Literature DB >> 25561034

Hitting the target without pulling the trigger.

Thierry VandenDriessche1, Marinee K Chuah1.   

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Year:  2015        PMID: 25561034      PMCID: PMC4426814          DOI: 10.1038/mt.2014.230

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


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  22 in total

Review 1.  Safe harbours for the integration of new DNA in the human genome.

Authors:  Michel Sadelain; Eirini P Papapetrou; Frederic D Bushman
Journal:  Nat Rev Cancer       Date:  2011-12-01       Impact factor: 60.716

2.  Gene targeting in vivo by adeno-associated virus vectors.

Authors:  Daniel G Miller; Pei-Rong Wang; Lisa M Petek; Roli K Hirata; Mark S Sands; David W Russell
Journal:  Nat Biotechnol       Date:  2006-07-30       Impact factor: 54.908

3.  Assessing the potential for AAV vector genotoxicity in a murine model.

Authors:  Hojun Li; Nirav Malani; Shari R Hamilton; Alexander Schlachterman; Giulio Bussadori; Shyrie E Edmonson; Rachel Shah; Valder R Arruda; Federico Mingozzi; J Fraser Wright; Frederic D Bushman; Katherine A High
Journal:  Blood       Date:  2010-11-24       Impact factor: 22.113

4.  Site-specific integration and tailoring of cassette design for sustainable gene transfer.

Authors:  Angelo Lombardo; Daniela Cesana; Pietro Genovese; Bruno Di Stefano; Elena Provasi; Daniele F Colombo; Margherita Neri; Zulma Magnani; Alessio Cantore; Pietro Lo Riso; Martina Damo; Oscar M Pello; Michael C Holmes; Philip D Gregory; Angela Gritti; Vania Broccoli; Chiara Bonini; Luigi Naldini
Journal:  Nat Methods       Date:  2011-08-21       Impact factor: 28.547

5.  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

6.  AAV vector integration sites in mouse hepatocellular carcinoma.

Authors:  Anthony Donsante; Daniel G Miller; Yi Li; Carole Vogler; Elizabeth M Brunt; David W Russell; Mark S Sands
Journal:  Science       Date:  2007-07-27       Impact factor: 47.728

7.  CD8(+) T-cell responses to adeno-associated virus capsid in humans.

Authors:  Federico Mingozzi; Marcela V Maus; Daniel J Hui; Denise E Sabatino; Samuel L Murphy; John E J Rasko; Margaret V Ragni; Catherine S Manno; Jurg Sommer; Haiyan Jiang; Glenn F Pierce; Hildegund C J Ertl; Katherine A High
Journal:  Nat Med       Date:  2007-03-18       Impact factor: 53.440

8.  Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response.

Authors:  Catherine S Manno; Glenn F Pierce; Valder R Arruda; Bertil Glader; Margaret Ragni; John J Rasko; John Rasko; Margareth C Ozelo; Keith Hoots; Philip Blatt; Barbara Konkle; Michael Dake; Robin Kaye; Mahmood Razavi; Albert Zajko; James Zehnder; Pradip K Rustagi; Hiroyuki Nakai; Amy Chew; Debra Leonard; J Fraser Wright; Ruth R Lessard; Jürg M Sommer; Michael Tigges; Denise Sabatino; Alvin Luk; Haiyan Jiang; Federico Mingozzi; Linda Couto; Hildegund C Ertl; Katherine A High; Mark A Kay
Journal:  Nat Med       Date:  2006-02-12       Impact factor: 53.440

9.  In vivo genome editing restores haemostasis in a mouse model of haemophilia.

Authors:  Hojun Li; Virginia Haurigot; Yannick Doyon; Tianjian Li; Sunnie Y Wong; Anand S Bhagwat; Nirav Malani; Xavier M Anguela; Rajiv Sharma; Lacramiora Ivanciu; Samuel L Murphy; Jonathan D Finn; Fayaz R Khazi; Shangzhen Zhou; David E Paschon; Edward J Rebar; Frederic D Bushman; Philip D Gregory; Michael C Holmes; Katherine A High
Journal:  Nature       Date:  2011-06-26       Impact factor: 49.962

10.  Hepatocyte-targeted expression by integrase-defective lentiviral vectors induces antigen-specific tolerance in mice with low genotoxic risk.

Authors:  Janka Mátrai; Alessio Cantore; Cynthia C Bartholomae; Andrea Annoni; Wei Wang; Abel Acosta-Sanchez; Ermira Samara-Kuko; Liesbeth De Waele; Ling Ma; Pietro Genovese; Martina Damo; Anne Arens; Kevin Goudy; Timothy C Nichols; Christof von Kalle; Marinee K L Chuah; Maria Grazia Roncarolo; Manfred Schmidt; Thierry Vandendriessche; Luigi Naldini
Journal:  Hepatology       Date:  2011-05       Impact factor: 17.425

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  1 in total

1.  Development of a Cell-Based Gene Therapy Approach to Selectively Turn Off Bone Formation.

Authors:  Pedro Alvarez-Urena; Banghe Zhu; Gabrielle Henslee; Corinne Sonnet; Eleanor Davis; Eva Sevick-Muraca; Alan Davis; Elizabeth Olmsted-Davis
Journal:  J Cell Biochem       Date:  2017-07-31       Impact factor: 4.429

  1 in total

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