Keynote lecture 'Gene therapy for severe immunodeficiencies'

Professor Bobby Gaspar

Keynote lecture 'Gene therapy for severe immunodeficiencies'

Professor Bobby Gaspar ot_0011.jpg
Professor of Paediatrics and Immunology at UCL Great Ormond Street Institute of Child Health and Honorary Consultant Paediatric Immunologist at Great Ormond Street Hospital for Children NHS Foundation Trust.

Professor Bobby Gaspar is initially trained in paediatrics and then became interested in primary immunodeficiencies at an early stage in his career and undertook a PhD at the Molecular Immunology Unit at ICH. From there he has continued his academic and clinical career. His interests are in many different aspects of primary immunodeficiency including understanding the molecular and cellular defects and disease pathogenesis, bone marrow transplantation for severe immunodeficiencies and the development of gene and cell therapy for these conditions. Over the last decade, his team have conducted clinical trials that have shown that gene therapy can successfully correct the immune defect in specific immunodeficiency conditions. He is leading UK and European initiatives for newborn screening for severe combined immunodeficiency. He is also Director Designate of the Zayed Centre for Research into Rare Diseases in Children (ZCRRDC), the new academic and clinical facility that will open at Great Ormond Street in 2018.

Selected publications:

  • Outcome following Gene Therapy in Patients with Severe Wiskott-Aldrich Syndrome
    Salima Hacein-Bey Abina, H. Bobby Gaspar, Johanna Blondeau, Laure Caccavelli, Sabine Charrier, Karen Buckland, Capucine Picard, Emmanuelle Six, Nourredine Himoudi, Kimberly Gilmour, Anne-Marie McNicol, Havinder Hara, Jinhua Xu-Bayford, Christine Rivat, Fabien Touzot, Fulvio Mavilio, Annick Lim, Jean-Marc Treluyer, Sébastien Héritier, Francois Lefrere, Jeremy Magalon, Isabelle Pengue-Koyi, Géraldine Honnet, Stéphane Blanche, Eric A. Sherman, Frances Male, Charles Berry, Nirav Malani, Frederic D. Bushman, Alain Fischer, Adrian J. Thrasher, Anne Galy, Marina Cavazzana
    JAMA. Author manuscript; available in PMC 2016 Jul 13. Published in final edited form as: JAMA. 2015 Apr 21; 313(15): 1550–1563. doi: 10.1001/jama.2015.3253 
  • Immunodeficiency and disseminated mycobacterial infection associated with homozygous nonsense mutation of IKKβ
    Siobhan O. Burns, Vincent Plagnol, Beatriz Morillo Gutierrez, Daifulah Al Zahrani, James Curtis, Miguel Gaspar, Amel Hassan, Alison M. Jones, Marian Malone, Dyanne Rampling, Alex McLatchie, Rainer Doffinger, Kimberly C. Gilmour, Frances Henriquez, Adrian J. Thrasher, H. Bobby Gaspar, Sergey Nejentsev
    J Allergy Clin Immunol. 2014 Jul; 134(1): 215–218.e3. doi: 10.1016/j.jaci.2013.12.1093 
  • Preclinical Demonstration of Lentiviral Vector-mediated Correction of Immunological and Metabolic Abnormalities in Models of Adenosine Deaminase Deficiency
    Denise A Carbonaro, Lin Zhang, Xiangyang Jin, Claudia Montiel-Equihua, Sabine Geiger, Marlene Carmo, Aaron Cooper, Lynette Fairbanks, Michael L Kaufman, Neil J Sebire, Roger P Hollis, Michael P Blundell, Shantha Senadheera, Pei-Yu Fu, Arineh Sahaghian, Rebecca Y Chan, Xiaoyan Wang, Kenneth Cornetta, Adrian J Thrasher, Donald B Kohn, H Bobby Gaspar
    Mol Ther. 2014 Mar; 22(3): 607–622. Prepublished online 2013 Nov 20. Published online 2014 Jan 21. doi: 10.1038/mt.2013.265
  • “Special Exemptions”: Should They Be Put on Trial?
    H Bobby Gaspar, Sue Swift, Adrian J Thrasher
    Mol Ther. 2013 Feb; 21(2): 261–262. Published online 2013 Feb 1. doi: 10.1038/mt.2013.1


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