Dr. Sandip K. Basu from University of Tennessee awarded $70,000 Knights Templar Eye Foundation Grant

Dr. Sandip K. Basu from the University of Tennessee Health Sciences Center was awarded a $70,000 grant titled: A novel anti-ceramide gene therapy for retinitis pigmentosa.

Retinitis Pigmentosa (RP) is an inherited disease that starts in childhood with night blindness and peripheral vision loss. The disease progresses with age and the affected individual completely loses vision by 40-50 years of age. The vision loss happens due to progressive death of the light sensing cells of the retina, called photoreceptors, due to mutation in more than 100 different genes essential for proper vision. Gene therapy is a very promising strategy to cure hereditary diseases, but the high heterogeneity of mutations that cause RP makes it difficult to develop a common therapy that could help affected people with different underlying mutations. Since the ultimate effect of all the different mutations is death of the photoreceptors, targeting a common factor of photoreceptor death could be beneficial for a diverse group of RP patients with different mutations.

In this proposal Dr. Basu intends to target one such common denominator of photoreceptor death by gene therapy in cultured cells and mouse model of RP. Ceramide is a bioactive lipid whose levels are shown to increase when photoreceptors die in many different models and reducing its levels can prevent their death. They will use an enzyme that converts ceramide to a non-toxic form and lower its levels. We believe that reducing ceramide levels will protect the photoreceptors from dying and restore the visual functions. This study will establish ceramide as a potentially important therapeutic target for RP and ways to lower ceramide levels as a possible therapeutic strategy for other retinal diseases.