Now showing items 1-6 of 6

    • Genetic Circuitry of Survival Motor Neuron, the Gene Underlying Spinal Muscular Atrophy 

      Sen, Anindya; Dimlich, Douglas; Guruharsha, K. G.; Kankel, Mark William; Hori, Kazuya; Yokokura, Takakazu; Brachat, Sophie; Richardson, Delwood; Loureiro, Joseph; Sivasankaran, Rajeev; Curtis, Daniel; Davidow, Lance Steven; Rubin, Lee; Hart, Anne C.; Van Vactor, David L.; Artavanis-Tsakonas, Spyros (Proceedings of the National Academy of Sciences, 2013)
      The clinical severity of the neurodegenerative disorder spinal muscular atrophy (SMA) is dependent on the levels of functional Survival Motor Neuron (SMN) protein. Consequently, current strategies for developing treatments ...
    • Glucocorticoid Compounds Modify Smoothened Localization and Hedgehog Pathway Activity 

      McMahon, Andrew P.; Davidow, Lance Steven; Wang, Yu; Blanchard, Joel; Lam, Kelvin; Xu, Ke; Oza, Vatsal U.; Woo Yoo, Jin; Ng, Jessica; Curran, Tom; Rubin, Lee; Arvanites, Anthony (Elsevier, 2012)
      The Hedgehog signaling pathway is linked to a variety of diseases, notably a range of cancers. The first generation of drug screens identified Smoothened (Smo), a membrane protein essential for signaling, as an attractive ...
    • Reversal of β cell de-differentiation by a small molecule inhibitor of the TGFβ pathway 

      Blum, Barak; Roose, Adam N; Barrandon, Ornella; Maehr, René; Arvanites, Anthony C; Davidow, Lance S; Davis, Jeffrey C; Peterson, Quinn P; Rubin, Lee L; Melton, Douglas A (eLife Sciences Publications, Ltd, 2014)
      Dysfunction or death of pancreatic β cells underlies both types of diabetes. This functional decline begins with β cell stress and de-differentiation. Current drugs for type 2 diabetes (T2D) lower blood glucose levels but ...
    • A screen for regulators of survival of motor neuron protein levels 

      Makhortova, Nina R; Hayhurst, Monica; Cerqueira, Antonio; Sinor-Anderson, Amy D; Zhao, Wen-Ning; Heiser, Patrick W; Arvanites, Arvanites; Davidow, Lance Steven; Waldon, Zachary O; Steen, Judith A; Lam, Kelvin; Ngo, Hien D; Rubin, Lee (Springer Nature, 2011)
      The motor neuron disease spinal muscular atrophy (SMA) results from mutations that lead to low levels of the ubiquitously expressed protein survival of motor neuron (SMN). An ever-increasing collection of data suggests ...
    • Selective Identification of Hedgehog Pathway Antagonists by Direct Analysis of Smoothened Ciliary Translocation 

      Wang, Yu; Arvanites, Anthony C.; Davidow, Lance Steven; Blanchard, Joel; Lam, Kelvin; Yoo, Jin Woo; Coy, Shannon; Rubin, Lee L.; McMahon, Andrew P. (American Chemical Society, 2012-05-11)
      Hedgehog (Hh) signaling promotes tumorigenesis. The accumulation of the membrane protein Smoothened (Smo) within the primary cilium (PC) is a key event in Hh signal transduction, and many pharmacological inhibitors identified ...
    • A small molecule that directs differentiation of human ESCs into the pancreatic lineage 

      Chen, Shuibing; Borowiak, Malgorzata; Maehr, René; Osafune, Kenji; Davidow, Lance Steven; Lam, Kelvin; Peng, Lee F; Schreiber, Stuart L.; Rubin, Lee; Melton, Douglas A. (Nature Publishing Group, 2009)
      Stepwise differentiation from embryonic stem cells (ESCs) to functional insulin-secreting beta cells will identify key steps in beta-cell development and may yet prove useful for transplantation therapy for diabetics. An ...