Gene therapy with secreted acid alpha-glucosidase rescues Pompe disease in a novel mouse model with early-onset spinal cord and respiratory defects.

Added 585 days ago (12.10.2020)
Authors: Colella P, Sellier P, Gomez MJ, Biferi MG, Tanniou G, Guerchet N, Cohen-Tannoudji M, Moya-Nilges M, van Wittenberghe L, Daniele N, Gjata B, Krijnse-Locker J, Collaud F, Simon-Sola M, Charles S, Cagin U, Mingozzi F
PubMed id: 33039711
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