The Fabulous Impact of CRISPR Method in Sickle Cell Disease Treatment

Abstract

Sickle cell diseases are the most prevalent monogenic blood diseases with complications such as severe end-organ harm, pain, and reduced life expectancy. Dealing options for sickle cell diseases are inadequate, as there are just two FDA-approved drugs to decrease acute manifestation. The only sickle cell diseases curative procedure is bone marrow transplantation, frequently from a harmonized, related donor. Ex vivo manipulation of autologous hematopoietic stem and progenitor cells and subsequent transplantation of genetically altered cells theoretically offer an everlasting therapy appropriate to all sickle cell anemia patients, regardless of the accessibility of fit donors and graft-versus-host disease. In this review, we emphasize applying CRISPR gene editing strategies for sickle cell anemia treatment, containing the genetic modification and rectification of sickle cell disease mutation in β-globin and the stimulation of fetal hemoglobin to protect cells against sickling. We summarize the importance of stem cell manipulation to cure sickle cell anemia having likely lifetime efficacy for cell and gene therapies. HIGHLIGHTS• Sickle cell disease (SCD) is a type of monogenic blood disorders.• The CRISPR/Cas9 technology can be used to treat SCD.• RBC sickling is reversed by mutation correction or fetal hemoglobin induction.