Full-length Dysferlin Transfer by the Hyperactive Sleeping Beauty Transposase Restores Dysferlin-deficient Muscle

Dysferlin-deficient muscular dystrophy is a progressive disease characterized by muscle weakness and wasting for which there is no treatment. It is caused by mutations in DYSF, a large, multiexonic gene that forms a coding sequence of 6.2 kb. Sleeping Beauty (SB) transposon is a nonviral gene transf...

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Bibliographic Details
Main Authors: Helena Escobar (Author), Verena Schöwel (Author), Simone Spuler (Author), Andreas Marg (Author), Zsuzsanna Izsvák (Author)
Format: Book
Published: Elsevier, 2016-01-01T00:00:00Z.
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3rd Floor Main Library

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Call Number: A1234.567
Copy 1 Available