Now showing items 1-17 of 17

    • Adenosine kinase inhibition selectively promotes rodent and porcine islet β-cell replication 

      Annes, J. P.; Ryu, J. H.; Lam, K.; Carolan, Peter Joseph; Utz, K.; Hollister-Lock, J.; Anthony, Anthony; Rubin, Lee; Weir, Gordon Campbell; Melton, Douglas A. (Proceedings of the National Academy of Sciences, 2012)
      Diabetes is a pathological condition characterized by relative insulin deficiency, persistent hyperglycemia, and, consequently, diffuse micro- and macrovascular disease. One therapeutic strategy is to amplify insulin-secretion ...
    • Clonal analyses and gene profiling identify genetic biomarkers of human brown and white preadipocyte thermogenic potential 

      Xue, Ruidan; Lynes, Matthew D.; Dreyfuss, Jonathan M.; Shamsi, Farnaz; Schulz, Tim J.; Zhang, Hongbin; Huang, Tian Lian; Townsend, Kristy L.; Li, Yiming; Takahashi, Hirokazu; Weiner, Lauren S.; White, Andrew P.; Lynes, Maureen S.; Rubin, Lee L.; Goodyear, Laurie J.; Cypess, Aaron M.; Tseng, Yu-Hua (2015)
      Targeting brown adipose tissue (BAT) content or activity has therapeutic potential for treating obesity and the metabolic syndrome by increasing energy expenditure. Both inter- and intra-individual differences contribute ...
    • A comparison of non-integrating reprogramming methods 

      Schlaeger, Thorsten M.; Daheron, Laurence Marie; Brickler, Thomas R; Entwisle, Samuel; Chan, Karrie; Cianci, Amelia; DeVine, Alexander; Ettenger, Andrew; Fitzgerald, Kelly; Godfrey, Michelle; Gupta, Dipti; McPherson, Jade; Malwadkar, Prerana; Gupta, Manav; Bell, Blair; Doi, Akiko; Jung, Namyoung; Li, Xin; Lynes, Maureen M.; Brookes, Emily; Cherry, Anne B C; Demirbas, Didem; Tsankov, Alexander; Zon, Leonard Ira; Rubin, Lee; Feinberg, Andrew P; Meissner, Alexander; Cowan, Chad A.; Daley, George Quentin (Nature Publishing Group, 2014)
      Human induced pluripotent stem cells (hiPSCs1, 2, 3) are useful in disease modeling and drug discovery, and they promise to provide a new generation of cell-based therapeutics. To date there has been no systematic evaluation ...
    • 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 ...
    • Identification of a Maleimide-Based Glycogen Synthase Kinase-3 (GSK-3) Inhibitor, BIP-135, That Prolongs the Median Survival Time of Δ7 SMA KO Mouse Model of Spinal Muscular Atrophy 

      Chen, Po C.; Gaisina, Irina N.; El-Khodor, Bassem F.; Ramboz, Sylvie; Makhortova, Nina R; Rubin, Lee; Kozikowski, Alan P. (American Chemical Society (ACS), 2013)
      The discovery of upregulated glycogen synthase kinase-3 (GSK-3) in various pathological conditions has led to the development of a host of chemically diverse small molecule GSK-3 inhibitors, such as BIP-135. GSK-3 inhibition ...
    • Modeling pain in vitro using nociceptor neurons reprogrammed from fibroblasts 

      Wainger, Brian J.; Buttermore, Elizabeth D.; Oliveira, Julia T.; Mellin, Cassidy; Lee, Seungkyu; Saber, Wardiya Afshar; Wang, Amy; Ichida, Justin K.; Chiu, Isaac M.; Barrett, Lee; Huebner, Eric A.; Bilgin, Canan; Tsujimoto, Naomi; Brenneis, Christian; Kapur, Kush; Rubin, Lee L.; Eggan, Kevin; Woolf, Clifford J. (2015)
      Reprogramming somatic cells from one cell fate to another can generate specific neurons suitable for disease modeling. To maximize the utility of patient-derived neurons, they must model not only disease-relevant cell ...
    • Notch Inhibition Allows Oncogene-Independent Generation of iPS Cells 

      Ichida, Justin K.; TCW, Julia; Williams Gonzalez, Luis Alberto; Carter, Ava; Shi, Yingxiao; Moura, Marcelo T.; Ziller, Michael Johannes; Singh, Sean; Amabile, Giovanni; Bock, Christoph; Umezawa, Akihiro; Rubin, Lee; Bradner, James Elliott; Akutsu, Hidenori; Meissner, Alexander; Eggan, Kevin Carl (Nature Publishing Group, 2014)
      The reprogramming of somatic cells to pluripotency using defined transcription factors holds great promise for biomedicine. However, human reprogramming remains inefficient and relies either on the use of the potentially ...
    • 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 ...
    • RNA and Protein Networks That Locally Control Brain Wiring During Development 

      Murphy, Alexander James (2015-05-14)
      The molecular machineries of growth cones control the formation of neural circuits in the developing brain. Although great progress has been made in elucidating axon guidance cues and their growth cone receptors, we still ...
    • 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 inhibitor of Tgf-β signaling replaces Sox2 in reprogramming by inducing Nanog 

      Ichida, Justin K.; Blanchard, Joel; Lam, Kelvin; Son, Esther Y.; Chung, Julia E.; Egli, Dieter; Loh, Kyle M.; Carter, Ava C.; Di Giorgio, Francesco P.; Koszka, Kathryn E; Huangfu, Danwei; Akutsu, Hidenori; Liu, David Ruchien; Rubin, Lee; Eggan, Kevin Carl (Elsevier BV, 2009)
      The combined activity of three transcription factors can reprogram adult cells into induced pluripotent stem (iPS) cells. However, the transgenic methods used to deliver reprogramming factors have raised concerns regarding ...
    • 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 ...
    • A TALEN Genome-Editing System for Generating Human Stem Cell-Based Disease Models 

      Ding, Qiurong; Lee, Youn-Kyoung; Schaefer, Esperance Anne Kreek; Peters, Derek Tilghman; Veres, Adrian; Kim, Kevin; Kuperwasser, Nicolas; Motola, Daniel L; Meissner, Torsten B; Hendriks, William; Trevisan, Marta; Gupta, Rajat; Moisan, Annie; Banks, Eric; Friesen, Max; Schinzel, Robert T.; Xia, Fang; Tang, Alexander; Xia, Yulei; Figueroa, Emmanuel; Wann, Amy; Ahfeldt, Tim; Daheron, Laurence Marie; Zhang, Feng; Rubin, Lee; Peng, Lee F; Chung, Raymond Taeyong; Musunuru, Kiran; Cowan, Chad A. (Elsevier BV, 2013)
      Transcription activator-like effector nucleases (TALENs) are a new class of engineered nucleases that are easier to design to cleave at desired sites in a genome than previous types of nucleases. We report here the use of ...
    • Targeting Superficial or Nodular Basal Cell Carcinoma with Topically Formulated Small Molecule Inhibitor of Smoothened 

      Tang, Tracy; Tang, Jean Y.; Li, Dongwei; Reich, Mike; Callahan, Christopher A.; Fu, Ling; Yauch, Robert L.; Wang, Frank; Kotkow, Karen; Chang, Kris S.; Shpall, Elana; Wu, Angela; Rubin, Lee; Marsters, James C., Jr.; Epstein, Ervin H., Jr.; Caro, Ivor; de Sauvage, Frederic J. (American Association for Cancer Research (AACR), 2011)
      Purpose: Inappropriate activation of the Hedgehog (Hh) signaling pathway in skin is critical for the development of basal cell carcinomas (BCC). We have investigated the anti-BCC efficacy of topically-applied CUR61414, an ...
    • Young systemic factors as a medicine for age-related neurodegenerative diseases 

      Katsimpardi, Lida; Rubin, Lee L (Taylor & Francis, 2015)
      It is widely known that neurogenesis, brain function and cognition decline with aging. Increasing evidence suggests that cerebrovascular dysfunction is a major cause of cognitive impairment in the elderly but is also ...