Target intelligence / Profile preview

B-cell CLL/lymphoma 11A (BCL11A)

Target
BCL11A
Molecular classification
Transcription factor, Proto-oncogene, C2H2-type zinc-finger DNA-binding protein
01

Overview

BCL11A is a gene encoding a regulatory protein that functions as a transcription factor. It plays critical roles in hematopoiesis, brain development, and fetal-to-adult hemoglobin switching. It is considered a proto-oncogene and is implicated in leukemia and lymphoma. Therapeutic targeting of BCL11A is under investigation for treating sickle cell disease and beta-thalassemia.

Other names
BCL11 transcription factor A
02

Mechanism of action

Targeting BCL11A to reactivate fetal hemoglobin

03

Biological functions

Transcriptional regulationHematopoietic system developmentBrain developmentFetal-to-adult hemoglobin switchingGene repressionCell differentiationRegulation of apoptosis
04

Disease associations

LeukemiaLymphomaβ-Hemoglobinopathies (Sickle cell disease, Beta-thalassemia)Intellectual Disability SyndromesAutism Spectrum DisorderType II Diabetes

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