Biallelic mutations in the 3' exonuclease TOE1 cause pontocerebellar hypoplasia and uncover a role in snRNA processing.

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Published in Nat Genet on January 16, 2017

Authors

Rea M Lardelli1, Ashleigh E Schaffer1,2,3, Veerle R C Eggens4, Maha S Zaki5, Stephanie Grainger6, Shashank Sathe3, Eric L Van Nostrand3, Zinayida Schlachetzki2, Basak Rosti2, Naiara Akizu2, Eric Scott2, Jennifer L Silhavy2, Laura Dean Heckman2, Rasim Ozgur Rosti2, Esra Dikoglu2, Anne Gregor2, Alicia Guemez-Gamboa2, Damir Musaev2, Rohit Mande2, Ari Widjaja2, Tim L Shaw1, Sebastian Markmiller3, Isaac Marin-Valencia2, Justin H Davies7, Linda de Meirleir8, Hulya Kayserili9, Umut Altunoglu10, Mary Louise Freckmann11, Linda Warwick12, David Chitayat13,14, Susan Blaser15, Ahmet Okay Çağlayan16,17, Kaya Bilguvar18, Huseyin Per19, Christina Fagerberg20, Henrik T Christesen21, Maria Kibaek21, Kimberly A Aldinger22, David Manchester23, Naomichi Matsumoto24, Kazuhiro Muramatsu25, Hirotomo Saitsu24,26, Masaaki Shiina27, Kazuhiro Ogata27, Nicola Foulds28, William B Dobyns22, Neil C Chi29, David Traver6, Luigina Spaccini30, Stefania Maria Bova31, Stacey B Gabriel32, Murat Gunel17, Enza Maria Valente33, Marie-Cecile Nassogne34, Eric J Bennett1, Gene W Yeo3,35, Frank Baas36, Jens Lykke-Andersen1, Joseph G Gleeson1,2

Author Affiliations

1: University of California San Diego, La Jolla, California, USA.
2: Laboratory of Pediatric Brain Disease and Howard Hughes Medical Institute, The Rockefeller University, New York, New York, USA.
3: Department of Cellular and Molecular Medicine, Stem Cell Program and Institute for Genomic Medicine, University of California San Diego, La Jolla, California, USA.
4: Department of Clinical Genetics, Academic Medical Center, Amsterdam, the Netherlands.
5: Clinical Genetics Department, Human Genetics and Genome Research Division, National Research Centre, Cairo, Egypt.
6: Department of Cellular and Molecular Medicine, University of California San Diego, La Jolla, California, USA.
7: Department of Paediatric Medicine, University Hospital Southampton NHS Foundation Trust, Southampton, UK.
8: Pediatric Neurology and Metabolic Diseases, Universitair Ziekenhuis Brussels, Vrije Universiteit Brussel, Brussels, Belgium.
9: Medical Genetics Department, Koc University School of Medicine, Istanbul, Turkey.
10: Medical Genetics Department, Istanbul Medical Faculty, Istanbul University, Istanbul Turkey.
11: Department of Clinical Genetics, The Canberra Hospital, Woden, Australian Capital Territory, Australia.
12: Australian Capital Territory Genetic Service, The Canberra Hospital, Canberra City, Australian Capital Territory, Australia.
13: Department of Pediatrics, Division of Clinical and Metabolic Genetics, The Hospital for Sick Children, Toronto, Ontario, Canada.
14: The Prenatal Diagnosis and Medical Genetics Program, Department of Obstetrics and Gynecology, Mount Sinai Hospital, University of Toronto, Toronto, Ontario, Canada.
15: Division of Neuroradiology, Department of Diagnostic Imaging, The Hospital for Sick Children, Toronto, Ontario, Canada.
16: Department of Medical Genetics, School of Medicine, Istanbul Bilim University, Istanbul, Turkey.
17: Yale Program on Neurogenetics, Departments of Neurosurgery, Neurobiology and Genetics, Yale University School of Medicine, New Haven, Connecticut, USA.
18: Department of Genetics, Yale Center for Genome Analysis, Yale University School of Medicine, New Haven, Connecticut, USA.
19: Division of Pediatric Neurology, Department of Pediatrics, Erciyes University School of Medicine, Kayseri, Turkey.
20: Department of Clinical Genetics, Odense University Hospital, Odense, Denmark.
21: Hans Christian Andersen Children's Hospital, Odense University Hospital, Odense, Denmark.
22: Center for Integrative Brain Research, Seattle Children's Research Institute, Seattle, Washington, USA.
23: Department of Pediatrics, Clinical Genetics and Metabolism, University of Colorado School of Medicine, Children's Hospital Colorado, Aurora, Colorado, USA.
24: Department of Human Genetics, Yokohama City University, Graduate School of Medicine, Yokohama, Japan.
25: Department of Pediatrics, Gunma University School of Medicine, Showa-machi, Maebashi City, Japan.
26: Department of Biochemistry, Hamamatsu University School of Medicine, Hamamatsu, Japan.
27: Department of Biochemistry, Yokohama City University Graduate School of Medicine, Yokohama, Japan.
28: Southampton University Hospitals Trust, Southampton, UK.
29: UCSD Cardiology, University of California San Diego, La Jolla, California, USA.
30: Clinical Genetics Unit, Department of Women, Mother and Neonates, "Vittore Buzzi" Children's Hospital, Istituti Clinici di Perfezionamento, Milan, Italy.
31: Child Neurology Unit, Department of Pediatrics, "Vittore Buzzi" Children Hospital, Istituti Clinici di Perfezionamento, Milan, Italy.
32: Broad Institute of Harvard and MIT, Cambridge, Massachusetts, USA.
33: Section of Neurosciences, Department of Medicine and Surgery, University of Salerno, Salerno, Italy.
34: Pediatric Neurology, Université Catholique de Louvain, Cliniques Universitaires Saint-Luc, Brussels, Belgium.
35: Department of Physiology, National University of Singapore and Molecular Engineering Laboratory, A*STAR, Singapore.
36: Department of Clinical Genetics, Academic Medical Centre, University of Amsterdam, Amsterdam, the Netherlands.

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