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...

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Библиографические подробности
Главные авторы:	Bowerman, M, Becker, C, Yáñez-Muñoz, R, Ning, K, Wood, M, Gillingwater, T, Talbot, K, UK SMA Research Consortium
Формат:	Journal article
Опубликовано:	Company of Biologists 2017

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