Therapeutic strategies for spinal muscular atrophy: SMN and beyond

Therapeutic strategies for spinal muscular atrophy: SMN and beyond

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Spinal muscular atrophy (SMA) is a devastating neuromuscular disorder characterized by loss of motor neurons and muscle atrophy, generally presenting in childhood. SMA is caused by low levels of the survival motor neuron protein (SMN) due to inactivating mutations in the encoding gene SMN1. A second...

書誌詳細
主要な著者:	Bowerman, M, Becker, C, Yáñez-Muñoz, R, Ning, K, Wood, M, Gillingwater, T, Talbot, K, UK SMA Research Consortium
フォーマット:	Journal article
出版事項:	Company of Biologists 2017

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