I型糖原贮积病

基因治疗

在小鼠和猫糖原贮积症Ia的模型中基因治疗均有效。[15]Ghosh A, Allamarvdasht M, Pan CJ, et al. Long-term correction of murine glycogen storage disease type Ia by recombinant adeno-associated virus-1-mediated gene transfer. Gene Ther. 2006;13:321-329.http://www.ncbi.nlm.nih.gov/pubmed/16195703?tool=bestpractice.com[16]Weinstein DA, Correia CE, Conlon T, et al. Adeno-associated virus-mediated correction of a canine model of glycogen storage disease type 1a. Hum Gene Ther. 2010;21:903-910.http://www.ncbi.nlm.nih.gov/pubmed/20163245?tool=bestpractice.com目前尚无针对人类的基因治疗。