Allele-specific invalidation of dominant-negative mutations in PRPH2 underlying CACD 

Antisense oligonucleotide therapy for CEP290-associated LCA

Antisense oligonucleotide therapy for ABCA4-associated Stargardt disease

Delivery of antisense oligonucleotides to the retina

Development of novel cellular models to assess therapeutic efficacy

Expanding the use of antisense oligonucleotide therapy for other subtypes of IRDs

Gene augmentation therapy for EYS- or PCARE-associated RP

Therapeutic genome editing for ABCA4-­associated Stargardt disease


Radboud university medical center

Department of Human Genetics

Route 855

Geert Grooteplein-Zuid 30

6525 GA Nijmegen

The Netherlands