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A recurrent COL6A1 pseudoexon insertion causes muscular dystrophy and is effectively targeted by splice-correction therapies

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Description scientific article published on 21 March 2019
Author/s

author: Eric Hanssen  Shireen R Lamandé  Haiyan Zhou  Daniel G. MacArthur  Steve D Wilton  Francesco Muntoni  Cecilia Jimenez-Mallebrera 

Publication date March 21, 2019
Language English
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