University of Rochester

Researchers Receive $2.3 Million for Gene Therapy Research

March 25, 1997

A gene therapy laboratory at the University of Rochester has received two grants totaling more than $2.3 million from the National Institutes of Health (NIH) to develop new ways to modify genes to eventually treat a range of neurodegenerative diseases such as Parkinson's and Alzheimer's diseases. The grants are to a group led by Howard Federoff, M.D./Ph.D., chief of the University's Division of Molecular Medicine and Gene Therapy and professor in the Department of Neurology.

A five-year, $1.4 million grant will allow Dr. Federoff to continue studying ways to improve control over genes of the nervous system. Dr. Federoff and one of his students, Andy Brooks, recently developed a new technology that allows scientists to turn on or off a gene in the adult nervous system of mice whenever and wherever they want. The grant allows the team to continue this basic research, a necessary step on the road to clinical use of gene therapy against disease.

The NIH also contributed a four-year, $916,000 grant toward Dr. Federoff's work developing improved "gene shuttles," ways to carry into cells genetic material that the brain needs to be healthy. Dr. Federoff will concentrate on the development of particles based on non-infectious herpes virus particles for use in the treatment of Parkinson's disease. Just last month a research team led by one of Dr. Federoff's colleagues, Dr. Martha Bohn, Ph.D., used gene therapy to protect brain cells in animals with Parkinson's-like symptoms. Dr. Federoff will explore new ways to protect these cells more effectively and for longer periods of time.