Faculty Opinions recommendation of Oligonucleotide-mediated survival of motor neuron protein expression in CNS improves phenotype in a mouse model of spinal muscular atrophy.

2014 ◽  
Author(s):  
Chris Henderson
Keyword(s):  
Mouse Model ◽  
Protein Expression ◽  
Spinal Muscular Atrophy ◽  
Motor Neuron ◽  
Muscular Atrophy ◽  
Motor Neuron Protein ◽  
Survival Of Motor Neuron

scholarly journals Survival of motor neuron protein downregulates miR-9 expression in patients with spinal muscular atrophy

2014 ◽  
Vol 30 (5) ◽  
pp. 229-234 ◽  
Author(s):  
Li-Ting Wang ◽  
Shyh-Shin Chiou ◽  
Yu-Mei Liao ◽  
Yuh-Jyh Jong ◽  
Shih-Hsien Hsu
Keyword(s):  
Spinal Muscular Atrophy ◽  
Motor Neuron ◽  
Muscular Atrophy ◽  
Motor Neuron Protein ◽  
Survival Of Motor Neuron

scholarly journals CIM Journal Club: Gene therapy for spinal muscular atrophy Comment on Mendell et al. N Engl J Med 2017;377:1713-22.

2018 ◽  
pp. E31-E33 ◽  
Author(s):  
Raphael Schneider
Keyword(s):  
Gene Therapy ◽  
Spinal Muscular Atrophy ◽  
Motor Neuron ◽  
Viral Vector ◽  
Muscular Atrophy ◽  
Journal Club ◽  
Dna Encoding ◽  
Motor Neuron Diseases ◽  
Motor Neuron Protein ◽  
Survival Of Motor Neuron

In their landmark paper, Mendell et al. show that infants with spinal muscular atrophy (SMA) reached important motor milestones and survived longer when treated with AVXS-101 (AveXis), a viral vector containing DNA encoding the survival of motor neuron protein (SMN). Patients not only crawled, stood and walked independently, but learned to speak. These results are very encouraging for patients with SMA and offer hope for pediatric and adult patients with other types of motor neuron diseases.

scholarly journals Interaction between the small‐nuclear‐RNA cap hypermethylase and the spinal muscular atrophy protein, survival of motor neuron

EMBO Reports ◽  
2003 ◽  
Vol 4 (6) ◽  
pp. 616-622 ◽  
Author(s):  
John Mouaikel ◽  
Usha Narayanan ◽  
Céline Verheggen ◽  
A Gregory Matera ◽  
Edouard Bertrand ◽  
...  
Keyword(s):  
Spinal Muscular Atrophy ◽  
Motor Neuron ◽  
Muscular Atrophy ◽  
Small Nuclear Rna ◽  
Nuclear Rna ◽  
Survival Of Motor Neuron

scholarly journals Salbutamol inhibits ubiquitin-mediated survival motor neuron protein degradation in spinal muscular atrophy cells

2015 ◽  
Vol 4 ◽  
pp. 351-356 ◽  
Author(s):  
Nur Imma Fatimah Harahap ◽  
Dian Kesumapramudya Nurputra ◽  
Mawaddah Ar Rochmah ◽  
Ai Shima ◽  
Naoya Morisada ◽  
...  
Keyword(s):  
Spinal Muscular Atrophy ◽  
Motor Neuron ◽  
Protein Degradation ◽  
Muscular Atrophy ◽  
Survival Motor Neuron ◽  
Survival Motor Neuron Protein ◽  
Motor Neuron Protein
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