Splicing Modulation
Splicing modulation applies short oligonucleotides that lead to skipping of the mutated region, thereby restoring the activity of the protein.
Prime Editing
Prime editing is a novel technology that can precisely correct mutations in USH2A or EYS.
Partial cDNA insertion
This technology aims to insert a part of the normal coding sequence just in front of the mutation to restore function.
Alternative genome editing
We explore novel gene editors that would allow targeted gene modifications for retinitis pigmentosa.
AAV split intein
Inteins can be used to split a large gene – such as EYS and USH2A – allowing for delivery with AAV vectors
Viral vectors
High capacity viral vectors could carry to full coding sequence of EYS and USH2A.
Non-viral vectors
For large genes, encapsulation in non-viral vectors is promising, as they do not suffer from the limited loading capacity of AAVs.