Correction in trans for Fabry disease: expression, secretion and uptake of alpha-galactosidase A in patient-derived cells driven by a high-titer recombinant retroviral vector
法布里病相关更正:高滴度重组逆转录病毒载体驱动患者来源细胞中α-半乳糖苷酶A的表达、分泌和摄取
期刊:Proceedings of the National Academy of Sciences of the United States of America
影响因子:9.1
doi:10.1073/pnas.93.15.7917
Medin, J A; Tudor, M; Simovitch, R; Quirk, J M; Jacobson, S; Murray, G J; Brady, R O