Successful reconstitution of immunity in ADA-SCID by stem cell gene therapy following cessation of PEG-ADA and use of mild preconditioning.
H. Bobby Gaspar,H. Bobby Gaspar,Emma Bjorkegren,Kate Parsley,Kate Parsley,Kimberly Gilmour,Kimberly Gilmour,Doug King,Joanna Sinclair,Fang Zhang,Aris Giannakopoulos,Stuart Adams,Lynette D. Fairbanks,Jane Gaspar,Lesley Henderson,Jin Hua Xu-Bayford,E. Graham Davies,Paul Veys,Christine Kinnon,Adrian J. Thrasher,Adrian J. Thrasher +20 more
TLDR
It is document successful reconstitution of immune function in a child with the adenosine deaminase (ADA)-deficient form of severe combined immunodeficiency (SCID) following hematopoietic stem cell (HSC) gene therapy.About:
This article is published in Molecular Therapy.The article was published on 2006-10-01 and is currently open access. It has received 230 citations till now. The article focuses on the topics: Genetic enhancement & Severe combined immunodeficiency.read more
Citations
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Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCID-X1 patients
Steven J. Howe,Marc R. Mansour,Kerstin Schwarzwaelder,Cynthia C. Bartholomae,Mike Hubank,Helena Kempski,Martijn H. Brugman,Karin Pike-Overzet,S Chatters,Dick de Ridder,Dick de Ridder,Kimberly 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 Webb,Manfred Schmidt,Christof von Kalle,H. Bobby Gaspar,Adrian J. Thrasher +27 more
TL;DR: The occurrence of clonal T cell acute lymphoblastic leukemia (T-ALL) promoted by insertional mutagenesis in a completed gene therapy trial of 10 SCID-X1 patients is described and a general toxicity of endogenous gammaretroviral enhancer elements is highlighted.
Journal ArticleDOI
Gene Therapy for Immunodeficiency Due to Adenosine Deaminase Deficiency
Alessandro Aiuti,Federica Cattaneo,Stefania Galimberti,Ulrike Benninghoff,Barbara Cassani,Luciano Callegaro,Samantha Scaramuzza,Grazia Andolfi,Massimiliano Mirolo,Immacolata Brigida,Antonella Tabucchi,Filippo Carlucci,Martha M. Eibl,Memet Aker,Shimon Slavin,Hamoud Al-Mousa,Abdulaziz Al Ghonaium,Alina Ferster,Andrea Duppenthaler,Luigi D. Notarangelo,Uwe Wintergerst,Rebecca H. Buckley,Marco Bregni,Sarah Marktel,Maria Grazia Valsecchi,Pier Luca Rossi,Fabio Ciceri,Roberto Miniero,Claudio Bordignon,Claudio Bordignon,Maria Grazia Roncarolo +30 more
TL;DR: Gene therapy, combined with reduced-intensity conditioning, is a safe and effective treatment for SCID in patients with ADA deficiency and effective protection against infections and improvement in physical development made a normal lifestyle possible.
Journal ArticleDOI
Gene therapy clinical trials worldwide to 2012 - an update
Samantha L. Ginn,Ian E. Alexander,Ian E. Alexander,Michael Edelstein,Mohammad R. Abedi,Jo Wixon +5 more
TL;DR: This database brings together global information on gene therapy clinical trials from official agency sources, published literature, conference presentations and posters kindly provided by individual investigators or trial sponsors.
Journal ArticleDOI
Genomic instability and myelodysplasia with monosomy 7 consequent to EVI1 activation after gene therapy for chronic granulomatous disease
Stefan Stein,Marion Ott,Stephan Schultze-Strasser,Anna Jauch,Barbara Burwinkel,Barbara Burwinkel,Andrea Kinner,Manfred Schmidt,Alwin Krämer,Joachim Schwäble,Hanno Glimm,Ulrike Koehl,Carolin Preiss,Claudia R. Ball,Hans Martin,Gudrun Göhring,Kerstin Schwarzwaelder,Wolf K. Hofmann,Kadin Karakaya,Sandrine Tchatchou,Sandrine Tchatchou,Rongxi Yang,Rongxi Yang,Petra Reinecke,Klaus Kühlcke,Brigitte Schlegelberger,Adrian J. Thrasher,Dieter Hoelzer,Reinhard Seger,Christof von Kalle,Manuel Grez +30 more
TL;DR: It is shown that forced overexpression of EVI1 in human cells disrupts normal centrosome duplication, linking EVI 1 activation to the development of genomic instability, monosomy 7 and clonal progression toward myelodysplasia.
Journal ArticleDOI
Gene therapy clinical trials worldwide to 2007--an update.
TL;DR: This database set up in 1997 to bring together global information on gene therapy clinical trials as comprehensively as possible is compiled and regularly updated.
References
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LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1.
Salima Hacein-Bey-Abina,C von Kalle,C von Kalle,Manfred Schmidt,Matthew P. McCormack,NM Wulffraat,Philippe Leboulch,Annick Lim,Cameron S. Osborne,R. Pawliuk,Estelle Morillon,R. Sorensen,A. Forster,Peter Fraser,Jeffrey I. Cohen,G de Saint Basile,Ian E. Alexander,Uwe Wintergerst,Thierry Frebourg,Alain Aurias,Dominique Stoppa-Lyonnet,Serge Romana,I. Radford-Weiss,Fabian Gross,Françoise Valensi,Eric Delabesse,Elizabeth Macintyre,F. Sigaux,Jean Soulier,L. E. Leiva,Manuela Wissler,Claudia Prinz,Terence H. Rabbitts,F. Le Deist,Alain Fischer,Marina Cavazzana-Calvo +35 more
TL;DR: Retrovirus vector insertion can trigger deregulated premalignant cell proliferation with unexpected frequency, most likely driven by retrovirus enhancer activity on the LMO2 gene promoter.
Journal ArticleDOI
Correction of ADA-SCID by Stem Cell Gene Therapy Combined with Nonmyeloablative Conditioning
Alessandro Aiuti,Shimon Slavin,Memet Aker,Francesca Ficara,Sara Deola,Alessandra Mortellaro,Shoshana Morecki,Grazia Andolfi,Antonella Tabucchi,Filippo Carlucci,Enrico Marinello,Federica Cattaneo,Sergio Vai,Paolo Servida,Roberto Miniero,Maria Grazia Roncarolo,Claudio Bordignon +16 more
TL;DR: Sustained engraftment of engineered HSCs with differentiation into multiple lineages resulted in increased lymphocyte counts, improved immune functions, and lower toxic metabolites, indicating the safety and efficacy of HSC gene therapy combined with nonmyeloablative conditioning for the treatment of SCID.
Journal ArticleDOI
Sustained Correction of X-Linked Severe Combined Immunodeficiency by ex Vivo Gene Therapy
Salima Hacein-Bey-Abina,Françoise Le Deist,Frédérique Carlier,Cécile Bouneaud,Christophe Hue,Jean-Pierre de Villartay,Adrian J. Thrasher,NM Wulffraat,Ricardo U. Sorensen,Sophie Dupuis-Girod,Alain Fischer,E. Graham Davies,Wietse Kuis,Lilly Leiva,Marina Cavazzana-Calvo +14 more
TL;DR: Ex vivo gene therapy with gamma(c) can safely correct the immune deficiency of patients with X-linked severe combined immunodeficiency and allow patients to have a normal life.
Journal ArticleDOI
Woodchuck Hepatitis Virus Posttranscriptional Regulatory Element Enhances Expression of Transgenes Delivered by Retroviral Vectors
TL;DR: It is demonstrated that the WPRE significantly improves the performance of retroviral vectors and emphasize that posttranscriptional regulation of gene expression should be taken into account in the design of gene delivery systems.
Journal ArticleDOI
Gene therapy in peripheral blood lymphocytes and bone marrow for ADA- immunodeficient patients
Claudio Bordignon,Luigi D. Notarangelo,Nadia Nobili,Giuliana Ferrari,Giulia Casorati,Paola Panina,Evelina Mazzolari,Daniela Maggioni,Claudia Rossi,Paolo Servida,Alberto G. Ugazio,Fulvio Mavilio +11 more
TL;DR: Results indicate successful gene transfer into long-lasting progenitor cells, producing a functional multilineage progeny.
Related Papers (5)
Correction of ADA-SCID by Stem Cell Gene Therapy Combined with Nonmyeloablative Conditioning
Correction of X-linked chronic granulomatous disease by gene therapy, augmented by insertional activation of MDS1-EVI1, PRDM16 or SETBP1.
Marion Ott,Manfred Schmidt,Kerstin Schwarzwaelder,Stefan Stein,Ulrich Siler,Ulrike Koehl,Hanno Glimm,K. Kühlcke,Andrea Schilz,Hana Kunkel,Sonja Naundorf,Andrea Brinkmann,Annette Deichmann,Marlene Fischer,Claudia R. Ball,Ingo H. Pilz,Cynthia E. Dunbar,Yang Du,Nancy A. Jenkins,Neal G. Copeland,Ursula Lüthi,Moustapha Hassan,Adrian J. Thrasher,Dieter Hoelzer,Christof von Kalle,Reinhard Seger,Manuel Grez +26 more
LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1.
Salima Hacein-Bey-Abina,C von Kalle,C von Kalle,Manfred Schmidt,Matthew P. McCormack,NM Wulffraat,Philippe Leboulch,Annick Lim,Cameron S. Osborne,R. Pawliuk,Estelle Morillon,R. Sorensen,A. Forster,Peter Fraser,Jeffrey I. Cohen,G de Saint Basile,Ian E. Alexander,Uwe Wintergerst,Thierry Frebourg,Alain Aurias,Dominique Stoppa-Lyonnet,Serge Romana,I. Radford-Weiss,Fabian Gross,Françoise Valensi,Eric Delabesse,Elizabeth Macintyre,F. Sigaux,Jean Soulier,L. E. Leiva,Manuela Wissler,Claudia Prinz,Terence H. Rabbitts,F. Le Deist,Alain Fischer,Marina Cavazzana-Calvo +35 more
T Lymphocyte-Directed Gene Therapy for ADA− SCID: Initial Trial Results After 4 Years
R. Michael Blaese,Kenneth W. Culver,A. Dusty Miller,Charles S. Carter,Thomas A. Fleisher,Mario Clerici,Gene M. Shearer,Lauren A. Chang,Yawen Chiang,Paul Tolstoshev,Jay J. Greenblatt,Steven A. Rosenberg,Harvey G. Klein,Melvin Berger,Craig A. Mullen,W. Jay Ramsey,Linda M. Muul,Richard A. Morgan,W. French Anderson +18 more