The new therapy could potentially replace interferon in future cocktails, since it provides a high barrier to resistance. "This antiviral could be used alone to treat disease progression and there are indications that it can convert interferon non-responders to responders, so that non-responders to the current therapy could be treated with the combination of this drug with interferon," Lanford said. It may also be a good therapy to use after transplant, since it may help suppress HCV in the new liver. It has no toxic or adverse reactions and this is critical in the transplant setting.

In the study, four HCV chronically infected chimpanzees were treated with the new antiviral. The two animals that received the higher dose had a drop in viral levels in the blood and liver of 2.5 orders of magnitude or approximately 350 fold. The surprising findings were the lack of antiviral resistant mutants and the fact that the therapy continued to work for several months after dosing stopped.

In one advance, the new study was a critical proof of concept that the LNA technology could work for HCV. It proved that miR-122 was truly required for HCV replication in an animal infected with HCV. Previously the role of miR-122 in HCV replication had only been shown in tissue culture. A second advance was the finding that LNA therapy could work against an important disease model, suggesting that the new technology can be applied to other diseases.

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