CRISPR Mediated Gene Therapy via HDR in Stem Cells for the Treatment of Sickle Cell Anemia
DOI:
https://doi.org/10.58445/rars.2229Keywords:
Biomedical and Health Sciences; Genetics and Molecular Biology of Disease, CRSIPR-Cas9, Sickle Cell Anemia, Hematopoietic Stem Cells, Homology Directed Repair, HemoglobinAbstract
Sickle cell anemia (SCA) is a genetic disorder caused by a single point mutation in the β-globin gene (HBB), leading to the production of abnormal hemoglobin S. CRISPR-Cas9 gene editing technology offers a promising approach to cure SCA by directly targeting and correcting this mutation. This review discusses the application of CRISPR-Cas9 in SCA treatment, focusing on the engineering of the Cas9 enzyme to specifically target the HBB gene mutation. Strategies to enhance Cas9 precision and minimize off-target effects are explored, along with the use of homology-directed repair (HDR) to repair the DNA after Cas9-mediated cleavage. Preclinical and clinical studies demonstrating the feasibility of this approach are reviewed, alongside the challenges that must be addressed for clinical application. CRISPR-Cas9 presents a potential revolution in SCA treatment, offering a potentially curative solution for patients.
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