Jaap Oostrik

This study develops patient-derived inner ear organoids to evaluate antisense oligonucleotide therapies for hereditary hearing loss, showing effective ASO uptake and splice correction in disease models.

This study demonstrates that antisense oligonucleotides targeting the c.151C>T mutation in COCH can reduce mutant transcript levels in transgenic cells, suggesting potential as a therapy for DFNA9.

The paper identifies an in-frame deletion in RIPOR2 as a frequent and highly penetrant cause of adult-onset hearing loss, with implications for genetic therapy.