Long-term complications of glycogen storage disease type Ia in the canine model treated with gene replacement therapy

ConclusionsHere, we show that the canine GSD Ia model demonstrates similar long-term complications as GSD Ia patients in spite of gene replacement therapy. Further development of gene therapy is needed to develop a more effective treatment to prevent long-term complications of GSD Ia.
Source: Journal of Inherited Metabolic Disease - Category: Internal Medicine Source Type: research