GET-READY develops therapies for retinitis pigmentosa.
In partnership with
GET-READY is an international consortium between seven research groups. It formed in 2021 under the European Joint Programme for Rare Diseases. Our collaboration aims to find treatments for retinitis pigmentosa, an unmet medical need that leads to vision loss. We focus on developing novel treatments for certain forms of retinitis pigmentosa, which are caused by mutations in the EYS and USH2A genes. We explore alternative and complementary approaches to maximize our chance of success and to rapidly bring potential therapies to the clinic.
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 ā such as EYS and USH2A ā 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.
Where can I get more Information?
Please watch our videos below in six different languages!
If you are interested, either contact us, or contact the respective patient organization in your country (here link to patient organisations)
