eng Oriental Scientific Publishing Company Biosciences Biotechnology Research Asia 0973-1245 2026-05-11 23 2 58894 article Vaishnavi Vinod Dere 1 Sushant Satappa Patil 1 Ajay Yeshawant Kale 1 Kishor Vasant Otari 1 Gene Therapy and CRISPR-based Intervention in Sickle Cell Anemia Sickle cell anemia is a severe inherited blood disorder caused by a mutation in the beta globin gene, leading to the formation of abnormal hemoglobin S and deformation of red blood cells into rigid, sickle-shaped structures. These altered cells impair blood flow within the microcirculation, resulting in recurrent vaso-occlusive episodes, chronic inflammation, progressive organ damage, and reduced quality of life. Conventional therapeutic approaches, including hydroxyurea therapy, repeated blood transfusions, and hematopoietic stem cell transplantation, can reduce disease severity but do not correct the underlying genetic defect.The objective of this review is to provide a comprehensive evaluation of gene therapy strategies, with particular emphasis on clustered regularly interspaced short palindromic repeats and CRISPR associated protein 9 based genome editing, in the treatment of sickle cell disease, focusing on their mechanisms, clinical outcomes, and associated challenges.Recent advances in gene editing enable targeted correction of the mutated beta globin gene or reactivation of fetal hemoglobin through modification of regulatory elements such as B cell lymphoma eleven A. The first approved gene editing therapy, Casgevy, has demonstrated substantial clinical benefits, including a marked reduction in vaso-occlusive episodes and improved transfusion independence.Despite these promising outcomes, several challenges remain, including unintended genomic modifications, toxicity associated with conditioning regimens, high treatment costs, limited accessibility, and ethical concerns related to genome integrity. Continued advancements in technology, ethical oversight, and healthcare policy are essential to ensure safe, effective, and equitable clinical implementation of gene therapy as a potentially curative approach for sickle cell disease. https://www.biotech-asia.org/vol23no2/gene-therapy-and-crispr-based-intervention-in-sickle-cell-anemia/ CRISPR/Cas9; Gene Therapy; Genome Editing; Hemoglobin; Sickle Cell Disease