Design of efficacious somatic cell genome editing strategies for recessive and polygenic diseases

This project demonstrates that CRISPR-Cas9-based S1mplex genome editors can be designed and developed to correct two distinct mutant alleles within a single human cell precisely.

本项目表明，基于CRISPR-Cas9的S1mplex基因组编辑器可被设计并开发，以精准校正单个人类细胞内的两种不同突变等位基因。