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S258 : RETROVIRAL GENE THERAPY FOR THE TREATMENT OF ADA-SCID: LONG-TERM FOLLOW UP AND FIRST CASE OF T-CELL ACUTE LEUKAEMIA DUE TO INSERTIONAL MUTAGENESIS

Researchers

Presenter

  • Maria Pia Cicalese

Principal Investigators

  • Daniela Cesana

  • Roberta Caruso

  • Andrea Calabria

  • Federica Barzaghi

  • Francesca Dionisio

  • Stefania Giannelli

  • Giulio Spinozzi

  • Fabrizio Benedicenti

  • Laura Rudilosso

  • Andrea Ciolfi

  • Alessandro Bruselles

  • Simone Pizzi

  • Miriam Casiraghi

  • Claudia Fossati

  • Stefano Zancan

  • Michela Gabaldo

  • Francesca Tucci

  • Luisa Strocchio

  • Maria Ester Bernardo

  • Luciana Vinti

  • Francesca Ferrua

  • Valeria Calbi

  • Vera Gallo

  • Filippo Carlucci

  • Andrea Guerra

  • Alan Richardson

  • Mahesh Kudari

  • Russell Jones

  • Caterina Cancrini

  • Fabio Ciceri

  • Luigi Naldini

  • Marco Tartaglia

  • Eugenio Montini

  • Franco Locatelli

  • Alessandro Aiuti

Medical Centers

  • Hematology and Bone Marrow Transplantation Unit, IRCCS San Raffaele Scientific Institute, Milano, Italy

  • Genetics and Rare Diseases Research Division,Bambino Gesù Children's Hospital, IRCCS,Rome,Italy

  • Fondazione Telethon,Milan,Italy

  • Department of Pediatrics,Sapienza University of Rome,Rome, Italy #Equal contribution

  • Department of Onco-haematology, Cell and Gene Therapy, Bambino Gesù Children’s Hospital-IRCCS, Rome, Italy

  • Department of Hematology, Oncology and Molecular Medicine, Istituto Superiore di Sanita, Rome, Italy

  • Department of Paediatrics,Ospedale Pediatrico Bambino Gesù and University of Rome 'Tor Vergata',Rome,Italy

  • San Raffaele Telethon Institute for Gene Therapy (HSR-TIGET), San Raffaele Scientific Institute, Milan, Italy

  • Department of Medical Biotechnologies, University of Siena, Siena, Italy

  • equal contribution, ., Italy

  • Universita Vita Salute San Raffaele, Milan, Italy

  • Hematology and Bone Marrow Transplantation Unit, IRCCS San Raffaele Scientific Institute, Milano, Italy

  • Orchard Therapeutics,London,United Kingdom

Locations

  • Italy

  • United Kingdom

Companies

  • N/A

Study Components

Therapeutic Area

  • Oncology (ONC)

  • Blood/haematological Diseases

Disease

  • Acute Leukemia

Biomarkers

  • Cluster of Differentiation 34

Drug/Treatment

  • Busulfan

Outcome

  • N/A

Study Design

  • Cohort

Phase

  • NA

Study Id's

  • N/A

Sponsors

  • N/A

Result

  • N/A

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