Dual SMN inducing therapies can rescue survival and motor unit function in symptomatic ∆7SMA mice

Dual SMN inducing therapies can rescue survival and motor unit function in symptomatic ∆7SMA mice

Spinal muscular atrophy (SMA) is an autosomal recessive disease characterized by survival motor neuron (SMN) protein deficiency which results in motor neuron loss and muscle atrophy. SMA is caused by a mutation or deletion of the survival motor neuron 1 (SMN1) gene and retention of the nearly identi...

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Bibliographic Details
Main Authors: Kaitlyn M. Kray, Vicki L. McGovern, Deepti Chugh, W. David Arnold, Arthur H.M. Burghes
Format: Article
Language:English
Published: Elsevier 2021-11-01
Series:Neurobiology of Disease
Subjects:
Spinal muscular atrophy (SMA)
Survival motor neuron (SMN)
Antisense oligonucleotides
Electrophysiology
Risdiplam
Online Access:http://www.sciencedirect.com/science/article/pii/S0969996121002370

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http://www.sciencedirect.com/science/article/pii/S0969996121002370

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