Literature DB >> 22134740

Gene therapy for SCID-X1: focus on clinical data.

Christopher Baum.   

Abstract

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Year:  2011        PMID： 22134740      PMCID： PMC3242671          DOI： 10.1038/mt.2011.252

Source DB:  PubMed          Journal:  Mol Ther        ISSN： 1525-0016            Impact factor:   11.454


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  14 in total

1.  Insertional transformation of hematopoietic cells by self-inactivating lentiviral and gammaretroviral vectors.

Authors:  Ute Modlich; Susana Navarro; Daniela Zychlinski; Tobias Maetzig; Sabine Knoess; Martijn H Brugman; Axel Schambach; Sabine Charrier; Anne Galy; Adrian J Thrasher; Juan Bueren; Christopher Baum
Journal:  Mol Ther       Date:  2009-08-11       Impact factor: 11.454

2.  Transplantation of hematopoietic stem cells and long-term survival for primary immunodeficiencies in Europe: entering a new century, do we do better?

Authors:  Andrew R Gennery; Mary A Slatter; Laure Grandin; Pierre Taupin; Andrew J Cant; Paul Veys; Persis J Amrolia; H Bobby Gaspar; E Graham Davies; Wilhelm Friedrich; Manfred Hoenig; Luigi D Notarangelo; Evelina Mazzolari; Fulvio Porta; Robbert G M Bredius; Arjen C Lankester; Nico M Wulffraat; Reinhard Seger; Tayfun Güngör; Anders Fasth; Petr Sedlacek; Benedicte Neven; Stephane Blanche; Alain Fischer; Marina Cavazzana-Calvo; Paul Landais
Journal:  J Allergy Clin Immunol       Date:  2010-07-31       Impact factor: 10.793

3.  Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia.

Authors:  Marina Cavazzana-Calvo; Emmanuel Payen; Olivier Negre; Gary Wang; Kathleen Hehir; Floriane Fusil; Julian Down; Maria Denaro; Troy Brady; Karen Westerman; Resy Cavallesco; Beatrix Gillet-Legrand; Laure Caccavelli; Riccardo Sgarra; Leila Maouche-Chrétien; Françoise Bernaudin; Robert Girot; Ronald Dorazio; Geert-Jan Mulder; Axel Polack; Arthur Bank; Jean Soulier; Jérôme Larghero; Nabil Kabbara; Bruno Dalle; Bernard Gourmel; Gérard Socie; Stany Chrétien; Nathalie Cartier; Patrick Aubourg; Alain Fischer; Kenneth Cornetta; Frédéric Galacteros; Yves Beuzard; Eliane Gluckman; Frederick Bushman; Salima Hacein-Bey-Abina; Philippe Leboulch
Journal:  Nature       Date:  2010-09-16       Impact factor: 49.962

4.  Long-term survival and transplantation of haemopoietic stem cells for immunodeficiencies: report of the European experience 1968-99.

Authors:  Corinne Antoine; Susanna Müller; Andrew Cant; Marina Cavazzana-Calvo; Paul Veys; Jaak Vossen; Anders Fasth; Carsten Heilmann; Nicolas Wulffraat; Reinhard Seger; Stéphane Blanche; Wilhelm Friedrich; Mario Abinun; Graham Davies; Robert Bredius; Ansgar Schulz; Paul Landais; Alain Fischer
Journal:  Lancet       Date:  2003-02-15       Impact factor: 79.321

5.  Efficacy of gene therapy for X-linked severe combined immunodeficiency.

Authors:  Salima Hacein-Bey-Abina; Julia Hauer; Annick Lim; Capucine Picard; Gary P Wang; Charles C Berry; Chantal Martinache; Frédéric Rieux-Laucat; Sylvain Latour; Bernd H Belohradsky; Lily Leiva; Ricardo Sorensen; Marianne Debré; Jean Laurent Casanova; Stephane Blanche; Anne Durandy; Frederic D Bushman; Alain Fischer; Marina Cavazzana-Calvo
Journal:  N Engl J Med       Date:  2010-07-22       Impact factor: 91.245

6.  Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1.

Authors:  Salima Hacein-Bey-Abina; Alexandrine Garrigue; Gary P Wang; Jean Soulier; Annick Lim; Estelle Morillon; Emmanuelle Clappier; Laure Caccavelli; Eric Delabesse; Kheira Beldjord; Vahid Asnafi; Elizabeth MacIntyre; Liliane Dal Cortivo; Isabelle Radford; Nicole Brousse; François Sigaux; Despina Moshous; Julia Hauer; Arndt Borkhardt; Bernd H Belohradsky; Uwe Wintergerst; Maria C Velez; Lily Leiva; Ricardo Sorensen; Nicolas Wulffraat; Stéphane Blanche; Frederic D Bushman; Alain Fischer; Marina Cavazzana-Calvo
Journal:  J Clin Invest       Date:  2008-09       Impact factor: 14.808

7.  The genotoxic potential of retroviral vectors is strongly modulated by vector design and integration site selection in a mouse model of HSC gene therapy.

Authors:  Eugenio Montini; Daniela Cesana; Manfred Schmidt; Francesca Sanvito; Cynthia C Bartholomae; Marco Ranzani; Fabrizio Benedicenti; Lucia Sergi Sergi; Alessandro Ambrosi; Maurilio Ponzoni; Claudio Doglioni; Clelia Di Serio; Christof von Kalle; Luigi Naldini
Journal:  J Clin Invest       Date:  2009-03-23       Impact factor: 14.808

8.  Long-term persistence of a polyclonal T cell repertoire after gene therapy for X-linked severe combined immunodeficiency.

Authors:  H Bobby Gaspar; Samantha Cooray; Kimberly C Gilmour; Kathryn L Parsley; Stuart Adams; Steven J Howe; Abdulaziz Al Ghonaium; Jinhua Bayford; Lucinda Brown; E Graham Davies; Christine Kinnon; Adrian J Thrasher
Journal:  Sci Transl Med       Date:  2011-08-24       Impact factor: 17.956

9.  Self-inactivating gammaretroviral vectors for gene therapy of X-linked severe combined immunodeficiency.

Authors:  Susannah I Thornhill; Axel Schambach; Steven J Howe; Meera Ulaganathan; Elke Grassman; David Williams; Bernhard Schiedlmeier; Neil J Sebire; H Bobby Gaspar; Christine Kinnon; Christopher Baum; Adrian J Thrasher
Journal:  Mol Ther       Date:  2008-01-08       Impact factor: 11.454

10.  Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCID-X1 patients.

Authors:  Steven J Howe; Marc R Mansour; Kerstin Schwarzwaelder; Cynthia Bartholomae; Michael Hubank; Helena Kempski; Martijn H Brugman; Karin Pike-Overzet; Stephen J Chatters; Dick de Ridder; Kimberly C Gilmour; Stuart Adams; Susannah I Thornhill; Kathryn L Parsley; Frank J T Staal; Rosemary E Gale; David C Linch; Jinhua Bayford; Lucie Brown; Michelle Quaye; Christine Kinnon; Philip Ancliff; David K Webb; Manfred Schmidt; Christof von Kalle; H Bobby Gaspar; Adrian J Thrasher
Journal:  J Clin Invest       Date:  2008-09       Impact factor: 14.808
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  3 in total

1.  Genomic discovery of potent chromatin insulators for human gene therapy.

Authors:  Mingdong Liu; Matthew T Maurano; Hao Wang; Heyuan Qi; Chao-Zhong Song; Patrick A Navas; David W Emery; John A Stamatoyannopoulos; George Stamatoyannopoulos
Journal:  Nat Biotechnol       Date:  2015-01-12       Impact factor: 54.908

2.  Gene correction of HAX1 reversed Kostmann disease phenotype in patient-specific induced pluripotent stem cells.

Authors:  Erik Pittermann; Nico Lachmann; Glenn MacLean; Stephan Emmrich; Mania Ackermann; Gudrun Göhring; Brigitte Schlegelberger; Karl Welte; Axel Schambach; Dirk Heckl; Stuart H Orkin; Tobias Cantz; Jan-Henning Klusmann
Journal:  Blood Adv       Date:  2017-06-02

3.  Development of safer gene delivery systems to minimize the risk of insertional mutagenesis-related malignancies: a critical issue for the field of gene therapy.

Authors:  Gaetano Romano
Journal:  ISRN Oncol       Date:  2012-11-22
  3 in total

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