- Genome editing for Rhodopsin associated dominant RP
- Genome editing for USH2A associated Usher Syndrome
- Viral or nonviral-based gene augmentation therapy and CRISPR/Cas9 genome editing for RP1 associated RP
- Transcript isoform as an alternative therapeutic strategy for RP1 associated RP
- Oligonucleotide-based gene editing for the treatment of ophthalmology indications