MILSOM, Michael,WILLIAMS, David A.,GREGORY, Richard
申请号：
DK15783357
公开号：
DK3134130T3
申请日:
2015.04.24
申请国别(地区):
DK
年份:
2019
代理人:
摘要：
Embodiments herein provide specially designed synthetic BCL11A-targeting microRNAs for RNA polymerase II expression, and methods of use to treat hemoglobinopathies such as sickle cell disease or thalassemia by increasing the expression levels of fetal hemoglobin levels.
