A Novel Gene Therapy Approach for GSD III Using an AAV Vector Encoding a Bacterial Glycogen Debranching Enzyme

Glycogen storage disease type III (GSD III) is an inherited disorder caused by a deficiency of glycogen debranching enzyme (GDE), which results in the accumulation of abnormal glycogen (limit dextrin) in the cytoplasm of liver, heart, and skeletal muscle cells. Currently, there is no curative treatm...

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Detalles Bibliográficos
Autores principales: Lim, Jeong-A, Choi, Su Jin, Gao, Fengqin, Kishnani, Priya S., Sun, Baodong
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2020
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7327847/
https://www.ncbi.nlm.nih.gov/pubmed/32637453
http://dx.doi.org/10.1016/j.omtm.2020.05.034

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7327847/
https://www.ncbi.nlm.nih.gov/pubmed/32637453
http://dx.doi.org/10.1016/j.omtm.2020.05.034

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