Tom Maniatis, Ph.D. spent most of his career as a self-described “basic scientist” studying genes. However, when his sister was diagnosed with ALS 12 years ago, Dr. Maniatis became involved in ALS research. Initially he served as an advisor to the ALS Association, but after ALS took his sister’s life he initiated research in his own laboratory.

His current ALS research focus is to identify early events in the ALS disease process. “Scientists have been studying the causes of ALS in mouse models for many years, and these studies have provided many insights. However, it has been very difficult to pinpoint the earliest events that trigger the disease” said Dr. Maniatis.

Dr. Maniatis' laboratory is exploiting new stem cell technology that has made it possible to reprogram skin cells from ALS patients to produce motor neurons for studies in cell culture. He is applying powerful tools for genome research to these cells in an effort to detect molecular differences between normal and ALS motor neurons.

“We hope that studies of motor neurons produced in this manner will provide clues to the cause of ALS”, said Dr. Maniatis. "In fact, preliminary data has revealed significant differences between motor neurons from control and ALS patient samples."

Dr. Maniatis is particularly excited about the recent discovery in other labs that two genes that cause ALS when altered by mutations (familial ALS) also happen to function in gene expression - the focus of his basic research program. Thus, his lab has combined the approaches to study ALS described above with those of his basic research to search for new clues to the causes of ALS.

Dr. Maniatis is the Isidore S. Edelman Professor and Chairman of Biochemistry and Molecular Biophysics at the Columbia University Medical Center. He received his bachelor’s degree from the University of Colorado at Boulder, and a doctorate in molecular biology from Vanderbilt University. His postdoctoral studies were carried out at Harvard University and the Medical Research Council for Molecular Biology in Cambridge, England. He’s held research and academic positions at Harvard University, California Institute of Technology and the Cold Spring Harbor Laboratory. He also serves on the Board of Directors of the ALS Therapy Alliance.

Dr. Maniatis has won numerous awards, including the Eli Lilly Award in Microbiology and Immunology early in his career, and more recently the Scientific Achievement Award of the American Medical Association. He is a member of the U.S. National Academy of Science.

He’s best known for pioneering the development and application of recombinant DNA methods to the study of gene regulation. His research has impacted a broad spectrum of biomedical fields, from basic mechanisms of gene expression to advances in understanding human genetic and inflammatory diseases. He has also been a pioneer in biotechnology, founding companies that developed several FDA approved drugs. His leadership was recognized by the Jacob Heskel Gabbay Award in Biotechnology and Medicine.

For more information about Dr. Maniatis, visit or Directors & Staff page.