A large animal model of spinal muscular atrophy and correction of phenotype
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.
Published in Annals of neurology (01.03.2015)
Published in Annals of neurology (01.03.2015)
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Journal Article
Somatic Therapy of a Mouse SMA Model with a U7 snRNA Gene Correcting SMN2 Splicing
Odermatt, Philipp, Trüb, Judith, Furrer, Lavinia, Fricker, Roger, Marti, Andreas, Schümperli, Daniel
Published in Molecular therapy (01.10.2016)
Published in Molecular therapy (01.10.2016)
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Journal Article
Splicing changes in SMA mouse motoneurons and SMN-depleted neuroblastoma cells: Evidence for involvement of splicing regulatory proteins
Huo, Qing, Kayikci, Melis, Odermatt, Philipp, Meyer, Kathrin, Michels, Olivia, Saxena, Smita, Ule, Jernej, Schümperli, Daniel
Published in RNA biology (02.11.2014)
Published in RNA biology (02.11.2014)
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Journal Article