This technology is a collection of optimized therapeutic prime-editing strategies targeting four common splicing mutations in the peripherin‐2 gene (PRPH2) that are associated with various retinal degenerations.
This technology is a modified CRISPR/Cas9 gene editing therapy that is mutation-independent for treating autosomal dominant disease-related genes, including retinitis pigmentosa.
This technology is an AAV-mediated BEST1 gene therapy for the treatment of retinal degenerative disorders associated with Bestrophin-1 (BEST1) dominant mutations.
This technology is a gene therapy which delivers CRB1 isoforms to their cell-specific localizations, for the treatment of retinopathies caused by CRB1 mutations.
This technology is a generally applicable gene therapy approach that restores metabolic dysregulation and treats retinal degenerative diseases regardless of genetic variation.
This technology is a method of reprogramming cellular metabolism to treat neurodegenerative disorders by downregulating catabolism and upregulating anabolism in cells.
This technology uses CRISPR-Cas gene editing to disrupt and correct ocular disease-associated mutated genes, resulting in improved protein function and disease treatment.
This technology is a DNA editing platform with a reduced payload size to allow adeno-associated virus (AAV) and nanoparticle packaging for gene therapy.
This technology is a method to treat and prevent blindness due to macular degeneration by regulating cholesterol efflux by preventing drusen formation.