Long‐term complications of glycogen storage disease type Ia in the canine model treated with gene replacement therapy
Brooks, E. D., Landau, D. J., Everitt, J. I., Brown, T. T., Grady, K. M., Waskowicz, L., … Koeberl, D. D. (2018, July 24). Journal of Inherited Metabolic Disease.
topics (OpenAlex): Glycogen Storage Diseases and Myoclonus; Genetics and Neurodevelopmental Disorders; Oral and gingival health research
TL;DR:
It is shown that the canine G SD Ia model demonstrates similar long-term complications as GSD Ia patients in spite of gene replacement therapy, and further development of gene therapy is needed to develop a more effective treatment to prevent long- term complications of GSDIa.
(via Semantic Scholar)
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