Long-term safety and efficacy of AAV gene therapy in the canine model of glycogen storage disease type Ia

ConclusionrAAV-GPE-hG6PC treatment in GSD-Ia dogs was found to be safe and efficacious. GSD-Ia is an attractive target for human gene therapy since it is a monogenic disorder with limited tissue involvement. Blood glucose and lactate monitoring can be used to assess effectiveness and as a biomarker of success. GSD-Ia can also serve as a model for other hepatic monogenic disorders.
Source: Journal of Inherited Metabolic Disease - Category: Internal Medicine Source Type: research