Therapeutic strategies for spinal muscular atrophy: SMN and beyond

• Main Authors: Melissa Bowerman (Author), Catherina G. Becker (Author), Rafael J. Yáñez-Muñoz (Author), Ke Ning (Author), Matthew J. A. Wood (Author), Thomas H. Gillingwater (Author), Kevin Talbot (Author), The UK SMA Research Consortium (Author)
• Format: Book
• Published: The Company of Biologists, 2017-08-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

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...