tins.2023.02.005_bb0075","doi-asserted-by":"crossref","first-page":"888","DOI":"10.1038\/nbt.4194","article-title":"Optimized base editors enable efficient editing in cells, organoids and mice","volume":"36","author":"Zafra","year":"2018","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.tins.2023.02.005_bb0080","doi-asserted-by":"crossref","first-page":"57","DOI":"10.1038\/nature26155","article-title":"Evolved Cas9 variants with broad PAM compatibility and high DNA specificity","volume":"556","author":"Hu","year":"2018","journal-title":"Nature"},{"key":"10.1016\/j.tins.2023.02.005_bb0085","doi-asserted-by":"crossref","first-page":"430","DOI":"10.1038\/s41587-019-0050-1","article-title":"Genome-wide target specificity of CRISPR RNA-guided adenine base editors","volume":"37","author":"Kim","year":"2019","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.tins.2023.02.005_bb0090","doi-asserted-by":"crossref","first-page":"169","DOI":"10.1016\/j.molp.2019.10.007","article-title":"Discriminated sgRNAs-based SurroGate system greatly enhances the screening efficiency of plant base-edited cells","volume":"13","author":"Xu","year":"2020","journal-title":"Mol. Plant"},{"key":"10.1016\/j.tins.2023.02.005_bb0095","doi-asserted-by":"crossref","first-page":"279","DOI":"10.1038\/nbt.2808","article-title":"Improving CRISPR-Cas nuclease specificity using truncated guide RNAs","volume":"32","author":"Fu","year":"2014","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.tins.2023.02.005_bb0100","doi-asserted-by":"crossref","first-page":"2697","DOI":"10.1038\/s41467-020-16542-9","article-title":"Suppression of unwanted CRISPR-Cas9 editing by co-administration of catalytically inactivating truncated guide RNAs","volume":"11","author":"Rose","year":"2020","journal-title":"Nat. Commun."},{"key":"10.1016\/j.tins.2023.02.005_bb0105","doi-asserted-by":"crossref","first-page":"15790","DOI":"10.1038\/ncomms15790","article-title":"Improving the DNA specificity and applicability of base editing through protein engineering and protein delivery","volume":"8","author":"Rees","year":"2017","journal-title":"Nat. Commun."},{"key":"10.1016\/j.tins.2023.02.005_bb0110","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.tins.2023.02.005_bb0115","doi-asserted-by":"crossref","first-page":"292","DOI":"10.1126\/science.aaw7166","article-title":"Cytosine, but not adenine, base editors induce genome-wide off-target mutations in rice","volume":"364","author":"Jin","year":"2019","journal-title":"Science"},{"key":"10.1016\/j.tins.2023.02.005_bb0120","doi-asserted-by":"crossref","first-page":"824","DOI":"10.1038\/s41587-020-0561-9","article-title":"Genome editing with CRISPR-Cas nucleases, base editors, transposases and prime editors","volume":"38","author":"Anzalone","year":"2020","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.tins.2023.02.005_bb0125","doi-asserted-by":"crossref","first-page":"D862","DOI":"10.1093\/nar\/gkv1222","article-title":"ClinVar: public archive of interpretations of clinically relevant variants","volume":"44","author":"Landrum","year":"2016","journal-title":"Nucleic Acids Res."},{"key":"10.1016\/j.tins.2023.02.005_bb0130","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.tins.2023.02.005_bb0135","doi-asserted-by":"crossref","first-page":"405","DOI":"10.1001\/jama.2019.21827","article-title":"DNA prime editing: a new CRISPR-based method to correct most disease-causing mutations","volume":"323","author":"Hampton","year":"2020","journal-title":"JAMA"},{"key":"10.1016\/j.tins.2023.02.005_bb0140","doi-asserted-by":"crossref","first-page":"528","DOI":"10.3389\/fgene.2020.00528","article-title":"Prime editing: genome editing for rare genetic diseases without double-strand breaks or donor DNA","volume":"11","author":"Matsoukas","year":"2020","journal-title":"Front. Genet."},{"key":"10.1016\/j.tins.2023.02.005_bb0145","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":"10.1016\/j.tins.2023.02.005_bb0150","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":"10.1016\/j.tins.2023.02.005_bb0155","doi-asserted-by":"crossref","first-page":"12584","DOI":"10.1021\/jacs.2c02633","article-title":"Chemical modifications of CRISPR RNAs to improve gene-editing activity and specificity","volume":"144","author":"Rozners","year":"2022","journal-title":"J. Am. Chem. Soc."},{"key":"10.1016\/j.tins.2023.02.005_bb0160","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":"10.1016\/j.tins.2023.02.005_bb0165","doi-asserted-by":"crossref","first-page":"345","DOI":"10.1016\/j.jconrel.2022.01.013","article-title":"Delivery of CRISPR-Cas tools for in vivo genome editing therapy: trends and challenges","volume":"342","author":"Taha","year":"2022","journal-title":"J. Control. Release"},{"key":"10.1016\/j.tins.2023.02.005_bb0170","doi-asserted-by":"crossref","first-page":"401","DOI":"10.1016\/j.jconrel.2022.08.042","article-title":"LNP-mediated delivery of CRISPR RNP for wide-spread in vivo genome editing in mouse cornea","volume":"350","author":"Mirjalili Mohanna","year":"2022","journal-title":"Control. Release"},{"key":"10.1016\/j.tins.2023.02.005_bb0175","doi-asserted-by":"crossref","first-page":"217","DOI":"10.1038\/nature25164","article-title":"Treatment of autosomal dominant hearing loss by in vivo delivery of genome editing agents","volume":"553","author":"Gao","year":"2018","journal-title":"Nature"},{"key":"10.1016\/j.tins.2023.02.005_bb0180","doi-asserted-by":"crossref","first-page":"497","DOI":"10.1038\/s41551-018-0252-8","article-title":"Nanoparticle delivery of CRISPR into the brain rescues a mouse model of fragile X syndrome from exaggerated repetitive behaviours","volume":"2","author":"Lee","year":"2018","journal-title":"Nat. Biomed. Eng."},{"key":"10.1016\/j.tins.2023.02.005_bb0185","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.tins.2023.02.005_bb0190","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.tins.2023.02.005_bb0195","doi-asserted-by":"crossref","first-page":"118","DOI":"10.1038\/s41591-019-0674-1","article-title":"Spinal subpial delivery of AAV9 enables widespread gene silencing and blocks motoneuron degeneration in ALS","volume":"26","author":"Bravo-Hernandez","year":"2020","journal-title":"Nat. Med."},{"key":"10.1016\/j.tins.2023.02.005_bb0200","doi-asserted-by":"crossref","first-page":"1177","DOI":"10.1016\/j.ymthe.2020.01.005","article-title":"Treatment of a mouse model of ALS by in vivo base editing","volume":"28","author":"Lim","year":"2020","journal-title":"Mol. Ther."},{"key":"10.1016\/j.tins.2023.02.005_bb0205","doi-asserted-by":"crossref","first-page":"4303","DOI":"10.1016\/j.celrep.2020.02.092","article-title":"Conditional single vector CRISPR\/SaCas9 viruses for efficient mutagenesis in the adult mouse nervous system","volume":"30","author":"Hunker","year":"2020","journal-title":"Cell Rep."},{"key":"10.1016\/j.tins.2023.02.005_bb0210","doi-asserted-by":"crossref","DOI":"10.1126\/sciadv.aay6687","article-title":"Development of a CRISPR-SaCas9 system for projection- and function-specific gene editing in the rat brain","volume":"6","author":"Sun","year":"2020","journal-title":"Sci. Adv."},{"key":"10.1016\/j.tins.2023.02.005_bb0215","doi-asserted-by":"crossref","first-page":"646","DOI":"10.1038\/s41434-021-00224-2","article-title":"Highly efficient neuronal gene knockout in vivo by CRISPR-Cas9 via neonatal intracerebroventricular injection of AAV in mice","volume":"28","author":"Hana","year":"2021","journal-title":"Gene Ther."},{"key":"10.1016\/j.tins.2023.02.005_bb0220","doi-asserted-by":"crossref","first-page":"755","DOI":"10.1016\/j.omtm.2021.02.012","article-title":"CRISPR-mediated rapid generation of neural cell-specific knockout mice facilitates research in neurophysiology and pathology","volume":"20","author":"Xiao","year":"2021","journal-title":"Mol. Ther. Methods Clin. Dev."},{"key":"10.1016\/j.tins.2023.02.005_bb0225","doi-asserted-by":"crossref","first-page":"447","DOI":"10.1038\/s41593-018-0077-5","article-title":"CRISPR interference-based specific and efficient gene inactivation in the brain","volume":"21","author":"Zheng","year":"2018","journal-title":"Nat. Neurosci."},{"key":"10.1016\/j.tins.2023.02.005_bb0230","doi-asserted-by":"crossref","DOI":"10.1523\/ENEURO.0188-21.2021","article-title":"A Cre-dependent CRISPR\/dCas9 system for gene expression regulation in neurons","volume":"8","author":"Carullo","year":"2021","journal-title":"eNeuro"},{"key":"10.1016\/j.tins.2023.02.005_bb0235","doi-asserted-by":"crossref","first-page":"993","DOI":"10.1038\/s41423-022-00897-8","article-title":"Virus-like particle vaccinology, from bench to bedside","volume":"19","author":"Mohsen","year":"2022","journal-title":"Cell. Mol. Immunol."},{"key":"10.1016\/j.tins.2023.02.005_bb0240","doi-asserted-by":"crossref","first-page":"372","DOI":"10.1016\/j.neuron.2016.09.021","article-title":"A designer AAV variant permits efficient retrograde access to projection neurons","volume":"92","author":"Tervo","year":"2016","journal-title":"Neuron"},{"key":"10.1016\/j.tins.2023.02.005_bb0245","doi-asserted-by":"crossref","first-page":"2719","DOI":"10.1172\/JCI92087","article-title":"CRISPR\/Cas9-mediated gene editing ameliorates neurotoxicity in mouse model of Huntington's disease","volume":"127","author":"Yang","year":"2017","journal-title":"J. Clin. Invest."},{"key":"10.1016\/j.tins.2023.02.005_bb0250","doi-asserted-by":"crossref","first-page":"3101","DOI":"10.1093\/brain\/awab190","article-title":"Huntingtin silencing delays onset and slows progression of Huntington's disease: a biomarker study","volume":"144","author":"Liu","year":"2021","journal-title":"Brain"},{"key":"10.1016\/j.tins.2023.02.005_bb0255","doi-asserted-by":"crossref","first-page":"829","DOI":"10.1016\/j.omtn.2019.07.009","article-title":"CRISPR-Cas9-mediated genome editing increases lifespan and improves motor deficits in a Huntington's disease mouse model","volume":"17","author":"Ekman","year":"2019","journal-title":"Mol. Ther. Nucleic Acids"},{"key":"10.1016\/j.tins.2023.02.005_bb0260","doi-asserted-by":"crossref","first-page":"75","DOI":"10.3389\/fnins.2018.00075","article-title":"Precise excision of the CAG tract from the Huntingtin gene by Cas9 nickases","volume":"12","author":"Dabrowska","year":"2018","journal-title":"Front. Neurosci."},{"key":"10.1016\/j.tins.2023.02.005_bb0265","doi-asserted-by":"crossref","first-page":"619","DOI":"10.1016\/j.stemcr.2017.01.022","article-title":"Reversal of phenotypic abnormalities by CRISPR\/Cas9-mediated gene correction in Huntington disease patient-derived induced pluripotent stem cells","volume":"8","author":"Xu","year":"2017","journal-title":"Stem Cell Rep."},{"key":"10.1016\/j.tins.2023.02.005_bb0270","doi-asserted-by":"crossref","first-page":"837","DOI":"10.1016\/S1474-4422(17)30280-6","article-title":"Therapies targeting DNA and RNA in Huntington's disease","volume":"16","author":"Wild","year":"2017","journal-title":"Lancet Neurol."},{"key":"10.1016\/j.tins.2023.02.005_bb0275","first-page":"4566","article-title":"Permanent inactivation of Huntington's disease mutation by personalized allele-specific CRISPR\/Cas9","volume":"25","author":"Shin","year":"2016","journal-title":"Hum. Mol. Genet."},{"key":"10.1016\/j.tins.2023.02.005_bb0280","doi-asserted-by":"crossref","first-page":"12","DOI":"10.1016\/j.ymthe.2016.11.010","article-title":"CRISPR\/Cas9 editing of the mutant huntingtin allele in vitro and in vivo","volume":"25","author":"Monteys","year":"2017","journal-title":"Mol. Ther."},{"key":"10.1016\/j.tins.2023.02.005_bb0285","doi-asserted-by":"crossref","DOI":"10.1371\/journal.pgen.1007308","article-title":"Comparison of phasing strategies for whole human genomes","volume":"14","author":"Choi","year":"2018","journal-title":"PLoS Genet."},{"key":"10.1016\/j.tins.2023.02.005_bb0290","doi-asserted-by":"crossref","first-page":"608","DOI":"10.1038\/s41586-020-03086-7","volume":"589","author":"Koblan","year":"2021","journal-title":"Nature"},{"key":"10.1016\/j.tins.2023.02.005_bb0295","doi-asserted-by":"crossref","first-page":"27","DOI":"10.1038\/s41593-022-01207-1","article-title":"An RNA-targeting CRISPR-Cas13d system alleviates disease-related phenotypes in Huntington\u2019s disease models","volume":"26","author":"Morelli","year":"2023","journal-title":"Nat. Neurosci."},{"key":"10.1016\/j.tins.2023.02.005_bb0300","doi-asserted-by":"crossref","first-page":"252","DOI":"10.1056\/NEJMoa2031054","article-title":"CRISPR-Cas9 gene editing for sickle cell disease and beta-thalassemia","volume":"384","author":"Frangoul","year":"2021","journal-title":"N. Engl. J. Med."},{"key":"10.1016\/j.tins.2023.02.005_bb0305","doi-asserted-by":"crossref","first-page":"4534","DOI":"10.3390\/ijms22094534","article-title":"An update on gene therapy for inherited retinal dystrophy: experience in Leber congenital amaurosis clinical trials","volume":"22","author":"Chiu","year":"2021","journal-title":"Int. J. Mol. Sci."},{"key":"10.1016\/j.tins.2023.02.005_bb0310","doi-asserted-by":"crossref","first-page":"2764","DOI":"10.1158\/1078-0432.CCR-20-3863","article-title":"CRISPR\/Cas9-engineered universal CD19\/CD22 dual-targeted CAR-T cell therapy for relapsed\/refractory B-cell acute lymphoblastic leukemia","volume":"27","author":"Hu","year":"2021","journal-title":"Clin. Cancer Res."},{"key":"10.1016\/j.tins.2023.02.005_bb0315","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.tins.2023.02.005_bb0320","doi-asserted-by":"crossref","DOI":"10.1126\/sciadv.aar3952","article-title":"In vivo genome editing improves motor function and extends survival in a mouse model of ALS","volume":"3","author":"Gaj","year":"2017","journal-title":"Sci. Adv."},{"key":"10.1016\/j.tins.2023.02.005_bb0325","doi-asserted-by":"crossref","first-page":"756","DOI":"10.1089\/scd.2017.0209","article-title":"CRISPR\/Cas9-targeted deletion of polyglutamine in spinocerebellar ataxia type 3-derived induced pluripotent stem cells","volume":"27","author":"Ouyang","year":"2018","journal-title":"Stem Cells Dev."},{"key":"10.1016\/j.tins.2023.02.005_bb0330","doi-asserted-by":"crossref","first-page":"234","DOI":"10.1016\/j.celrep.2015.08.084","article-title":"Reversion of FMR1 methylation and silencing by editing the triplet repeats in fragile x IPSC-derived neurons","volume":"13","author":"Park","year":"2015","journal-title":"Cell Rep."},{"key":"10.1016\/j.tins.2023.02.005_bb0335","article-title":"CRISPR-Cas9 targeted deletion of the C9orf72 repeat expansion mutation corrects cellular phenotypes in patient-derived IPS cells","author":"Pribadi","year":"2016","journal-title":"bioRxiv"},{"key":"10.1016\/j.tins.2023.02.005_bb0340","doi-asserted-by":"crossref","first-page":"1131","DOI":"10.1038\/s41591-019-0478-3","article-title":"Allele-selective transcriptional repression of mutant HTT for the treatment of Huntington\u2019s disease","volume":"25","author":"Zeitler","year":"2019","journal-title":"Nat. Med."},{"key":"10.1016\/j.tins.2023.02.005_bb0345","doi-asserted-by":"crossref","first-page":"571","DOI":"10.3233\/JAD-171058","article-title":"Generation of a new tau knockout (tau\u03b4ex1) line using CRISPR\/cas9 genome editing in mice","volume":"62","author":"Tan","year":"2018","journal-title":"J. Alzheimers Dis."},{"key":"10.1016\/j.tins.2023.02.005_bb0350","doi-asserted-by":"crossref","first-page":"95","DOI":"10.1089\/crispr.2021.0025","article-title":"CRISPR-Cas9 gene editing protects from the A53T-SNCA overexpression-induced pathology of Parkinson's disease in vivo","volume":"5","author":"Yoon","year":"2022","journal-title":"CRISPR J."},{"key":"10.1016\/j.tins.2023.02.005_bb0355","doi-asserted-by":"crossref","DOI":"10.3389\/fcell.2020.576592","article-title":"Mitochondrial and redox modifications in Huntington disease induced pluripotent stem cells rescued by CRISPR\/Cas9 cags targeting","volume":"8","author":"Lopes","year":"2020","journal-title":"Front. Cell Dev. Biol."},{"key":"10.1016\/j.tins.2023.02.005_bb0360","doi-asserted-by":"crossref","first-page":"2200","DOI":"10.1093\/hmg\/ddaa106","article-title":"Correction of amyotrophic lateral sclerosis related phenotypes in induced pluripotent stem cell-derived motor neurons carrying a hexanucleotide expansion mutation in C9orf72 by CRISPR\/Cas9 genome editing using homology-directed repair","volume":"29","author":"Ababneh","year":"2020","journal-title":"Hum. Mol. Genet."},{"key":"10.1016\/j.tins.2023.02.005_bb0365","doi-asserted-by":"crossref","first-page":"157","DOI":"10.1038\/s41434-019-0116-1","article-title":"The deletion of mutant SOD1 via CRISPR\/Cas9\/sgrna prolongs survival in an amyotrophic lateral sclerosis mouse model","volume":"27","author":"Duan","year":"2020","journal-title":"Gene Ther."},{"key":"10.1016\/j.tins.2023.02.005_bb0370","doi-asserted-by":"crossref","first-page":"892","DOI":"10.1016\/j.stemcr.2020.03.023","article-title":"Impairment of mitochondrial calcium buffering links mutations in C9ORF72 and TARDBP in IPS-derived motor neurons from patients with ALS\/FTD","volume":"14","author":"Dafinca","year":"2020","journal-title":"Stem Cell Rep."},{"key":"10.1016\/j.tins.2023.02.005_bb0375","doi-asserted-by":"crossref","first-page":"396","DOI":"10.1038\/s42003-021-01942-4","article-title":"Efficacy and long-term safety of CRISPR\/Cas9 genome editing in the SOD1-linked mouse models of ALS","volume":"4","author":"Deng","year":"2021","journal-title":"Commun. Biol."},{"key":"10.1016\/j.tins.2023.02.005_bb0380","doi-asserted-by":"crossref","first-page":"168","DOI":"10.1038\/s41551-021-00759-0","article-title":"Brain-wide Cas9-mediated cleavage of a gene causing familial Alzheimer's disease alleviates amyloid-related pathologies in mice","volume":"6","author":"Duan","year":"2022","journal-title":"Nat. Biomed. Eng."}],"container-title":["Trends in Neurosciences"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S0166223623000462?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S0166223623000462?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2025,9,28]],"date-time":"2025-09-28T12:23:07Z","timestamp":1759062187000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S0166223623000462"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2023,5]]},"references-count":76,"journal-issue":{"issue":"5","published-print":{"date-parts":[[2023,5]]}},"alternative-id":["S0166223623000462"],"URL":"https:\/\/doi.org\/10.1016\/j.tins.2023.02.005","relation":{},"ISSN":["0166-2236"],"issn-type":[{"value":"0166-2236","type":"print"}],"subject":[],"published":{"date-parts":[[2023,5]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"The potential of gene editing for Huntington\u2019s disease","name":"articletitle","label":"Article Title"},{"value":"Trends in Neurosciences","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.tins.2023.02.005","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"\u00a9 2023 Elsevier Ltd. All rights reserved.","name":"copyright","label":"Copyright"}]}}