AAV gene therapy corrects OTC deficiency and prevents liver fibrosis in aged OTC-knock out heterozygous mice
AAV基因疗法可纠正老年OTC敲除杂合子小鼠的OTC缺陷并预防肝纤维化。
期刊:Molecular Genetics and Metabolism
影响因子:3.5
doi:10.1016/j.ymgme.2017.02.011
Wang, Lili; Bell, Peter; Morizono, Hiroki; He, Zhenning; Pumbo, Elena; Yu, Hongwei; White, John; Batshaw, Mark L; Wilson, James M