Intravenous Injection of an AAV-PHP.B Vector Encoding Human Acid α-Glucosidase Rescues Both Muscle and CNS Defects in Murine Pompe Disease

Pompe disease, a severe and often fatal neuromuscular disorder, is caused by a deficiency of the lysosomal enzyme acid alpha-glucosidase (GAA). The disease is characterized by the accumulation of excess glycogen in the heart, skeletal muscle, and CNS. Currently approved enzyme replacement therapy or...

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Bibliografiska uppgifter
I publikationen:Mol Ther Methods Clin Dev
Huvudupphovsmän: Lim, Jeong-A, Yi, Haiqing, Gao, Fengqin, Raben, Nina, Kishnani, Priya S., Sun, Baodong
Materialtyp: Artigo
Språk:Inglês
Publicerad: American Society of Gene & Cell Therapy 2019
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Article
Länkar:https://ncbi.nlm.nih.gov/pmc/articles/PMC6376130/
https://ncbi.nlm.nih.gov/pubmed/30809555
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtm.2019.01.006
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