Rescue of Advanced Pompe Disease in Mice with Hepatic Expression of Secretable Acid α-Glucosidase

Pompe disease is a neuromuscular disorder caused by disease-associated variants in the gene encoding for the lysosomal enzyme acid α-glucosidase (GAA), which converts lysosomal glycogen to glucose. We previously reported full rescue of Pompe disease in symptomatic 4-month-old Gaa knockout (Gaa(−/−))...

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Detalles Bibliográficos
Autores principales: Cagin, Umut, Puzzo, Francesco, Gomez, Manuel Jose, Moya-Nilges, Maryse, Sellier, Pauline, Abad, Catalina, Van Wittenberghe, Laetitia, Daniele, Nathalie, Guerchet, Nicolas, Gjata, Bernard, Collaud, Fanny, Charles, Severine, Sola, Marcelo Simon, Boyer, Olivier, Krijnse-Locker, Jacomina, Ronzitti, Giuseppe, Colella, Pasqualina, Mingozzi, Federico
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2020
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7474269/
https://www.ncbi.nlm.nih.gov/pubmed/32526204
http://dx.doi.org/10.1016/j.ymthe.2020.05.025

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