Browsing Faculty of Arts and Sciences by Keyword "disease model"

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    • Generation of pluripotent stem cells from patients with type 1 diabetes 

      Maehr, René; Chen, Shuibing; Snitow, Melinda; Ludwig, Thomas; Yagasaki, Lisa; Goland, Robin; Leibel, Rudolph L.; Melton, Douglas A. (Proceedings of the National Academy of Sciences, 2009)
      Type 1 diabetes (T1D) is the result of an autoimmune destruction of pancreatic β cells. The cellular and molecular defects that cause the disease remain unknown. Pluripotent cells generated from patients with T1D would ...

    • 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 ...