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Literature DB >> 20436489

Lentiviral vector gene therapy: effective and safe?

Derek A Persons.

Abstract

Mesh：

Year: 2010 PMID： 20436489 PMCID： PMC2890097 DOI： 10.1038/mt.2010.70

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

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17 in total

1. Gammaretrovirus-mediated correction of SCID-X1 is associated with skewed vector integration site distribution in vivo.

Authors: Kerstin Schwarzwaelder; Steven J Howe; Manfred Schmidt; Martijn H Brugman; Annette Deichmann; Hanno Glimm; Sonja Schmidt; Claudia Prinz; Manuela Wissler; Douglas J S King; Fang Zhang; Kathryn L Parsley; Kimberly C Gilmour; Joanna Sinclair; Jinhua Bayford; Rachel Peraj; Karin Pike-Overzet; Frank J T Staal; Dick de Ridder; Christine Kinnon; Ulrich Abel; Gerard Wagemaker; H Bobby Gaspar; Adrian J Thrasher; Christof von Kalle
Journal: J Clin Invest Date: 2007-08 Impact factor: 14.808

2. Vector integration is nonrandom and clustered and influences the fate of lymphopoiesis in SCID-X1 gene therapy.

Authors: Annette Deichmann; Salima Hacein-Bey-Abina; Manfred Schmidt; Alexandrine Garrigue; Martijn H Brugman; Jingqiong Hu; Hanno Glimm; Gabor Gyapay; Bernard Prum; Christopher C Fraser; Nicolas Fischer; Kerstin Schwarzwaelder; Maria-Luise Siegler; Dick de Ridder; Karin Pike-Overzet; Steven J Howe; Adrian J Thrasher; Gerard Wagemaker; Ulrich Abel; Frank J T Staal; Eric Delabesse; Jean-Luc Villeval; Bruce Aronow; Christophe Hue; Claudia Prinz; Manuela Wissler; Chuck Klanke; Jean Weissenbach; Ian Alexander; Alain Fischer; Christof von Kalle; Marina Cavazzana-Calvo
Journal: J Clin Invest Date: 2007-08 Impact factor: 14.808

3. Overcoming promoter competition in packaging cells improves production of self-inactivating retroviral vectors.

Authors: A Schambach; D Mueller; M Galla; M M A Verstegen; G Wagemaker; R Loew; C Baum; J Bohne
Journal: Gene Ther Date: 2006-06-08 Impact factor: 5.250

4. Physiological promoters reduce the genotoxic risk of integrating gene vectors.

Authors: Daniela Zychlinski; Axel Schambach; Ute Modlich; Tobias Maetzig; Johann Meyer; Elke Grassman; Anjali Mishra; Christopher Baum
Journal: Mol Ther Date: 2008-03-04 Impact factor: 11.454

5. 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

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

7. 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

8. Hematopoietic stem cell gene transfer in a tumor-prone mouse model uncovers low genotoxicity of lentiviral vector integration.

Authors: Eugenio Montini; Daniela Cesana; Manfred Schmidt; Francesca Sanvito; Maurilio Ponzoni; Cynthia Bartholomae; Lucia Sergi Sergi; Fabrizio Benedicenti; Alessandro Ambrosi; Clelia Di Serio; Claudio Doglioni; Christof von Kalle; Luigi Naldini
Journal: Nat Biotechnol Date: 2006-05-28 Impact factor: 54.908

9. 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

10. Identification and characterization of cell type-specific and ubiquitous chromatin regulatory structures in the human genome.

Authors: Hualin Xi; Hennady P Shulha; Jane M Lin; Teresa R Vales; Yutao Fu; David M Bodine; Ronald D G McKay; Josh G Chenoweth; Paul J Tesar; Terrence S Furey; Bing Ren; Zhiping Weng; Gregory E Crawford
Journal: PLoS Genet Date: 2007-07-02 Impact factor: 5.917

24 in total

1. Assessing the risk of T-cell malignancies in mouse models of SCID-X1.

Authors: Brian Sorrentino
Journal: Mol Ther Date: 2010-05 Impact factor: 11.454

2. Genetic engineering of murine CD8+ and CD4+ T cells for preclinical adoptive immunotherapy studies.

Authors: Sid P Kerkar; Luis Sanchez-Perez; Shicheng Yang; Zachary A Borman; Pawel Muranski; Yun Ji; Dhanalakshmi Chinnasamy; Andrew D M Kaiser; Christian S Hinrichs; Christopher A Klebanoff; Christopher D Scott; Luca Gattinoni; Richard A Morgan; Steven A Rosenberg; Nicholas P Restifo
Journal: J Immunother Date: 2011-05 Impact factor: 4.456

3. Multiplex CRISPR/Cas9-based genome engineering from a single lentiviral vector.

Authors: Ami M Kabadi; David G Ousterout; Isaac B Hilton; Charles A Gersbach
Journal: Nucleic Acids Res Date: 2014-08-13 Impact factor: 16.971

Review 4. Recent advances in RNAi-based strategies for therapy and prevention of HIV-1/AIDS.

Authors: Manjunath N Swamy; Haoquan Wu; Premlata Shankar
Journal: Adv Drug Deliv Rev Date: 2016-03-21 Impact factor: 15.470

Review 5. Stem cell gene therapy: the risks of insertional mutagenesis and approaches to minimize genotoxicity.

Authors: Chuanfeng Wu; Cynthia E Dunbar
Journal: Front Med Date: 2011-12-27 Impact factor: 4.592

6. Therapeutic levels of fetal hemoglobin in erythroid progeny of β-thalassemic CD34+ cells after lentiviral vector-mediated gene transfer.

Authors: Andrew Wilber; Phillip W Hargrove; Yoon-Sang Kim; Janice M Riberdy; Vijay G Sankaran; Eleni Papanikolaou; Maria Georgomanoli; Nicholas P Anagnou; Stuart H Orkin; Arthur W Nienhuis; Derek A Persons
Journal: Blood Date: 2010-12-14 Impact factor: 22.113

7. Transplantation of mouse HSCs genetically modified to express a CD4-restricted TCR results in long-term immunity that destroys tumors and initiates spontaneous autoimmunity.

Authors: Sung P Ha; Nicholas D Klemen; Garrett H Kinnebrew; Andrew G Brandmaier; Jon Marsh; Giao Hangoc; Douglas C Palmer; Nicholas P Restifo; Kenneth Cornetta; Hal E Broxmeyer; Christopher E Touloukian
Journal: J Clin Invest Date: 2010-11-15 Impact factor: 14.808