BBS Faculty Member - Lee Rubin

Lee Rubin

Department of Stem Cell and Regenerative Biology

Harvard University
Sherman Fairchild Building, Room G60
7 Divinity Ave.
Cambridge, MA 02138
Tel: 617-384-5799
Fax: 617-495-3961
Visit my lab page here.

My laboratory uses a stem cell based approach to understand neurodegenerative diseases, particularly orphan diseases such as Spinal Muscular Atrophy, so that we can identify potentially new therapeutic targets. Our work is based on using disease relevant cells that we can produce in large numbers from stem-like cells. However, because our fundamental interest relates to drug discovery, we have configured a high throughput screening laboratory that combines stem cells, particular sets of drug-like small molecules, and automated confocal imaging. This combination is quite versatile and has allowed us to identify reagents that regulate cell differentiation, reprogramming, transdifferentiation, proliferation and survival. In addition, we have gained a great deal of information concerning pathways that regulate fate and behavior of many different types of cells. Along the way, we have established collaborations with many labs in the Harvard community who share our interest in stem cells, differentiation and disease.

Last Update: 8/22/2013


For a complete listing of publications click here.



Wang Y, Davidow L, Arvanites AC, Blanchard J, Lam K, Xu K, Oza V, Yoo JW, Ng JMY, Curran T, Rubin LL, McMahon AP. Glucocorticoid Compounds Modify Smoothened Localization and Hedgehog Pathway Activity. Chemistry & Biology
2012 (in press).

Hayhurst, M, Wagner A, Cerletti M, Wagers AJ, Rubin LL. A cell-autonomous defect in skeletal muscle satellite cells expressing low levels of Survival of Motor Neuron protein. Dev Biol 2012; 368:323-34.

Wang, Yu, Arvanites AC, Davidow L, Blanchard J, Lam K, Yoo JW, Coy S, Rubin LL, McMahon, AP. Selective Identification of Hedgehog Pathway Antagonists By Direct Analysis of Smoothened Ciliary Translocation. ACS Chem Biol 2012; 7:1040-8.

Chen PC, Gaisina IN, El-Khodor BF, Ramboz S, Makhortova NR, Rubin LL, Kozikowski AP. Identification of a Maleimide-Based Glycogen Synthase
Kinase-3 (GSK-3) Inhibitor, BIP-135, that Prolongs the Median Survival Time of 7 SMA KO Mouse Model of Spinal Muscular Atrophy. ACS Chem Neurosci 2012; 3:5-11.

Annes JP, Ryu JH, Lam K, Carolan PJ, Utz K, Hollister-Lock J, Arvanites AC, Rubin LL, Weir G, Melton DA. Adenosine kinase inhibition selectively promotes rodent and porcine islet ß-cell replication. Proc Natl Acad Sci U S A 2012; 109:3915-20.

Makhortova NR, Hayhurst M, Cerqueira A, Sinor-Anderson A, Zhao W-N, Heiser P, Arvanites AC, Davidow LS, Waldon ZO, Steen JA, Lam K, Ngo H, Rubin LL.
A Screen for Regulators of Survival of Motor Neuron Protein Levels. Nature Chem Biol 2011; 7:544-52.

Rubin LL, Haston KM. Stem cells and drug discovery. BMC Biol 2011; 9:42.

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