Progress of CRISPR/Cas9 in the treatment of sickle cell disease

Abstract

Sickle cell disease (SCD) is a hereditary monogenic disease, which is characterized by the substitution of glutamic acid at the sixth position of β-peptide chain by valine, resulting in insufficient hemoglobin synthesis and a serious threats to human health. SCD causes about 80% of mortality and morbidity worldwide. Patients with SCD who are homozygous usually die before the age of 30. Patients with SCD who are heterozygous usually have a better prognosis because of their low intraerythrocytic hemoglobin S. However, FDA-approved drugs such as hydroxyurea and L-glutamine can reduce the severity of the disease, and there is no definite cure for all patients with SCD. At this stage, many scholars use creation and correction methods to induce pluripotent stem cells (IPSCs) to treat sickle cell disease. Gene editing is currently considered to be one of the most potential methods for the treatment of SCD. Among them CRISPR/Cas9 gene editing engineering technology has already made an abyss contribution to the transformation of genome engineering and its application in clinical or medical experiments. This review systematically introduces the gene therapy of CRISPR/Cas9, and summarizes the challenges and prospects of this technique in the treatment of sickle cell anemia.