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Cambridge, MA, United States

Kole R.,Sarepta Therapeutics
Discovery medicine | Year: 2012

Several clinical trials have recently demonstrated that oligonucleotide-based drugs induced targeted exon skipping in dystrophin pre-mRNA in Duchenne muscular dystrophy patients, resulting in novel expression of a truncated but functional isoform of the dystrophin protein. Such exon skipping therapy has the potential to convert the lethal Duchenne phenotype into the less severe Becker phenotype. This splice switching technology has been shown to be very well tolerated and may become the first gene-specific therapy, if approved, for the treatment of Duchenne muscular dystrophy. Source


Kole R.,Sarepta Therapeutics | Krieg A.M.,Checkmate Pharmaceuticals
Advanced Drug Delivery Reviews | Year: 2015

Duchenne muscular dystrophy (DMD) is caused mostly by internal deletions in the gene for dystrophin, a protein essential for maintaining muscle cell membrane integrity. These deletions abrogate the reading frame and the lack of dystrophin results in progressive muscle deterioration. DMD patients experience progressive loss of ambulation, followed by a need for assisted ventilation, and eventual death in mid-twenties. By the method of exon skipping in dystrophin pre-mRNA the reading frame is restored and the internally deleted but functional dystrophin is produced. Two oligonucleotide drugs that induce desired exon skipping are currently in advanced clinical trials. © 2015 Elsevier B.V. Source


Patent
Sarepta Therapeutics | Date: 2015-06-18

Antisense molecules capable of binding to a selected target site in the human dystrophin gene to induce exon 53 skipping are described.


Patent
Sarepta Therapeutics | Date: 2015-09-17

Provided are antisense molecules capable of binding to a selected target site in the human dystrophin gene to induce exon skipping, and methods of use thereof to treat muscular dystrophy.


Patent
Sarepta Therapeutics | Date: 2015-09-18

Provided are antisense molecules capable of binding to a selected target site in the human dystrophin gene to induce exon skipping, and methods of use thereof to treat muscular dystrophy.

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