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

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...

Fuld beskrivelse

Na minha lista:
Bibliografiske detaljer
Udgivet i:Mol Ther Nucleic Acids
Main Authors: Panagiotopoulos, Anna-Lena, Karguth, Nina, Pavlou, Marina, Böhm, Sybille, Gasparoni, Gilles, Walter, Jörn, Graf, Alexander, Blum, Helmut, Biel, Martin, Riedmayr, Lisa Maria, Becirovic, Elvir
Format: Artigo
Sprog:Inglês
Udgivet: American Society of Gene & Cell Therapy 2020
Fag:
Article
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC7452116/
https://ncbi.nlm.nih.gov/pubmed/32841912
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtn.2020.07.036
Tags: Tilføj Tag
Ingen Tags, Vær først til at tagge denne postø!

Lignende værker