MORAWA, Ewelina,CHAKRABORTY, Tirtha,LUNDBERG, Ante Sven,HO, Tony,SANDLER, Laura,EUSTACE, Brenda,ROSSERT, Jerome,KAUFFMAN, Robert
申请号：
AU2018378479
公开号：
AU2018378479A1
申请日:
2018.12.05
申请国别(地区):
AU
年份:
2020
代理人:
摘要：
Provided herein, in some embodiments, are methods and compositions for treatment of subjects with β-thalassemia and subjects with severe sickle cell disease using autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem and progenitor cells.
