Replacing the enzyme alpha-L-iduronidase at birth ameliorates symptoms in the brain and periphery of dogs with mucopolysaccharidosis type I
出生时补充α-L-艾杜糖醛酸酶可改善I型粘多糖贮积症犬的脑部和外周症状。
期刊:Science Translational Medicine
影响因子:14.6
doi:10.1126/scitranslmed.3001380
Dierenfeld, Ashley D; McEntee, Michael F; Vogler, Carole A; Vite, Charles H; Chen, Agnes H; Passage, Merry; Le, Steven; Shah, Sahil; Jens, Jackie K; Snella, Elizabeth M; Kline, Karen L; Parkes, Jennifer D; Ware, Wendy A; Moran, Lori E; Fales-Williams, Amanda J; Wengert, Jane A; Whitley, R David; Betts, Daniel M; Boal, Amy M; Riedesel, Elizabeth A; Gross, William; Ellinwood, N Matthew; Dickson, Patricia I