Lentiviral Hematopoietic Stem Cell Gene Therapy Corrects Murine Pompe Disease

Pompe disease is an autosomal recessive lysosomal storage disorder characterized by progressive muscle weakness. The disease is caused by mutations in the acid α-glucosidase (GAA) gene. Despite the currently available enzyme replacement therapy (ERT), roughly half of the infants with Pompe disease d...

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Detalhes bibliográficos
Publicado no:Mol Ther Methods Clin Dev
Main Authors: Stok, Merel, de Boer, Helen, Huston, Marshall W., Jacobs, Edwin H., Roovers, Onno, Visser, Trudi P., Jahr, Holger, Duncker, Dirk J., van Deel, Elza D., Reuser, Arnold J.J., van Til, Niek P., Wagemaker, Gerard
Formato: Artigo
Idioma:Inglês
Publicado em: American Society of Gene & Cell Therapy 2020
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Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC7240064/
https://ncbi.nlm.nih.gov/pubmed/32462050
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtm.2020.04.023
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