bcl11a shrna

BCL11a shRNA is a gene therapy approach that uses a short hairpin RNA (shRNA) embedded in a microRNA (miR) backbone to specifically knock down the expression of the B-cell lymphoma/leukemia 11A (BCL11A) gene. This strategy is designed to reactivate fetal hemoglobin (HbF) production by suppressing BCL11A, which normally represses γ-globin expression after birth. By reducing BCL11A levels in erythroid cells, this therapy induces HbF and reduces sickling in red blood cells—offering therapeutic potential for β-hemoglobinopathies such as sickle cell disease and β-thalassemia[1][2][5]. The lineage-specific delivery of the shRNA minimizes toxicity and preserves hematopoietic stem cell function[5]. The drug is being developed by bluebird bio and Boston Children's Hospital for intravenous administration[1].

Other names

bcl11a shrnabcl-11a shrnabcl 11a shrnaBCL11A-based gene therapyBCL-11A-based gene therapyBCL 11A-based gene therapy