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:

Peter Quinn, Ph.D.

Patent Information:

Patent Pending

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