Matthew Hebden PORTEUS,Melanie Ruth ALLEN,Chad Albert COWAN,Ante Sven LUNDBERG,Michelle I-Ching LIN,Jeffrey LI,Thao Thi NGUYEN
申请号:
US15762700
公开号:
US20180273609A1
申请日:
2016.11.04
申请国别(地区):
US
年份:
2018
代理人:
摘要:
The present application provides materials and methods for treating hemoglobinopathies. More specifically, the application provides methods for producing progenitor cells that are genetically modified via genome editing to increase the production of fetal hemoglobin (HbF), as well as modified progenitor cells (including, for example, CD34+ human hematopoietic stem cells) producing increased levels of HbF, and methods of using such cells for treating hemoglobinopathies such as sickle cell anemia and β-thalassemia.