This technology uses CRISPR-Cas9 gene editing to target prolyl hydroxylase (PHD2) to slow the progression of retinitis pigmentosa (RP).
Retinitis pigmentosa (RP) is a group of genetic neurodegenerative eye diseases in which metabolic dysregulation in photoreceptors results in their degeneration, leading to vision impairment. Similar patterns of metabolic dysregulation can also be found in other neurodegenerative diseases, including age-related macular degeneration, Alzheimer’s Disease, and Parkinson’s Disease. There are currently no effective treatment options for most types of RP, as well as for these related neurodegenerative diseases. As such, there is a clear need for novel approaches for treating neurodegeneration.
This technology describes the use of CRISPR gene editing to target metabolic dysregulation in retinitis pigmentosa (RP). Specifically, this technology uses an adeno-associated viral (AAV) vector to deliver CRISPR-Cas9 guide RNAs targeting the prolyl hydroxylase (PHD2) gene in the retina. Inhibiting this gene promotes the survival of rod and cone cells by reprogramming metabolic pathways dysregulated in RP. Through this reprogramming process, this technology slows the progression of RP disease without inducing cytotoxicity.
This technology has been validated in RP preclinical mouse models.
Patent Pending
IR CU23293
Licensing Contact: Kristin Neuman
