Pathogenesis of growth failure and partial reversal with gene therapy in murine and canine Glycogen Storage Disease type Ia

Glycogen Storage Disease type Ia (GSD-Ia) in humans frequently causes delayed bone maturation, decrease in final adult height, and decreased growth velocity. This study evaluates the pathogenesis of growth failure and the effect of gene therapy on growth in GSD-Ia affected dogs and mice. Here we fou...

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Main Authors: Brooks, Elizabeth Drake, Little, Dianne, Arumugam, Ramamani, Sun, Baodong, Curtis, Sarah, DeMaster, Amanda, Maranzano, Michael, Jackson, Mark W., Kishnani, Priya, Freemark, Michael S., Koeberl, Dwight D.
Format: Artigo
Jezik:Inglês
Izdano: 2013
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Article
Online dostop:https://ncbi.nlm.nih.gov/pmc/articles/PMC3764490/
https://ncbi.nlm.nih.gov/pubmed/23623482
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymgme.2013.03.018
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