2020年12月5日 · BCL11A is a transcription factor that represses γ-globin expression and fetal hemoglobin in erythroid cells. We performed electroporation of CD34+ hematopoietic stem and progenitor cells obtained...

2021年1月21日 · This study validates BCL11A inhibition as an effective target for HbF induction and provides preliminary evidence that shmiR-based gene knockdown offers a favorable risk-benefit profile in sickle cell disease.

2020年12月5日 · We report findings in six patients with severe sickle cell disease treated with therapy directed against BCL11A, in this case through infusion with BCH-BB694 BCL11A shmiR transduced CD34+...

2019年11月21日 · BCL11A regulates the fetal-adult hemoglobin switch by repressing expression at the gamma (γ)-globin locus (Sankaran et al., Science, 2008), and thus it represents an appealing therapeutic target for sickle cell disease (SCD).

2016年9月6日 · A precision-engineered gene therapy virus, inserted into blood stem cells that are then transplanted, markedly reduced sickle-induced red-cell damage in mice with sickle cell disease, according to a study in the Journal of Clinical Investigation.

These data demonstrate that reducing, but not eliminating, BCL11A expression represents a viable approach for the treatment of sickle cell disease and other β-hemoglobinopathies. Chang et al. 3 demonstrated further that edited HSPCs with a disrupted BCL11A enhancer were efficient in long-term engraftment in Nod-Scid-Gamma (NSG ...

BCL11A enhancer reduces expression of BCL11A sufficiently to achieve therapeutic levels of g-globin expression without impairing the maturation of red blood cells.

2020年12月5日 · BCL11A is a repressor of γ-globin expres-sion and HbF production in adult erythrocytes. Its down-regulation is a promising therapeutic strategy for induction of HbF. We enrolled patients with...

A BCL11A-targeting gene therapy in patients with sickle cell disease (SCD) led to higher levels of fetal hemoglobin (HbF) and reductions in the severity of disease, according to findings from a small pilot study.

2013年11月15日 · These data indicate that targeting BCL11A and/or KLF1 with ASO treatment can cause an increase in γ-globin expression that is necessary for the upregulation of fetal hemoglobin and may be used for the treatment of sickle-cell anemia and β-thalassemia.