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Cas9-specific immune responses compromise local and systemic AAV CRISPR therapy in multiple dystrophic canine models.
Hakim CH, Kumar SRP, Pérez-López DO, Wasala NB, Zhang D, Yue Y, Teixeira J, Pan X, Zhang K, Million ED, Nelson CE, Metzger S, Han J, Louderman JA, Schmidt F, Feng F, Grimm D, Smith BF, Yao G, Yang NN, Gersbach CA, Chen SJ, Herzog RW, Duan D. Hakim CH, et al. Among authors: pan x. Nat Commun. 2021 Nov 24;12(1):6769. doi: 10.1038/s41467-021-26830-7. Nat Commun. 2021. PMID: 34819506 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: pan x. 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.
A Five-Repeat Micro-Dystrophin Gene Ameliorated Dystrophic Phenotype in the Severe DBA/2J-mdx Model of Duchenne Muscular Dystrophy.
Hakim CH, Wasala NB, Pan X, Kodippili K, Yue Y, Zhang K, Yao G, Haffner B, Duan SX, Ramos J, Schneider JS, Yang NN, Chamberlain JS, Duan D. Hakim CH, et al. Among authors: pan x. Mol Ther Methods Clin Dev. 2017 Jul 27;6:216-230. doi: 10.1016/j.omtm.2017.06.006. eCollection 2017 Sep 15. Mol Ther Methods Clin Dev. 2017. PMID: 28932757 Free PMC article.
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