Erwin van Wijk
Dr. Erwin van Wijk is an Associate Professor at the Department of Otorhinolaryngology and the Donders Institute for Brain, Cognition and Behaviour (Radboud University Medical Center, Nijmegen, The Netherlands). He is head of the Usher Syndrome & Hereditary Hearing Loss Therapeutics Research Unit. From 2012 onwards he is exploring different therapeutic strategies to develop a genetic therapy for USH2A-associated retinal degeneration thereby employing the zebrafish model for evaluating therapeutic efficacy. Using the method of antisense oligonucleotide-based splice modulation he obtained pre-clinical proof of concept for the redirection of aberrant splicing caused by a common deep-intronic mutation in the USH2A gene. Furthermore he explored the use of antisense oligonucleotides for the purpose of skipping of the most frequently mutated exon in the USH2A gene, for which a phase 1/2 clinical trial was just successfully completed. The current work of his research team is dedicated to better understand the molecular mechanisms underlying different clinical types of Usher syndrome, non-syndromic inherited retinal dystrophies and inherited hearing impairment, and to directly apply that knowledge for the development of new treatment strategies for the many visually and hearing impaired individuals worldwide.
Patients suffering from hereditary forms of hearing loss are currently being provided with hearing aids or cochlear implants. Although these devices have undergone several major technical improvements overtime, they will never be able to compete with the quality of natural hearing. In the past couple of years several new developments in the area of genetic therapies have emerged. Although these developments are still in early days, some significant progress has already been made. In this session we will highlight some recent developments in the field of RNA therapies and gene augmentation, but also emphasize the hurdles that scientists and clinicians still have to overcome, such as delivery and identification of suitable clinical endpoints to determine the efficacy of treatments.