Advances in Gene Therapy Delivery Systems:Promising Prospects for the Treatment of Inherited Retinal Diseases
10.3870/j.issn.1672-0741.23.05.002
- VernacularTitle:基因治疗递送系统的研究新进展:遗传性视网膜疾病治疗的曙光
- Author:
Yang ZHAI
1
;
Zixuan SU
;
Xinghua WANG
Author Information
1. 华中科技大学同济医学院第一临床学院,武汉 430030
- Keywords:
inherited retinal disease;
retinal gene therapy;
gene delivery system;
viral vector;
non-viral vector
- From:
Acta Medicinae Universitatis Scientiae et Technologiae Huazhong
2024;53(3):414-419
- CountryChina
- Language:Chinese
-
Abstract:
Inherited retinal disease encompasses a range of congenital retinal neurodegenerative conditions,characterized by symptoms such as night blindness,progressive visual field defect,vision loss,and even blindness.The main cause of the disease is gene mutation.Through gene therapy,that is the application of exogenous nucleotides to replace or silence the disease-causing genes in the genetically defective cells,so that the cells can express the correct protein and restore the function of cells.Therefore,it is possible to cure the disease.The immune-privileged status of the eye makes it an ideal organ for gene thera-py.However,successful gene therapy requires delivery systems that can carry therapeutic nucleotides into the cells.This review focuses on the progress and challenges of gene therapy delivery systems for inherited retinal diseases,including viral and non-vi-ral vectors.
