CRISPR/Cas9 genome editing to treat sickle cell disease

Abstract

The most prevalent inherited blood condition, Sickle Cell Disease (SCD) causes excruciating pain, permanent organ damage including liver, heart, and kidney also causes an untimely death. There is currently a dearth of treatment options for SCD. In order to lessen the severity of acute problems, only four medications have been licensed by the Food and Drug Administration (FDA). Hematopoietic stem cell transplantation, is the curative treatment for SCD, that is related to matched donor. Recent and significant advancements in genome editing techniques have shown promise as a curative option, with the potential to fix the mutation in patient-derived Hematopoietic Stem/Progenitor Cells (HSPCs), to increase Fetal Hemoglobin (HbF) expression to avoid sickling of Red Blood Cells (RBCs), and generate corrected induced Pluripotent Stem Cells (iPSCs). CRISPR/Cas9, which was discovered relatively recently, has not only changed the face of genome engineering, but also made it possible to use these ideas in the clinic. In this review, we provide an overview of CRISPR/Cas9 applications in genome engineering and discuss the advantages, disadvantages, how it is superior to other gene editing technologies such as Zinc Finger Nucleases (ZFNs) and Transcription Activator-Like Effector Nucleases (TALENs) and potential of this technology as a treatment for SCD.