Lee L. Rubin, Ph.D.
Principal Faculty, Harvard Stem Cell Institute
Co-chair, HSCI Nervous System Disease Program
Lee Rubin investigates the key molecular mediators of different neurodegenerative diseases, with the ultimate goal of finding effective preclinical therapeutic candidates.
Dr. Lee Rubin, co-leader of the HSCI Nervous System Diseases Program, has a broad experience in translational neuroscience research in both academia and industry. Earlier in Rubin's career, his group investigated the cell biology of the blood-brain barrier (BBB) and its modulation. Some of this work led to the discovery of the multiple sclerosis drug Tysabri, an antibody that blocks the trafficking of activated T-lymphocytes in the brain. Subsequently, his laboratory discovered the first small molecule agonists and antagonists of the hedgehog-signaling pathway. One of the antagonists was partnered with Genentech and is now approved as a treatment for metastatic basal cell carcinoma. One of the agonists is now widely used by stem cell investigators to produce motor neurons and other types of neurons from stem cells.
Since coming to Harvard, Rubin's work is focused on producing differentiated patient-specific neurons from induced pluripotent stem cells to model disease and discover therapeutics, including Parkinson’s disease (PD) and the neuromuscular disorders Amyotrophic Lateral Sclerosis (ALS) and Spinal Muscular Atrophy (SMA). His lab has initiated a series of experiments focused on identifying aging related factors, such as GDF11, that may stimulate functional improvement in patients suffering from neurodegenerative diseases.
Dr. Rubin received his Ph.D. in neuroscience from the Rockefeller University and had postdoctoral training, also in Neuroscience, at Harvard Medical School and Stanford University School of Medicine.