microRNA Therapy More Effective than Short Hairpin RNA

Researchers at Amsterdam Molecular Therapeutics (AMT) have announced the results of an in vivo study comparing the two as treatment for high cholesterol.  The molecules were delivered with an injection of their adeno-associated viral vectors programmed with a liver-specific  promoter for expression.  The miRNA therapy was able to significantly lower plasma cholesterol levels over a period of 18 weeks, while the shRNA therapy began to wear off after 8 weeks.

“Certainly of the two approaches, the natural characteristics of miRNA, such as prolonged stable long term expression, undetectable toxicity profile, tissue specificity, as well as the significant efficacy, suggest a more favorable route to a viable treatment for high cholesterol,” stated Jorn Aldag, CEO of AMT.

(Read the press release… )

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