[HTML][HTML] Correction of glycogen storage disease type 1a in a mouse model by gene therapy
Glycogen storage disease type 1a (GSD-1a), characterized by hypoglycemia, liver and
kidney enlargement, growth retardation, hyperlipidemia, and hyperuricemia, is caused by a
deficiency in glucose-6-phosphatase (G6Pase), a key enzyme in glucose homeostasis. To
evaluate the feasibility of gene replacement therapy for GSD-1a, we have infused adenoviral
vector containing the murine G6Pase gene (Ad-mG6Pase) into G6Pase-deficient
(G6Pase−/−) mice that manifest symptoms characteristic of human GSD-1a. Whereas< 15 …
kidney enlargement, growth retardation, hyperlipidemia, and hyperuricemia, is caused by a
deficiency in glucose-6-phosphatase (G6Pase), a key enzyme in glucose homeostasis. To
evaluate the feasibility of gene replacement therapy for GSD-1a, we have infused adenoviral
vector containing the murine G6Pase gene (Ad-mG6Pase) into G6Pase-deficient
(G6Pase−/−) mice that manifest symptoms characteristic of human GSD-1a. Whereas< 15 …