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

Panagiotopoulos AL, Karguth N, Pavlou M, Bohm S, Gasparoni G, Walter J, Graf A, Blum H, Biel M, Riedmayr LM, Becirovic E
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