A Review of CRISPR Cas9 for SCA: Treatment Strategies and Could Target β-globin Gene and BCL11A Gene using CRISPR Cas9 Prevent the Patient from Sickle Cell Anemia?

Abstract

BACKGROUND: Sickle cell anemia is a hereditary globin chain condition that leads to hemolysis and persistent organ damage. Chronic hemolytic anemia, severe acute and chronic pain, and end-organ destruction occur throughout the lifespan of sickle cell anemia. SCD is associated with a higher risk of mortality. Genome editing with CRISPR-associated regularly interspersed short palindromic repeats (CRISPR/Cas9) have therapeutic potential for sickle cell anemia thala. AIM: This research aimed to see if using CRISPR/Cas9 to target β-globin gene is an effective therapeutic and if it has a long-term effect on Sickle Cell Anemia. METHODS: The method used in this study summarizes the article by looking for keywords that have been determined in the title and abstract. The authors used official guidelines from Science Direct, PubMed, Google Scholar, and Journal Molecular Biology to select full-text articles published within the last decade, prioritizing searches within the past 10 years. RESULTS: CRISPR/Cas9-mediated genome editing in clinical trials contributes to α-globin gene deletion correcting β-thalassemia through balanced α- and β-globin ratios and inhibiting disease progression. CONCLUSION: HBB and BCL11A targeting by CRISPR/Cas9 deletion effectively inactivate BCL11A, a repressor of fetal hemoglobin production. However, further research is needed to determine its side effects and safety.