Progress and opportunities of gene and stem cell therapy on hereditary ocular fundus diseases
10.3760/cma.j.issn.1005-1015.2018.06.001
- VernacularTitle:遗传性眼底病基因和干细胞治疗趋势与面临的挑战和机遇
- Author:
Ruyi HAN
1
;
Zibing JIN
Author Information
1. 325027,温州医科大学附属眼视光医院遗传眼病专科、视网膜再生医疗研究组温州医科大学干细胞研究所眼视光学与视觉科学国家重点实验室国家再生医学与神经遗传国际联合研究中心
- Keywords:
Retinal diseases/genetics;
Gene therapy;
Stem cell therapy;
Editorial
- From:
Chinese Journal of Ocular Fundus Diseases
2018;34(6):519-525
- CountryChina
- Language:Chinese
-
Abstract:
Hereditary ocular fundus disease is an important cause of irreversible damage to patients' visual acuity.It has attracted much attention due to its poor prognosis and lack of effective clinical interventions.With the discovery of a large number of hereditary ocular fundus genes and the development of gene editing technology and stem cell technology,gene and stem cell therapy emerged as the new hope for curing such diseases.Gene therapy is more directed at early hereditary ocular fundus diseases,using wild-type gene fragments to replace mutant genes to maintain existing retinal cell viability.Stem cell therapy is more targeted at advanced hereditary ocular fundus diseases,replacing and filling the disabled retinal cell with healthy stem cells.Although gene and stem cell therapy still face many problems such as gene off-target,differentiation efficiency,cell migration and long-term efficacy,the results obtained in preclinical and clinical trials should not be underestimated.With the emergence of various new technologies and new materials,it is bound to further assist gene and stem cell therapy,bringing unlimited opportunities and possibilities for the clinical cure of hereditary ocular fundus diseases.
