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Spinal muscular atrophy: mechanisms and therapeutic strategies

Spinal muscular atrophy (SMA) is an autosomal recessive neurodegenerative disorder and a leading genetic cause of infantile mortality. SMA is caused by mutation or deletion of Survival Motor Neuron-1 (SMN1). The clinical features of the disease are caused by specific degeneration of α-motor neurons...

Ausführliche Beschreibung

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Bibliographische Detailangaben
Hauptverfasser: Lorson, Christian L., Rindt, Hansjorg, Shababi, Monir
Format: Artigo
Sprache:Inglês
Veröffentlicht: Oxford University Press 2010
Schlagworte:
Online Zugang:https://ncbi.nlm.nih.gov/pmc/articles/PMC2875050/
https://ncbi.nlm.nih.gov/pubmed/20392710
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1093/hmg/ddq147
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