Rochester Scientist Leads Federal Gene Therapy Advisory Group

September 08, 2006

A neuroscientist at the University of Rochester Medical Center has been named head of a Federal panel that oversees human gene therapy experiments throughout the nation.

Howard J. Federoff, M.D., Ph.D., senior associate dean for basic research, is the new chair of the Recombinant DNA Advisory Committee, a panel created by the National Institutes of Health that helps guide the nation’s approach to complex genetics studies involving people. The panel makes recommendations to NIH and the U.S. Food & Drug Administration about which studies should proceed as plan and which should be the subject of further public review; the goal is to assure that all studies are safe and conducted ethically.

Such experiments – efforts by scientists and physicians to cure or treat disease by manipulating our genes– offer great promise as more and more of our genetic code is worked out. Gene therapy generally involves replacing a defective gene with a working copy, or turning off a defective gene, or sometimes adding and then turning on several copies of a “good” gene designed to stop disease. The effort is extraordinarily complex: In many diseases dozens or even hundreds of genes play a role, while in others a single gene or protein holds the key to health. The burgeoning knowledge of our gene code is offering tantalizing new targets every day to scientists who are increasingly equipped with the means to change our genes and maybe save lives.

Despite the great promise, true gains to the health of large numbers of people have yet to be realized. Scientists cite the incredible complexity of human genetics, and the need for the highest safety standards, as reasons why the field has advanced more slowly than many people expected 20 years ago.

The group Federoff heads is comprised of physicians, scientists, medical ethicists, and other citizens who work together to provide the Federal government with broad, outside advice on proposals involving gene therapy. The group meets four times a year to review research proposals, and it also produces a set of research guidelines that scientists doing recombinant DNA research are expected to follow. Another Medical Center scientist, Stephen Dewhurst, Ph.D., professor of Microbiology and Immunology, is also a member of the committee.

Federoff is a pioneer in the world of gene therapy. He founded the Parkinson’s Disease Gene Therapy Study Group, an effort that has brought together experts around the nation to evaluate the most promising strategies to fight the disease using genetic manipulation. Parkinson’s is one of the most common diseases widely considered to be a good target for gene therapy, and Federoff leads the largest effort to date to bring gene therapy to the realm of Parkinson’s. Earlier this year, he helped to organize one of the largest international meetings ever to focus on Parkinson’s disease, the first world Parkinson Congress.

More broadly, he has helped create the field of gene therapy for diseases of the central nervous system. He has pioneered the use of a harmless form of the herpes virus as a new way to treat Parkinson’s and other diseases, and his group has discovered many of the molecular steps that occur as Parkinson’s progresses. His work on using gene therapy to create vaccines against AIDS, Alzheimer’s, and prion diseases has shown promise in initial testing, and studies are continuing. He is also an expert on identifying biomarkers, such as genes or proteins, which indicate that a person might develop diseases like Parkinson’s and Alzheimer’s.

A professor of Neurology, Medicine, and Microbiology & Immunology, Federoff is director of the University’s Center for Aging and Developmental Biology. He received his master's, doctoral and medical degrees from Albert Einstein College of Medicine in New York City, and did his internship and residency at Massachusetts General Hospital of Harvard Medical School.

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