Therapeutic strategies for spinal muscular atrophy: SMN and beyond

Therapeutic strategies for spinal muscular atrophy: SMN and beyond

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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Bibliographic Details
Main Authors: Melissa Bowerman, Catherina G. Becker, Rafael J. Yáñez-Muñoz, Ke Ning, Matthew J. A. Wood, Thomas H. Gillingwater, Kevin Talbot, The UK SMA Research Consortium
Format: Article
Language:English
Published: The Company of Biologists 2017-08-01
Series:Disease Models & Mechanisms
Subjects:
Animal models
Cellular models
Combinatorial therapies
Skeletal muscle
Spinal muscular atrophy
Survival motor neuron
Online Access:http://dmm.biologists.org/content/10/8/943

Internet

http://dmm.biologists.org/content/10/8/943

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