GORI, JENNIFER LEAH,AUPEPIN DE LAMOTHE-DREUZY, EDOUARD,HEATH, JACK,ZURIS, JOHN ANTHONY,CHANG, KAIHSIN
申请号：
CA3093702
公开号：
CA3093702A1
申请日:
2019.03.14
申请国别(地区):
CA
年份:
2019
代理人:
摘要：
Genome editing systems, guide RNAs, and CRISPR-mediated methods are provided for altering portions of the HBG1 and HBG2 loci, portions of the erythroid specific enhancer of the BCL11A gene, or a combination thereof, in cells and increasing expression of fetal hemoglobin.
