|1.||Schümperli, Daniel: 4 articles (01/2012 - 08/2007)|
|2.||Meyer, Kathrin: 3 articles (11/2010 - 08/2007)|
|3.||Marquis, Julien: 2 articles (02/2009 - 08/2007)|
|4.||Beley, Cyriaque: 1 article (03/2012)|
|5.||Wilson, James M: 1 article (03/2012)|
|6.||Bogan, Janet: 1 article (03/2012)|
|7.||Vandenborne, Krista: 1 article (03/2012)|
|8.||Sleeper, Meg M: 1 article (03/2012)|
|9.||Bish, Lawrence T: 1 article (03/2012)|
|10.||Forbes, Sean C: 1 article (03/2012)|
|1.||Muscular Dystrophies (Muscular Dystrophy)
03/01/2012 - "In this study, we sought to optimize restoration of cardiac dystrophin expression in the golden retriever muscular dystrophy (GRMD) model using percutaneous transendocardial delivery of recombinant AAV6 (rAAV6) to deliver a modified U7 small nuclear RNA (snRNA) carrying antisense sequence to target the exon splicing enhancers of exons 6 and 8 and correct the disrupted reading frame. "
|2.||Spinal Muscular Atrophy (Progressive Muscular Atrophy)
01/01/2012 - "More specifically, in the context of the fatal neuromuscular disorder Spinal Muscular Atrophy (SMA), we have shown that U7 snRNA constructs can restore the inclusion of exon 7 in the SMN2 gene and thereby alleviate or even fully cure disease symptoms in a severe mouse model for SMA. "
11/01/2010 - "Ultrastructural changes in diaphragm neuromuscular junctions in a severe mouse model for Spinal Muscular Atrophy and their prevention by bifunctional U7 snRNA correcting SMN2 splicing."
02/01/2009 - "Rescue of a severe mouse model for spinal muscular atrophy by U7 snRNA-mediated splicing modulation."
08/01/2007 - "Spinal muscular atrophy: SMN2 pre-mRNA splicing corrected by a U7 snRNA derivative carrying a splicing enhancer sequence."
|3.||Duchenne Muscular Dystrophy (Muscular Dystrophy, Becker)
|2.||Small Nuclear RNA (snRNA)
|3.||RNA Precursors (Precursor, mRNA)
|5.||Proteins (Proteins, Gene)