MORAWA, EWELINA,CHAKRABORTY, TIRTHA,LUNDBERG, ANTE SVEN,HO, TONY,SANDLER, LAURA,EUSTACE, BRENDA,ROSSERT, JEROME,KAUFFMAN, ROBERT
申请号：
CA3084955
公开号：
CA3084955A1
申请日:
2018.12.05
申请国别(地区):
CA
年份:
2019
代理人:
摘要：
Provided herein, in some embodiments, are methods and compositions for treatment of subjects with ß-thalassemia and subjects with severe sickle cell disease using autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem and progenitor cells.
