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Stem Cells and Eye Disease

The potential for using stem cells to cure certain types of vision loss has been a hot topic for a while, as it seems like a promising area of research that could yield great results for people with vision loss. One way to utilize stem cells is by manipulating them with CRISPR technology, which is a tool which allows scientists to edit genomes, altering DNA sequences and modifying gene function. These genetically edited stem cells can then be used to help treat and hopefully cure diseases.

Excitingly, this is currently being trialed with Leber congenital amaurosis 10 (LCA 10), a congenital eye disease which affects the retina and leads to severe vision loss early in life. A researcher in Oregon is currently testing for the safety of this treatment, with the ultimate hope of being able to restore vision for individuals with LCA.  Below is a posting that was shared by the Foundation fighting Blindness:

Clinical researchers at Casey Eye Institute, Oregon Health & Science University (OHSU), have dosed the first patient with an experimental CRISPR/Cas9 therapy in the BRILLIANCE Phase 1/2 clinical trial for people with Leber congenital amaurosis 10 (LCA 10). The emerging treatment targets a specific mutation (c.2991+1655A>G in Intron 26) of the gene CEP290. For more information click here.

This is an exciting development for individuals with genetic retinal diseases and emphasizes the importance of genetic testing for eye diseases, as these studies require that participants have detailed genetic profiles. Genetic testing for inherited retinal diseases is becoming much more available and can often be completed at no cost. Both the Eye Institute and the UW IRD clinics have options for free genetic testing so if you have an inherited retinal disease make sure to take advantage!