Literature DB >> 24870243

Gene therapy: Repair and replace.

Alain Fischer1.   

Abstract

Mesh：

Year:  2014        PMID： 24870243     DOI： 10.1038/nature13344

Source DB:  PubMed          Journal:  Nature        ISSN： 0028-0836            Impact factor:   49.962


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  11 in total

Review 1.  A guide to genome engineering with programmable nucleases.

Authors:  Hyongbum Kim; Jin-Soo Kim
Journal:  Nat Rev Genet       Date:  2014-04-02       Impact factor: 53.242

2.  Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease.

Authors:  M Cavazzana-Calvo; S Hacein-Bey; G de Saint Basile; F Gross; E Yvon; P Nusbaum; F Selz; C Hue; S Certain; J L Casanova; P Bousso; F L Deist; A Fischer
Journal:  Science       Date:  2000-04-28       Impact factor: 47.728

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.  LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1.

Authors:  S Hacein-Bey-Abina; C Von Kalle; M Schmidt; M P McCormack; N Wulffraat; P Leboulch; A Lim; C S Osborne; R Pawliuk; E Morillon; R Sorensen; A Forster; P Fraser; J I Cohen; G de Saint Basile; I Alexander; U Wintergerst; T Frebourg; A Aurias; D Stoppa-Lyonnet; S Romana; I Radford-Weiss; F Gross; F Valensi; E Delabesse; E Macintyre; F Sigaux; J Soulier; L E Leiva; M Wissler; C Prinz; T H Rabbitts; F Le Deist; A Fischer; M Cavazzana-Calvo
Journal:  Science       Date:  2003-10-17       Impact factor: 47.728

5.  Gene therapy for human genetic disease?

Authors:  T Friedmann; R Roblin
Journal:  Science       Date:  1972-03-03       Impact factor: 47.728

6.  Hematopoietic stem cell gene therapy with a lentiviral vector in X-linked adrenoleukodystrophy.

Authors:  Nathalie Cartier; Salima Hacein-Bey-Abina; Cynthia C Bartholomae; Gabor Veres; Manfred Schmidt; Ina Kutschera; Michel Vidaud; Ulrich Abel; Liliane Dal-Cortivo; Laure Caccavelli; Nizar Mahlaoui; Véronique Kiermer; Denice Mittelstaedt; Céline Bellesme; Najiba Lahlou; François Lefrère; Stéphane Blanche; Muriel Audit; Emmanuel Payen; Philippe Leboulch; Bruno l'Homme; Pierre Bougnères; Christof Von Kalle; Alain Fischer; Marina Cavazzana-Calvo; Patrick Aubourg
Journal:  Science       Date:  2009-11-06       Impact factor: 47.728

7.  Lentiviral hematopoietic stem cell gene therapy in patients with Wiskott-Aldrich syndrome.

Authors:  Alessandro Aiuti; Luca Biasco; Samantha Scaramuzza; Francesca Ferrua; Maria Pia Cicalese; Cristina Baricordi; Francesca Dionisio; Andrea Calabria; Stefania Giannelli; Maria Carmina Castiello; Marita Bosticardo; Costanza Evangelio; Andrea Assanelli; Miriam Casiraghi; Sara Di Nunzio; Luciano Callegaro; Claudia Benati; Paolo Rizzardi; Danilo Pellin; Clelia Di Serio; Manfred Schmidt; Christof Von Kalle; Jason Gardner; Nalini Mehta; Victor Neduva; David J Dow; Anne Galy; Roberto Miniero; Andrea Finocchi; Ayse Metin; Pinaki P Banerjee; Jordan S Orange; Stefania Galimberti; Maria Grazia Valsecchi; Alessandra Biffi; Eugenio Montini; Anna Villa; Fabio Ciceri; Maria Grazia Roncarolo; Luigi Naldini
Journal:  Science       Date:  2013-07-11       Impact factor: 47.728

8.  Correction of ADA-SCID by stem cell gene therapy combined with nonmyeloablative conditioning.

Authors:  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
Journal:  Science       Date:  2002-06-28       Impact factor: 47.728

9.  Lentiviral hematopoietic stem cell gene therapy benefits metachromatic leukodystrophy.

Authors:  Alessandra Biffi; Eugenio Montini; Laura Lorioli; Martina Cesani; Francesca Fumagalli; Tiziana Plati; Cristina Baldoli; Sabata Martino; Andrea Calabria; Sabrina Canale; Fabrizio Benedicenti; Giuliana Vallanti; Luca Biasco; Simone Leo; Nabil Kabbara; Gianluigi Zanetti; William B Rizzo; Nalini A L Mehta; Maria Pia Cicalese; Miriam Casiraghi; Jaap J Boelens; Ubaldo Del Carro; David J Dow; Manfred Schmidt; Andrea Assanelli; Victor Neduva; Clelia Di Serio; Elia Stupka; Jason Gardner; Christof von Kalle; Claudio Bordignon; Fabio Ciceri; Attilio Rovelli; Maria Grazia Roncarolo; Alessandro Aiuti; Maria Sessa; Luigi Naldini
Journal:  Science       Date:  2013-07-11       Impact factor: 47.728

10.  Targeted genome editing in human repopulating haematopoietic stem cells.

Authors:  Giulia Schiroli; Giulia Escobar; Pietro Genovese; Tiziano Di Tomaso; Claudia Firrito; Andrea Calabria; Davide Moi; Roberta Mazzieri; Chiara Bonini; Michael C Holmes; Philip D Gregory; Mirjam van der Burg; Bernhard Gentner; Eugenio Montini; Angelo Lombardo; Luigi Naldini
Journal:  Nature       Date:  2014-05-28       Impact factor: 49.962
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  2 in total

1.  Modified Genomic Sequencing PCR Using the MiSeq Platform to Identify Retroviral Integration Sites.

Authors:  Dustin T Rae; Casey P Collins; Jonah D Hocum; Diana L Browning; Grant D Trobridge
Journal:  Hum Gene Ther Methods       Date:  2015-10-20       Impact factor: 2.396

2.  BTK gene targeting by homologous recombination using a helper-dependent adenovirus/adeno-associated virus hybrid vector.

Authors:  H Yamamoto; M Ishimura; M Ochiai; H Takada; K Kusuhara; Y Nakatsu; T Tsuzuki; K Mitani; T Hara
Journal:  Gene Ther       Date:  2015-08-17       Impact factor: 5.250
  2 in total

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