scholarly journals Adeno-Associated Virus-Mediated Correction of a Canine Model of Glycogen Storage Disease Type Ia

2010 ◽  
Vol 21 (7) ◽  
pp. 903-910 ◽  
Author(s):  
David A. Weinstein ◽  
Catherine E. Correia ◽  
Thomas Conlon ◽  
Andrew Specht ◽  
John Verstegen ◽  
...  
Keyword(s):  
Glycogen Storage Disease ◽  
Storage Disease ◽  
Glycogen Storage Disease Type ◽  
Canine Model ◽  
Glycogen Storage ◽  
Disease Type ◽  
Adeno Associated Virus ◽  
Type Ia

scholarly journals Canine Model and Genomic Structural Organization of Glycogen Storage Disease Type Ia (GSD Ia)

2001 ◽  
Vol 38 (1) ◽  
pp. 83-91 ◽  
Author(s):  
P. S. Kishnani ◽  
E. Faulkner ◽  
S. VanCamp ◽  
M. Jackson ◽  
T. Brown ◽  
...  
Keyword(s):  
Glycogen Storage Disease ◽  
Structural Organization ◽  
Storage Disease ◽  
Glycogen Storage Disease Type ◽  
Canine Model ◽  
Glycogen Storage ◽  
Disease Type ◽  
Type Ia

scholarly journals Glycogen Storage Disease Type Ia in Canines: A Model for Human Metabolic and Genetic Liver Disease

2011 ◽  
Vol 2011 ◽  
pp. 1-9 ◽  
Author(s):  
Andrew Specht ◽  
Laurie Fiske ◽  
Kirsten Erger ◽  
Travis Cossette ◽  
John Verstegen ◽  
...  
Keyword(s):  
Glycogen Storage Disease ◽  
Storage Disease ◽  
Clinical Manifestations ◽  
Glycogen Storage Disease Type ◽  
Canine Model ◽  
Glycogen Storage ◽  
Disease Type ◽  
Hepatic Diseases ◽  
Type Ia ◽  
Preclinical Trials

A canine model of Glycogen storage disease type Ia (GSDIa) is described. Affected dogs are homozygous for a previously described M121I mutation resulting in a deficiency of glucose-6-phosphatase-α. Metabolic, clinicopathologic, pathologic, and clinical manifestations of GSDIa observed in this model are described and compared to those observed in humans. The canine model shows more complete recapitulation of the clinical manifestations seen in humans including “lactic acidosis”, larger size, and longer lifespan compared to other animal models. Use of this model in preclinical trials of gene therapy is described and briefly compared to the murine model. Although the canine model offers a number of advantages for evaluating potential therapies for GSDIa, there are also some significant challenges involved in its use. Despite these challenges, the canine model of GSDIa should continue to provide valuable information about the potential for generating curative therapies for GSDIa as well as other genetic hepatic diseases.

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