Ask about this productRelated genes to: BCL11A Blocking Peptide
- Gene:
- BCL11A NIH gene
- Name:
- BAF chromatin remodeling complex subunit BCL11A
- Previous symbol:
- EVI9
- Synonyms:
- BCL11A-XL, BCL11A-L, BCL11A-S, CTIP1, HBFQTL5, ZNF856
- Chromosome:
- 2p16.1
- Locus Type:
- gene with protein product
- Date approved:
- 2001-02-28
- Date modifiied:
- 2019-01-25
Related products to: BCL11A Blocking Peptide
Related articles to: BCL11A Blocking Peptide
- Temozolomide (TMZ) is used to treat glioblastoma cancer and it is essential to identify key genes and investigate the molecular mechanisms of glioblastoma cancer cells resistant to temozolomide. - Source: PubMed
Publication date: 2026/06/05
Alamholo MostafaTarinejad Alireza - This study aimed to evaluate the role of two genetic polymorphisms (rs9399137 and rs4895441) in the HBS1L-MYB and rs766432 of BCL11A genes as determinant HbF level and disease severity in Egyptian β-thalassemia patients. - Source: PubMed
Publication date: 2026/06/03
Abdelrahman Abdelrahman ATalaat Randa MEl-Halfawy Ibrahim AbdoAlalwi Mohamed RamzyRizk Dina Malak Yousef - Sickle cell disease (SCD) is characterized by chronic hemolysis, painful vaso-occlusive episodes (VOE) and end organ damage. High levels of fetal hemoglobin (HbF) attenuate the disease phenotype. We used a lentivirus vector (LVV) expressing an shRNA embedded in a microRNA (shmiR) targeting BCL11A in erythrocytes to induce HbF in a first-in-human pilot study in SCD. The purpose of the study was to assess hematopoietic stem/progenitor cells (HSPCs) collection, transduction parameters, safety, HbF induction and durability. Eleven eligible patients with SCD had HSC collection. Plerixafor-mobilized peripheral blood HSCs required for manufacturing were obtained in one mobilization cycle for 10/11 subjects and 11/11 patient products were successfully manufactured with a median time to release of product of 39 days. Ten patients were infused with autologous HSCs transduced with the shmiR vector. Engraftment occurred in all 10 patients. With a median follow-up of 58 months (range: 35-82) after infusion, no adverse events attributed to the gene vector have occurred. Transduction efficiency was 93.1%. One patient demonstrated low engraftment of transduced cells and had suboptimal HbF induction. In the remaining 9 patients, at 2 years post-treatment peripheral blood demonstrated 71% F cells with 11.9 pg HbF/F cells, both stable in 9 patients with ≥48 months follow-up. All patients who had VOEs prior to gene therapy demonstrated sustained mitigation of pain events. These data demonstrate excellent manufacturing efficiency and safety, with efficacy of targeting BCL11A using a shmiR LVV, and long-term durability of the shmiR vector, leading to a pivotal multi-site phase 2 trial currently underway (NCT05353647). - Source: PubMed
Publication date: 2026/06/02
Esrick Erica BLehmann LeslieFederico AmyVincon HeleneLiu BoyaDaley HeatherDansereau ColleenDe Oliveira SatiroEverett John KKao Pei-ChiMoore Theodore BMorris EmilyTrebeden-Negre HeleneShaw Kit LRoach Gavin DRitz JeromeRoche Aoife MSilva OliviaGrant Patricia EllenBushman Frederic DKohn Donald BJain AkshatLondon Wendy BJustus David GArmant MyriamManis John PWilliams David A - Bariatric surgery, particularly Roux-en-Y gastric bypass (RYGB), is an effective treatment for extreme obesity, but some patients experience inadequate weight loss or weight regain. This study aimed to identify blood biomarkers that predict effective weight loss following RYGB. - Source: PubMed
Publication date: 2026/03/09
Mahjoubin-Tehran MaryamJamialahmadi TannazSukhorukov Vasily NAlmahmeed WaelKroh MatthewSahebkar Amirhossein - Sickle cell disease (SCD) is a monogenic disorder responsible for recurrent vaso-occlusive crises, progressive organ damage, and shortened life expectancy. For decades, allogeneic hematopoietic stem cell transplantation from a matched sibling donor has been the only established cure, but its reach remains limited by donor availability and transplant-related toxicity. The approval of two autologous gene therapy products in 2023, exagamglogene autotemcel (exa-cel) and lovotibeglogene autotemcel (lovo-cel), marked a turning point for the SCD population and the gene therapy field in general. This review proposes a molecular rationale for fetal hemoglobin reactivation and β-globin gene addition, describes the engineering of lentiviral and CRISPR-based platforms, and highlights the clinical evidence accumulated to date that demonstrated durable disease modification with acceptable short-term toxicity. We then assess the clinical positioning of gene therapy within the broader spectrum of curative options compared to current available treatments and address the financial, ethical and psychosocial barriers that limit access to gene therapy both within high-income countries and globally. Critical research priorities include long-term safety surveillance, comparative effectiveness studies, pediatric trials below 12 years, and validated patient-reported outcome instruments. Base editing, non-genotoxic conditioning, and in vivo delivery represent the most promising avenues to broaden access and reduce treatment burden. - Source: PubMed
Publication date: 2026/05/20
Tardif MagalieSaby ManonForté StéphaniePincez Thomas