![]() ![]() Safety and vision outcomes of subretinal gene therapy targeting cone photoreceptors in achromatopsia: a nonrandomized controlled trial. Human gene therapy for RPE65 isomerase deficiency activates the retinoid cycle of vision but with slow rod kinetics. Safety and efficacy of gene transfer for Leber’s congenital amaurosis. Effect of gene therapy on visual function in Leber’s congenital amaurosis. Gene therapy restores vision in a canine model of childhood blindness. Retinoic acid induces hyperactivity, and blocking its receptor unmasks light responses and augments vision in retinal degeneration. Meclofenamic acid improves the signal to noise ratio for visual responses produced by ectopic expression of human rod opsin. Persistent remodeling and neurodegeneration in late-stage retinal degeneration. Retinal remodeling: concerns, emerging remedies and future prospects. Retinal remodeling in human retinitis pigmentosa. ![]() Cellular responses following retinal injuries and therapeutic approaches for neurodegenerative diseases. Neural reprogramming in retinal degeneration. Retinal remodeling during retinal degeneration. Neural remodeling in retinal degeneration. Retinal remodeling in inherited photoreceptor degenerations. ![]() Retinal remodeling triggered by photoreceptor degenerations. Transplantation of retinal ganglion cells derived from male germline stem cell as a potential treatment to glaucoma. Cell transplantation of retinal ganglion cells derived from hESCs. Induced pluripotent stem cells promote retinal ganglion cell survival after transplant. Safety of rAAV2/2-ND4 gene therapy for Leber hereditary optic neuropathy. Gene therapy for Leber hereditary optic neuropathy: low- and medium-dose visual results. Development of a gene-editing approach to restore vision loss in Leber congenital amaurosis type 10. Genome-editing strategies for treating human retinal degenerations. ![]() CRISPR genome engineering for retinal diseases. Initial results from a first-in-human gene therapy trial on X-linked retinitis pigmentosa caused by mutations in RPGR. Retinal AAV8-RS1 gene therapy for X-linked retinoschisis: initial findings from a phase I/IIa trial by intravitreal delivery. Beneficial effects on vision in patients undergoing retinal gene therapy for choroideremia. Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65-mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial. Global prevalence of age-related macular degeneration and disease burden projection for 20: a systematic review and meta-analysis. A comparison of the causes of blindness certifications in England and Wales in working age adults (16–64 years), 1999–2000 with 2009–2010. Prevalence of mutations causing retinitis pigmentosa and other inherited retinopathies. In this Review, we highlight the most promising gene therapies, cell therapies and retinal prostheses for the treatment of retinal disease, discuss the benefits and drawbacks of each treatment strategy and the factors influencing whether functional tissue is reconstructed and repaired or replaced with an electronic device, and summarize upcoming technologies for enhancing the restoration of vision. And gene-editing strategies aim to correct the underlying genetic defects. In the earlier stages of degeneration, gene-replacement therapy could attenuate retinal-disease progression and reverse loss of function. In age-related macular degeneration, some cell-based therapies also aim to restore photoreceptor-supporting tissue to prevent complete photoreceptor loss. These approaches aim to restore light sensitivity to the retina as well as visual perception by integrating neuronal responses for transmission to the cortex. Late-stage retinal degenerative disease involving photoreceptor loss can be treated by optogenetic therapy, cell transplantation and retinal prostheses. ![]()
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