CRISPR-based genomic modification method in the treatment of hematological diseases

Abstract

Mutations in hematopoietic stem cells are the main cause of most blood diseases. Hematopoietic stem cell transplantation, which is the main treatment for hematopoietic stem cell disorders ,brings many negative effects according to the clinical cases, such as high toxic side effects, limited donor source, high recurrence rate, and high treatment cost .CRISPR/Cas acts as an immune system in prokaryote that grants them an ability to resist invasion by exogenous mobile genetic elements (MGE), which is a new generation of gene editing technology after the appearance of TALEN and ZFN. The immune system functions in three steps: adaptation, expression, and interference. It enables genomic modification by unwinding the double strands of DNA and inducing DNA repair processes such as homologydirected repair (HDR) and/or non-homologous end joining (NHEJ). So, this paper discusses the utilization of Crispr system in the treatment of -hemoglobinopathy, Chronic myeloid leukemia (CML) , Hemophilia and summarizes the current limitations of this technology, such as the editing efficiency, potential immunogenicity of editing tools and off-target effects , while it still remains promising as to its future prospects for research. This technology is very likely to replace traditional therapies and achieve a complete cure for the blood disease.