Literature DB >> 25295939

Genome editing: a tool for research and therapy: targeted genome editing hits the clinic.

Angelo Lombardo1, Luigi Naldini1.   

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Year:  2014        PMID： 25295939     DOI： 10.1038/nm.3721

Source DB:  PubMed          Journal:  Nat Med        ISSN： 1078-8956            Impact factor:   53.440


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  12 in total

1.  Gene editing in human stem cells using zinc finger nucleases and integrase-defective lentiviral vector delivery.

Authors:  Angelo Lombardo; Pietro Genovese; Christian M Beausejour; Silvia Colleoni; Ya-Li Lee; Kenneth A Kim; Dale Ando; Fyodor D Urnov; Cesare Galli; Philip D Gregory; Michael C Holmes; Luigi Naldini
Journal:  Nat Biotechnol       Date:  2007-10-28       Impact factor: 54.908

2.  An unbiased genome-wide analysis of zinc-finger nuclease specificity.

Authors:  Richard Gabriel; Angelo Lombardo; Anne Arens; Jeffrey C Miller; Pietro Genovese; Christine Kaeppel; Ali Nowrouzi; Cynthia C Bartholomae; Jianbin Wang; Geoffrey Friedman; Michael C Holmes; Philip D Gregory; Hanno Glimm; Manfred Schmidt; Luigi Naldini; Christof von Kalle
Journal:  Nat Biotechnol       Date:  2011-08-07       Impact factor: 54.908

3.  Homozygous defect in HIV-1 coreceptor accounts for resistance of some multiply-exposed individuals to HIV-1 infection.

Authors:  R Liu; W A Paxton; S Choe; D Ceradini; S R Martin; R Horuk; M E MacDonald; H Stuhlmann; R A Koup; N R Landau
Journal:  Cell       Date:  1996-08-09       Impact factor: 41.582

Review 4.  A guide to genome engineering with programmable nucleases.

Authors:  Hyongbum Kim; Jin-Soo Kim
Journal:  Nat Rev Genet       Date:  2014-04-02       Impact factor: 53.242

5.  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

6.  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

7.  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

Review 8.  The DNA damage response: making it safe to play with knives.

Authors:  Alberto Ciccia; Stephen J Elledge
Journal:  Mol Cell       Date:  2010-10-22       Impact factor: 17.970

9.  Human hematopoietic stem/progenitor cells modified by zinc-finger nucleases targeted to CCR5 control HIV-1 in vivo.

Authors:  Nathalia Holt; Jianbin Wang; Kenneth Kim; Geoffrey Friedman; Xingchao Wang; Vanessa Taupin; Gay M Crooks; Donald B Kohn; Philip D Gregory; Michael C Holmes; Paula M Cannon
Journal:  Nat Biotechnol       Date:  2010-07-02       Impact factor: 54.908

10.  Targeted genome editing in human repopulating haematopoietic stem cells.

Authors:  Giulia Schiroli; Giulia Escobar; Pietro Genovese; Tiziano Di Tomaso; Claudia Firrito; Andrea Calabria; Davide Moi; Roberta Mazzieri; Chiara Bonini; Michael C Holmes; Philip D Gregory; Mirjam van der Burg; Bernhard Gentner; Eugenio Montini; Angelo Lombardo; Luigi Naldini
Journal:  Nature       Date:  2014-05-28       Impact factor: 49.962
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  8 in total

Review 1.  Gene therapy using haematopoietic stem and progenitor cells.

Authors:  Giuliana Ferrari; Adrian J Thrasher; Alessandro Aiuti
Journal:  Nat Rev Genet       Date:  2020-12-10       Impact factor: 53.242

Review 2.  Cure for thalassemia major - from allogeneic hematopoietic stem cell transplantation to gene therapy.

Authors:  Alok Srivastava; Ramachandran V Shaji
Journal:  Haematologica       Date:  2016-12-01       Impact factor: 9.941

3.  Targeted Integration and High-Level Transgene Expression in AAVS1 Transgenic Mice after In Vivo HSC Transduction with HDAd5/35++ Vectors.

Authors:  Chang Li; Arpit Suresh Mishra; Sucheol Gil; Meng Wang; Aphrodite Georgakopoulou; Thalia Papayannopoulou; R David Hawkins; André Lieber
Journal:  Mol Ther       Date:  2019-08-19       Impact factor: 11.454

4.  Genetic engineering in primary human B cells with CRISPR-Cas9 ribonucleoproteins.

Authors:  Chung-An M Wu; Theodore L Roth; Yuriy Baglaenko; Dario M Ferri; Patrick Brauer; Juan Carlos Zuniga-Pflucker; Kristina W Rosbe; Joan E Wither; Alexander Marson; Christopher D C Allen
Journal:  J Immunol Methods       Date:  2018-03-31       Impact factor: 2.303

5.  Hereditary Angioedema as a Metabolic Liver Disorder: Novel Therapeutic Options and Prospects for Cure.

Authors:  Rohan Ameratunga; Adam Bartlett; John McCall; Richard Steele; See-Tarn Woon; Constance H Katelaris
Journal:  Front Immunol       Date:  2016-11-30       Impact factor: 7.561

6.  A Novel High-Throughput Screening Platform Reveals an Optimized Cytokine Formulation for Human Hematopoietic Progenitor Cell Expansion.

Authors:  Marina Tarunina; Diana Hernandez; Barbara Kronsteiner-Dobramysl; Philip Pratt; Thomas Watson; Peng Hua; Francesca Gullo; Mark van der Garde; Youyi Zhang; Lilian Hook; Yen Choo; Suzanne M Watt
Journal:  Stem Cells Dev       Date:  2016-09-30       Impact factor: 3.272

Review 7.  Neurogenethics: An emerging discipline at the intersection of ethics, neuroscience, and genomics.

Authors:  Turhan Canli
Journal:  Appl Transl Genom       Date:  2015-05-15

8.  Multilineage polyclonal engraftment of Cal-1 gene-modified cells and in vivo selection after SHIV infection in a nonhuman primate model of AIDS.

Authors:  Christopher W Peterson; Kevin G Haworth; Bryan P Burke; Patricia Polacino; Krystin K Norman; Jennifer E Adair; Shiu-Lok Hu; Jeffrey S Bartlett; Geoff P Symonds; Hans-Peter Kiem
Journal:  Mol Ther Methods Clin Dev       Date:  2016-02-24       Impact factor: 6.698
  8 in total

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