Large animal models and new therapies for glycogen storage disease
Author:
Publisher
Wiley
Subject
Genetics(clinical),Genetics
Link
http://link.springer.com/content/pdf/10.1007/s10545-014-9766-8
Reference30 articles.
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3. Brooks ED, Little D, Arumugam R et al (2013) Pathogenesis of growth failure and partial reversal with gene therapy in murine and canine glycogen storage disease type Ia. Mol Genet Metab 109(2):161–170
4. Crane B, Luo X, Demaster A et al (2012) Rescue administration of a helper-dependent adenovirus vector with long-term efficacy in dogs with glycogen storage disease type Ia. Gene Ther 19(4):443–452
5. Cunningham SC, Dane AP, Spinoulas A, Logan GJ, Alexander IE (2008) Gene delivery to the juvenile mouse liver using AAV2/8 vectors. Mol Ther 16(6):1081–1088
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