This technology is a modified CRISPR/Cas9 gene editing therapy that is mutation-independent for treating autosomal dominant disease-related genes, including retinitis pigmentosa.
There are currently no known cures for autosomal dominant rhodopsin diseases including retinitis pigmentosa. Management of the disease are limited to vitamin supplements, protection from sunlight, and visual aids. Due to the diversity of underlying mutations in retinitis pigmentosa, traditional patient-specific therapy targeting specific mutations is impractical. Thus, there is a need for mutation-independent treatment of retinitis pigmentosa.
This technology is a modified CRISPR/Cas9 gene editing system to overcome limitations of mutation-specific treatment of autosomal dominant disease-related genes. By using a pair of viral vectors, both wild-type and mutated autosomal dominant disease-related gene are removed and replaced by a modified autosomal dominant disease-related gene to restore normal gene function. This technology provides greater long-term efficacy. As such, this simple mutation-independent gene therapy system can treat autosomal dominant diseases including the autosomal dominant disease-related gene in retinitis pigmentosa.
Patent Pending (US 20210017509)
IR CU18283
Licensing Contact: Kristin Neuman