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

25 May 2018

Abstract excerpt

BACKGROUND: Viral mediated gene therapy has progressed after overcoming early failures, and gene therapy has now been approved for several conditions in Europe and the USA. Glycogen storage disease (GSD) type Ia, caused by a deficiency of glucose-6-phosphatase-α, has been viewed as an outstanding candidate for gene therapy. This follow-up report describes the long-term outcome for the naturally occurring GSD-Ia...

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PMID: 29802554
DOI: 10.1007/s10545-018-0199-7

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