Antisense Oligonucleotide based Therapies for Inherited Retinal Diseases From Mutation to Clinical

The eye is at the forefront of therapeutic development for its unique properties. Local delivery of ASOs to the retina has shown to be well tolerated and safe. During this webinar, Prof. Alex Garanto will explain the steps they are following to identify pathogenic variants, model the defect in human-based cellular models and the use of ASOs to correct splicing defects in IRDs. Prof. Garanto will also touch upon on how antisense technology can be used in the clinics using ongoing clinical trials as an example.