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AAV CRISPR editing rescues cardiac and muscle function for 18 months in dystrophic mice.
JCI Insight. 2018 Dec 6;3(23):e124297. doi: 10.1172/jci.insight.124297.
JCI Insight. 2018.
PMID: 30518686
Free PMC article.
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: louderman ja.
Nat Commun. 2021 Nov 24;12(1):6769. doi: 10.1038/s41467-021-26830-7.
Nat Commun. 2021.
PMID: 34819506
Free PMC article.
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