Antisense Oligonucleotide-based Splice Correction for USH2A-associated Retinal Degeneration Caused by a Frequent Deep-intronic Mutation

• Main Authors: Radulfus WN Slijkerman (Author), Christel Vaché (Author), Margo Dona (Author), Gema García-García (Author), Mireille Claustres (Author), Lisette Hetterschijt (Author), Theo A Peters (Author), Bas P Hartel (Author), Ronald JE Pennings (Author), José M Millan (Author), Elena Aller (Author), Alejandro Garanto (Author), Rob WJ Collin (Author), Hannie Kremer (Author), Anne-Françoise Roux (Author), Erwin Van Wijk (Author)
• Format: Book
• Published: Elsevier, 2016-01-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

Usher syndrome (USH) is the most common cause of combined deaf-blindness in man. The hearing loss can be partly compensated by providing patients with hearing aids or cochlear implants, but the loss of vision is currently untreatable. In general, mutations in the USH2A gene are the most frequent cau...