{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,7]],"date-time":"2026-10-07T04:16:30Z","timestamp":1791346590699,"version":"4.3.1"},"reference-count":84,"publisher":"Oxford University Press (OUP)","issue":"1","license":[{"start":{"date-parts":[[2022,4,7]],"date-time":"2022-04-07T00:00:00Z","timestamp":1649289600000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/academic.oup.com\/pages\/standard-publication-reuse-rights"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":[],"published-print":{"date-parts":[[2023,3,17]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:p>CRISPR technologies have progressed by leaps and bounds over the past decade, not only having a transformative effect on biomedical research but also yielding new therapies that are poised to enter the clinic. In this review, I give an overview of (i) the various CRISPR DNA-editing technologies, including standard nuclease gene editing, base editing, prime editing, and epigenome editing, (ii) their impact on cardiovascular basic science research, including animal models, human pluripotent stem cell models, and functional screens, and (iii) emerging therapeutic applications for patients with cardiovascular diseases, focusing on the examples of hypercholesterolaemia, transthyretin amyloidosis, and Duchenne muscular dystrophy.<\/jats:p>","DOI":"10.1093\/cvr\/cvac048","type":"journal-article","created":{"date-parts":[[2022,4,7]],"date-time":"2022-04-07T09:31:36Z","timestamp":1649323896000},"page":"79-93","source":"Crossref","is-referenced-by-count":27,"title":["CRISPR and cardiovascular diseases"],"prefix":"10.1093","volume":"119","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-3298-0368","authenticated-orcid":false,"given":"Kiran","family":"Musunuru","sequence":"first","affiliation":[{"name":"Cardiovascular Institute, Department of Medicine, Perelman School of Medicine at the University of Pennsylvania , Philadelphia, PA , USA"},{"name":"Department of Genetics, Perelman School of Medicine at the University of Pennsylvania , Philadelphia, PA , USA"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"286","published-online":{"date-parts":[[2022,4,7]]},"reference":[{"key":"2023031717462827900_","author":"Stein"},{"key":"2023031717462827900_","first-page":"p1","volume-title":"Genome Editing: A Practical Guide to Research and Clinical Applications","author":"Musunuru","year":"2021","edition":"1st ed"},{"key":"2023031717462827900_","first-page":"8096","article-title":"Introduction of double-strand breaks into the genome of mouse cells by expression of a rare-cutting endonuclease","volume":"14","author":"Rouet","year":"1994","journal-title":"Mol Cell Biol"},{"key":"2023031717462827900_","doi-asserted-by":"crossref","first-page":"1169","DOI":"10.1093\/genetics\/161.3.1169","article-title":"Targeted chromosomal cleavage and mutagenesis in Drosophila using zinc-finger nucleases","volume":"161","author":"Bibikova","year":"2002","journal-title":"Genetics"},{"key":"2023031717462827900_","doi-asserted-by":"crossref","first-page":"289","DOI":"10.1128\/MCB.21.1.289-297.2001","article-title":"Stimulation of homologous recombination through targeted cleavage by chimeric nucleases","volume":"21","author":"Bibikova","year":"2001","journal-title":"Mol Cell Biol"},{"key":"2023031717462827900_","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":"2023031717462827900_","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":"2023031717462827900_","doi-asserted-by":"crossref","first-page":"186","DOI":"10.1038\/nature14299","article-title":"In vivo genome editing using Staphylococcus aureus Cas9","volume":"520","author":"Ran","year":"2015","journal-title":"Nature"},{"key":"2023031717462827900_","doi-asserted-by":"crossref","first-page":"290","DOI":"10.1126\/science.aba8853","article-title":"Unconstrained genome targeting with near-PAMless engineered CRISPR-Cas9 variants","volume":"368","author":"Walton","year":"2020","journal-title":"Science"},{"key":"2023031717462827900_","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":"2023031717462827900_","doi-asserted-by":"crossref","first-page":"212","DOI":"10.1038\/s41467-018-08224-4","article-title":"Engineering of CRISPR-Cas12b for human genome editing","volume":"10","author":"Strecker","year":"2019","journal-title":"Nat Commun"},{"key":"2023031717462827900_","doi-asserted-by":"crossref","first-page":"218","DOI":"10.1038\/s41586-019-0908-x","article-title":"CasX enzymes comprise a distinct family of RNA-guided genome editors","volume":"566","author":"Liu","year":"2019","journal-title":"Nature"},{"key":"2023031717462827900_","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":"2023031717462827900_","doi-asserted-by":"crossref","first-page":"p81","DOI":"10.1016\/B978-0-12-823484-6.00001-3","volume-title":"Genome Editing: A Practical Guide to Research and Clinical Applications","author":"Musunuru","year":"2021","edition":"1st ed"},{"key":"2023031717462827900_","doi-asserted-by":"crossref","first-page":"490","DOI":"10.1038\/nature16526","article-title":"High-fidelity CRISPR-Cas9 nucleases with no detectable genome-wide off-target effects","volume":"529","author":"Kleinstiver","year":"2016","journal-title":"Nature"},{"key":"2023031717462827900_","doi-asserted-by":"crossref","first-page":"84","DOI":"10.1126\/science.aad5227","article-title":"Rationally engineered Cas9 nucleases with improved specificity","volume":"351","author":"Slaymaker","year":"2016","journal-title":"Science"},{"key":"2023031717462827900_","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":"2023031717462827900_","doi-asserted-by":"crossref","first-page":"311","DOI":"10.1038\/nchembio.2559","article-title":"Partial DNA-guided Cas9 enables genome editing with reduced off-target activity","volume":"14","author":"Yin","year":"2018","journal-title":"Nat Chem Biol"},{"key":"2023031717462827900_","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":"2023031717462827900_","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":"2023031717462827900_","doi-asserted-by":"crossref","first-page":"1414","DOI":"10.1038\/s41587-021-00938-z","article-title":"Efficient C\u2022G-to-G\u2022C base editors developed using CRISPRi screens, target-library analysis, and machine learning","volume":"39","author":"Koblan","year":"2021","journal-title":"Nat Biotechnol"},{"key":"2023031717462827900_","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":"2023031717462827900_","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":"2023031717462827900_","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":"2023031717462827900_","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":"2023031717462827900_","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":"2023031717462827900_","doi-asserted-by":"crossref","first-page":"218","DOI":"10.1038\/s41587-021-01025-z","article-title":"Precise genomic deletions using paired prime editing","volume":"40","author":"Choi","year":"2022","journal-title":"Nat Biotechnol"},{"key":"2023031717462827900_","doi-asserted-by":"crossref","first-page":"227","DOI":"10.1038\/s41587-021-01026-y","article-title":"Deletion and replacement of long genomic sequences using prime editing","volume":"40","author":"Jiang","year":"2022","journal-title":"Nat Biotechnol"},{"key":"2023031717462827900_","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":"2023031717462827900_","doi-asserted-by":"crossref","first-page":"1173","DOI":"10.1016\/j.cell.2013.02.022","article-title":"Repurposing CRISPR as an RNA-guided platform for sequence-specific control of gene expression","volume":"152","author":"Qi","year":"2013","journal-title":"Cell"},{"key":"2023031717462827900_","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":"2023031717462827900_","doi-asserted-by":"crossref","first-page":"647","DOI":"10.1016\/j.cell.2014.09.029","article-title":"Genome-scale CRISPR-mediated control of gene repression and activation","volume":"159","author":"Gilbert","year":"2014","journal-title":"Cell"},{"issue":"184","key":"2023031717462827900_","doi-asserted-by":"crossref","first-page":"2503","DOI":"10.1016\/j.cell.2021.03.025","article-title":"Genome-wide programmable transcriptional memory by CRISPR-based epigenome editing","volume":"202","author":"Nu\u00f1ez","year":"2021","journal-title":"Cell"},{"key":"2023031717462827900_","doi-asserted-by":"crossref","first-page":"910","DOI":"10.1016\/j.cell.2013.04.025","article-title":"One-step generation of mice carrying mutations in multiple genes by CRISPR\/Cas-mediated genome engineering","volume":"153","author":"Wang","year":"2013","journal-title":"Cell"},{"key":"2023031717462827900_","doi-asserted-by":"crossref","first-page":"1370","DOI":"10.1016\/j.cell.2013.08.022","article-title":"One-step generation of mice carrying reporter and conditional alleles by CRISPR\/Cas-mediated genome engineering","volume":"154","author":"Yang","year":"2013","journal-title":"Cell"},{"key":"2023031717462827900_","doi-asserted-by":"crossref","first-page":"171","DOI":"10.1186\/s13059-019-1776-2","article-title":"Reproducibility of CRISPR-Cas9 methods for generation of conditional mouse alleles: a multi-center evaluation","volume":"20","author":"Gurumurthy","year":"2019","journal-title":"Genome Biol"},{"key":"2023031717462827900_","doi-asserted-by":"crossref","first-page":"191","DOI":"10.1007\/978-1-4939-8831-0_11","article-title":"Generation of conditional knockout mice by sequential insertion of two loxP sites in cis using CRISPR\/Cas9 and single-stranded DNA oligonucleotides","volume":"1874","author":"Liu","year":"2019","journal-title":"Methods Mol Biol"},{"key":"2023031717462827900_","doi-asserted-by":"crossref","first-page":"1668","DOI":"10.3390\/ijms17101668","article-title":"Porcine zygote injection with Cas9\/sgRNA results in DMD-modified pig with muscle dystrophy","volume":"17","author":"Yu","year":"2016","journal-title":"Int J Mol Sci"},{"key":"2023031717462827900_","doi-asserted-by":"crossref","first-page":"e021631","DOI":"10.1161\/JAHA.121.021631","article-title":"Cardiovascular development and congenital heart disease modeling in the pig","volume":"10","author":"Gabriel","year":"2021","journal-title":"J Am Heart Assoc"},{"key":"2023031717462827900_","doi-asserted-by":"crossref","first-page":"868","DOI":"10.1038\/nmeth.3993","article-title":"A multifunctional AAV-CRISPR-Cas9 and its host response","volume":"13","author":"Chew","year":"2016","journal-title":"Nat Methods"},{"key":"2023031717462827900_","doi-asserted-by":"crossref","first-page":"440","DOI":"10.1016\/j.cell.2014.09.014","article-title":"CRISPR-Cas9 knockin mice for genome editing and cancer modeling","volume":"159","author":"Platt","year":"2014","journal-title":"Cell"},{"key":"2023031717462827900_","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":"2023031717462827900_","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":"2023031717462827900_","doi-asserted-by":"crossref","first-page":"338","DOI":"10.1073\/pnas.1523918113","article-title":"A mouse model for adult cardiac-specific gene deletion with CRISPR\/Cas9","volume":"113","author":"Carroll","year":"2016","journal-title":"Proc Natl Acad Sci USA"},{"key":"2023031717462827900_","doi-asserted-by":"crossref","first-page":"1874","DOI":"10.1161\/CIRCRESAHA.116.310283","article-title":"Analysis of cardiac myocyte maturation using CASAAV, a platform for rapid dissection of cardiac myocyte gene function in vivo","volume":"120","author":"Guo","year":"2017","journal-title":"Circ Res"},{"key":"2023031717462827900_","first-page":"e000043","article-title":"Induced pluripotent stem cells for cardiovascular disease modeling and precision medicine: a scientific statement from the American heart association","volume":"11","author":"Musunuru","year":"2018","journal-title":"Circ Genom Precis Med"},{"key":"2023031717462827900_","doi-asserted-by":"crossref","first-page":"2666","DOI":"10.1161\/CIRCULATIONAHA.117.032273","article-title":"Determining the pathogenicity of a genomic variant of uncertain significance using CRISPR\/Cas9 and human-induced pluripotent stem cells","volume":"138","author":"Ma","year":"2018","journal-title":"Circulation"},{"key":"2023031717462827900_","doi-asserted-by":"crossref","first-page":"2852","DOI":"10.1161\/CIRCULATIONAHA.118.035028","article-title":"Functional annotation of TNNT2 variants of uncertain significance with genome-edited cardiomyocytes","volume":"138","author":"Lv","year":"2018","journal-title":"Circulation"},{"key":"2023031717462827900_","doi-asserted-by":"crossref","first-page":"e002327","DOI":"10.1161\/CIRCGEN.118.002327","article-title":"Confirmation of causal rs9349379-PHACTR1 expression quantitative trait locus in human-induced pluripotent stem cell endothelial cells","volume":"11","author":"Wang","year":"2018","journal-title":"Circ Genom Precis Med"},{"key":"2023031717462827900_","doi-asserted-by":"crossref","first-page":"67","DOI":"10.1038\/nrg.2017.97","article-title":"Am I ready for CRISPR? A user\u2019s guide to genetic screens","volume":"19","author":"Doench","year":"2018","journal-title":"Nat Rev Genet"},{"key":"2023031717462827900_","doi-asserted-by":"crossref","first-page":"e38839","DOI":"10.7554\/eLife.38839","article-title":"The cargo receptor SURF4 promotes the efficient cellular secretion of PCSK9","volume":"7","author":"Emmer","year":"2018","journal-title":"Elife"},{"key":"2023031717462827900_","doi-asserted-by":"crossref","first-page":"e1009285","DOI":"10.1371\/journal.pgen.1009285","article-title":"Genome-scale CRISPR screening for modifiers of cellular LDL uptake","volume":"17","author":"Emmer","year":"2021","journal-title":"PLoS Genet"},{"key":"2023031717462827900_","doi-asserted-by":"crossref","first-page":"100160","DOI":"10.1016\/j.jlr.2021.100160","article-title":"Transgelin: a new gene involved in LDL endocytosis identified by a genome-wide CRISPR-Cas9 screen","volume":"63","author":"Lucero","year":"2021","journal-title":"J Lipid Res"},{"key":"2023031717462827900_","article-title":"CSDE1 is a post-transcriptional regulator of the LDL receptor","author":"Smith","year":"2021","journal-title":"bioRxiv"},{"key":"2023031717462827900_","doi-asserted-by":"crossref","first-page":"4442","DOI":"10.1038\/s41467-021-24743-z","article-title":"Massively parallel in vivo CRISPR screening identifies RNF20\/40 as epigenetic regulators of cardiomyocyte maturation","volume":"12","author":"VanDusen","year":"2021","journal-title":"Nat Commun"},{"key":"2023031717462827900_","doi-asserted-by":"crossref","first-page":"132","DOI":"10.1016\/j.cell.2019.02.015","article-title":"Genetics of common, complex coronary artery disease","volume":"177","author":"Musunuru","year":"2019","journal-title":"Cell"},{"key":"2023031717462827900_","doi-asserted-by":"crossref","first-page":"488","DOI":"10.1161\/CIRCRESAHA.115.304351","article-title":"Permanent alteration of PCSK9 with in vivo CRISPR-Cas9 genome editing","volume":"115","author":"Ding","year":"2014","journal-title":"Circ Res"},{"key":"2023031717462827900_","doi-asserted-by":"crossref","first-page":"416","DOI":"10.1038\/s41586-018-0500-9","article-title":"In vivo CRISPR editing with no detectable genome-wide off-target mutations","volume":"561","author":"Akcakaya","year":"2018","journal-title":"Nature"},{"key":"2023031717462827900_","doi-asserted-by":"crossref","first-page":"783","DOI":"10.1161\/ATVBAHA.116.307227","article-title":"CRISPR-Cas9 targeting of PCSK9 in human hepatocytes in vivo","volume":"36","author":"Wang","year":"2016","journal-title":"Arterioscler Thromb Vasc Biol"},{"key":"2023031717462827900_","article-title":"Global-scale CRISPR gene editor specificity profiling by ONE-seq identifies population-specific, variant off-target effects","author":"Petri","journal-title":"bioRxiv"},{"key":"2023031717462827900_","doi-asserted-by":"crossref","first-page":"440","DOI":"10.1038\/cr.2017.16","article-title":"A non-viral CRISPR\/Cas9 delivery system for therapeutically targeting HBV DNA and pcsk9 in vivo","volume":"27","author":"Jiang","year":"2017","journal-title":"Cell Res"},{"key":"2023031717462827900_","doi-asserted-by":"crossref","DOI":"10.1073\/pnas.2020401118","article-title":"Lipid nanoparticle-mediated codelivery of Cas9 mRNA and single-guide RNA achieves liver-specific in vivo genome editing of Angptl3","volume":"118","author":"Qiu","year":"2021","journal-title":"Proc Natl Acad Sci USA"},{"key":"2023031717462827900_","doi-asserted-by":"crossref","first-page":"1741","DOI":"10.1161\/ATVBAHA.117.309881","article-title":"In vivo base editing of PCSK9 (proprotein convertase subtilisin\/kexin type 9) as a therapeutic alternative to genome editing","volume":"37","author":"Chadwick","year":"2017","journal-title":"Arterioscler Thromb Vasc Biol"},{"key":"2023031717462827900_","doi-asserted-by":"crossref","first-page":"975","DOI":"10.1161\/CIRCULATIONAHA.117.031335","article-title":"Reduced blood lipid levels with in vivo CRISPR-Cas9 base editing of ANGPTL3","volume":"137","author":"Chadwick","year":"2018","journal-title":"Circulation"},{"key":"2023031717462827900_","doi-asserted-by":"crossref","first-page":"1674","DOI":"10.1038\/s41467-018-04048-4","article-title":"RNA-guided transcriptional silencing in vivo with S. aureus CRISPR-Cas9 repressors","volume":"9","author":"Thakore","year":"2018","journal-title":"Nat Commun"},{"key":"2023031717462827900_","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":"2023031717462827900_","first-page":"949","article-title":"In vivo adenine base editing of PCSK9 in macaques reduces LDL cholesterol levels","volume":"39","author":"Rothgangl","year":"2021","journal-title":"."},{"key":"2023031717462827900_","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":"2023031717462827900_","doi-asserted-by":"crossref","first-page":"11","DOI":"10.1056\/NEJMoa1716153","article-title":"Patisiran, an RNAi therapeutic, for hereditary transthyretin amyloidosis","volume":"379","author":"Adams","year":"2018","journal-title":"N Engl J Med"},{"key":"2023031717462827900_","doi-asserted-by":"crossref","first-page":"22","DOI":"10.1056\/NEJMoa1716793","article-title":"Inotersen treatment for patients with hereditary transthyretin amyloidosis","volume":"379","author":"Benson","year":"2018","journal-title":"N Engl J Med"},{"key":"2023031717462827900_","doi-asserted-by":"crossref","first-page":"2227","DOI":"10.1016\/j.celrep.2018.02.014","article-title":"A single administration of CRISPR\/Cas9 lipid nanoparticles achieves robust and persistent in vivo genome editing","volume":"22","author":"Finn","year":"2018","journal-title":"Cell Rep"},{"key":"2023031717462827900_","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":"2023031717462827900_","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":"2023031717462827900_","doi-asserted-by":"crossref","first-page":"407","DOI":"10.1126\/science.aad5177","article-title":"In vivo gene editing in dystrophic mouse muscle and muscle stem cells","volume":"351","author":"Tabebordbar","year":"2016","journal-title":"Science"},{"key":"2023031717462827900_","doi-asserted-by":"crossref","first-page":"923","DOI":"10.1161\/CIRCRESAHA.117.310996","article-title":"In vivo genome editing restores dystrophin expression and cardiac function in dystrophic mice","volume":"121","author":"El Refaey","year":"2017","journal-title":"Circ Res"},{"key":"2023031717462827900_","doi-asserted-by":"crossref","first-page":"400","DOI":"10.1126\/science.aad5725","article-title":"Postnatal genome editing partially restores dystrophin expression in a mouse model of muscular dystrophy","volume":"351","author":"Long","year":"2016","journal-title":"Science"},{"key":"2023031717462827900_","doi-asserted-by":"crossref","first-page":"427","DOI":"10.1038\/s41591-019-0344-3","article-title":"Long-term evaluation of AAV-CRISPR genome editing for Duchenne muscular dystrophy","volume":"25","author":"Nelson","year":"2019","journal-title":"Nat Med"},{"key":"2023031717462827900_","doi-asserted-by":"crossref","first-page":"eabg4910","DOI":"10.1126\/sciadv.abg4910","article-title":"Precise correction of Duchenne muscular dystrophy exon deletion mutations by base and prime editing","volume":"7","author":"Chemello","year":"2021","journal-title":"Sci Adv"},{"key":"2023031717462827900_","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":"2023031717462827900_","doi-asserted-by":"crossref","first-page":"86","DOI":"10.1126\/science.aau1549","article-title":"Gene editing restores dystrophin expression in a canine model of Duchenne muscular dystrophy","volume":"362","author":"Amoasii","year":"2018","journal-title":"Science"},{"key":"2023031717462827900_","doi-asserted-by":"crossref","first-page":"207","DOI":"10.1038\/s41591-019-0738-2","article-title":"Somatic gene editing ameliorates skeletal and cardiac muscle failure in pig and human models of Duchenne muscular dystrophy","volume":"26","author":"Moretti","year":"2020","journal-title":"Nat Med"},{"key":"2023031717462827900_","doi-asserted-by":"crossref","DOI":"10.1038\/s41467-021-26830-7","article-title":"Cas9-specific immune responses compromise local and systemic AAV CRISPR therapy in multiple dystrophic canine models","volume":"12","author":"Hakim","year":"2021","journal-title":"Nat Commun"},{"key":"2023031717462827900_","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":"2023031717462827900_","doi-asserted-by":"crossref","first-page":"4919","DOI":"10.1016\/j.cell.2021.08.028","article-title":"Directed evolution of a family of AAV capsid variants enabling potent muscle-directed gene delivery across species","volume":"184","author":"Tabebordbar","year":"2021","journal-title":"Cell"}],"container-title":["Cardiovascular Research"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/academic.oup.com\/cardiovascres\/advance-article-pdf\/doi\/10.1093\/cvr\/cvac048\/43513393\/cvac048.pdf","content-type":"application\/pdf","content-version":"am","intended-application":"syndication"},{"URL":"https:\/\/academic.oup.com\/cardiovascres\/article-pdf\/119\/1\/79\/49554623\/cvac048.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"syndication"},{"URL":"https:\/\/academic.oup.com\/cardiovascres\/article-pdf\/119\/1\/79\/49554623\/cvac048.pdf","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2023,11,19]],"date-time":"2023-11-19T19:17:21Z","timestamp":1700421441000},"score":1,"resource":{"primary":{"URL":"https:\/\/academic.oup.com\/cardiovascres\/article\/119\/1\/79\/6564520"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2022,4,7]]},"references-count":84,"journal-issue":{"issue":"1","published-online":{"date-parts":[[2022,4,7]]},"published-print":{"date-parts":[[2023,3,17]]}},"URL":"https:\/\/doi.org\/10.1093\/cvr\/cvac048","relation":{},"ISSN":["0008-6363","1755-3245"],"issn-type":[{"value":"0008-6363","type":"print"},{"value":"1755-3245","type":"electronic"}],"subject":[],"published-other":{"date-parts":[[2023,1,1]]},"published":{"date-parts":[[2022,4,7]]}}}