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

Elizabeth D BrooksDustin J LandauJeffrey I EverittTalmage T BrownKylie M GradyLauren WaskowiczCameron R BassJohn D'AngeloYohannes G AsfawKyha WilliamsPriya S KishnaniDwight D Koeberl

Published in: Journal of inherited metabolic disease (2018)

Here, 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.

Keyphrases

replacement therapy
smoking cessation
gene therapy
end stage renal disease
risk factors
newly diagnosed
genome wide
ejection fraction
copy number
chronic kidney disease
peritoneal dialysis
prognostic factors
gene expression
transcription factor
genome wide analysis