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

BACKGROUND: Pompe disease (PD) is a neuromuscular disorder caused by deficiency of acidalpha-glucosidase (GAA), leading to motor and respiratory dysfunctions. Available Gaa knock-out (KO) mouse models do not accurately mimic PD, particularly its highly impaired respiratory phenotype. METHODS: Here w...

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Gepubliceerd in:EBioMedicine
Hoofdauteurs: Colella, Pasqualina, Sellier, Pauline, Gomez, Manuel J., Biferi, Maria G., Tanniou, Guillaume, Guerchet, Nicolas, Cohen-Tannoudji, Mathilde, Moya-Nilges, Maryse, van Wittenberghe, Laetitia, Daniele, Natalie, Gjata, Bernard, Krijnse-Locker, Jacomina, Collaud, Fanny, Simon-Sola, Marcelo, Charles, Severine, Cagin, Umut, Mingozzi, Federico
Formaat: Artigo
Taal:Inglês
Gepubliceerd in: Elsevier 2020
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Research Paper
Online toegang:https://ncbi.nlm.nih.gov/pmc/articles/PMC7553357/
https://ncbi.nlm.nih.gov/pubmed/33039711
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ebiom.2020.103052
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