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Next, we boosted muscle specificity by displaying a myotropic peptide on the capsid surface. In a mouse model of X-linked myotubular myopathy, the best vectors\u2014AAVMYO2 and AAVMYO3\u2014prolonged survival, corrected growth, restored strength, and ameliorated muscle fiber size and centronucleation. In a mouse model of Duchenne muscular dystrophy, our lead capsid induced robust microdystrophin expression and improved muscle function. Our pipeline is compatible with complementary AAV genome bioengineering strategies, as demonstrated here with two promoters, and could benefit many clinical applications beyond muscle gene therapy.<\/jats:p>","DOI":"10.1126\/sciadv.abn4704","type":"journal-article","created":{"date-parts":[[2022,9,21]],"date-time":"2022-09-21T13:58:34Z","timestamp":1663768714000},"source":"Crossref","is-referenced-by-count":96,"title":["Semirational bioengineering of AAV vectors with increased potency and specificity for systemic gene therapy of muscle disorders"],"prefix":"10.1126","volume":"8","author":[{"ORCID":"https:\/\/orcid.org\/0000-0001-5502-6214","authenticated-orcid":true,"given":"Jihad","family":"El Andari","sequence":"first","affiliation":[{"name":"Medical Faculty, Department of Infectious Diseases\/Virology, Section Viral Vector Technologies, Cluster of Excellence CellNetworks, University of Heidelberg, 69120 Heidelberg, Germany."},{"name":"BioQuant, University of Heidelberg, 69120 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