Research progress of gene therapy for chronic granulomatosis disease / 国际儿科学杂志

ABSTRACT

Chronic granulomatous disease (CGD) is a rare type of primary phagocytic immunodeficiency disease.CGD is caused by a defective gene that encodes the components of reduced nicotinamide adenine dinucleotide phosphate (NADPH) in phagocytes.Due to genetic mutations causing phagocytic respiratory dysfunction,phagocytic cells are not effective in killing peroxidase-positive bacteria and fungi,and clinical manifestations are characterized by repeated severe bacterial,fungal infections and granuloma formation.The current clinical treatments include routine therapy and hematopoietic stem cell transplantation (HSCT).With the research development of CGD in genetics,molecular biology and vector science,clinical research on gene therapy of the disease has been carried out,in which CRISPR/Cas9 system has a good application prospect in gene therapy for genetic diseases.This article reviews the progress of gene therapy.