Lee Rubin investigates the key molecular mediators of different neurodegenerative diseases, with the ultimate goal of finding effective preclinical therapeutic candidates. His research group discovered that a circulating protein, GDF11, has the ability to reverse some of the changes in the CNS associated with aging. They are actively exploring the therapeutic implications of these observations.
The Rubin group takes advantage of their ability to produce large numbers of patient-derived induced pluripotent stem (iPS) cell lines and of effective means of deriving large numbers of differentiated neurons from them. They have set up an array of techniques that allow them to identify early cellular and physiological changes in neurons as they become diseased. For example, they have identified new targets for the treatment of the motor neuron disorders Spinal Muscular Atrophy (SMA) and Amyotrophic Lateral Sclerosis (ALS). They are also studying Autism Spectrum Disorders, Parkinson’s disease and Alzheimer’s disease.
Dr. Rubin received his Ph.D. in Neuroscience from The Rockefeller University and completed postdoctoral fellowships in Pharmacology from Harvard Medical School and in Neurobiology from Stanford University School of Medicine. He has a broad experience in both academia and industry, particularly in the realms of cell-based assays and drug discovery. Prior to coming to Harvard, Rubin was Chief Scientific Officer of Curis, Inc., a Cambridge-based biotechnology company, where his group identified the first small molecule regulators of the hedgehog signaling pathway. One of their antagonists was developed by Genentech and is now (as Erivedge) approved as the first oral treatment for metastatic basal cell carcinoma.
The Translational Science of Stem Cells
Through a series of lectures and assigned papers, students will be introduced to a broad view of the ways in which stem cells can be used for translational research. This will include human disease modeling, identifying drugs that target endogenous stem cells, or otherwise promote tissue repair, and regenerative medicine (cell-based therapies).