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måndag 17 november 2014

Antisense PMO


Warfield and colleagues designed antisense Phosphorodiamidate Morpholino Oligomers (PMOs) — oligonucleotide analogues incorporating a phosphorodiamidate link and a morpholine ring — to target EBOV VP24, VP35 and RNA-dependent RNA polymerase (L protein).
 In contrast to oligonucleotides, PMOs have several drug-like properties — for example, they are non-ionic in character and therefore permeate cells more easily, are resistant to degradation in cells and do not seem to induce an interferon response, all of which make them attractive as potential therapeutic agents.

Antisense antiviral compounds and methods for treating a filovirus infection
EP 2603220 A1
The present invention provides antisense antiviral compounds, compositions, and methods of their use and production, mainly for inhibiting the replication of viruses of the Filoviridae family, including Ebola and Marburg viruses. 
The compounds, compositions, and methods also relate to the treatment of viral infections in mammals including primates by Ebola and Marburg viruses. 
The antisense antiviral compounds include phosphorodiamidate morpholino oligonucleotides (PMO plus) having a nuclease resistant backbone, about 15-40 nucleotide bases, at least two but typically no more than half piperazine-containing intersubunit linkages,
and a targeting sequence that is targeted against the AUG start site region of  Ebola virus VP35,
 Ebola virus VP24, Marburg virus VP24, or Marburg virus NP,
including combinations and mixtures thereof.

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