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Expert Opinion on Biological Therapy Volume 8, 2008 - Issue 4
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Gene therapy of inherited immunodeficiencies

Giorgia Santilli Senior Research Fellow University College London, Institute of Child Health, Centre for Immunodeficiency, Molecular Immunology Unit, 30 Guilford Street, London, WC1N 1EH, UK
, PhD,
Susannah I Thornhill Research Fellow University College London, Institute of Child Health, Centre for Immunodeficiency, Molecular Immunology Unit, 30 Guilford Street, London, WC1N 1EH, UK
, PhD,
Christine Kinnon Professor of Molecular Immunology University College London, Institute of Child Health, Centre for Immunodeficiency, Molecular Immunology Unit, 30 Guilford Street, London, WC1N 1EH, UK
, PhD &
Adrian J Thrasher Professor of Paediatric Immunology University College London, Institute of Child Health, Centre for Immunodeficiency, Molecular Immunology Unit, 30 Guilford Street, London, WC1N 1EH, UK; Great Ormond Street Hospital NHS Trust, Department of Clinical Immunology, London WC1N 3RJ, UK +44 0207 813 8490; +44 0207 905 2810; [email protected]
, PhD FRCP FMedSci
Pages 397-407 | Published online: 19 Mar 2008

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Makoto Hayashi. (2010) Associating Gene Therapy with the Human Genome Project. East Asian Science, Technology and Society: An International Journal 4:1, pages 77-97.
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Articles from other publishers (23)

Elena Blanco, Natalia Izotova, Claire Booth & Adrian James Thrasher. (2020) Immune Reconstitution After Gene Therapy Approaches in Patients With X-Linked Severe Combined Immunodeficiency Disease. Frontiers in Immunology 11.
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Laura Garcia-Perez, Marja van Eggermond, Lieke van Roon, Sandra A. Vloemans, Martijn Cordes, Axel Schambach, Michael Rothe, Dagmar Berghuis, Chantal Lagresle-Peyrou, Marina Cavazzana, Fang Zhang, Adrian J. Thrasher, Daniela Salvatori, Pauline Meij, Anna Villa, Jacques J.M. Van Dongen, Jaap-Jan Zwaginga, Mirjam van der Burg, H. Bobby Gaspar, Arjan Lankester, Frank J.T. Staal & Karin Pike-Overzet. (2020) Successful Preclinical Development of Gene Therapy for Recombinase-Activating Gene-1-Deficient SCID. Molecular Therapy - Methods & Clinical Development 17, pages 666-682.
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Alfred Pingoud, George H. Silva & Wolfgang Wende. 2013. Modern Biopharmaceuticals. Modern Biopharmaceuticals 165 181 .
Sean Knight, Fang Zhang, Uta Mueller-Kuller, Marieke Bokhoven, Abhinav Gupta, Thomas Broughton, Sha Sha, Michael N. Antoniou, Christian Brendel, Manuel Grez, Adrian J. Thrasher, Mary Collins & Yasuhiro Takeuchi. (2012) Safer, Silencing-Resistant Lentiviral Vectors: Optimization of the Ubiquitous Chromatin-Opening Element through Elimination of Aberrant Splicing. Journal of Virology 86:17, pages 9088-9095.
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Karen Beemon & Naomi Rosenberg. 2012. Cancer Associated Viruses. Cancer Associated Viruses 677 704 .
Rohan Ameratunga, See-Tarn Woon, Maia Brewerton, Wikke Koopmans, Anthony Jordan, Shannon Brothers & Ranjeeta Singh. (2011) Primary immune deficiency disorders in the South Pacific: the clinical utility of a customized genetic testing program in New Zealand. Annals of the New York Academy of Sciences 1238:1, pages 53-64.
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E. AlmarzaF. ZhangG. SantilliM.P. BlundellS.J. HoweS.I. ThornhillJ.A. BuerenA.J. Thrasher. (2011) Correction of SCID-X1 Using an Enhancerless Vav Promoter . Human Gene Therapy 22:3, pages 263-270.
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H. Akita, H. Hatakeyama, I.A. Khalil, Y. Yamada & H. Harashima. 2011. Comprehensive Biomaterials. Comprehensive Biomaterials 411 444 .
Tanya Lynn Applegate, Donald John Birkett, Glen John Mcintyre, Angel Belisario Jaramillo, Geoff Symonds & John Michael Murray. (2010) In silico modeling indicates the development of HIV-1 resistance to multiple shRNA gene therapy differs to standard antiretroviral therapy. Retrovirology 7:1.
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Rohan Ameratunga, See-Tarn Woon, Katherine Neas & Donald R Love. (2010) The clinical utility of molecular diagnostic testing for primary immune deficiency disorders: a case based review. Allergy, Asthma & Clinical Immunology 6:1.
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Simon Urschel. (2010) Otitis Media in Children with Congenital Immunodeficiencies. Current Allergy and Asthma Reports 10:6, pages 425-433.
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Hannah M. KernsByoung Y. RyuBrigid V. StirlingBlythe D. SatherAlexander AstrakhanStephanie Humblet-Baron, Denny Liggitt & David J. Rawlings. (2010) B cell–specific lentiviral gene therapy leads to sustained B-cell functional recovery in a murine model of X-linked agammaglobulinemia. Blood 115:11, pages 2146-2155.
Crossref
Makoto Hayashi. (2010) Associating Gene Therapy with the Human Genome Project. East Asian Science, Technology and Society: an International Journal 4:1, pages 77-97.
Crossref
Javier Chinen & Rebecca H. Buckley. (2010) Transplantation immunology: Solid organ and bone marrow. Journal of Allergy and Clinical Immunology 125:2, pages S324-S335.
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Eliot Ward, Emma Chan, Kenth Gustafsson & Suwan N. Jayasinghe. (2010) Combining bio-electrospraying with gene therapy: a novel biotechnique for the delivery of genetic material via living cells. The Analyst 135:5, pages 1042.
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Yngvar Fløisand & Mouldy Sioud. 2010. RNA Therapeutics. RNA Therapeutics 505 521 .
Naomi Rosenberg. 2011. Retroviruses and Insights into Cancer. Retroviruses and Insights into Cancer 1 30 .
Rachael Hough, Nicola Cooper & Paul Veys. (2009) Allogeneic haemopoietic stem cell transplantation in children: what alternative donor should we choose when no matched sibling is available?. British Journal of Haematology 147:5, pages 593-613.
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. (2009) Current World Literature. Current Opinion in Allergy & Clinical Immunology 9:6, pages 574-578.
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Mirjam van der Burg, Jacques JM van Dongen & Dik C van Gent. (2009) DNA-PKcs deficiency in human: long predicted, finally found. Current Opinion in Allergy & Clinical Immunology 9:6, pages 503-509.
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Maheswaran ManiShivkumar Venkatasubrahmanyam, Mrinmoy Sanyal, Shoshana Levy, Atul Butte, Kenneth WeinbergThomas Jahn. (2009) Wiskott-Aldrich syndrome protein is an effector of Kit signaling. Blood 114:14, pages 2900-2908.
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Christian J. Buchholz, Michael D. Mühlebach & Klaus Cichutek. (2009) Lentiviral vectors with measles virus glycoproteins – dream team for gene transfer?. Trends in Biotechnology 27:5, pages 259-265.
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Suk See De Ravin & Harry L. Malech. (2008) Partially corrected X-linked severe combined immunodeficiency: long-term problems and treatment options. Immunologic Research 43:1-3, pages 223-242.
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