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Glycogen Reduction in Myotubes of Late-Onset Pompe Disease Patients Using Antisense Technology

Glycogen storage disease type II (GSDII) is a lysosomal disorder caused by the deficient activity of acid alpha-glucosidase (GAA) enzyme, leading to the accumulation of glycogen within the lysosomes. The disease has been classified in infantile and late-onset forms. Most late-onset patients share a...

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Detalhes bibliográficos
Publicado no:Mol Ther
Main Authors: Goina, Elisa, Peruzzo, Paolo, Bembi, Bruno, Dardis, Andrea, Buratti, Emanuele
Formato: Artigo
Idioma:Inglês
Publicado em: American Society of Gene & Cell Therapy 2017
Assuntos:
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC5589062/
https://ncbi.nlm.nih.gov/pubmed/28629821
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymthe.2017.05.019
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