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...

Descripción completa

Detalles Bibliográficos
Autores principales: Goina, Elisa, Peruzzo, Paolo, Bembi, Bruno, Dardis, Andrea, Buratti, Emanuele
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2017
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5589062/
https://www.ncbi.nlm.nih.gov/pubmed/28629821
http://dx.doi.org/10.1016/j.ymthe.2017.05.019

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5589062/
https://www.ncbi.nlm.nih.gov/pubmed/28629821
http://dx.doi.org/10.1016/j.ymthe.2017.05.019

Ejemplares similares