Progress and opportunities of gene and stem cell therapy on hereditary ocular fundus diseases / 中华眼底病杂志

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.