Peer-reviewed veterinary case report
Gene therapy helped dogs with glycogen storage disease type Ia live
By Crane, B et al.·Published in Gene therapy·2012·College of Veterinary Medicine, United States·View original on PubMed →
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Original publication title: Rescue administration of a helper-dependent adenovirus vector with long-term efficacy in dogs with glycogen storage disease type Ia.
- Species:
- dog
Plain-English summary
Three dogs with glycogen storage disease type Ia (GSD-Ia), which causes low blood sugar and liver problems, were treated with a special virus designed to help their bodies produce a missing enzyme. Initially, this treatment helped keep their blood sugar levels normal. However, after several months, the dogs started showing signs of low blood sugar again, along with loss of appetite and lethargy. A second treatment with a different version of the virus helped reverse the low blood sugar in two of the dogs, allowing one to live up to 36 months, but ongoing health issues made management difficult.
People also search for: dog glycogen storage disease treatment · low blood sugar in dogs · GSD-Ia symptoms in dogs
Abstract
Glycogen storage disease type Ia (GSD-Ia) stems from glucose-6-phosphatase (G6Pase) deficiency and causes hypoglycemia, hepatomegaly, hypercholesterolemia and lactic acidemia. Three dogs with GSD-Ia were initially treated with a helper-dependent adenovirus encoding a human G6Pase transgene (HDAd-cG6Pase serotype 5) on postnatal day 3. Unlike untreated dogs with GSD-Ia, all three dogs initially maintained normal blood glucose levels. After 6-22 months, vector-treated dogs developed hypoglycemia, anorexia and lethargy, suggesting that the HDAd-cG6Pase serotype 5 vector had lost efficacy. Liver biopsies collected at this time revealed significantly elevated hepatic G6Pase activity and reduced glycogen content, when compared with affected dogs treated only by frequent feeding. Subsequently, the HDAd-cG6Pase serotype 2 vector was administered to two dogs, and hypoglycemia was reversed; however, renal dysfunction and recurrent hypoglycemia complicated their management. Administration of a serotype 2 HDAd vector prolonged survival in one GSD-Ia dog to 12 months of age and 36 months of age in the other, but the persistence of long-term complications limited HDAd vectors in the canine model for GSD-Ia.
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Search related cases →Original publication on PubMed: https://pubmed.ncbi.nlm.nih.gov/21654821/