Therapeutic approaches for sickle cell disease by targeting BCL11A

QingDao No.19 High School, QingDao, ShanDong, 266000, China

^* Corresponding vclarkedx52604@student.napavalley.edu

Abstract

Sickle cell disease (SCD) is one of the most serious diseases in the world caused by gene mutat ions. People with SCD have sickle-shaped red blood cells. BCL11A can inhibit the formation of γ-protein which can lead to the reduction of HbF,the reduction of the ability of hemoglobin to carry oxygen. Thus, d ecreasing the quantity of BCL11A can be a potential target to treat SCD.One of the potentially effective tr eatment approaches is to edit BCL11A genes with CRISPR technology.CRISPR is a gene-editing technolo gy,which can precisely and efficiently locate the treatment targets.With CRISPR technology,the gene expr ession of BCL11A can be blocked,and γ-protein can be switched to form more HbF.Hemoglobin levels for transporting oxygen can return to normal.This review introduces and summarizes the mechanisms to treat SCD andtheir limitations.Previous research papers revealed that the treatments of SCD are one-dimension al and these treatments just can inhibit disease.Fundamental problems of SCD remain to be solved.