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Micro-dystrophin AAV Vectors Made by Transient Transfection and Herpesvirus System Are Equally Potent in Treating mdx Mouse Muscle Disease.
Hakim CH, Clément N, Wasala LP, Yang HT, Yue Y, Zhang K, Kodippili K, Adamson-Small L, Pan X, Schneider JS, Yang NN, Chamberlain JS, Byrne BJ, Duan D. Hakim CH, et al. Among authors: wasala lp. Mol Ther Methods Clin Dev. 2020 Jul 9;18:664-678. doi: 10.1016/j.omtm.2020.07.004. eCollection 2020 Sep 11. Mol Ther Methods Clin Dev. 2020. PMID: 32775499 Free PMC article.
Dystrophin contains multiple independent membrane-binding domains.
Zhao J, Kodippili K, Yue Y, Hakim CH, Wasala L, Pan X, Zhang K, Yang NN, Duan D, Lai Y. Zhao J, et al. Among authors: wasala l. Hum Mol Genet. 2016 Sep 1;25(17):3647-3653. doi: 10.1093/hmg/ddw210. Epub 2016 Jul 4. Hum Mol Genet. 2016. PMID: 27378693 Free PMC article.
AAV CRISPR editing rescues cardiac and muscle function for 18 months in dystrophic mice.
Hakim CH, Wasala NB, Nelson CE, Wasala LP, Yue Y, Louderman JA, Lessa TB, Dai A, Zhang K, Jenkins GJ, Nance ME, Pan X, Kodippili K, Yang NN, Chen SJ, Gersbach CA, Duan D. Hakim CH, et al. Among authors: wasala lp. JCI Insight. 2018 Dec 6;3(23):e124297. doi: 10.1172/jci.insight.124297. JCI Insight. 2018. PMID: 30518686 Free PMC article.
18 results