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• W2064841089 abstract "The emergence of life threatening long-term complications in glycogen storage disease type Ia (GSD-Ia) has emphasized the need for new therapies, such as gene therapy, which could achieve biochemical correction of glucose-6-phosphatase deficiency and reverse clinical involvement. We have developed gene therapy with a novel adeno-associated virus (AAV) vector that: 1) prevented mortality and corrected glycogen storage in the liver, 2) corrected hypoglycemia during fasting, and 3) achieved efficacy with a low number of vector particles in G6Pase-deficient mice and dogs. However, the gradual loss of transgene expression from episomal AAV vector genomes eventually necessitated the administration of a different pseudotype of the AAV vector to sustain dogs with GSD-Ia. Further preclinical development of AAV vector-mediated gene therapy is therefore warranted in GSD-Ia." @default.
• W2064841089 created "2016-06-24" @default.
• W2064841089 creator A5045419573 @default.
• W2064841089 date "2012-02-07" @default.
• W2064841089 modified "2023-09-25" @default.
• W2064841089 title "In search of proof‐of‐concept: gene therapy for glycogen storage disease type Ia" @default.
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• W2064841089 doi "https://doi.org/10.1007/s10545-012-9454-5" @default.
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