Page 4 - RNA Biology | University of Rochester Medical Center

A World Leader in RNA Biology

The Center for RNA Biology boasts one of the largest concentrations of RNA biology experts of any center in the world.
Lynne Maquat, Ph.D., is director of the center and the J. Lowell Orbison Distinguished Service Alumni Professor. She is

one of the world’s premier RNA researchers and was recently elected to the prestigious National Academy of Sciences for
her groundbreaking work.

The research being done—with five areas featured below—is wide-ranging: from averting the health care catastrophe of
drug-resistant bacteria to developing RNA-based treatments for dementia and Lou Gehrig’s disease, to creating defenses
against bio-terrorist threats from arenaviruses. This research is also highly collaborative, drawing not just from the
resources and experts of the Medical Center, but from throughout the University, including the College of Arts, Sciences
and Engineering.

MAKING SENSE OUT OF “NONSENSE”

One of the most damaging things that can happen in the production
of RNA is a “nonsense” mutation, a mutation that tells the cell’s

ribosomes to stop making a protein partway through the process.
The resulting proteins are too short, so they cannot do their jobs
properly. One-third of all inherited and acquired diseases are caused by
nonsense or “frameshift” mutations. Dr. Maquat’s work has provided
an understanding of the process the body uses to get rid of RNA with
nonsense mutations. By controlling the process better, her work holds
the potential for making current drugs far more effective, while also

reducing their toxicity.

CHANGING THE BLUEPRINT OF DISEASE

Messenger RNA carries “blueprints” from DNA. These blueprints are
then used to build proteins that carry out many biological functions.
But when an RNA blueprint contains a mistake, it produces defective
proteins which cause diseases such as cystic fibrosis, muscular dystrophy,
and many forms of cancer. The work of Yi-Tao Yu, Ph.D., is showing
that abnormal RNA can be modified before it has a chance to cause
disease. His work, covered widely in national media, is revealing ways to

modify specific sites on the RNA molecule so that it does the work it was
intended to do. This research promises to give us a better understanding
of aging, and new treatment approaches for various diseases.