Gene therapy for Crb1 retinopathies
Current treatments for retinal disease include laser surgeries, injectable corticosteroids, and other medications which may slow the process of retinal degeneration but are not able to stop it. The Crb1 protein has been implicated in the development of retinal diseases, but technical barriers in delivering gene therapies have since provided the largest hurdle in bringing this treatment to patients.
Duke inventors have reported a gene therapy intended to treat the causes of retinal disease. This invention utilizes a novel Crb1 protein isoform, much smaller than the original protein, as part of a targeted Crb1 retinopathy gene therapy. This isoform has been found to be critical in the retina and in retinal development and is significant because it can be effectively packaged into a viral vector for gene delivery to patients.
- This invention provides a strategy for stopping retinal disease progression rather than simply slowing it
- The truncated Crb1 protein isoform can be packaged with an AAV vector
- The protein isoform has been shown to be expressed exclusively in photoreceptors, the vital cell type in retinal dystrophies