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

Young Mok LeeThomas J ConlonAndrew SpechtKirsten E ColemanLaurie M BrownAna M EstrellaMonika DambskaKathryn R DahlbergWeinstein DA
Published in: Journal of inherited metabolic disease (2018)
rAAV-GPE-hG6PC treatment in GSD-Ia dogs was found to be safe and efficacious. GSD-Ia is an attractive target for human gene therapy since it is a monogenic disorder with limited tissue involvement. Blood glucose and lactate monitoring can be used to assess effectiveness and as a biomarker of success. GSD-Ia can also serve as a model for other hepatic monogenic disorders.
Keyphrases
  • gene therapy
  • blood glucose
  • endothelial cells
  • randomized controlled trial
  • glycemic control
  • type diabetes
  • blood pressure
  • induced pluripotent stem cells
  • skeletal muscle
  • combination therapy
  • smoking cessation