Compositions and methods to treating hemoglobinopathies
Abstract:
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.
Public/Granted literature
Information query
Patent Agency Ranking
0/0