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Surgical resection and liver transplantation are currently the best curative options to treat liver cancer. However, recurrence or metastasis is common. Hence, there is a need to develop new therapeutic strategies for treating this type of cancer effectively. A role for microRNAs miRNAs in cancer cell proliferation, survival, migration, and cell death has been extensively documented.

Previous studies from our laboratory and others provide evidence for miRNA replacement as an efficacious, non-toxic anti-cancer therapeutic strategy. In order to assess whether miR downregulation functionally contributes to hepatocarcinogenesis, we delivered 1e12vg of scAAV8.

Interestingly, miR treated animals showed significantly lower tumor burden 7. Moreover, therapeutic strategies to reduce endogenous miR levels using antisense oligonucleotides are currently being clinically investigated. Nevertheless, the consequences of sustained loss of miR have not been yet been determined. To understand the biological function of miR, we generated germ-line and liver- specific knockout mice.