申请人:Sangamo BioSciences, Inc.
公开号:US10648001B2
公开(公告)日:2020-05-12
Nucleases and methods of using these nucleases for inserting a sequence encoding a therapeutic protein such as an enzyme into a cell, thereby providing proteins or cell therapeutics for treatment and/or prevention of a lysosomal storage disease.
核酸酶以及使用这些核酸酶将编码治疗性蛋白质(如酶)的序列插入细胞的方法,从而提供用于治疗和/或预防溶酶体贮积症的蛋白质或细胞疗法。