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Imagine treating a genetic disease not by managing symptoms for life, but by correcting the underlying genetic error itself. Inventors at NIH are advancing that vision with a gene editing technology designed to deliver highly precise genetic corrections, opening new possibilities for therapies that
Currently, there is no effective treatment for many types of retinal degeneration. Nor are there sufficient treatments for autoimmune uveitis, as current uveitis treatment primarily uses steroidal anti-inflammatory medication, which can produce significant unwanted side effects with long-term use
NIH inventors have developed an image-based machine learning system that is able to validate functional cell phenotypes. The system may be trained to automatically recognize image features that correlate with a desired cell-type or properties for research, diagnostic, and therapeutic purposes. This
NIH inventors have developed a mutation-independent method to treat Leber congenital amaurosis (LCA), retinitis pigmentosa (RP), other inherited retinal degenerations (IRDs), and age-related macular degeneration (AMD) using the FDA-approved small molecule, Halofantrine. NIH inventors found