About the Authors

John F. Staropoli

Affiliations Molecular Neurogenetics Unit, Center for Human Genetic Research, Massachusetts General Hospital, Boston, Massachusetts, United States of America, Department of Pathology, Massachusetts General Hospital, Boston, Massachusetts, United States of America

Larissa Haliw

Affiliation Molecular Neurogenetics Unit, Center for Human Genetic Research, Massachusetts General Hospital, Boston, Massachusetts, United States of America

Sunita Biswas

Affiliation Molecular Neurogenetics Unit, Center for Human Genetic Research, Massachusetts General Hospital, Boston, Massachusetts, United States of America

Lillian Garrett

Affiliation Institute of Developmental Genetics, Helmholtz Zentrum München, Neuherberg/Munich, Germany

Sabine M. Hölter

Affiliation Institute of Developmental Genetics, Helmholtz Zentrum München, Neuherberg/Munich, Germany

Lore Becker

Affiliations Department of Neurology, Friedrich-Baur-Institut, Ludwig-Maximilians-Universität München, Munich, Germany, German Mouse Clinic, Institute of Experimental Genetics, Helmholtz Zentrum München, Neuherberg/Munich, Germany

Sergej Skosyrski

Affiliation Charité-Eye Hospital, Campus Virchow-Klinikum, Berlin, Germany

Patricia Da Silva-Buttkus

Affiliation Institute of Pathology, Helmholtz Zentrum München, Neuherberg/Munich, Germany

Julia Calzada-Wack

Affiliation Institute of Pathology, Helmholtz Zentrum München, Neuherberg/Munich, Germany

Frauke Neff

Affiliation Institute of Pathology, Helmholtz Zentrum München, Neuherberg/Munich, Germany

Birgit Rathkolb

Affiliations German Mouse Clinic, Institute of Experimental Genetics, Helmholtz Zentrum München, Neuherberg/Munich, Germany, Chair for Molecular Animal Breeding and Biotechnology, Gene Center, Ludwig-Maximilians-Universität München, Munich, Germany

Jan Rozman

Affiliations German Mouse Clinic, Institute of Experimental Genetics, Helmholtz Zentrum München, Neuherberg/Munich, Germany, Molecular Nutritional Medicine, Else Kröner-Fresenius Center, TUM, Freising-Weihenstephan, Germany

Anja Schrewe

Affiliation German Mouse Clinic, Institute of Experimental Genetics, Helmholtz Zentrum München, Neuherberg/Munich, Germany

Thure Adler

Affiliations German Mouse Clinic, Institute of Experimental Genetics, Helmholtz Zentrum München, Neuherberg/Munich, Germany, Institute of Medical Microbiology, Immunology, and Hygiene, TUM, München, Germany

Oliver Puk

Affiliation Institute of Developmental Genetics, Helmholtz Zentrum München, Neuherberg/Munich, Germany

Minxuan Sun

Affiliation Institute of Developmental Genetics, Helmholtz Zentrum München, Neuherberg/Munich, Germany

Jack Favor

Affiliation Institute of Human Genetics, Helmholtz Zentrum München, Neuherberg/Munich, Germany

Ildikó Racz

Affiliation Institute of Molecular Psychiatry, University of Bonn, Bonn, Germany

Raffi Bekeredjian

Affiliation Department of Medicine III, Division of Cardiology, University of Heidelberg, Otto-Meyerhof-Zentrum, Heidelberg, Germany

Dirk H. Busch

Affiliation Institute of Medical Microbiology, Immunology, and Hygiene, TUM, München, Germany

Jochen Graw

Affiliation Institute of Developmental Genetics, Helmholtz Zentrum München, Neuherberg/Munich, Germany

Martin Klingenspor

Affiliation Molecular Nutritional Medicine, Else Kröner-Fresenius Center, TUM, Freising-Weihenstephan, Germany

Thomas Klopstock

Affiliation Department of Neurology, Friedrich-Baur-Institut, Ludwig-Maximilians-Universität München, Munich, Germany

Eckhard Wolf

Affiliation Chair for Molecular Animal Breeding and Biotechnology, Gene Center, Ludwig-Maximilians-Universität München, Munich, Germany

Wolfgang Wurst

Affiliations Institute of Developmental Genetics, Helmholtz Zentrum München, Neuherberg/Munich, Germany, Lehrstuhl für Entwicklungsgenetik, TUM, Freising-Weihenstephan, Germany, Max-Planck-Institute of Psychiatry, Munich, Germany, Deutsches Zentrum für Neurodegenerative Erkrankungen e. V. Site Munich, Munich, Germany

Andreas Zimmer

Affiliation Institute of Molecular Psychiatry, University of Bonn, Bonn, Germany

Edith Lopez

Affiliation Molecular Neurogenetics Unit, Center for Human Genetic Research, Massachusetts General Hospital, Boston, Massachusetts, United States of America

Hayat Harati

Affiliations Molecular Neurogenetics Unit, Center for Human Genetic Research, Massachusetts General Hospital, Boston, Massachusetts, United States of America, Neurogenetics Program and Division of Pediatric Neurology, Departments of Pediatrics and Biochemistry, American University of Beirut, Beirut, Lebanon

Eric Hill

Affiliation Center for Systems Biology, Program in Membrane Biology/Nephrology Division, Massachusetts General Hospital, Boston, Massachusetts, United States of America

Daniela S. Krause

Affiliation Department of Pathology, Massachusetts General Hospital, Boston, Massachusetts, United States of America

Jolene Guide

Affiliation Molecular Neurogenetics Unit, Center for Human Genetic Research, Massachusetts General Hospital, Boston, Massachusetts, United States of America

Ella Dragileva

Affiliation Molecular Neurogenetics Unit, Center for Human Genetic Research, Massachusetts General Hospital, Boston, Massachusetts, United States of America

Evan Gale

Affiliation Molecular Neurogenetics Unit, Center for Human Genetic Research, Massachusetts General Hospital, Boston, Massachusetts, United States of America

Vanessa C. Wheeler

Affiliation Molecular Neurogenetics Unit, Center for Human Genetic Research, Massachusetts General Hospital, Boston, Massachusetts, United States of America

Rose-Mary Boustany

Affiliation Neurogenetics Program and Division of Pediatric Neurology, Departments of Pediatrics and Biochemistry, American University of Beirut, Beirut, Lebanon

Diane E. Brown

Affiliations Department of Pathology, Massachusetts General Hospital, Boston, Massachusetts, United States of America, Center for Comparative Medicine, Massachusetts General Hospital, Boston, Massachusetts, United States of America

Sylvie Breton

Affiliation Center for Systems Biology, Program in Membrane Biology/Nephrology Division, Massachusetts General Hospital, Boston, Massachusetts, United States of America

Klaus Ruether

Affiliation Augenabteilung Sankt Gertrauden Krankenhaus, Berlin, Germany

Valérie Gailus-Durner

Affiliation German Mouse Clinic, Institute of Experimental Genetics, Helmholtz Zentrum München, Neuherberg/Munich, Germany

Helmut Fuchs

Affiliation German Mouse Clinic, Institute of Experimental Genetics, Helmholtz Zentrum München, Neuherberg/Munich, Germany

Martin Hrabě de Angelis

Affiliations German Mouse Clinic, Institute of Experimental Genetics, Helmholtz Zentrum München, Neuherberg/Munich, Germany, Lehrstuhl für Experimentelle Genetik, TUM, Freising-Weihenstephan, Germany

Susan L. Cotman

cotman@helix.mgh.harvard.edu

Affiliation Molecular Neurogenetics Unit, Center for Human Genetic Research, Massachusetts General Hospital, Boston, Massachusetts, United States of America

Competing Interests

The authors have read the journal’s policy and have the following declarations: SLC is a member of the Scientific Advisory Board of the Batten Disease Support and Research Association (BDSRA). R-MB is a member of the Medical Advisory Board of the BDSRA, and Associate Editor of the Annals of Neurology. R-MB also holds the following patents, issued or under examination: Methods of Screening for Risk of Proliferative Disease and Methods for the Treatment of Proliferative Disease, issued June 6, 2006 (US Patent # 60 105 262); Application for Method of Treating Batten Disease, issued November 23, 2004 (US Patent # 6 821 995); Methods and compositions for treating disorders caused by deficiency in a gene product of a CLN gene, patent under examination. Klaus Ruether is employed by Augenabteilung Sankt Gertrauden Krankenhaus. This does not alter the authors’ adherence to all the PLoS ONE policies on sharing data and materials.

Author Contributions

Conceived and designed the experiments: KR VG-D HF MHdA VCW SLC. Performed the experiments: JFS LH SB LG SMH LB SS PDS-B JC-W FN BR JR AS TA OP MS JF IR RB DHB JG MK TK EW WW AZ EL HH EH DK JG ED EG DEB SB KR VG-D HF. Analyzed the data: JFS LH SB LG SMH LB SS PDS-B JC-W FN BR JR AS TA OP MS JF IR RB DHB JG MK TK EW WW AZ EL HH R-MB EH DK JG ED EG DEB SB KR VG-D HF MHdA VCW SLC. Contributed reagents/materials/analysis tools: JFS SB KR VG-D HF MHdA SLC. Wrote the paper: SLC JFS.