GeneticsbioRxivPreprint
Allele-specific antisense oligonucleotide therapy for dominantly inherited hearing impairment DFNA9.
Erik de Vrieze, Jolien Peijnenborg, Jorge Cañas-Martín…·Erwin van Wijk
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.
DOI 10.1101/2020.09.29.316364