Antisense Oligonucleotide- and CRISPR-Cas9-Mediated Rescue of mRNA Splicing for a Deep Intronic CLRN1 Mutation

• Main Authors: Anna-Lena Panagiotopoulos (Author), Nina Karguth (Author), Marina Pavlou (Author), Sybille Böhm (Author), Gilles Gasparoni (Author), Jörn Walter (Author), Alexander Graf (Author), Helmut Blum (Author), Martin Biel (Author), Lisa Maria Riedmayr (Author), Elvir Becirovic (Author)
• Format: Book
• Published: Elsevier, 2020-09-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

Mutations in CLRN1 cause Usher syndrome (USH) type III (USH3A), a disease characterized by progressive hearing impairment, retinitis pigmentosa, and vestibular dysfunction. Due to the lack of appropriate disease models, no efficient therapy for retinitis pigmentosa in USH patients exists so far. In...