Antisense oligonucleotide therapy for genetic heterogeneous rare disorders Ghent University
About 350 million people worldwide suffer from a rare disease, which is defined as a disorder that affects less than 1 in 2,000 individuals. Over 7,000 different rare disease entities have been identified, 80% of which have a genetic cause. Recent large-scale sequencing technologies enable fast and accurate genetic diagnosis, which is a prerequisite for genomic-based precision medicine. Major efforts are currently ongoing for therapy ...