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Targeting BCL11A with CRISPR-Cas9 Gene Editing Technology to Treat Sickle Cell Disease
- 1 Guanghua Cambridge International School, Shanghai, China
* Author to whom correspondence should be addressed.
Abstract
An enormous portion of people from all over the world are afflicted by sickle cell disease (SCD), which is a hereditary blood malady that has a severe negative influence on quality of life. Despite advancements in supportive care, a definitive cure remains elusive. The transcriptional repressor BCL11A has surfaced as an enticing target for novel SCD therapies. The prospect of applying CRISPR-Cas9 gene editing to alter BCL11A promoter and optimize SCD pathogenesis is examined in this paper. By suppressing BCL11A, which normally inhibits fetal hemoglobin (HbF) expression, HbF levels can be elevated in adult red blood cells. Increased HbF mitigates the deleterious effects of sickle hemoglobin polymerization, the root cause of SCD manifestations. Preclinical investigations deploying CRISPR-Cas9 to disrupt BCL11A in hematopoietic stem cells have yielded promising results, paving the way for pioneering clinical trials. Although in its early stages of development, this gene editing method has great potential as a revolutionary, one-time therapeutic intervention for SCD. As research progresses, meticulous evaluation of safety, efficacy, and long-term outcomes can be paramount in translating this groundbreaking strategy into clinical reality
Keywords
Sickle cell anemia, BCL11A repressor, CRISPR-Cas9 gene editing, fetal hemoglobin induction
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Cite this article
Wang,Z. (2025). Targeting BCL11A with CRISPR-Cas9 Gene Editing Technology to Treat Sickle Cell Disease. Theoretical and Natural Science,75,161-165.
Data availability
The datasets used and/or analyzed during the current study will be available from the authors upon reasonable request.
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Volume title: Proceedings of ICBioMed 2024 Workshop: Computational Proteomics in Drug Discovery and Development from Medicinal Plants
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