Literature DB >> 19179320

Gene therapy fulfilling its promise.

Donald B Kohn, Fabio Candotti.   

Abstract

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Year:  2009        PMID： 19179320     DOI： 10.1056/NEJMe0809614

Source DB:  PubMed          Journal:  N Engl J Med        ISSN： 0028-4793            Impact factor:   91.245


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  22 in total

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2.  Live and let die: a new suicide gene therapy moves to the clinic.

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Journal:  Mol Ther       Date:  2012-01       Impact factor: 11.454

Review 3.  The use of cell-delivered gene therapy for the treatment of HIV/AIDS.

Authors:  Geoff P Symonds; Helen A Johnstone; Michelle L Millington; Maureen P Boyd; Bryan P Burke; Louis R Breton
Journal:  Immunol Res       Date:  2010-12       Impact factor: 2.829

4.  Stable transgene expression in primitive human CD34+ hematopoietic stem/progenitor cells, using the Sleeping Beauty transposon system.

Authors:  Teiko Sumiyoshi; Nathalia G Holt; Roger P Hollis; Shundi Ge; Paula M Cannon; Gay M Crooks; Donald B Kohn
Journal:  Hum Gene Ther       Date:  2009-12       Impact factor: 5.695

Review 5.  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

6.  Genome editing of the germline: broadening the discussion.

Authors:  Matthew H Porteus; Christina T Dann
Journal:  Mol Ther       Date:  2015-06       Impact factor: 11.454

7.  Genomic discovery of potent chromatin insulators for human gene therapy.

Authors:  Mingdong Liu; Matthew T Maurano; Hao Wang; Heyuan Qi; Chao-Zhong Song; Patrick A Navas; David W Emery; John A Stamatoyannopoulos; George Stamatoyannopoulos
Journal:  Nat Biotechnol       Date:  2015-01-12       Impact factor: 54.908

8.  Correct mRNA processing at a mutant TT splice donor in FANCC ameliorates the clinical phenotype in patients and is enhanced by delivery of suppressor U1 snRNAs.

Authors:  Linda Hartmann; Kornelia Neveling; Stephanie Borkens; Hildegard Schneider; Marcel Freund; Elke Grassman; Stephan Theiss; Angela Wawer; Stefan Burdach; Arleen D Auerbach; Detlev Schindler; Helmut Hanenberg; Heiner Schaal
Journal:  Am J Hum Genet       Date:  2010-10-08       Impact factor: 11.025

9.  Dynamics of HSPC repopulation in nonhuman primates revealed by a decade-long clonal-tracking study.

Authors:  Sanggu Kim; Namshin Kim; Angela P Presson; Mark E Metzger; Aylin C Bonifacino; Mary Sehl; Samson A Chow; Gay M Crooks; Cynthia E Dunbar; Dong Sung An; Robert E Donahue; Irvin S Y Chen
Journal:  Cell Stem Cell       Date:  2014-04-03       Impact factor: 24.633

10.  Metabolic correction of congenital erythropoietic porphyria with iPSCs free of reprogramming factors.

Authors:  Aurélie Bedel; Miguel Taillepierre; Véronique Guyonnet-Duperat; Eric Lippert; Pierre Dubus; Sandrine Dabernat; Thibaud Mautuit; Bruno Cardinaud; Catherine Pain; Benoît Rousseau; Magalie Lalanne; Cécile Ged; Yann Duchartre; Emmanuel Richard; Hubert de Verneuil; François Moreau-Gaudry
Journal:  Am J Hum Genet       Date:  2012-07-13       Impact factor: 11.025
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