Gene therapy research demonstrates promising results that can be applied to reversing and treating blindness. Viral delivery of certain therapies shows effectiveness in treating vision loss in multiple mammals including human beings. These therapies include:
AAV-RPGRIP
AAV2-hRPE65v2
AAV2-ChR2V
AAV2/5-hIRPB-hRPGR
AAV8-Y733F-mGRM6-SV40-hChR2-heGFP
CMV-MYO7A
rAAV-Lrat
rAAV2-CBSB-hRPE65
RPGRORF15-XLRP
References:
AAV2 gene therapy readministration in three adults with congenital blindness. (Link)
Age-dependent effects of RPE65 gene therapy for Leber’s congenital amaurosis: a phase 1 dose-escalation trial. (Link)
Channelrhodopsin-2 gene transduced into retinal ganglion cells restores functional vision in genetically blind rats. (Link)
Gene replacement therapy rescues photoreceptor degeneration in a murine model of Leber congenital amaurosis lacking RPGRIP. (Link)
Gene therapy rescues photoreceptor blindness in dogs and paves the way for treating human X-linked retinitis pigmentosa. (Link)
Human gene therapy for RPE65 isomerase deficiency activates the retinoid cycle of vision but with slow rod kinetics. (Link)
In utero gene therapy rescues vision in a murine model of congenital blindness. (Link)
Lentiviral gene replacement therapy of retinas in a mouse model for Usher syndrome type 1B. (Link)
Long-term restoration of rod and cone vision by single dose rAAV-mediated gene transfer to the retina in a canine model of childhood blindness. (Link)
Pharmacological and rAAV gene therapy rescue of visual functions in a blind mouse model of Leber congenital amaurosis. (Link)
Restoration of vision in RPE65-deficient Briard dogs using an AAV serotype 4 vector that specifically targets the retinal pigmented epithelium. (Link)
Safety and efficacy of subretinal readministration of a viral vector in large animals to treat congenital blindness. (Link)
Treatment of leber congenital amaurosis due to RPE65 mutations by ocular subretinal injection of adeno-associated virus gene vector: short-term results of a phase I trial. (Link)
Virally delivered channelrhodopsin-2 safely and effectively restores visual function in multiple mouse models of blindness. (Link)
