Posts Tagged ‘ gene therapy ’
The Complexity Of Gene Therapy For Congenital Blindness
Independent clinical trials, including one conducted at the Scheie Eye Institute at the Perelman School of Medicine, have reported safety and efficacy for Leber congenital amaurosis (LCA), a congenital form of blindness caused by mutations in a gene (RPE65) required for recycling vitamin A in the retina. Inherited retinal degenerative diseases were previously considered untreatable and incurable. There were early improvements in vision observed in the trials, but a key question about the long-te...
Potential Major Breakthrough in Treatment of Wet Age-Related Macular Degeneration
A single injection of a new gene therapy treatment from Avalanche Biotech could possibly stop the advance of neovascularization in the wet form of AMD for several years. Human clinical trials are currently underway.
Here is how I commented about this breakthrough in my writeup:...
A Novel Gene Therapy Approach to Treating the Wet Form of AMD: The BioFactoryTM From Avalanche Biotech
I originally contacted this company in November 2010, when they were still in “stealth mode” and weren't able to share details about what they were doing. Recently, the company got back in touch to provide an update, having announced, in December 2011, a clinical trial of their gene therapy approach to treating the wet form of AMD.
Gene Therapy in Ophthalmology Update 6: First-Ever Clinical Trial for the Autosomal Recessive Form of Retinitis Pigmentosa (arRP) is Underway
It has been difficult keeping up with the changing world of gene therapy in ophthalmology, but thanks to the Foundation Fighting Blindness, I learned yesterday about this new, and first clinical study for treating a rare form of retinitis pigmentosa, underway in Saudi Arabia.
The Use of Gene Therapy in Treating Retinitis Pigmentosa and Dry AMD
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After several discussions with Sean Ainsworth, the founder of RetroSense, and much online research, I think I have learned a little about what gene therapy is about, and its application in ophthalmology, especially in the possible restoration of vision in those who suffer from retinitis pigmentosa (RP). Thanks to Sean for whetting my appetite -- here is what I have learned.
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