BioCentury
ARTICLE | Translation in Brief

Setting boundaries

Stanford group finds toxicity ceiling for shRNA therapeutics

June 2, 2016 7:00 AM UTC

The Stanford University laboratory that first showed exogenous shRNA can cause liver damage by overloading the microRNA machinery has found the molecular culprit behind the toxicity, and proposed quantitative guidelines for safer shRNA therapeutics. While the researchers' new study in Nature Medicine suggests exogenous shRNA levels should be capped at 12% of total liver microRNA to avoid toxicity, they believe that still leaves a large window for therapeutic effects.

"We can piggyback on cellular microRNA machinery and use exogenous RNAi to treat a whole host of disorders, but obviously there'll be a limit to any compound," said Mark Kay, professor of pediatrics and genetics at Stanford and principal investigator on the study...