This technology describes a metabolism regulator gene, which can be manipulated to improve neuronal survival in retinal degeneration and other neurodegenerative diseases.
Retinitis pigmentosa is a progressive genetic neurodegenerative disorder that causes impaired vision and blindness. Current treatment options are extremely limited and specific gene therapies would only be effective in a fraction of patients due to the large number of genes associated with retinitis pigmentosa. There is an urgent need to develop therapies that could halt the deterioration of neurons in this disease and related neurodegenerative conditions.
This technology describes a gene target that can be inhibited to improve the rate of cellular metabolism for preventing neuronal death. Recent research has implicated metabolic dysregulation as a critical factor leading to neurodegeneration. By selectively manipulating the gene von Hippel-Lindau (VHL), glycolysis can be promoted, which subsequently improves neuronal survival in retinitis pigmentosa. The ability to correct metabolic function through selectively targeting this gene could potentially treat a wide range of neurogenerative conditions that involve metabolic dysfunction such as age-related macular degeneration (AMD), Alzheimer’s disease, and Parkinson’s disease.
This technology has been successfully used to preserve the visual function in a mouse model of retinitis pigmentosa.
IR CU17321
Licensing Contact: Ron Katz