CHILDRENS MEDICAL CENTER CORPORATION;CHILDREN'S MEDICAL CENTER CORPORATION
发明人:
MILSOM, MICHAEL,WILLIAMS, DAVID A.,GREGORY, RICHARD
申请号：
CA2946309
公开号：
CA2946309A1
申请日:
2015.04.24
申请国别(地区):
CA
年份:
2015
代理人:
摘要：
Embodiment herein provide specially designed synthetic BCL11A-targeting microRNAs for RNA polymerase II expression, and methods o use to treat hemoglobinopathies such as sickle cell disease or thalassemia by increasing the expression levels of fetal hemoglobin levels. In particular illustrative embodiment, the present invention provides, in part, improved compositions and methods for achieving gene therapy in hematopoietic cells and hematopoietic precursor cells, including erythrocytes, erythroid progenitors, and embryonic stem cells. The invention further provides improved gene therapy methods for treating hematoppictic-related disorders.
