Adenine base editing of the DUX4 polyadenylation signal for targeted genetic therapy in facioscapulohumeral muscular dystrophy
Šikrová, Darina, Cadar, Vlad A., Ariyurek, Yavuz, Laros, Jeroen F.J., Balog, Judit, van der Maarel, Silvère M.
Published in Molecular therapy. Nucleic acids (03.09.2021)
Published in Molecular therapy. Nucleic acids (03.09.2021)
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