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...
Հիմնական հեղինակներ: | Bowerman, M, Becker, C, Yáñez-Muñoz, R, Ning, K, Wood, M, Gillingwater, T, Talbot, K, UK SMA Research Consortium |
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Ձևաչափ: | Journal article |
Հրապարակվել է: |
Company of Biologists
2017
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Նմանատիպ նյութեր
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Therapeutic strategies for spinal muscular atrophy: SMN and beyond
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Հրապարակվել է: (2021) -
Targeting the 5′ untranslated region of SMN2 as a therapeutic strategy for spinal muscular atrophy
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SMN deficiency disrupts brain development in a mouse model of severe spinal muscular atrophy.
: Wishart, T, և այլն
Հրապարակվել է: (2010)