Grant to develop new therapies for inherited blindness

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Alex Garanto (photo up) and Rob Collin from the Dept. of Human Genetics, theme Sensory disorders were recently awarded with € 241,074 to conduct their research project entitled “Expanding Splice Modulation Therapies for Inherited Retinal Dystrophies”.  

Previously, the group already showed the therapeutic potential of antisense oligonucleotides (AONs) to correct the splicing defects resulting from a recurrent deep-intronic mutation in CEP290 underlying congenital blindness. Based on these promising results, the aim of this research project is to further expand AON-based therapy to other genes and different types of mutations underlying inherited retinal disorders.

This grant was awarded by the Uitzicht call 2014/2015, and contributions were received from the Stichting Maculafonds, Stichting Oogfonds Nederland, Algemene Nederlandse Vereniging ter Voorkoming van Blindheid, Landelijke Stichting voor Blinden en Slechtzienden, Stichting Blinden-Penning, the Retina Fonds, the Rotterdamse Stichting Blindenbelangen, Stichting Blindenhulp, Stichting tot Verbetering van het Lot der Blinden, Stichting Dowilvo and Stichting voor Ooglijders.

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