Literature DB >> 24014892

Hematopoietic stem cell gene therapy:assessing the relevance of preclinical models.

Andre Larochelle, Cynthia E Dunbar.   

Abstract

Mesh：

Year:  2013        PMID： 24014892      PMCID： PMC3761403          DOI： 10.1053/j.seminhematol.2013.03.025

Source DB:  PubMed          Journal:  Semin Hematol        ISSN： 0037-1963            Impact factor:   3.851


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  305 in total

1.  Efficient retrovirus-mediated transfer of the multidrug resistance 1 gene into autologous human long-term repopulating hematopoietic stem cells.

Authors:  R Abonour; D A Williams; L Einhorn; K M Hall; J Chen; J Coffman; C M Traycoff; A Bank; I Kato; M Ward; S D Williams; R Hromas; M J Robertson; F O Smith; D Woo; B Mills; E F Srour; K Cornetta
Journal:  Nat Med       Date:  2000-06       Impact factor: 53.440

2.  Transduction of human CD34+ CD38- bone marrow and cord blood-derived SCID-repopulating cells with third-generation lentiviral vectors.

Authors:  G Guenechea; O I Gan; T Inamitsu; C Dorrell; D S Pereira; M Kelly; L Naldini; J E Dick
Journal:  Mol Ther       Date:  2000-06       Impact factor: 11.454

3.  Lymphoid development and function in X-linked severe combined immunodeficiency mice after stem cell gene therapy.

Authors:  M Otsu; S M Anderson; D M Bodine; J M Puck; J J O'Shea; F Candotti
Journal:  Mol Ther       Date:  2000-02       Impact factor: 11.454

4.  Lentiviral gene transfer into primary and secondary NOD/SCID repopulating cells.

Authors:  N B Woods; C Fahlman; H Mikkola; I Hamaguchi; K Olsson; R Zufferey; S E Jacobsen; D Trono; S Karlsson
Journal:  Blood       Date:  2000-12-01       Impact factor: 22.113

5.  Genomic and functional assays demonstrate reduced gammaretroviral vector genotoxicity associated with use of the cHS4 chromatin insulator.

Authors:  Chang Long Li; Ding Xiong; George Stamatoyannopoulos; David W Emery
Journal:  Mol Ther       Date:  2009-02-24       Impact factor: 11.454

6.  Retroviral gene transduction of adult peripheral blood or marrow-derived CD34+ cells for six hours without growth factors or on autologous stroma does not improve marking efficiency assessed in vivo.

Authors:  R V Emmons; S Doren; J Zujewski; M Cottler-Fox; C S Carter; K Hines; J A O'Shaughnessy; S F Leitman; J J Greenblatt; K Cowan; C E Dunbar
Journal:  Blood       Date:  1997-06-01       Impact factor: 22.113

7.  Colocalization of retrovirus and target cells on specific fibronectin fragments increases genetic transduction of mammalian cells.

Authors:  H Hanenberg; X L Xiao; D Dilloo; K Hashino; I Kato; D A Williams
Journal:  Nat Med       Date:  1996-08       Impact factor: 53.440

8.  Anti-human immunodeficiency virus hematopoietic progenitor cell-delivered ribozyme in a phase I study: myeloid and lymphoid reconstitution in human immunodeficiency virus type-1-infected patients.

Authors:  Rafael G Amado; Ronald T Mitsuyasu; Joseph D Rosenblatt; Frances K Ngok; Andreas Bakker; Steve Cole; Nathalie Chorn; Lii-Shin Lin; Gregory Bristol; Maureen P Boyd; Janet L MacPherson; Gregory C Fanning; Alison V Todd; Julie A Ely; Jerome A Zack; Geoff P Symonds
Journal:  Hum Gene Ther       Date:  2004-03       Impact factor: 5.695

9.  Hematopoietic stem cell gene transfer in a tumor-prone mouse model uncovers low genotoxicity of lentiviral vector integration.

Authors:  Eugenio Montini; Daniela Cesana; Manfred Schmidt; Francesca Sanvito; Maurilio Ponzoni; Cynthia Bartholomae; Lucia Sergi Sergi; Fabrizio Benedicenti; Alessandro Ambrosi; Clelia Di Serio; Claudio Doglioni; Christof von Kalle; Luigi Naldini
Journal:  Nat Biotechnol       Date:  2006-05-28       Impact factor: 54.908

10.  A nuclear localization signal within HIV-1 matrix protein that governs infection of non-dividing cells.

Authors:  M I Bukrinsky; S Haggerty; M P Dempsey; N Sharova; A Adzhubel; L Spitz; P Lewis; D Goldfarb; M Emerman; M Stevenson
Journal:  Nature       Date:  1993-10-14       Impact factor: 69.504
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  11 in total

1.  CRISPR/Cas9 PIG -A gene editing in nonhuman primate model demonstrates no intrinsic clonal expansion of PNH HSPCs.

Authors:  Tae-Hoon Shin; Eun Jung Baek; Marcus A F Corat; Shirley Chen; Jean-Yves Metais; Aisha A AlJanahi; Yifan Zhou; Robert E Donahue; Kyung-Rok Yu; Cynthia E Dunbar
Journal:  Blood       Date:  2019-04-19       Impact factor: 22.113

2.  Genome editing and stem cell therapy pave the path for new treatment of sickle-cell disease.

Authors:  Mary Katherine Randolph; Wanke Zhao
Journal:  Stem Cell Investig       Date:  2015-11-30

3.  The impact of aging on primate hematopoiesis as interrogated by clonal tracking.

Authors:  Kyung-Rok Yu; Diego A Espinoza; Chuanfeng Wu; Lauren Truitt; Tae-Hoon Shin; Shirley Chen; Xing Fan; Idalia M Yabe; Sandhya Panch; So Gun Hong; Samson Koelle; Rong Lu; Aylin Bonifacino; Allen Krouse; Mark Metzger; Robert E Donahue; Cynthia E Dunbar
Journal:  Blood       Date:  2018-01-02       Impact factor: 22.113

4.  Collection of hematopoietic CD34 stem cells in rhesus macaques using Spectra Optia.

Authors:  Lynn D Haynes; Jennifer Coonen; Jennifer Post; Kevin Brunner; Debra Bloom; Peiman Hematti; Dixon B Kaufman
Journal:  J Clin Apher       Date:  2016-08-31       Impact factor: 2.821

Review 5.  CRISPR/Cas9 ribonucleoprotein-mediated genome and epigenome editing in mammalian cells.

Authors:  Hanan Bloomer; Jennifer Khirallah; Yamin Li; Qiaobing Xu
Journal:  Adv Drug Deliv Rev       Date:  2021-12-20       Impact factor: 15.470

Review 6.  Understanding and overcoming adverse consequences of genome editing on hematopoietic stem and progenitor cells.

Authors:  Byung-Chul Lee; Richard J Lozano; Cynthia E Dunbar
Journal:  Mol Ther       Date:  2021-09-10       Impact factor: 11.454

7.  Clonal Dominance With Retroviral Vector Insertions Near the ANGPT1 and ANGPT2 Genes in a Human Xenotransplant Mouse Model.

Authors:  Reinhard Haemmerle; Ruhi Phaltane; Michael Rothe; Simon Schröder; Axel Schambach; Thomas Moritz; Ute Modlich
Journal:  Mol Ther Nucleic Acids       Date:  2014-10-07       Impact factor: 10.183

8.  Barcoding of Macaque Hematopoietic Stem and Progenitor Cells: A Robust Platform to Assess Vector Genotoxicity.

Authors:  Idalia M Yabe; Lauren L Truitt; Diego A Espinoza; Chuanfeng Wu; Samson Koelle; Sandhya Panch; Marcus A F Corat; Thomas Winkler; Kyung-Rok Yu; So Gun Hong; Aylin Bonifacino; Allen Krouse; Mark Metzger; Robert E Donahue; Cynthia E Dunbar
Journal:  Mol Ther Methods Clin Dev       Date:  2018-10-25       Impact factor: 6.698

9.  Transplantation of T-cell receptor α/β-depleted allogeneic bone marrow in nonhuman primates.

Authors:  Saritha S D'Souza; Sarah Bennett; Akhilesh Kumar; Laurel E Kelnhofer; Jason Weinfurter; Kran Suknuntha; Jennifer Coonen; Andres Mejia; Heather Simmons; Thaddeus Golos; Peiman Hematti; Christian M Capitini; Matthew R Reynolds; Igor I Slukvin
Journal:  Exp Hematol       Date:  2020-11-08       Impact factor: 3.084

10.  Prediction and validation of hematopoietic stem and progenitor cell off-target editing in transplanted rhesus macaques.

Authors:  Aisha A AlJanahi; Cicera R Lazzarotto; Shirley Chen; Tae-Hoon Shin; Stefan Cordes; Xing Fan; Isabel Jabara; Yifan Zhou; David J Young; Byung-Chul Lee; Kyung-Rok Yu; Yuesheng Li; Bradley Toms; Ilker Tunc; So Gun Hong; Lauren L Truitt; Julia Klermund; Geoffroy Andrieux; Miriam Y Kim; Toni Cathomen; Saar Gill; Shengdar Q Tsai; Cynthia E Dunbar
Journal:  Mol Ther       Date:  2021-06-24       Impact factor: 11.454
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