上海科技大学知识管理系统

提供了基因治疗技术，包括针对改进的碱基编辑器特别设计和测试的指导RNA序列，其可用于增加γ-珠蛋白基因的表达。 指导RNA序列可以同时靶向BCL11A红系增强子或γ-珠蛋白启动子，或两者。 碱基编辑器可以包括核碱基脱氨酶抑制剂，其具有碱基编辑器的编辑活性，直到它们与靶位点结合。 这些基因治疗技术可用于治疗疾病，包括β-地中海贫血和镰状细胞贫血等。

Provided are gene therapy technologies, including specifically designed and tested guide RNA sequences for improved base editors, useful for increasing the expression of the gamma-globin gene. The guide RNA sequences may target the BCL11A erythroid enhancer or the gamma-globin promoter, or both at the same time. The base editors can include nucleobase deaminase inhibitor that inhibits the editing activity of the base editors until they are bound to the target sites. These gene therapy technologies are useful for treating diseases including beta-thalassemia and sickle cell anemia, among others.