In-vivo gene therapy for Retinitis Pigmentosa

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Retinitis Pigmentosa (RP) is one of the leading causes of blindness worldwide. RP is a highly heterogeneous disease caused by mutations in one of over 60 genes. Due to the genetic nature of the disease, in-vivo gene therapy is considered to be the most promising approach to treating RP.However, the challenge of developing gene therapies for RP is that many of the underlying gene mutations are ultra-rare making them unattractive for therapeutic development. An approach that allows the development of a single precision therapy that would benefit a larger patient population irrespective of the underlying genetic cause is highly desirable. The proposed project will develop a proof of concept for a gene agnostic therapy for RP that will benefit a broader patient population.