Neonatal Gene Therapy of Glycogen Storage Disease Type Ia Using a Feline Immunodeficiency Virus–based Vector

Glycogen storage disease type Ia (GSD-Ia), also known as von Gierke disease, is caused by a deficiency of glucose-6-phosphatase-α (G6Pase), a key enzyme in glucose homeostasis. From birth, affected individuals cannot maintain normal blood glucose levels and suffer from a variety of metabolic disorde...

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Bibliographische Detailangaben
Hauptverfasser: Grinshpun, Albert, Condiotti, Reba, N Waddington, Simon, Peer, Michael, Zeig, Eli, Peretz, Sima, Simerzin, Alina, Chou, Janice, Pann, Chi-Jiunn, Giladi, Hilla, Galun, Eithan
Format: Artigo
Sprache:Inglês
Veröffentlicht: Nature Publishing Group 2010
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Original Article
Online Zugang:https://ncbi.nlm.nih.gov/pmc/articles/PMC2956916/
https://ncbi.nlm.nih.gov/pubmed/20571544
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2010.119
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