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Both conventional gene therapy and genome editing approaches are being developed, and a great number of human clinical trials are ongoing. Already 2 years ago, the first gene therapy for a neurodegenerative disease, spinal muscular atrophy type 1 (SMA1), obtained market approval. To realize such innovative strategies, gene therapy delivery tools are key assets. Here, we focus on recombinant adeno-associated virus (AAV) vectors and report on strategies to improve first-generation vectors. Current efforts focus on the viral capsid to modify the host\u2013vector interaction aiming at increasing the efficacy of target cell transduction, at simplifying vector administration, and at reducing the risk of vector dose-related side effects.<\/jats:p>","DOI":"10.1089\/hum.2021.169","type":"journal-article","created":{"date-parts":[[2021,10,18]],"date-time":"2021-10-18T19:34:29Z","timestamp":1634585669000},"page":"1096-1119","update-policy":"https:\/\/doi.org\/10.1177\/sage-journals-update-policy","source":"Crossref","is-referenced-by-count":16,"title":["Capsid-Engineering for Central Nervous System-Directed Gene Therapy with Adeno-Associated Virus Vectors"],"prefix":"10.1177","volume":"32","author":[{"given":"Josephine","family":"Macdonald","sequence":"first","affiliation":[{"name":"Institute of Experimental Hematology, Hannover Medical School, Hannover, Germany."},{"name":"REBIRTH Research Center for Translational Regenerative Medicine, Hannover Medical School, Hannover, Germany."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jennifer","family":"Marx","sequence":"additional","affiliation":[{"name":"Institute of Experimental Hematology, Hannover Medical School, Hannover, Germany."},{"name":"REBIRTH Research Center for Translational Regenerative Medicine, Hannover Medical School, Hannover, Germany."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-3365-6933","authenticated-orcid":false,"given":"Hildegard","family":"B\u00fcning","sequence":"additional","affiliation":[{"name":"Institute of Experimental Hematology, Hannover Medical School, Hannover, Germany."},{"name":"REBIRTH Research Center for Translational Regenerative Medicine, Hannover Medical School, Hannover, Germany."}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"179","published-online":{"date-parts":[[2021,10,18]]},"reference":[{"key":"e_1_3_5_2_2","doi-asserted-by":"publisher","DOI":"10.1038\/nature15818"},{"key":"e_1_3_5_3_2","doi-asserted-by":"publisher","DOI":"10.1161\/ATVBAHA.108.175620"},{"key":"e_1_3_5_4_2","doi-asserted-by":"publisher","DOI":"10.3389\/fimmu.2014.00082"},{"key":"e_1_3_5_5_2","unstructured":"Stanton D. 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