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Breuel, Saskia and Vorm, Mariann and Bräuer, Anja U. and Owczarek-Lipska, Marta and Neidhardt, John (2019) Combining engineered U1 snRNA and antisense oligonucleotides to improve the treatment of a BBS1 splice site mutation. Molecular Therapy / Nucleic Acids, 17. pp. 123-130. ISSN 2162-2531