Intronic SMCHD1 variants in FSHD: Testing the potential for CRISPR-Cas9 genome editing

Remko Goossens, Marlinde L. Van Den Boogaard, Richard J.L.F. Lemmers, Judit Balog, Patrick J. Van Der Vliet, Iris M. Willemsen, Julie Schouten, Ignazio Maggio, Nienke Van Der Stoep, Rob C. Hoeben, Stephen J. Tapscott, Niels Geijsen, Manuel A.F.V. Gonçalves, Sabrina Sacconi, Rabi Tawil, Silvère M. Van Der Maarel

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29 Citations (Scopus)

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Biochemistry, Genetics and Molecular Biology