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AssigneeSarepta Therapeutics, Inc.
InventorsDavid A. Stein, Richard K. Bestwick, Patrick L. Iversen, Dwight D. Weller
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The invention provides antisense antiviral compounds and methods of their use and production in inhibition of growth of viruses of the Picornaviridae family and in the treatment of a viral infection. The compounds are particularly useful in the treatment of Enterovirus and/or Rhinovirus infection in a mammal. The antisense antiviral compounds are substantially uncharged, morpholino oligonucleotides have a sequence of 12-40 subunits, including at least 12 subunits having a targeting sequence that is complementary to a region associated with viral RNA sequences within a 32 nucleotide region of the viral 5′ untranslated region identified by SEQ ID NO:7.
A method of treating viral infection of mammalian cells by an Enterovirus or Rhinovirus, comprising exposing host cells to an effective amount of an antiviral compound that comprises a morpholino antisense oligonucleotide of between 15 and 25 morpholino subunits having a substantially uncharged, nuclease-resistant backbone containing phosphorodiamidate intersubunit linkages joining a morpholino nitrogen of one subunit to a 5′ exocyclic carbon of an adjacent subunit, wherein the morpholino antisense oligonucleotide comprises a targeting sequence of at least 15 nucleotide bases complementary to SEQ ID NO:7 and, conjugated to the morpholino antisense oligonucleotide, an arginine-rich polypeptide effective to promote uptake of the antiviral compound into the host cells, wherein the arginine-rich polypeptide has the sequence of SEQ ID NO:15.
Dependencies are inferred from the claim text.