Article
Genetic circuitry of Survival motor neuron, the gene underlying spinal muscular atrophy.
Proceedings of the National Academy of Sciences of the United States of America - 25 Jun 2013
Sen Anindya, Dimlich Douglas N, Guruharsha K G, Kankel Mark W, Hori Kazuya, Yokokura Takakazu, Brachat Sophie, Richardson Delwood, Loureiro Joseph, Sivasankaran Rajeev, Curtis Daniel, Davidow Lance S, Rubin Lee L, Hart Anne C, Van Vactor David, Artavanis-Tsakonas Spyros
Abstract excerpt
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 for SMA generally focus on augmenting SMN levels. To identify additional potential therapeutic avenues and achieve a greater understanding of SMN, we applied in vivo, in vitro, and in silico...
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