Dr. Lee Rubin

Biography

Lee Rubin investigates key molecular mediators of neurodegenerative diseases with the ultimate goal of finding effective preclinical therapeutic candidates. As co-leader of the Harvard Stem Cell Institute's Nervous System Diseases Program, he has extensive experience in translational neuroscience research across academia and industry. Rubin's group has explored cell biology related to the modulation of the blood-brain barrier (BBB), leading to the discovery of Tysabri, a drug that blocks the trafficking of activated T-lymphocytes to the brain in multiple sclerosis. His laboratory has also uncovered small molecule agonists and antagonists of the hedgehog-signaling pathway, with the antagonist partnered with Genentech as a treatment for metastatic basal cell carcinoma. The agonists are widely utilized by stem cell researchers to generate motor neurons from stem cells. Since joining Harvard, Rubin has focused on generating differentiated patient-specific neurons from induced pluripotent stem cells to model diseases and discover therapies for Parkinson’s disease (PD) and neuromuscular disorders like Amyotrophic Lateral Sclerosis (ALS) and Spinal Muscular Atrophy (SMA). His lab has begun a series of experiments aimed at identifying aging-related factors, such as GDF11, to stimulate functional improvements in patients suffering from neurodegenerative diseases.

Research Interests