Targeted gene therapy for retinopathies
This technology is a gene therapy which delivers CRB1 isoforms to their cell-specific localizations, for the treatment of retinopathies caused by CRB1 mutations.
Unmet Need: Targeted gene therapy against multiple retinal disorder mutations
Mutations in the CRB1 gene have been identified as a cause of retinopathies like retinitis pigmentosa and Leber’s congenital amaurosis. CRB1 inherited retinal disorders affect 1 in 86,500 people in the United States, yet there are no approved treatments. This is due largely to the existence of multiple CRB1 isoforms with cell-type specific roles, making genotype-phenotype correlation difficult to discern.
The Technology: CRB1 cell-specific targeted gene therapy for the treatment of retinopathies
This technology describes a gene therapy for autosomal recessive retinal dystrophies caused by mutations in CRB1. Approximately 70% of CRB1 mutations affect both isoform CRB1-A, which is predominantly expressed in Müller glia cells, and isoform CRB1-B which is expressed in photoreceptor cells. This technology used vector-mediated targeted delivery of one or both of these isoforms to their cell-specific localizations to induce gene expression, and treat CRB1 related disorders.
Applications:
- Therapeutic for retinitis pigmentosa, Leber’s congenital amaurosis, and other CRB1 mutation related retinopathies
- Research tool for studying CRB1 function
Advantages:
- Targets one or multiple cell types
- Enhances specificity using cell-specific promoters
- Targets two CRB1 isoforms
Lead Inventor:
Patent Information:
Patent Pending
Related Publications:
Tech Ventures Reference:
IR CU21122
Licensing Contact: Kristin Neuman