dysferlin

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1
Antisense-Mediated Skipping of Dysferlin Exons in Control and Dysferlinopathy Patient-Derived Cells.
Verwey, Nisha;Gazzoli, Isabella;Krause, Sabine;Mamchaoui, Kamel;Mouly, Vincent;Aartsma-Rus, Annemiek...
2019 nucleic acid therapeutics DOI: 10.1089/nat.2019.078... 250 views
2
full-length dysferlin transfer by the hyperactive sleeping beauty transposase restores dysferlin-deficient muscle
;Helena Escobar;Verena Schöwel;Simone Spuler;Andreas Marg;Zsuzsanna Izsvák
2016 coordination chemistry reviews DOI: 10.1038/mtna.2015.52 188 views

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nucleic acid therapeutics
ISSN: 2159-3345 271 views

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