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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Pubblicato in:J Inherit Metab Dis
Autori principali: 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.
Natura: Artigo
Lingua:Inglês
Pubblicazione: 2018
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Article
Accesso online: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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