A long-term study of AAV gene therapy in dogs with hemophilia A identifies clonal expansions of transduced liver cells
对患有血友病 A 的狗进行 AAV 基因治疗的长期研究发现，转导肝细胞出现克隆扩增
期刊:Nature Biotechnology
影响因子:33.1
doi:10.1038/s41587-020-0741-7
Giang N Nguyen #, John K Everett #, Samita Kafle, Aoife M Roche, Hayley E Raymond, Jacob Leiby, Christian Wood, Charles-Antoine Assenmacher, Elizabeth P Merricks, C Tyler Long, Haig H Kazazian, Timothy C Nichols, Frederic D Bushman, Denise E Sabatino
