Phase 2/3 anti-sense treatment (”QR-421a”) of retinitis pigmentosa (RP) aims to use exon skipping within the USH2A gene to repair the shortened usherin protein.
Dutch researchers at the Radboud University Medical Centre, Nijmegen, and ProQR Therapeutics, a company, based in Leiden, the Netherlands, are enrolling up to 200 patients… Read More »Phase 2/3 anti-sense treatment (”QR-421a”) of retinitis pigmentosa (RP) aims to use exon skipping within the USH2A gene to repair the shortened usherin protein.