AAV9-DOK7 gene therapy reduces disease severity in Smn2B/- SMA model mice

Spinal Muscular Atrophy (SMA) is an autosomal recessive neuromuscular disease caused by deletions or mutations in the survival motor neuron (SMN1) gene. An important hallmark of disease progression is the pathology of neuromuscular junctions (NMJs). Affected NMJs in the SMA context exhibit delayed m...

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Veröffentlicht in:	Biochemical and biophysical research communications 2020-09, Vol.530 (1), p.107-114
Hauptverfasser:	Kaifer, Kevin A., Villalón, Eric, Smith, Caley E., Simon, Madeline E., Marquez, Jose, Hopkins, Abigail E., Morcos, Toni I., Lorson, Christian L.
Format:	Artikel
Sprache:	eng
Schlagworte:	Adeno associate virus Gene Therapy Neurodegeneration SMA Spinal muscular atrophy
Online-Zugang:	Volltext
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