full-length dysferlin transfer by the hyperactive sleeping beauty transposase restores dysferlin-deficient muscle
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ID: 198614
2016
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Abstract
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 transfer vector, already used in clinical trials. The hyperactive SB system consists of a transposon DNA sequence and a transposase protein, SB100X, that can integrate DNA over 10 kb into the target genome. We constructed an SB transposon-based vector to deliver full-length human DYSF cDNA into dysferlin-deficient H2K A/J myoblasts. We demonstrate proper dysferlin expression as well as highly efficient engraftment (>1,100 donor-derived fibers) of the engineered myoblasts in the skeletal muscle of dysferlin- and immunodeficient B6. Cg-Dysfprmd Prkdcscid/J (Scid/BLA/J) mice. Nonviral gene delivery of full-length human dysferlin into muscle cells, along with a successful and efficient transplantation into skeletal muscle are important advances towards successful gene therapy of dysferlin-deficient muscular dystrophy.
| Reference Key |
escobar2016molecularfull-length
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| Authors | ;Helena Escobar;Verena Schöwel;Simone Spuler;Andreas Marg;Zsuzsanna Izsvák |
| Journal | coordination chemistry reviews |
| Year | 2016 |
| DOI |
10.1038/mtna.2015.52
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