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Friday Evening Lecture Series
08/17/07
Return to the RNAi World: Rethinking Gene Expression, Evolution and Medicine
Craig Mello, University of Massachusetts Medical Center, 2006 Nobel Laureate
Introduction by Helen M. Blau
Lecture Abstract:
While investigating the genetic workings of the microscopic worm, C. elegans, Mello and colleague Andrew Fire, Ph.D., of the Carnegie Institution of Washington, discovered RNAi, a natural but previously unrecognized process by which a certain form of RNA can be manipulated to silence—or interfere with—the expression of a selected gene. The discovery, published in the journal Nature in 1998, 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 out 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. RNAi is now showing promising in the clinic as a new class of gene-specific therapeutics.
Craig C. Mello is the Blais University Chair in Molecular Medicine at the University of Massachusetts Medical School (UMMS) and is an investigator of the Howard Hughes Medical Institute. Dr. Mello received his B.S. in biochemistry from Brown University and his Ph.D. in Cellular and Developmental Biology from Harvard University. He was a postdoctoral fellow at the Fred Hutchinson Cancer Research Center before coming to UMMS in 1995. He was also a 1995 Pew Scholar in the Biomedical Sciences.
Dr. Mello and his colleague Andrew Fire of Stanford, formerly of the Carnegie Institution of Washington, received the 2006 Nobel Prize in Medicine for their discovery of RNA interference (RNAi). They demonstrated that a certain form of RNA had the unanticipated property of silencing 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 offers astounding potential for understanding and manipulating the cellular basis of human disease, 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.
Given the fundamental and broad-based impact of RNAi, a patent, "Genetic Inhibition by Double-Stranded RNA," (US Patent 6,506,559 B1) issued to UMMS and Carnegie, is expected to have far-reaching licensing potential both in the lab and in drug development.
Dr. Helen M. Blau will introduce Dr. Mello. Dr. Blau is the Donald E. and Delia B. Baxter Professor, Director of the Baxter Laboratory in Genetic Pharmacology, and member of the Stem Cell Institute at Stanford University School of Medicine. She received a B.A. from the University of York in England, and an M.A. and Ph.D. from Harvard University. She completed her postdoctoral research at the University of California, San Francisco. Dr. Blau accepted the position of Assistant Professor in the Department of Pharmacology at the Stanford University School of Medicine in 1978, rising through the ranks of Associate Professor (1986 – 1991), Professor (1991 – 1999), and Chair (1997 – 2002) of the department. She has served on the editorial boards of a number of journals and is currently Associate Editor of The FASEB Journal. Dr. Blau has been the recipient of many honors and awards over the course of her career, including presentation of the Nobel Forum Lecture, Karolinska Institutet, Sweden (1995), a MERIT Award from the National Institutes of Health (1995-2005), recipient of a FASEB Excellence in Science Award (1999), an Honorary Doctorate from the University of Nijmegen, Holland (2003), and a Fulbright Senior Specialist Award (2007). She serves on the Scientific Advisory Board of the Ellison Medical Foundation and is an elected member of the American Association for the Advancement of Science, the Institute of Medicine of the National Academy of Sciences, the American Academy of Arts and Sciences, and Harvard University's Board of Overseer’s.
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