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Issue DateTitleAuthor(s)
19-Apr-2018Gene therapy in patients with transfusion-dependent β-thalassemiaA. A. Thompson; M. C. Walters; J. Kwiatkowski; J. E.J. Rasko; J. A. Ribeil; S. Hongeng; E. Magrin; G. J. Schiller; E. Payen; M. Semeraro; D. Moshous; F. Lefrere; H. Puy; P. Bourget; A. Magnani; L. Caccavelli; J. S. Diana; F. Suarez; F. Monpoux; V. Brousse; C. Poirot; C. Brouzes; J. F. Meritet; C. Pondarré; Y. Beuzard; S. Chrétien; T. Lefebvre; D. T. Teachey; U. Anurathapan; P. J. Ho; C. Von Kalle; M. Kletzel; E. Vichinsky; S. Soni; G. Veres; O. Negre; R. W. Ross; D. Davidson; A. Petrusich; L. Sandler; M. Asmal; O. Hermine; M. De Montalembert; S. Hacein-Bey-Abina; S. Blanche; P. Leboulch; M. Cavazzana; Bluebird Bio, Inc.; Lucille Packard Children's Hospital; Universite Paris Descartes; Hôpital Necker Enfants Malades; The University of Sydney; Hopital Cochin AP-HP; Royal Prince Alfred Hospital; German Cancer Research Center; Brigham and Women's Hospital; Faculty of Medicine, Ramathibodi Hospital, Mahidol University; Hopital de Bicetre; Northwestern University Feinberg School of Medicine; CEA Fontenay aux Roses; UCSF Benioff Children's Hospital Oakland; University of Pennsylvania; Universite Paris 7- Denis Diderot; David Geffen School of Medicine at UCLA; Centenary Institute of Cancer Medicine and Cell Biology; Hopital Louis-Mourier; Sorbonne Universite; Centre Hospitalier Intercommunal Creteil; Imagine Institute; Groupe Hospitalier Universitaire Ouest
1-Oct-2017Mutations in KEOPS-complex genes cause nephritic syndrome with primary microcephalyDaniela A. Braun; Jia Rao; Geraldine Mollet; David Schapiro; Marie Claire Daugeron; Weizhen Tan; Olivier Gribouval; Olivia Boyer; Patrick Revy; Tilman Jobst-Schwan; Johanna Magdalena Schmidt; Jennifer A. Lawson; Denny Schanze; Shazia Ashraf; Jeremy F.P. Ullmann; Charlotte A. Hoogstraten; Nathalie Boddaert; Bruno Collinet; Gaelle Martin; Dominique Liger; Svjetlana Lovric; Monica Furlano; I. Chiara Guerrera; Oraly Sanchez-Ferras; Jennifer F. Hu; Anne Claire Boschat; Sylvia Sanquer; Björn Menten; Sarah Vergult; Nina De Rocker; Merlin Airik; Tobias Hermle; Shirlee Shril; Eugen Widmeier; Heon Yung Gee; Won Il Choi; Carolin E. Sadowski; Werner L. Pabst; Jillian K. Warejko; Ankana Daga; Tamara Basta; Verena Matejas; Karin Scharmann; Sandra D. Kienast; Babak Behnam; Brendan Beeson; Amber Begtrup; Malcolm Bruce; Gaik Siew Ch'Ng; Shuan Pei Lin; Jui Hsing Chang; Chao Huei Chen; Megan T. Cho; Patrick M. Gaffney; Patrick E. Gipson; Chyong Hsin Hsu; Jameela A. Kari; Yu Yuan Ke; Cathy Kiraly-Borri; Wai Ming Lai; Emmanuelle Lemyre; Rebecca Okashah Littlejohn; Amira Masri; Mastaneh Moghtaderi; Kazuyuki Nakamura; Fatih Ozaltin; Marleen Praet; Chitra Prasad; Agnieszka Prytula; Elizabeth R. Roeder; Patrick Rump; Rhonda E. Schnur; Takashi Shiihara; Manish D. Sinha; Neveen A. Soliman; Children's Hospital Boston; Inserm; Universite Paris Descartes; Universite Paris-Sud XI; Hôpital Necker Enfants Malades; Medizinische Fakultät und Uniklinikum Magdeburg; Harvard Medical School; Sorbonne Universite; Universidad Autónoma de Barcelona, Facultad de Medicina; McGill University, Rosalind and Morris Goodman Cancer Research Centre; Massachusetts Institute of Technology; Imagine Institute; University Hospital of Ghent; Universität Freiburg im Breisgau; Yonsei University College of Medicine; Friedrich-Alexander-Universität Erlangen-Nürnberg; Max Planck Institut für molekulare Biomedizin; Westfälische Wilhelms-Universität Münster; Iran University of Medical Sciences; National Human Genome Research Institute; King Edward Memorial Hospital for Women; GeneDX, Inc.; Kuala Lumpur Hospital; MacKay Children's Hospital; Mackay Medical College; Veterans General Hospital-Taichung Taiwan; Oklahoma Medical Research Foundation; University of Michigan, Ann Arbor; King Abdulaziz University; Princess Margaret Hospital Hong Kong; University of Montreal; Baylor College of Medicine; The University of Jordan; Tehran University of Medical Sciences; Yamagata University Faculty of Medicine; Hacettepe University, Faculty of Medicine; Hacettepe Üniversitesi; Western University; University of Groningen, University Medical Center Groningen; Guy's and St Thomas' NHS Foundation Trust; Cairo University; Egyptian Group for Orphan Renal Diseases; Centre Hospitalier Universitaire Ibn-Rochd; Massachusetts General Hospital; Chi Mei Medical Center; Taipei Medical University; Universitäts Klinikum Essen und Medizinische Fakultät; University of Utah, School of Medicine; Mahidol University; University of Oklahoma Health Sciences Center; UT Southwestern Medical School; Tuen Mun Hospital; Singapore-MIT Alliance; Yale University School of Medicine; Rockefeller University; Hospital for Sick Children University of Toronto; Pediatric Nephrology Institute