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Current Clinical Applications of In Vivo Gene Therapy with AAVs.
Mendell JR, Al-Zaidy SA, Rodino-Klapac LR, Goodspeed K, Gray SJ, Kay CN, Boye SL, Boye SE, George LA, Salabarria S, Corti M, Byrne BJ, Tremblay JP. Mendell JR, et al. Among authors: tremblay jp. Mol Ther. 2021 Feb 3;29(2):464-488. doi: 10.1016/j.ymthe.2020.12.007. Epub 2020 Dec 10. Mol Ther. 2021. PMID: 33309881 Free PMC article. Review.
Expression of dog microdystrophin in mouse and dog muscles by gene therapy.
Pichavant C, Chapdelaine P, Cerri DG, Dominique JC, Quenneville SP, Skuk D, Kornegay JN, Bizario JC, Xiao X, Tremblay JP. Pichavant C, et al. Among authors: tremblay jp. Mol Ther. 2010 May;18(5):1002-9. doi: 10.1038/mt.2010.23. Epub 2010 Feb 23. Mol Ther. 2010. PMID: 20179674 Free PMC article.
294 results