Literature DB >> 27281416

Gene therapy's out-of-body experience.

Christopher Thomas Scott, Laura DeFrancesco1.   

Abstract

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Year:  2016        PMID： 27281416     DOI： 10.1038/nbt.3592

Source DB:  PubMed          Journal:  Nat Biotechnol        ISSN： 1087-0156            Impact factor:   54.908


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  31 in total

1.  An erythroid enhancer of BCL11A subject to genetic variation determines fetal hemoglobin level.

Authors:  Daniel E Bauer; Sophia C Kamran; Samuel Lessard; Jian Xu; Yuko Fujiwara; Carrie Lin; Zhen Shao; Matthew C Canver; Elenoe C Smith; Luca Pinello; Peter J Sabo; Jeff Vierstra; Richard A Voit; Guo-Cheng Yuan; Matthew H Porteus; John A Stamatoyannopoulos; Guillaume Lettre; Stuart H Orkin
Journal:  Science       Date:  2013-10-11       Impact factor: 47.728

2.  Correction of the sickle cell disease mutation in human hematopoietic stem/progenitor cells.

Authors:  Megan D Hoban; Gregory J Cost; Matthew C Mendel; Zulema Romero; Michael L Kaufman; Alok V Joglekar; Michelle Ho; Dianne Lumaquin; David Gray; Georgia R Lill; Aaron R Cooper; Fabrizia Urbinati; Shantha Senadheera; Allen Zhu; Pei-Qi Liu; David E Paschon; Lei Zhang; Edward J Rebar; Andrew Wilber; Xiaoyan Wang; Philip D Gregory; Michael C Holmes; Andreas Reik; Roger P Hollis; Donald B Kohn
Journal:  Blood       Date:  2015-03-02       Impact factor: 22.113

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.  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

5.  Long-term control of HIV by CCR5 Delta32/Delta32 stem-cell transplantation.

Authors:  Gero Hütter; Daniel Nowak; Maximilian Mossner; Susanne Ganepola; Arne Müssig; Kristina Allers; Thomas Schneider; Jörg Hofmann; Claudia Kücherer; Olga Blau; Igor W Blau; Wolf K Hofmann; Eckhard Thiel
Journal:  N Engl J Med       Date:  2009-02-12       Impact factor: 91.245

6.  Gene editing of CCR5 in autologous CD4 T cells of persons infected with HIV.

Authors:  Pablo Tebas; David Stein; Winson W Tang; Ian Frank; Shelley Q Wang; Gary Lee; S Kaye Spratt; Richard T Surosky; Martin A Giedlin; Geoff Nichol; Michael C Holmes; Philip D Gregory; Dale G Ando; Michael Kalos; Ronald G Collman; Gwendolyn Binder-Scholl; Gabriela Plesa; Wei-Ting Hwang; Bruce L Levine; Carl H June
Journal:  N Engl J Med       Date:  2014-03-06       Impact factor: 91.245

7.  Corepressor-dependent silencing of fetal hemoglobin expression by BCL11A.

Authors:  Jian Xu; Daniel E Bauer; Marc A Kerenyi; Thuy D Vo; Serena Hou; Yu-Jung Hsu; Huilan Yao; Jennifer J Trowbridge; Gail Mandel; Stuart H Orkin
Journal:  Proc Natl Acad Sci U S A       Date:  2013-04-01       Impact factor: 11.205

8.  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

Review 9.  Gene therapy on the move.

Authors:  Kerstin B Kaufmann; Hildegard Büning; Anne Galy; Axel Schambach; Manuel Grez
Journal:  EMBO Mol Med       Date:  2013-09-17       Impact factor: 12.137

10.  Homology-driven genome editing in hematopoietic stem and progenitor cells using ZFN mRNA and AAV6 donors.

Authors:  Jianbin Wang; Colin M Exline; Joshua J DeClercq; G Nicholas Llewellyn; Samuel B Hayward; Patrick Wai-Lun Li; David A Shivak; Richard T Surosky; Philip D Gregory; Michael C Holmes; Paula M Cannon
Journal:  Nat Biotechnol       Date:  2015-11-09       Impact factor: 54.908
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  5 in total

1.  Efficient Transduction of Human and Rhesus Macaque Primary T Cells by a Modified Human Immunodeficiency Virus Type 1-Based Lentiviral Vector.

Authors:  Huan He; Jing Xue; Weiming Wang; Lihong Liu; Chaobaihui Ye; Zhe Cong; Jason T Kimata; Chuan Qin; Paul Zhou
Journal:  Hum Gene Ther       Date:  2016-12-29       Impact factor: 5.695

2.  Massively-Parallelized, Deterministic Mechanoporation for Intracellular Delivery.

Authors:  Harish G Dixit; Renate Starr; Morgan L Dundon; Pranee I Pairs; Xin Yang; Yanyan Zhang; Daniel Nampe; Christopher B Ballas; Hideaki Tsutsui; Stephen J Forman; Christine E Brown; Masaru P Rao
Journal:  Nano Lett       Date:  2019-10-28       Impact factor: 11.189

3.  Modeling Anti-HIV-1 HSPC-Based Gene Therapy in Humanized Mice Previously Infected with HIV-1.

Authors:  Wannisa Khamaikawin; Saki Shimizu; Masakazu Kamata; Ruth Cortado; Yujin Jung; Jennifer Lam; Jing Wen; Patrick Kim; Yiming Xie; Sanggu Kim; Hubert Arokium; Angela P Presson; Irvin S Y Chen; Dong Sung An
Journal:  Mol Ther Methods Clin Dev       Date:  2017-12-01       Impact factor: 6.698

Review 4.  Acid ceramidase deficiency: Farber disease and SMA-PME.

Authors:  Fabian P S Yu; Samuel Amintas; Thierry Levade; Jeffrey A Medin
Journal:  Orphanet J Rare Dis       Date:  2018-07-20       Impact factor: 4.123

5.  Engineered Zinc Finger Protein Targeting 2LTR Inhibits HIV Integration in Hematopoietic Stem and Progenitor Cell-Derived Macrophages: In Vitro Study.

Authors:  Koollawat Chupradit; Wannisa Khamaikawin; Supachai Sakkhachornphop; Chaniporn Puaninta; Bruce E Torbett; Suparerk Borwornpinyo; Suradej Hongeng; Methichit Wattanapanitch; Chatchai Tayapiwatana
Journal:  Int J Mol Sci       Date:  2022-02-19       Impact factor: 5.923
  5 in total

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