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    • 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 ...