{"doi":"10.1126/sciadv.aay6812","title":"Enhanced CRISPR-Cas9 correction of Duchenne muscular dystrophy in mice by a self-complementary AAV delivery system","abstract":"). Previously, we applied CRISPR-Cas9-mediated \"single-cut\" genome editing to correct diverse genetic mutations in animal models of DMD. However, high doses of adeno-associated virus (AAV) are required for efficient in vivo genome editing, posing challenges for clinical application. In this study, we packaged Cas9 nuclease in single-stranded AAV (ssAAV) and CRISPR single guide RNAs in self-complementary AAV (scAAV) and delivered this dual AAV system into a mouse model of DMD. The dose of scAAV required for efficient genome editing were at least 20-fold lower than with ssAAV. Mice receiving systemic treatment showed restoration of dystrophin expression and improved muscle contractility. These findings show that the efficiency of CRISPR-Cas9-mediated genome editing can be substantially improved by using the scAAV system. This represents an important advancement toward therapeutic translation of genome editing for DMD.","journal":"Science Advances","year":2020,"id":51255,"datarank":0.0,"base_score":0.0,"endowment":0.0,"self_citation_contribution":0.0,"citation_network_contribution":0.0,"self_endowment_contribution":0.0,"citer_contribution":0.0,"corpus_percentile":null,"corpus_rank":null,"citation_count":162,"citer_count":0,"citers_with_citation_signal":0,"citers_with_endowment":0,"datacite_reuse_total":0,"is_dataset":false,"is_dataset_confidence":0.9421,"is_data_producer":false,"deposit_databanks":null,"is_oa":true,"file_count":0,"downloads":0,"has_version_chain":false,"published_date":"2020-01-01","fair_score":null,"fair_percentile":null,"algorithm_id":"datarank_citation_only_1hop_v6","ranking_scope":"data_only","authors":[{"id":251166,"name":"Hui Li","orcid":"0000-0002-4114-8349","position":1,"is_corresponding":false},{"id":225270,"name":"Yi-Li Min","orcid":"0000-0001-6205-9428","position":2,"is_corresponding":false},{"id":251165,"name":"Efrain Sanchez‐Ortiz","orcid":"0000-0001-9358-1630","position":3,"is_corresponding":false},{"id":255297,"name":"Jian Huang","orcid":"0000-0003-3282-8892","position":4,"is_corresponding":false},{"id":255298,"name":"Alex A. Mireault","orcid":"0000-0002-7534-636X","position":5,"is_corresponding":false},{"id":255299,"name":"John M. Shelton","orcid":"0000-0002-2949-1638","position":6,"is_corresponding":false},{"id":255300,"name":"Jiwoong Kim","orcid":"0000-0003-0220-9329","position":7,"is_corresponding":false},{"id":255301,"name":"Pradeep P.A. Mammen","orcid":"0000-0001-5688-7091","position":8,"is_corresponding":false},{"id":251128,"name":"Rhonda Bassel‐Duby","orcid":"0000-0001-7568-410X","position":9,"is_corresponding":false},{"id":225271,"name":"Eric N. Olson","orcid":"0000-0003-1151-8262","position":10,"is_corresponding":false},{"id":255296,"name":"Yu Zhang","orcid":"0000-0003-3384-8887","position":0,"is_corresponding":true}],"reference_count":58,"raw_metadata":null,"created_at":"2026-07-18T20:40:28.278215Z","pmid":"32128412","pmcid":null,"fwci":null,"citation_percentile":null,"influential_citations":0,"oa_status":null,"license":null,"views":0,"total_file_size_bytes":0,"version_count":0,"fair_f":null,"fair_a":null,"fair_i":null,"fair_r":null,"fair_zscore":null,"fair_rationale":null,"fair_model":null,"fair_agent_version":null,"fair_fulltext_source":null,"fair_has_llm":null,"fair_computed_at":null,"clinical_trials":[],"software_tools":[],"db_accessions":[],"linked_datasets":[],"topics":[]}