Sandra Dabora, M.D., Ph.D.

Assistant Professor of Medicine

Translational Medicine Division
Brigham & Women’s Hospital
Harvard Medical School
One Blackfan Circle
Karp Family Research Building, 6th Floor
Boston, MA 02115

Tel 617 355 9004
Fax 617 355 9016
sdabora@partners.org

> Selected Papers


Research Overview
The goal of my research program is to investigate novel therapeutic approaches for tuberous sclerosis (TSC) using animal models, and translate these findings to patient care by implementing multi-center clinical trials. TSC is an autosomal dominant disorder characterized by the development of benign tumors (hamartomas) in the brain, kidneys, skin and heart. Although TSC related tumors appear benign by histopathology, there is significant morbidity due to neurologic, renal, pulmonary and skin manifestations. Hamartin and tuberin, the products of the TSC genes (TSC1 and TSC2), play an important role in the regulation of protein translation and cell proliferation by inhibiting mTOR kinase. There are currently two major ongoing projects: 1) preclinical studies evaluating mTOR inhibitors, interferon-gamma and other novel therapies in TSC mouse models; 2) Phase II multi-center trial of sirolimus for the treatment of kidney angiomyolipomas associated with TSC and the related lung disorder, lymphangioleiomyomatosis (http://clinicaltrials.gov/show/NCT00126672).