CHILDRENS MEDICAL CENTER CORPORATION;CHILDREN'S MEDICAL CENTER CORPORATION
发明人:
申请号：
IL24846916
公开号：
IL248469D0
申请日:
2016.10.25
申请国别(地区):
IL
年份:
2016
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.
