About the Authors

Marcel Veltrop

Roles Investigation, Methodology, Writing – original draft

Affiliation Department of Human Genetics, Leiden University Medical Center, Leiden, RC, the Netherlands

Laura van Vliet

Roles Investigation, Methodology, Visualization

Affiliation Department of Human Genetics, Leiden University Medical Center, Leiden, RC, the Netherlands

Margriet Hulsker

Roles Formal analysis, Investigation, Methodology

Affiliation Department of Human Genetics, Leiden University Medical Center, Leiden, RC, the Netherlands

Jill Claassens

Roles Investigation

Affiliation Transgenic Facility, Leiden University Medical Center, Leiden, RC, the Netherlands

Conny Brouwers

Roles Investigation

Affiliation Transgenic Facility, Leiden University Medical Center, Leiden, RC, the Netherlands

Cor Breukel

Roles Investigation

Affiliation Transgenic Facility, Leiden University Medical Center, Leiden, RC, the Netherlands

Jos van der Kaa

Roles Investigation, Methodology

Affiliation Transgenic Facility, Leiden University Medical Center, Leiden, RC, the Netherlands

Margot M. Linssen

Roles Investigation

Affiliation Transgenic Facility, Leiden University Medical Center, Leiden, RC, the Netherlands

Johan T. den Dunnen

Roles Methodology

Affiliations Department of Human Genetics, Leiden University Medical Center, Leiden, RC, the Netherlands, Department of Clinical Genetics, Leiden University Medical Center, Leiden, RC, the Netherlands

Sjef Verbeek

Roles Methodology

Affiliations Department of Human Genetics, Leiden University Medical Center, Leiden, RC, the Netherlands, Transgenic Facility, Leiden University Medical Center, Leiden, RC, the Netherlands

Annemieke Aartsma-Rus

Roles Conceptualization, Funding acquisition, Methodology, Resources, Supervision, Writing – review & editing

a.m.rus@lumc.nl

Affiliation Department of Human Genetics, Leiden University Medical Center, Leiden, RC, the Netherlands

Maaike van Putten

Roles Formal analysis, Investigation, Methodology, Supervision, Validation, Visualization, Writing – review & editing

Affiliation Department of Human Genetics, Leiden University Medical Center, Leiden, RC, the Netherlands

Competing Interests

I have read the journal's policy and the authors of this manuscript have the following competing interests: AAR discloses being employed by LUMC, which has patents on exon skipping technology, some of which have been licensed to BioMarin. As co-inventor on some of these patents AAR is entitled to a share of potential royalties. AAR further discloses being on the scientific advisory board of ProQR (the Netherlands) and Philae Pharmaceuticals (the Netherlands) and being ad hoc consultant for PTC Therapeutics, BioMarin Pharmaceuticals Inc, GLC consulting, Global Guidepoint, Deerfield, Grunenthal, Summit PLc and being a consultant for BioClinica. Remuneration for these activities goes to her employer. AAR also discloses that LUMC has received speaker honoraria from BioMarin and PTC Therapeutics for presentations at satellite symposia. We do not have a patent related to this manuscript, however other patents that we have on exon skipping for the sake of transparency are: 1. Modulation of exon recognition in pre-mrna by interfering with the secondary RNA structure (WO2004083446); Gert-Jan B van Ommen, Judith C.T. van Deutekom, Johan T den Dunnen and Annemieke Aartsma-Rus, Publication date: September 30 2004; 2. Modulation of exon recognition in pre-mRNA by interfering with the binding of SR proteins and by interfering with secondary structures (WO2006112705) Judith CT van Deutekom, Annemieke Aartsma-Rus and Gert-Jan B van Ommen, Publication date: October 26, 2006; 3. Means and methods for inducing exon skipping (WO2007135105) Annemieke Aartsma-Rus, Judith CT van Deutekom and Gert-Jan B van Ommen, Publication date: November 29, 2007; 4. Modulation of exon recognition in pre-mrna by interfering with the secondary rna structure (WO2004083446) Gert-Jan B van Ommen, Judith CT van Deutekom, Johan T den Dunnen and Annemieke Aartsma-Rus, Publication date: January 31, 2008; 5. Methods and means for efficient skipping of at least one of the following exons of the human Duchenne muscular dystrophy gene: 43, 46, 50-53 (WO2010050802) Sjef J de Kimpe, Gerard Platenburg, Judith CT van Deutekom, Annemieke Aartsma-Rus and Gert-Jan B van Ommen, Publication date: May 6, 2010. There are no further patents, products in development or marketed products to declare. This does not alter our adherence to all the PLOS ONE policies on sharing data and materials, as detailed online in the guide for authors.