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Dual exon skipping in myostatin and dystrophin for Duchenne muscular dystrophy

Dwi U Kemaladewi12, Willem MH Hoogaars1, Sandra H van Heiningen1, Samuel Terlouw1, David JJ de Gorter23, Johan T den Dunnen1, Gert Jan B van Ommen1, Annemieke Aartsma-Rus1, Peter ten Dijke2 and Peter AC 't Hoen1*

Author Affiliations

1 Center for Human and Clinical Genetics, Leiden University Medical Center, Postzone S4-P, PO Box 9600, Leiden, 2300RC, the Netherlands

2 Department of Molecular and Cell Biology, Leiden University Medical Center, Postzone S1-P, PO Box 9600, Leiden, 2300RC, the Netherlands

3 Institute for Molecular Cell Biology, University of Münster, Schlossplatz 5, Münster, D-48149, Germany

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BMC Medical Genomics 2011, 4:36  doi:10.1186/1755-8794-4-36

Published: 20 April 2011

Additional files

Additional file 1:

Human primary control (KM109) myoblasts were differentiated for 7 days before transfection with 3 different MSTN AONs at 500 and 100 nM concentrations. All AONs were designed to target exon 2 of the myostatin gene. The sequences are listed in table 1. RNA was isolated 2 days post-transfection. cDNA was synthesized using random hexamer (N6) primers and subjected for PCR using primers in exon 1 and 3.

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