Warning: Undefined array key "mm" in /www/wwwroot/www.ai-bt.com/si.php on line 10 Deprecated: trim(): Passing null to parameter #1 ($string) of type string is deprecated in /www/wwwroot/www.ai-bt.com/si.php on line 10 Challenges in vector and trial design using retroviral vectors for long-term gene correction in hematopoietic stem cell gene therapy.

Literature DB >> 22652996

Challenges in vector and trial design using retroviral vectors for long-term gene correction in hematopoietic stem cell gene therapy.

Jacqueline Corrigan-Curay¹, Odile Cohen-Haguenauer, Marina O'Reilly, Susan R Ross, Hung Fan, Naomi Rosenberg, Nikunj Somia, Nancy King, Theodore Friedmann, Cynthia Dunbar, Alessandro Aiuti, Luigi Naldini, Christopher Baum, Christof von Kalle, Hans-Peter Kiem, Eugenio Montini, Frederic Bushman, Brian P Sorrentino, Manuel Carrondo, Harry Malech, Gösta Gahrton, Robyn Shapiro, Linda Wolff, Eugene Rosenthal, Robert Jambou, John Zaia, Donald B Kohn.

Abstract

Entities: Chemical

Mesh：

Year: 2012 PMID： 22652996 PMCID： PMC3369284 DOI： 10.1038/mt.2012.93

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

× No keyword cloud information.

58 in total

Review 1. Defining and describing benefit appropriately in clinical trials.

Authors: N M King
Journal: J Law Med Ethics Date: 2000 Impact factor: 1.718

Review 2. Challenges with advanced therapy medicinal products and how to meet them.

Authors: Christian K Schneider; Paula Salmikangas; Bernd Jilma; Bruno Flamion; Lyubina Racheva Todorova; Anna Paphitou; Ivana Haunerova; Toivo Maimets; Jean-Hugues Trouvin; Egbert Flory; Asterios Tsiftsoglou; Balázs Sarkadi; Kolbeinn Gudmundsson; Maura O'Donovan; Giovanni Migliaccio; Jānis Ancāns; Romaldas Maciulaitis; Jean-Louis Robert; Anthony Samuel; Johannes H Ovelgönne; Marit Hystad; Andrzej Mariusz Fal; Beatriz Silva Lima; Anca Stela Moraru; Peter Turcáni; Robert Zorec; Sol Ruiz; Lennart Akerblom; Gopalan Narayanan; Alastair Kent; Fabrizia Bignami; J George Dickson; Dietger Niederwieser; María-Angeles Figuerola-Santos; Ilona G Reischl; Claire Beuneu; Rosen Georgiev; Maria Vassiliou; Alena Pychova; Mette Clausen; Taina Methuen; Sophie Lucas; Martina Schüssler-Lenz; Vasilios Kokkas; Zsuzsanna Buzás; Niall MacAleenan; Maria Cristina Galli; Aija Linē; Jolanta Gulbinovic; Guy Berchem; Mariusz Fraczek; Margarida Menezes-Ferreira; Nela Vilceanu; Mikulás Hrubisko; Petra Marinko; Marcos Timón; Wing Cheng; George Andrew Crosbie; Nick Meade; Michelino Lipucci di Paola; Thierry VandenDriessche; Per Ljungman; Lucia D'Apote; Olga Oliver-Diaz; Isabel Büttel; Patrick Celis
Journal: Nat Rev Drug Discov Date: 2010-03 Impact factor: 84.694

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. Identification of hematopoietic stem cell-specific miRNAs enables gene therapy of globoid cell leukodystrophy.

Authors: Bernhard Gentner; Ilaria Visigalli; Hidefumi Hiramatsu; Eric Lechman; Silvia Ungari; Alice Giustacchini; Giulia Schira; Mario Amendola; Angelo Quattrini; Sabata Martino; Aldo Orlacchio; John E Dick; Alessandra Biffi; Luigi Naldini
Journal: Sci Transl Med Date: 2010-11-17 Impact factor: 17.956

5. Gene therapy for immunodeficiency due to adenosine deaminase deficiency.

Authors: 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 Eibl; Memet Aker; Shimon Slavin; Hamoud Al-Mousa; Abdulaziz Al Ghonaium; Alina Ferster; Andrea Duppenthaler; Luigi Notarangelo; Uwe Wintergerst; Rebecca H Buckley; Marco Bregni; Sarah Marktel; Maria Grazia Valsecchi; Paolo Rossi; Fabio Ciceri; Roberto Miniero; Claudio Bordignon; Maria-Grazia Roncarolo
Journal: N Engl J Med Date: 2009-01-29 Impact factor: 91.245

6. 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

Review 7. Roles of HMGA proteins in cancer.

Authors: Alfredo Fusco; Monica Fedele
Journal: Nat Rev Cancer Date: 2007-12 Impact factor: 60.716

8. Evidence that hematopoiesis may be a stochastic process in vivo.

Authors: J L Abkowitz; S N Catlin; P Guttorp
Journal: Nat Med Date: 1996-02 Impact factor: 53.440

9. Methods for integration site distribution analyses in animal cell genomes.

Authors: Angela Ciuffi; Keshet Ronen; Troy Brady; Nirav Malani; Gary Wang; Charles C Berry; Frederic D Bushman
Journal: Methods Date: 2008-11-25 Impact factor: 3.608

10. Molecular evolution of a novel hyperactive Sleeping Beauty transposase enables robust stable gene transfer in vertebrates.

Authors: Lajos Mátés; Marinee K L Chuah; Eyayu Belay; Boris Jerchow; Namitha Manoj; Abel Acosta-Sanchez; Dawid P Grzela; Andrea Schmitt; Katja Becker; Janka Matrai; Ling Ma; Ermira Samara-Kuko; Conny Gysemans; Diana Pryputniewicz; Csaba Miskey; Bradley Fletcher; Thierry VandenDriessche; Zoltán Ivics; Zsuzsanna Izsvák
Journal: Nat Genet Date: 2009-05-03 Impact factor: 38.330

15 in total

Review 1. Development of gene therapy for blood disorders: an update.

Authors: Arthur W Nienhuis
Journal: Blood Date: 2013-07-10 Impact factor: 22.113

2. Genome editing technologies: defining a path to clinic.

Authors: Jacqueline Corrigan-Curay; Marina O'Reilly; Donald B Kohn; Paula M Cannon; Gang Bao; Frederic D Bushman; Dana Carroll; Toni Cathomen; J Keith Joung; David Roth; Michel Sadelain; Andrew M Scharenberg; Christof von Kalle; Feng Zhang; Robert Jambou; Eugene Rosenthal; Morad Hassani; Aparna Singh; Matthew H Porteus
Journal: Mol Ther Date: 2015-05 Impact factor: 11.454

Review 3. Center for fetal monkey gene transfer for heart, lung, and blood diseases: an NHLBI resource for the gene therapy community.

Authors: Alice F Tarantal; Sonia I Skarlatos
Journal: Hum Gene Ther Date: 2012-10-19 Impact factor: 5.695

4. Gene Therapy for Respiratory Diseases: Progress and a Changing Context.

Authors: Eric W F W Alton; A Christopher Boyd; Jane C Davies; Deborah R Gill; Uta Griesenbach; Tracy E Harman; Stephen Hyde; Gerry McLachlan
Journal: Hum Gene Ther Date: 2020-09 Impact factor: 5.695

Review 5. Evaluating risks of insertional mutagenesis by DNA transposons in gene therapy.

Authors: Perry B Hackett; David A Largaespada; Kirsten C Switzer; Laurence J N Cooper
Journal: Transl Res Date: 2013-01-10 Impact factor: 7.012

6. Long-term in vivo monitoring of mouse and human hematopoietic stem cell engraftment with a human positron emission tomography reporter gene.

Authors: Melissa N McCracken; Eric H Gschweng; Evan Nair-Gill; Jami McLaughlin; Aaron R Cooper; Mireille Riedinger; Donghui Cheng; Christopher Nosala; Donald B Kohn; Owen N Witte
Journal: Proc Natl Acad Sci U S A Date: 2013-01-14 Impact factor: 11.205

7. No impact of lentiviral transduction on hematopoietic stem/progenitor cell telomere length or gene expression in the rhesus macaque model.

Authors: Stephanie E Sellers; Bogdan Dumitriu; Mary J Morgan; William M Hughes; Colin O Wu; Nalini Raghavarchari; Yanqin Yang; Naoya Uchida; John F Tisdale; Dong S An; Irvin S Chen; Peiman Hematti; Robert E Donahue; Andre Larochelle; Neal S Young; Rodrigo T Calado; Cynthia E Dunbar
Journal: Mol Ther Date: 2013-07-18 Impact factor: 11.454

8. 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

Review 9. Lentiviral vectors for the treatment of primary immunodeficiencies.

Authors: Giada Farinelli; Valentina Capo; Samantha Scaramuzza; Alessandro Aiuti
Journal: J Inherit Metab Dis Date: 2014-03-12 Impact factor: 4.982

10. Preclinical demonstration of lentiviral vector-mediated correction of immunological and metabolic abnormalities in models of adenosine deaminase deficiency.

Authors: 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
Journal: Mol Ther Date: 2013-11-20 Impact factor: 11.454