검색어 : 통합검색[Benioff David]
총 273건 중 273건 출력
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21
- Long-Term Clinical Outcomes of Lentiglobin Gene Therapy for Transfusion-Dependent β-Thalassemia in the Northstar (HGB-204) Study
- Kwiatkowski, Janet L.; Thompson, Alexis A.; Rasko, John E.J.; Hongeng, Suradej; Schiller, Gary J.; Anurathapan, Usanarat; Cavazzana, Marina; Ho, P. Joy; Schmidt, Manfred; Kletzel, Morris; Vichinsky, Elliott P.; Deary, Briana; Chen, Ying; Petrusich, Alexandria; Walters, Mark C.; Department of Pediatrics, University of Pennsylvania Perelman School of Medicine, Philadelphia, PA; Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, IL; Sydney Medical School, University of Sydney, Camperdown, Australia; Mahidol University, Ramathibodi Hospital, Bangkok, Thailand; David Geffen School of Medicine at UCLA, Los Angeles, CA; Mahidol University, Ramathibodi Hospital, Bangkok, Thailand; IMAGINE Institute, Paris Descartes-Sorbonne Paris Cité University, Paris, France; Institute of Haematology, Royal Prince Alfred Hospital, Sydney, Australia; GeneWerk GmbH, Heidelberg, Germany; Northwestern University Feinberg School of Medicine, Chicago, IL; UCSF Benioff Children's Hospital, Oakland, CA; bluebird bio, Inc., Cambridge, MA; bluebird bio, Inc., Cambridge, MA; bluebird bio, Inc., Cambridge, MA; UCSF Benioff Children's Hospital, Oakland, CA; (Blood, v.134, 2019, pp.4628-4628)
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- Clinical Outcomes of Lentiglobin Gene Therapy for Transfusion-Dependent β-Thalassemia Following Completion of the Northstar HGB-204 Study
- Rasko, John E.J.; Thompson, Alexis A.; Kwiatkowski, Janet L.; Hongeng, Suradej; Schiller, Gary J.; Anurathapan, Usanarat; Cavazzana, Marina; Ho, P. Joy; Schmidt, Manfred; Kletzel, Morris; Leboulch, Philippe; Vichinsky, Elliott; Deary, Briana; Chen, Ying; Asmal, Mohammed; Walters, Mark C.; Royal Prince Alfred Hospital, Camperdown, Australia; Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, IL; Division of Hematology, Children's Hospital of Philadelphia, Philadelphia, PA; Mahidol University, Ramathibodi Hospital, Bangkok, Thailand; David Geffen School of Medicine at UCLA, Los Angeles, CA; Mahidol University, Ramathibodi Hospital, Bangkok, Thailand; Biotherapy Department, Hopital Necker - Enfants Malades, Paris, France; Royal Prince Alfred Hospital, Camperdown, Australia; GeneWerk GmbH, Heidelberg, Germany; Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, IL; Brigham & Women's Hospital and Harvard Medical School, Boston, MA; UCSF Benioff Children's Hospital, Oakland, CA; bluebird bio, Inc., Cambridge, MA; bluebird bio, Inc., Cambridge, MA; bluebird bio, Inc., Cambridge, MA; UCSF Benioff Children's Hospital, Oakland, CA; (Blood, v.132, 2018, pp.167-167)
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- Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia
- Thompson, Alexis A.; Walters, Mark C.; Kwiatkowski, Janet; Rasko, John E.J.; Ribeil, Jean-Antoine; Hongeng, Suradej; Magrin, Elisa; Schiller, Gary J.; Payen, Emmanuel; Semeraro, Michaela; Moshous, Despina; Lefrere, Francois; Puy, Hervé ; Bourget, Philippe; Magnani, Alessandra; Caccavelli, Laure; Diana, Jean-Sé bastien; Suarez, Felipe; Monpoux, Fabrice; Brousse, Valentine; Poirot, Catherine; Brouzes, Chantal; Meritet, Jean-Franç ois; Pondarré , Corinne; Beuzard, Yves; Chré tien, Stany; Lefebvre, Thibaud; Teachey, David T.; Anurathapan, Usanarat; Ho, P. Joy; von Kalle, Christof; Kletzel, Morris; Vichinsky, Elliott; Soni, Sandeep; Veres, Gabor; Negre, Olivier; Ross, Robert W.; Davidson, David; Petrusich, Alexandria; Sandler, Laura; Asmal, Mohammed; Hermine, Olivier; De Montalembert, Mariane; Hacein-Bey-Abina, Salima; Blanche, Sté phane; Leboulch, Philippe; Cavazzana, Marina; From the Ann and Robert H. Lurie Children’s Hospital of Chicago, Northwestern University Feinberg School of Medicine, Chicago (A.A.T., M.K.); University of California, San Francisco, Benioff Children’s Hospital, Oakland (M.C.W., E.V.), Lucile Salter Packard Children’s Hospital at Stanford, Palo Alto (S.S.), and David Geffen School of Medicine, University of California, Los Angeles, Los Angeles (G.J.S.) - all in California; Children’s Hospital of Philadelphia and University of Pennsylvania Perelman...; From the Ann and Robert H. Lurie Children’s Hospital of Chicago, Northwestern University Feinberg School of Medicine, Chicago (A.A.T., M.K.); University of California, San Francisco, Benioff Children’s Hospital, Oakland (M.C.W., E.V.), Lucile Salter Packard Children’s Hospital at Stanford, Palo Alto (S.S.), and David Geffen School of Medicine, University of California, Los Angeles, Los Angeles (G.J.S.) - all in California; Children’s Hospital of Philadelphia and Universit; (The New England journal of medicine, v.378, 2018, pp.1479-1493)
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- An exploration of concomitant psychiatric disorders in children with autism spectrum disorder
- Lecavalier, Luc; McCracken, Courtney E.; Aman, Michael G.; McDougle, Christopher J.; McCracken, James T.; Tierney, Elaine; Smith, Tristram; Johnson, Cynthia; King, Bryan; Handen, Benjamin; Swiezy, Naomi B.; Eugene Arnold, L.; Bearss, Karen; Vitiello, Benedetto; Scahill, Lawrence; Ohio State University, Nisonger Center; Emory University School of Medicine, Department of Pediatrics; Ohio State University, Nisonger Center; Lurie Center for Autism, Massachusetts General Hospital; Department of Psychiatry and Biobehavioral Sciences, David Geffen School of Medicine at University of California at Los Angeles; Johns Hopkins University School of Medicine; University of Rochester Medical Center; Cleveland Clinic and Learner College of Medicine; University of California, San Francisco, Department of Psychiatry, Benioff Children's Hospitals; University of Pittsburgh School of; (Comprehensive psychiatry, v.88, 2019, pp.57-64)
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- A Comparison of Attitudes and Practice between Pediatric Nurses and Respiratory Therapists in Addressing Tobacco Smoke Exposure during an Inpatient Pediatric Hospital Stay
- Williams, Brian; Marbin, Jyothi; Huang, Maria; Kosack, Amanda; Garell, Cambria; Gribben, Valerie; Ross, Javay; Shaikh, Ulfat; Tebb, Kathleen; (1)University of Wisconsin School of Medicine and Public Health, Madison, WI; (2)Department of Pediatrics, UCSF, San Francisco, CA; (3)UC-San Diego, Rady Children's Hospital-San Diego, San Diego, CA; (4)Department of Pediatrics, UCLA, Los Angeles, CA; (5)Department of Pediatrics, David Geffen School of Medicine at UCLA, Los Angeles, CA; (6)UC San Francisco, San Francisco, CA; (7)UCSF Benioff Children's Hospital Oakland, Oakland, CA; (8)Department of Pediatrics, UCD, Sacramento, CA; (2)Department of Pediatrics, UCSF, San Francisco, CA; (Pediatrics, v.144, 2019, pp.850-850)
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- Updated Results of a Phase 1/2 Clinical Study of Zinc Finger Nuclease-Mediated Editing of <i>BCL11A</i> in Autologous Hematopoietic Stem Cells for Transfusion-Dependent Beta Thalassemia
- Walters, Mark C.; Smith, Angela R.; Schiller, Gary J.; Esrick, Erica B.; Williams, David A.; Gogoleva, Tatiana; Rouy, Didier; Cockroft, Bettina M.; Vercellotti, Gregory M.; Division of Hematology, UCSF Benioff Children's Hospital Oakland, Oakland, CA; Pediatric Blood and Marrow Transplantation, University of Minnesota, Minneapolis, MN; Division of Hematology and Oncology, Department of Medicine, David Geffen School of Medicine at the University of California Los Angeles, Los Angeles, CA; Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Boston, MA; Division of Hematology/Oncology, Boston Children's Hospital, Boston,; Sangamo Therapeutics, Brisbane, CA, Brisbane, CA; Sangamo Therapeutics, Brisbane, CA; Sangamo Therapeutics, Brisbane, CA; Division of Hematology, Oncology and Transplantation, Department of Medicine, University of Minnesota Medical School, Minneapolis, MN; (Blood, v.138, 2021, pp.3974-3974)
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- A Psychometric Evaluation of the Motor-Behavioral Assessment Scale for Use as an Outcome Measure in Rett Syndrome Clinical Trials
- Raspa, Melissa; Bann, Carla M.; Gwaltney, Angela; Benke, Timothy A.; Fu, Cary; Glaze, Daniel G.; Haas, Richard; Heydemann, Peter; Jones, Mary; Kaufmann, Walter E.; Lieberman, David; Marsh, Eric; Peters, Sarika; Ryther, Robin; Standridge, Shannon; Skinner, Steven A.; Percy, Alan K.; Neul, Jeffrey L.; Melissa Raspa, Carla M. Bann, and Angela Gwaltney, RTI International; Melissa Raspa, Carla M. Bann, and Angela Gwaltney, RTI International; Melissa Raspa, Carla M. Bann, and Angela Gwaltney, RTI International; Timothy A. Benke, University of Colorado School of Medicine; Cary Fu, Vanderbilt Kennedy Center; Daniel G. Glaze, Baylor College of Medicine; Richard Haas, University of California San Diego; Peter Heydemann, Rush University Medical Center; Mary Jones, Benioff Children's Hospital; Walter E. Kaufmann, Greenwood Genetic Center; David Lieberman and Eric Marsh, Children's Hospital Boston; David Lieberman and Eric Marsh, Children's Hospital Boston; Sarika Peters, Vanderbilt Kennedy Center; Robin Ryther, Washington University School of Medicine; Shannon Standridge, Cincinnati Children's Hospital; Steven A. Skinner, Greenwood Genetic Center; Alan K. Percy, University of Alabama at Birmingham; Jeffrey L. Neul, Vanderbilt Kennedy Center; (American journal on intellectual and developmental disabilities, v.125, 2020, pp.493-509)
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- Nijmegen Breakage Syndrome Detected by Newborn Screening for T Cell Receptor Excision Circles (TRECs)
- Patel, Jay P.; Puck, Jennifer M.; Srinivasan, Rajgopal; Brown, Christina; Sunderam, Uma; Kundu, Kunal; Brenner, Steven E.; Gatti, Richard A.; Church, Joseph A.; Division of General Pediatrics, Children’s Hospital of Los Angeles, Los Angeles, CA USA; Department of Pediatrics and Institute for Human Genetics, University of California, San Francisco and UCSF Benioff Children’s Hospital, San Francisco, CA USA; Tata Consultancy Services Ltd., Hyderabad, Telengana India; Departments of Human Genetics and Pathology & Laboratory Medicine, David Geffen School of Medicine, University of California, Los Angeles, CA USA; Tata Consultancy Services Ltd., Hyderabad, Telengana India; Tata Consultancy Services Ltd., Hyderabad, Telengana India; Department of Plant and Microbial Biology, University of California, Berkeley, CA USA; Departments of Human Genetics and Pathology & Laboratory Medicine, David Geffen School of Medicine, University of California, Los Angeles, CA USA; Division of Clinical Immunology and Allergy, Children’s Hospital of Los Angeles, Keck School of Medicine, University of Southern California, Los Angeles, CA USA; (Journal of clinical immunology, v.35, 2015, pp.227-233)
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- ACEP SimBox: A Pediatric Simulation-Based Training Innovation
- Vora, Samreen; Li, Joyce; Kou, Maybelle; Ng, Vivienne; Price, Amanda; Claudius, Ilene; Kant, Shruti; Sanseau, Elizabeth; Madhok, Manu; Auerbach, Marc; Department of Emergency Medicine, Children’s Minnesota Hospital; Division of Emergency Medicine, Boston Children’s Hospital, Harvard Medical School; Department of Emergency Medicine, Inova Fairfax Medical Campus/Inova Children’s Hospital; Department of Emergency Medicine, The University of Arizona; Division of Pediatric Emergency Medicine, Medical University South Carolina; Department of Emergency Medicine, David Geffen School of Medicine at UCLA; Department of Emergency Medicine, Benioff Children’s Hospital; Division of Emergency Medicine, Children’s Hospital of Philadelphia; Department of Emergency Medicine, Children’s Minnesota Hospital; Department of Pediatrics, Department of Emergency Medicine, Yale University School of Medicine; (Annals of emergency medicine, v.78, 2021, pp.346-354)
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- Initial Results from the Northstar Study (HGB-204): A Phase 1/2 Study of Gene Therapy for β-Thalassemia Major Via Transplantation of Autologous Hematopoietic Stem Cells Transduced Ex Vivo with a Lentiviral βΑ-T87Q -Globin Vector (LentiGlobin BB305 Drug Product)
- Thompson, Alexis A.; Rasko, John E; Hongeng, Suradej; Kwiatkowski, Janet L.; Schiller, Gary; von Kalle, Christof; Cavazzana, Marina; Leboulch, Philippe; Petrusich, Alexandria; Soni, Sandeep; Walters, Mark C.; Ann and Robert H. Lurie Children's Hospital of Chicago, Northwestern University, Chicago, IL; Royal Prince Alfred Hospital, Sydney, Australia; Faculty of Medicine, Ramathibodi Hospital, Bangkok, Thailand; Children's Hospital of Philadelphia and Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA; David Geffen School of Medicine at UCLA, Los Angeles, CA; National Center for Tumor Diseases (NCT) and German Cancer Research Center (DKFZ), Heidelberg, Germany; Hô pital Universitaire Necker - Enfants Malades, Paris, France; Brigham & Women's Hospital and Harvard Medical School, Boston, MA; bluebird bio, Inc., Cambridge, MA; bluebird bio, Inc., Cambridge, MA; UCSF Benioff Children's Hospital, Oakland, CA; (Blood, v.124, 2014, pp.549-549)
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