Long-term Complications of Glycogen Storage Disease Type Ia in the Canine Model Treated with Gene Replacement Therapy

BACKGROUND: Glycogen storage disease type Ia (GSD Ia) in dogs closely resembles human GSD Ia. Untreated patients with GSD Ia develop complications associated with glucose-6-phosphatase (G6Pase) deficiency. Survival of human patients on intensive nutritional management has improved; however, long-ter...

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發表在:J Inherit Metab Dis
Main Authors: Brooks, Elizabeth D., Landau, Dustin J., Everitt, Jeffrey I., Brown, Talmage T., Grady, Kylie M., Waskowicz, Lauren, Bass, Cameron R., D’Angelo, John, Asfaw, Yohannes G., Williams, Kyha, Kishnani, Priya S., Koeberl, Dwight D.
格式: Artigo
語言:Inglês
出版: 2018
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Article
在線閱讀:https://ncbi.nlm.nih.gov/pmc/articles/PMC6328337/
https://ncbi.nlm.nih.gov/pubmed/30043186
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1007/s10545-018-0223-y
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