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Member Research and Reports

Member Research and Reports

Johns Hopkins: A Fundamental Discovery About How Gene Activity Is Regulated

Researchers at Johns Hopkins Bloomberg School of Public Health have discovered a fundamental mechanism that regulates gene activity in cells. The newly discovered mechanism targets RNA, or ribonucleic acid, a close cousin of DNA that plays an important role in cellular activity.

The discovery, detailed in a paper published February 3 in the journal Molecular Cell, is a significant addition to the foundational understanding of how gene activity is regulated, and may ultimately lead to powerful new medical treatments.

The newly discovered mechanism effectively silences or dials down certain active genes as a basic cellular regulatory or quality control system. It may even act as a defense against viruses. When genes are active, they are copied out into strands of RNA. These RNA strands perform cellular functions on their own or are translated into proteins. The new mechanism destroys RNA strands that have excessively folded over and stuck to themselves to form knots, hairpins, and other structures. These highly structured RNAs can occur during normal processing but could possibly also be caused by misfolding.

The finding is likely to have implications for medical research because many human disorders, including cancers and neurodegenerative diseases, such as ALS (Amyotrophic Lateral Sclerosis) and Huntington disease-like syndromes, involve failures of normal RNA regulation and/or the accumulation of abnormally folded or tangled RNA in affected cells.

Dr. Anthony K. L. Leung, associate professor in the Bloomberg School’s department of biochemistry and molecular biology, was the principal investigator.

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