Literature DB >> 24981438

Charting a clear path: the ASGCT Standardized Pathways Conference.

Hans-Peter Kiem1, Christopher Baum2, Frederic D Bushman3, Barry J Byrne4, Barrie J Carter5, Joy Cavagnaro6, Harry L Malech7, Jerry R Mendell8, Luigi M Naldini9, Brian P Sorrentino10, David A Williams11, Terence R Flotte12.   

Abstract

Mesh:

Year:  2014        PMID: 24981438      PMCID: PMC4088998          DOI: 10.1038/mt.2014.95

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


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  16 in total

1.  A self-inactivating lentiviral vector for SCID-X1 gene therapy that does not activate LMO2 expression in human T cells.

Authors:  Sheng Zhou; Disha Mody; Suk See DeRavin; Julia Hauer; Taihe Lu; Zhijun Ma; Salima Hacein-Bey Abina; John T Gray; Michael R Greene; Marina Cavazzana-Calvo; Harry L Malech; Brian P Sorrentino
Journal:  Blood       Date:  2010-05-10       Impact factor: 22.113

2.  Gene therapy of X-linked severe combined immunodeficiency by use of a pseudotyped gammaretroviral vector.

Authors:  H Bobby Gaspar; Kathryn L Parsley; Steven Howe; Doug King; Kimberly C Gilmour; Joanna Sinclair; Gaby Brouns; Manfred Schmidt; Christof Von Kalle; Torben Barington; Marianne A Jakobsen; Hans O Christensen; Abdulaziz Al Ghonaium; Harry N White; John L Smith; Roland J Levinsky; Robin R Ali; Christine Kinnon; Adrian J Thrasher
Journal:  Lancet       Date:  2004 Dec 18-31       Impact factor: 79.321

3.  CTF/NF1 transcription factors act as potent genetic insulators for integrating gene transfer vectors.

Authors:  A Gaussin; U Modlich; C Bauche; N J Niederländer; A Schambach; C Duros; A Artus; C Baum; O Cohen-Haguenauer; N Mermod
Journal:  Gene Ther       Date:  2011-05-12       Impact factor: 5.250

4.  Preclinical characterization of a recombinant adeno-associated virus type 1-pseudotyped vector demonstrates dose-dependent injection site inflammation and dissemination of vector genomes to distant sites.

Authors:  Terence R Flotte; Thomas J Conlon; Amy Poirier; Martha Campbell-Thompson; Barry J Byrne
Journal:  Hum Gene Ther       Date:  2007-03       Impact factor: 5.695

5.  Cell-culture assays reveal the importance of retroviral vector design for insertional genotoxicity.

Authors:  Ute Modlich; Jens Bohne; Manfred Schmidt; Christof von Kalle; Sabine Knöss; Axel Schambach; Christopher Baum
Journal:  Blood       Date:  2006-07-06       Impact factor: 22.113

6.  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

7.  An experimental system for the evaluation of retroviral vector design to diminish the risk for proto-oncogene activation.

Authors:  Byoung Y Ryu; Marguerite V Evans-Galea; John T Gray; David M Bodine; Derek A Persons; Arthur W Nienhuis
Journal:  Blood       Date:  2007-11-08       Impact factor: 22.113

8.  Genomic instability and myelodysplasia with monosomy 7 consequent to EVI1 activation after gene therapy for chronic granulomatous disease.

Authors:  Stefan Stein; Marion G Ott; Stephan Schultze-Strasser; Anna Jauch; Barbara Burwinkel; Andrea Kinner; Manfred Schmidt; Alwin Krämer; Joachim Schwäble; Hanno Glimm; Ulrike Koehl; Carolin Preiss; Claudia Ball; Hans Martin; Gudrun Göhring; Kerstin Schwarzwaelder; Wolf-Karsten Hofmann; Kadin Karakaya; Sandrine Tchatchou; Rongxi Yang; Petra Reinecke; Klaus Kühlcke; Brigitte Schlegelberger; Adrian J Thrasher; Dieter Hoelzer; Reinhard Seger; Christof von Kalle; Manuel Grez
Journal:  Nat Med       Date:  2010-01-24       Impact factor: 53.440

9.  Gene therapy for Wiskott-Aldrich syndrome--long-term efficacy and genotoxicity.

Authors:  Christian Jörg Braun; Kaan Boztug; Anna Paruzynski; Maximilian Witzel; Adrian Schwarzer; Michael Rothe; Ute Modlich; Rita Beier; Gudrun Göhring; Doris Steinemann; Raffaele Fronza; Claudia Regina Ball; Reinhard Haemmerle; Sonja Naundorf; Klaus Kühlcke; Martina Rose; Chris Fraser; Liesl Mathias; Rudolf Ferrari; Miguel R Abboud; Waleed Al-Herz; Irina Kondratenko; László Maródi; Hanno Glimm; Brigitte Schlegelberger; Axel Schambach; Michael Heinrich Albert; Manfred Schmidt; Christof von Kalle; Christoph Klein
Journal:  Sci Transl Med       Date:  2014-03-12       Impact factor: 17.956

10.  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

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  6 in total

1.  Rethinking the Regulatory Infrastructure for Human Gene Transfer Clinical Trials.

Authors:  Christopher L Jenkins; Mark L Bagarazzi; Hans-Peter Kiem; Arnold J Levine; Dale Ando; Carl H June; Tony Reid; David W Emery
Journal:  Mol Ther       Date:  2016-08       Impact factor: 11.454

2.  Foamy Virus Vector Carries a Strong Insulator in Its Long Terminal Repeat Which Reduces Its Genotoxic Potential.

Authors:  Michael Aaron Goodman; Paritha Arumugam; Devin Marie Pillis; Anastacia Loberg; Mohammed Nasimuzzaman; Danielle Lynn; Johannes Christiaan Maria van der Loo; Phillip Joseph Dexheimer; Mehdi Keddache; Thomas Roy Bauer; Dennis Durand Hickstein; David William Russell; Punam Malik
Journal:  J Virol       Date:  2017-12-14       Impact factor: 5.103

3.  Hurdles Associated with the Translational Use of Genetically Modified Cells.

Authors:  Sunil S Raikar; H Trent Spencer
Journal:  Curr Stem Cell Rep       Date:  2018-02-17

4.  Safe and Effective Gene Therapy for Murine Wiskott-Aldrich Syndrome Using an Insulated Lentiviral Vector.

Authors:  Swati Singh; Iram Khan; Socheath Khim; Brenda Seymour; Karen Sommer; Matthew Wielgosz; Zachary Norgaard; Hans-Peter Kiem; Jennifer Adair; Denny Liggitt; Arthur Nienhuis; David J Rawlings
Journal:  Mol Ther Methods Clin Dev       Date:  2016-12-18       Impact factor: 6.698

5.  Two-Plasmid Packaging System for Recombinant Adeno-Associated Virus.

Authors:  Qiushi Tang; Allison M Keeler; Songbo Zhang; Qin Su; Zhuoyao Lyu; Yangfan Cheng; Guangping Gao; Terence R Flotte
Journal:  Biores Open Access       Date:  2020-10-16

6.  Evaluating the Safety of Retroviral Vectors Based on Insertional Oncogene Activation and Blocked Differentiation in Cultured Thymocytes.

Authors:  Sheng Zhou; Soghra Fatima; Zhijun Ma; Yong-Dong Wang; Taihe Lu; Laura J Janke; Yang Du; Brian P Sorrentino
Journal:  Mol Ther       Date:  2016-03-09       Impact factor: 11.454

  6 in total

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