About the Authors

Marcelina Malinowska

Contributed equally to this work with: Marcelina Malinowska, Fiona L. Wilkinson

Affiliations Mucopolysaccharidosis (MPS) Stem Cell Research Group, Biomedicine, Faculty of Medical and Human Sciences, University of Manchester, Manchester, United Kingdom, Department of Molecular Biology, Faculty of Biology, University of Gdansk, Gdansk, Poland

Fiona L. Wilkinson

Contributed equally to this work with: Marcelina Malinowska, Fiona L. Wilkinson

Affiliation Mucopolysaccharidosis (MPS) Stem Cell Research Group, Biomedicine, Faculty of Medical and Human Sciences, University of Manchester, Manchester, United Kingdom

Kia J. Langford-Smith

Affiliation Mucopolysaccharidosis (MPS) Stem Cell Research Group, Biomedicine, Faculty of Medical and Human Sciences, University of Manchester, Manchester, United Kingdom

Alex Langford-Smith

Affiliation Mucopolysaccharidosis (MPS) Stem Cell Research Group, Biomedicine, Faculty of Medical and Human Sciences, University of Manchester, Manchester, United Kingdom

Jillian R. Brown

Affiliation Zacharon Pharmaceuticals Inc., San Diego, California, United States of America

Brett E. Crawford

Affiliation Zacharon Pharmaceuticals Inc., San Diego, California, United States of America

Marie T. Vanier

Affiliation Institut National de la Santé et de la Recherche Médicale (INSERM) Unit 820, Lyon University, Lyon, France

Grzegorz Grynkiewicz

Affiliation Pharmaceutical Research Institute, Warsaw, Poland

Rob F. Wynn

Affiliation Bone Marrow Transplant Unit, Royal Manchester Children's Hospital, Manchester Academic Health Science Centre, Central Manchester University Hospitals NHS Foundation Trust, Manchester, United Kingdom

J. Ed Wraith

Affiliation Genetic Medicine, St. Mary's Hospital, Manchester Academic Health Science Centre, Central Manchester University Hospitals NHS Foundation Trust, Manchester, United Kingdom

Grzegorz Wegrzyn

Affiliation Department of Molecular Biology, Faculty of Biology, University of Gdansk, Gdansk, Poland

Brian W. Bigger

Brian.Bigger@manchester.ac.uk

Affiliations Mucopolysaccharidosis (MPS) Stem Cell Research Group, Biomedicine, Faculty of Medical and Human Sciences, University of Manchester, Manchester, United Kingdom, Bone Marrow Transplant Unit, Royal Manchester Children's Hospital, Manchester Academic Health Science Centre, Central Manchester University Hospitals NHS Foundation Trust, Manchester, United Kingdom

Competing Interests

Jillian R. Brown and Brett E. Crawford are affiliated to Zacharon Pharmaceuticals Inc. It could be reasonably perceived by an outside party that Zacharon Parmaceuticals could have interfered with objective analysis of the data they produced. However, all samples from the University of Manchester were blinded before sending to Zacharon Pharmaceuticals for biochemical analysis. Therefore Zacharon Pharmaceuticals had no control over the interpretation of the data produced. This does not alter the authors' adherence to the PLoS policy on data sharing.

Author Contributions

Conceived and designed the experiments: MM FLW KJLS ALS JRB BEC MTV GW BB. Performed the experiments: MM FLW KJLS ALS JRB BEC MTV. Analyzed the data: MM FLW KJLS ALS BEC MTV GW BB. Contributed reagents/materials/analysis tools: GG. Wrote the paper: MM FLW KJLS ALS MTV RFW JEW GW BB. Critically appraised the manuscript: MM FLW KJLS ALS JRB BEC MTV GG RFW JEW GW.