Work also hailed as 2002 “Breakthrough of the Year” in Science magazine

January 23, 2003

WORCESTER, Mass. -- Craig C. Mello, PhD, a cell biologist at the University of Massachusetts Medical School, has won the prestigious Award in Molecular Biology from the National Academy of Sciences for his work in discovering a process by which a particular form of ribonucleic acid—or RNA, the cellular material responsible for the transmission of genetic information—can silence targeted genes. The process, now known as RNA interference, or RNAi, offers astounding potential for understanding and manipulating the cellular basis of human disease.   Dr. Mello’s work in RNAi, hailed as “an electrifying discovery,” has also been named the 2002 “Breakthrough of the Year” by Science magazine.

“It has been extremely exciting and rewarding to see RNA interference grow as a research tool and field of study—from a strange phenomenon in a simple microscopic worm into so many exciting new discoveries and whole new research fields,” said Mello.

“Craig Mello is a brilliant example of the UMass Medical School researcher—innovative and passionate about his work,” said UMMS Chancellor and Dean Aaron Lazare. “We are thrilled to see him receive such distinguished recognition.”

Mello, a Howard Hughes Medical Institute Investigator and associate professor of molecular medicine and cell biology, and colleague Andrew Fire, PhD, of the Carnegie Institution of Washington in Baltimore, Maryland, demonstrated that a certain form of RNA had the unanticipated property of silencing—or interfering with—the expression of a gene whose coding sequence of DNA was similar to that of the RNA they tested.  The RNAi mechanism—a natural response of an organism to double-stranded RNA, of which many viruses are comprised—destroys the gene products that a virus needs to replicate itself, essentially halting the progression of the invading viral infection.

The discovery has had two extraordinary impacts on biological science. One is as a research tool: RNAi is now the state-of-the-art method by which scientists can knock down the expression of specific genes in cells, to thus define the biological functions of those genes.  But just as important has been the finding that RNA interference is a normal process of genetic regulation that takes place during development.  Thus, RNAi has provided not only a powerful research tool for experimentally knocking out the expression of specific genes, but has opened a completely new and totally unanticipated window on developmental gene regulation.

The National Academy of Science is a private, non-profit, self-perpetuating society of distinguished scholars engaged in scientific and engineering research, dedicated to the furtherance of science and technology and to their use for the general welfare. The NAS also advises the federal government on scientific and technical matters, as mandated by a charter granted in 1863 by Congress. The NAS selects just 18 individual researchers to receive awards honoring their outstanding scientific achievements; this year’s awards will be presented April 28 at a ceremony during the NAS 140th annual meeting, in Washington DC.

Mello’s award, which includes a medal and $25,000 prize, is awarded annually for a recent notable discovery in molecular biology by a young scientist.  Mello, who holds degrees from Brown University (BS in Biochemistry) and Harvard University (PhD in Cellular and Developmental Biology), was a postdoctoral fellow at the Fred Hutchinson Cancer Research Center before coming to UMMS in 1990.  In 2000 he was named an Investigator of the Howard Hughes Medical Institute (HHMI), one of the most prestigious and sought-after scientific awards in the world. HHMI is a $13 billion medical research organization that employs more than 350 eminent researchers at 72 medical schools, universities and research institutes worldwide. Mello, whose research into the genetics of the worm C. elegans provides important insights into human development and cancer, is the third HHMI researcher at UMMS, joining Michael R. Green, MD, PhD, the Lambi and Sarah Adams Chair in Genetic Research and  professor of molecular medicine, biochemistry & molecular pharmacology and surgery and director of the Program in Gene Function & Expression; and Roger J. Davis, PhD, the H. Arthur Smith Chair in Cancer Research and professor of molecular medicine and biochemistry & molecular pharmacology.

In addition to Mello’s work in establishing RNAi as a field of study, several other scientists at UMMS are also working in RNAi, particularly Phillip D. Zamore, PhD, associate professor of biochemistry & molecular pharmacology, whose research is focused on identifying the biochemical machinery that brings about RNAi. Specifically, his team is using extracts from fly embryos and human cells, purified in the test tube, in hopes of understanding the phenomena’s components at the molecular level― research that may eventually form the basis for a new class of drugs to treat human disease, including viral infections and some genetic disorders. Mario Stevenson, PhD, the David J. Freelander Professor of AIDS Research and professor of molecular medicine and molecular genetics & microbiology, is examining the use of one form of RNAi, small interfering or siRNA, to block HIV infection, while Tariq M. Rana, PhD, professor of biochemistry & molecular pharmacology, is studying RNAi in mammalian cells.

The University of Massachusetts Medical School, one of the fastest growing academic health centers in the country, has built a reputation as a world-class research institution, consistently producing noteworthy advances in clinical and basic research.  The Medical School attracts more than $143 million in research funding annually, 80 percent of which comes from federal funding sources.  Research funding enables UMMS scientists to explore human disease from the molecular level to large-scale clinical trials.  Basic and clinical research leads to new approaches for diagnosis, treatment and prevention of disease. Visit for additional information.

See the full article from the December 2002 issue of Science magazine. Additional information about the National Academy of Science is available at .



Alison Duffy