Large animal models and new therapies for glycogen storage disease

Correction of glycogen storage disease type III with rapamycin in a canine model

(2014) Haiqing Yi et al.   JOURNAL OF MOLECULAR MEDICINE-JMM

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

(2013) Elizabeth Drake Brooks et al.   MOLECULAR GENETICS AND METABOLISM

Phosphorylase re-expression, increase in the force of contraction and decreased fatigue following notexin-induced muscle damage and regeneration in the ovine model of McArdle disease

(2013) J.McC. Howell et al.   NEUROMUSCULAR DISORDERS

A Nonsense Mutation in the Acid α-Glucosidase Gene Causes Pompe Disease in Finnish and Swedish Lapphunds

(2013) Eija H. Seppälä et al.   PLoS One

Characterization of a canine model of glycogen storage disease type IIIa

(2012) H. Yi et al.   Disease Models & Mechanisms

Rescue administration of a helper-dependent adenovirus vector with long-term efficacy in dogs with glycogen storage disease type Ia

(2011) B Crane et al.   GENE THERAPY

Long-Term Efficacy Following Readministration of an Adeno-Associated Virus Vector in Dogs with Glycogen Storage Disease Type Ia

(2011) Amanda Demaster et al.   HUMAN GENE THERAPY

Hepatorenal Correction in Murine Glycogen Storage Disease Type I With a Double-stranded Adeno-associated Virus Vector

(2011) Xiaoyan Luo et al.   MOLECULAR THERAPY

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

(2010) David A. Weinstein et al.   HUMAN GENE THERAPY

Complete Normalization of Hepatic G6PC Deficiency in Murine Glycogen Storage Disease Type Ia Using Gene Therapy

(2010) Wai Han Yiu et al.   MOLECULAR THERAPY

Gene Delivery to the Juvenile Mouse Liver Using AAV2/8 Vectors

(2008) Sharon C Cunningham et al.   MOLECULAR THERAPY

AAV Vector-mediated Reversal of Hypoglycemia in Canine and Murine Glycogen Storage Disease Type Ia

(2008) Dwight D Koeberl et al.   MOLECULAR THERAPY

G.P.16.06. Investigation of possible treatment regimes for McArdle’s disease using the sheep model of the disease

(2008) J.M. Howell et al.   NEUROMUSCULAR DISORDERS

Adenovirus and adeno-associated virus-mediated delivery of human myophosphorylase cDNA and LacZ cDNA to muscle in the ovine model of McArdle’s disease: Expression and re-expression of glycogen phosphorylase

(2008) J.McC. Howell et al.   NEUROMUSCULAR DISORDERS