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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Publicado en:J Inherit Metab Dis
Autores principales: 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.
Formato: Artigo
Lenguaje:Inglês
Publicado: 2018
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Article
Acceso en línea: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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