A large animal model of Spinal Muscular Atrophy and correction of phenotype

OBJECTIVES: Spinal muscular atrophy (SMA) is caused by reduced levels of SMN which results in motoneuron loss. Therapeutic strategies to increase SMN levels including drug compounds, antisense oligonucleotides or scAAV9 gene therapy have proved effective in mice. We wished to determine whether reduc...

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Gepubliceerd in:Ann Neurol
Hoofdauteurs: Duque, Sandra I., Arnold, W. David, Odermatt, Philipp, Li, Xiaohui, Porensky, Paul N., Schmelzer, Leah, Meyer, Kathrin, Kolb, Stephen J., Schümperli, Daniel, Kaspar, Brian K., Burghes, Arthur H. M.
Formaat: Artigo
Taal:Inglês
Gepubliceerd in: 2015
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Online toegang:https://ncbi.nlm.nih.gov/pmc/articles/PMC4453930/
https://ncbi.nlm.nih.gov/pubmed/25516063
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/ana.24332
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