| 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. |
