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

por Panagiotopoulos, Anna-Lena, Karguth, Nina, Pavlou, Marina, Böhm, Sybille, Gasparoni, Gilles, Walter, Jörn, Graf, Alexander, Blum, Helmut, Biel, Martin, Riedmayr, Lisa Maria, Becirovic, Elvir
Publicado 2020