Radboudumc
The "Molecular Therapy for Inherited Retinal Diseases" group, headed by Prof. dr. Rob Collin and his colleague Dr. Alex Garanto, aim to develop various therapeutic interventions for inherited retinal diseases, with a strong focus on splice modulation and RNA therapies.
Stargardt disease is a progressive retinal disease that initially affects central vison, and later in life can lead to complete blindness. Stargardt disease can be caused by numerous different mutations in the
ABCA4 gene, some of which cause pre-mRNA splicing defects in
ABCA4. Over the last decade, our group has demonstrated pre-clinical proof-of-concept for splicing modulation therapy using antisense oligonucleotides (AONs) for several different subtypes of retinal disease, including Stargardt disease. In addition, the first application of AONs in the human eye have revealed encouraging results, with improved visual function upon ocular administration of AONs in patients with retinal disease.
The research is embedded at the
Department of Human Genetics within the Radboudumc in Nijmegen, which is internationally renowned for its research and diagnostics on a variety of genetic and genomic disorders. The Department offers an exciting work environment with enthusiastic people from many parts of the world enjoying high quality research.
RadboudumcRadboud university medical center is a university medical center for patient care, scientific research, and education in Nijmegen. Radboud university medical center strives to be at the forefront of shaping the healthcare of the future. We do this in a person-centered and innovative way, and in close collaboration with our network. We want to have
a significant impact on healthcare. We want to improve with each passing day, continuously working towards better healthcare, research, and education. And gaining a better understanding of how diseases arise and how we can prevent, treat, and cure them, day in and day out. This way, every patient always receives the best healthcare, now and in the future. Because that is why we do what we do.
Read more about our
strategy and what
working at Radboud university medical center means.
Our colleagues would be happy to tell you about it.
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