Xu, Jian;Cong, Peng;Childrens Medical Center Corporation;Children's Medical Center Corporation;Orkin, Stuart H.
发明人:
申请号：
EP12840096.7
公开号：
EP2766041B1
申请日:
2012.10.12
申请国别(地区):
EP
年份:
2018
代理人:
摘要：
Provided herein are compositions and methods for increasing fetal hemoglobin expression (HbF) for the treatment and/or amelioration of the symptoms of a hemoglobinpathy in a mammal. The compositions comprise a combination of one or more inhibitors of BCL11A expression or activity with epigenetic modifiers, such as inhibitors of DNA methylation and/or one or more inhibitors of histone deacetylases.
