The role of CRISPR in the treatment of sickle cell anemia

Abstract

Sickle cell anemia (SCA) is an autosomal recessive disease that gives rise to abnormal hemoglobin, thus impairing the oxygen carrying ability of the red blood cells (RBCs).Up till this point, SCA treatment has been mostly pharmaceutical accompanied by lifestyle modifications. However, since the advent of more precise genome editing tools like CRISPR, there are new, more promising possibilities for SCA therapy.CRISPR-Cas9 genome editing is a technique, based on a bacterial defense system, that can be adapted to modify genes of living organisms. With this tool, a programmed complex consisting of Cas9 nuclease coupled with a single-guide RNA (sgRNA) is delivered into a cell using a vector, and this complex then cuts the genome at a specified location, giving certain desired outcomes based on the induced mutation.