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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: salabarria s. 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.
Advancements in AAV-mediated Gene Therapy for Pompe Disease.
Salabarria SM, Nair J, Clement N, Smith BK, Raben N, Fuller DD, Byrne BJ, Corti M. Salabarria SM, et al. J Neuromuscul Dis. 2020;7(1):15-31. doi: 10.3233/JND-190426. J Neuromuscul Dis. 2020. PMID: 31796685 Free PMC article. Review.