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Cambridge, MA, United States

Patent
Sarepta Therapeutics | Date: 2014-06-04

A method and oligonucleotide compound for inhibiting replication of a nidovirus in virus-infected animal cells are disclosed. The compound (i) has a nuclease-resistant backbone, (ii) is capable of uptake by the infected cells, (iii) contains between 8-25 nucleotide bases, and (iv) has a sequence capable of disrupting base pairing between the transcriptional regulatory sequences in the 5 leader region of the positive-strand viral genome and negative-strand 3 subgenomic region. In practicing the method, infected cells are exposed to the compound in an amount effective to inhibit viral replication.


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.


Patent
Sarepta Therapeutics | Date: 2015-05-11

Provided are LMNA-targeted antisense oligonucleotides for reducing expression of one or more aberrantly spliced LMNA mRNA isoforms that encode progerin.

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