ONGOING RESEARCH LINES

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

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Antisense oligonucleotide therapy for CEP290-associated LCA

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Antisense oligonucleotide therapy for ABCA4-associated Stargardt disease

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Delivery of antisense oligonucleotides to the retina

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Development of novel cellular models to assess therapeutic efficacy

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

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Gene augmentation therapy for EYS- or PCARE-associated RP

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Therapeutic genome editing for ABCA4-­associated Stargardt disease

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Contact

Radboud university medical center

Department of Human Genetics

Route 855

Geert Grooteplein-Zuid 30

6525 GA Nijmegen

The Netherlands