[Gene therapy for hereditary ophthalmological diseases: Advances and future perspectives].
Gene therapy is a promising new therapeutic strategy that could provide a novel and more effective way of targeting hereditary ophthalmological diseases. The eye is easily accessible, highly compartmentalized, and an immune-privileged organ that gives advantages as an ideal gene therapy target. Recently, important advances in the availability of various intraocular vector delivery routes and viral vectors that are able to efficiently transduce specific ocular cell types have been described. Gene therapy has advanced in some retinal inherited dystrophies; in this way, preliminary success is now being reported for the treatment of Leber congenital amaurosis (LCA). This review will provide an update in the field of gene therapy for the treatment of ocular inherited diseases.
Unidad de Investigación, Servicio de Genética, Instituto de Oftalmología Conde de Valenciana, México, D.F., México.
Óscar Francisco
ÓF
Chacón-Camacho
Unidad de Investigación, Servicio de Genética, Instituto de Oftalmología Conde de Valenciana, México, D.F., México.
Aline
A
Astorga-Carballo
Unidad de Investigación, Servicio de Genética, Instituto de Oftalmología Conde de Valenciana, México, D.F., México.|Facultad de Medicina, Departamento de Bioquímica, UNAM, México, D.F., México.
Juan Carlos
JC
Zenteno
D005128:Eye Diseases / Q000235:genetics* / Q000628:therapy*; D005544:Forecasting; D015316:Genetic Therapy / Q000379:methods; D006801:Humans