BCL11A - Wikipedia
Clinical significance. The corresponding Bcl11a mouse gene is a common site of retroviral integration in myeloid leukemia, and may function as a leukemia disease gene, in part, through its interaction with BCL6. During hematopoietic cell differentiation, this gene is down-regulated.
CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β …
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...
BCL11A: a potential diagnostic biomarker and therapeutic target …
2019年11月29日 · BCL11A functions mainly as a transcriptional repressor that is crucial in brain, hematopoietic system development, as well as fetal-to-adult hemoglobin switching. The expression of this gene is regulated by microRNAs, transcription factors and genetic variations.
BCL11A Gene - GeneCards | BC11A Protein | BC11A Antibody
2024年10月1日 · BCL11A (BCL11 Transcription Factor A) is a Protein Coding gene. Diseases associated with BCL11A include Intellectual Developmental Disorder With Persistence Of Fetal Hemoglobin and Isolated Corpus Callosum Agenesis.
Post-Transcriptional Genetic Silencing of BCL11A to Treat …
2020年12月5日 · BCL11A is a repressor of γ-globin expression and HbF production in adult erythrocytes. Its down-regulation is a promising therapeutic strategy for induction of HbF.
Transcription factor competition at the γ-globin promoters ... - Nature
2021年3月1日 · BCL11A, the major regulator of fetal hemoglobin (HbF, α 2 γ 2) level, represses γ-globin expression through direct promoter binding in adult erythroid cells in a switch to adult...
BCL11A enhancer dissection by Cas9-mediated in situ saturating ...
2015年9月16日 · BCL11A is a transcriptional repressor that inhibits expression of fetal globin genes in adults, and is a potential therapeutic target for the treatment of β-globinopathies such as β-thalassemia...
CRISPR gets the glory in landmark approval, but haemoglobin …
2023年11月27日 · Stuart Orkin, of Harvard Medical School and HHMI, discusses how the discovery of BCL11A paved the way for Vertex and CRISPR Therapeutics’ exa-cel therapy for haemoglobinopathies.
Direct Promoter Repression by BCL11A Controls the Fetal to Adult ...
BCL11A acts at distal TGACCA in human γ-globin promoters to silence expression. •. TGACCA motif is mutated in individuals with HPFH syndrome. •. Editing the distal TGACCA prevents BCL11A binding and reactivates γ-globin expression. Summary.
Exagamglogene Autotemcel for Severe Sickle Cell Disease
2024年4月24日 · Exagamglogene autotemcel (exa-cel) is a nonviral, autologous cell therapy that is designed to reactivate fetal hemoglobin production by means of ex vivo clustered regularly interspaced...