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Isis researchers identify microRNA target

Isis Pharmaceuticals has revealed results from preclinical studies demonstrating that inhibition of miR-122, a liver-specific microRNA, by a second-generation antisense drug significantly improves high cholesterol, fatty liver, and liver function without affecting blood glucose levels in diabetic mouse models.

MicroRNAs (miRNAs) are a recently discovered class of natural antisense RNAs that are important in the regulation of cellular functions.

These data demonstrate that antisense inhibition is a powerful technique to regulate the function of miRNAs and suggest that miR-122 may be an attractive therapeutic target for cardiovascular and metabolic diseases. The data were presented at the American Diabetes Association annual sessions in Washington.

“Inhibiting miRNAs using antisense drugs has the potential to lead to exciting new therapeutics. The specificity and efficiency of antisense technology allow us to make great progress in converting this opportunity into new therapeutics, just as they do with mechanisms such as RNAi and alternative splicing,” said Dr Sanjay Bhanot, vice president of metabolic disease research and development at Isis Pharmaceuticals.