Literature DB >> 11999349

Hematopoietic stem cell gene therapy.

David W Emery1, Tamon Nishino, Ken Murata, Michalis Fragkos, George Stamatoyannopoulos.   

Abstract

Gene therapy applications that target hematopoietic stem cells (HSCs) offer great potential for the treatment of hematologic disease. Despite this promise, clinical success has been limited by poor rates of gene transfer, poor engraftment of modified cells, and poor levels of gene expression. We describe here the basic approach used for HSC gene therapy, briefly review some of the seminal clinical trials in the field, and describe several recent advances directed toward overcoming these limitations.

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Year:  2002        PMID： 11999349     DOI： 10.1007/bf02982035

Source DB:  PubMed          Journal:  Int J Hematol        ISSN： 0925-5710            Impact factor:   2.490


  105 in total

1.  Nonmyeloablative HPC transplantation.

Authors:  T Repka; D J Weisdorf
Journal:  Transfusion       Date:  2000-07       Impact factor: 3.157

2.  Integration of adeno-associated virus vectors in CD34+ human hematopoietic progenitor cells after transduction.

Authors:  G Fisher-Adams; K K Wong; G Podsakoff; S J Forman; S Chatterjee
Journal:  Blood       Date:  1996-07-15       Impact factor: 22.113

3.  Combination of interleukins 3 and 6 preserves stem cell function in culture and enhances retrovirus-mediated gene transfer into hematopoietic stem cells.

Authors:  D M Bodine; S Karlsson; A W Nienhuis
Journal:  Proc Natl Acad Sci U S A       Date:  1989-11       Impact factor: 11.205

4.  Adeno-associated virus vector integration junctions.

Authors:  E A Rutledge; D W Russell
Journal:  J Virol       Date:  1997-11       Impact factor: 5.103

5.  The effect of multidrug-resistance 1 gene versus neo transduction on ex vivo and in vivo expansion of rhesus macaque hematopoietic repopulating cells.

Authors:  S E Sellers; J F Tisdale; B A Agricola; M E Metzger; R E Donahue; C E Dunbar; B P Sorrentino
Journal:  Blood       Date:  2001-03-15       Impact factor: 22.113

6.  Gene transfer in intact animals.

Authors:  M J Cline; H Stang; K Mercola; L Morse; R Ruprecht; J Brown; W Salser
Journal:  Nature       Date:  1980-04-03       Impact factor: 49.962

7.  Sustained multilineage gene persistence and expression in dogs transplanted with CD34(+) marrow cells transduced by RD114-pseudotype oncoretrovirus vectors.

Authors:  M Goerner; P A Horn; L Peterson; P Kurre; R Storb; J E Rasko; H P Kiem
Journal:  Blood       Date:  2001-10-01       Impact factor: 22.113

8.  Ligand-inducible and liver-specific target gene expression in transgenic mice.

Authors:  Y Wang; F J DeMayo; S Y Tsai; B W O'Malley
Journal:  Nat Biotechnol       Date:  1997-03       Impact factor: 54.908

9.  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

10.  Helper-free foamy virus vectors.

Authors:  G D Trobridge; D W Russell
Journal:  Hum Gene Ther       Date:  1998-11-20       Impact factor: 5.695
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  8 in total

Review 1.  Stem cell and precursor cell therapy.

Authors:  Jingli Cai; Mahendra S Rao
Journal:  Neuromolecular Med       Date:  2002       Impact factor: 3.843

Review 2.  Selective expansion of transduced cells for hematopoietic stem cell gene therapy.

Authors:  Akihiro Kume; Yutaka Hanazono; Hiroaki Mizukami; Takashi Okada; Keiya Ozawa
Journal:  Int J Hematol       Date:  2002-11       Impact factor: 2.490

3.  Recombinant AAV2 transduction of primitive human hematopoietic stem cells capable of serial engraftment in immune-deficient mice.

Authors:  Leah Santat; Helicia Paz; Christie Wong; Lijing Li; James Macer; Stephen Forman; K K Wong; Saswati Chatterjee
Journal:  Proc Natl Acad Sci U S A       Date:  2005-07-25       Impact factor: 11.205

4.  A functional screen for regulatory elements that improve retroviral vector gene expression.

Authors:  Amy C Groth; David W Emery
Journal:  Blood Cells Mol Dis       Date:  2010-09-16       Impact factor: 3.039

5.  Toward a stem cell gene therapy for breast cancer.

Authors:  ZongYi Li; Ying Liu; Sebastian Tuve; Ye Xun; Xiaolong Fan; Liang Min; Qinghua Feng; Nancy Kiviat; Hans-Peter Kiem; Mary Leonora Disis; André Lieber
Journal:  Blood       Date:  2009-03-27       Impact factor: 22.113

6.  Recombinant adeno-associated viral vectors are deficient in provoking a DNA damage response.

Authors:  Michalis Fragkos; Madlaina Breuleux; Nathalie Clément; Peter Beard
Journal:  J Virol       Date:  2008-05-07       Impact factor: 5.103

7.  Identification of parameters required for efficient lentiviral vector transduction and engraftment of human cord blood CD34(+) NOD/SCID-repopulating cells.

Authors:  Ying Liu; Giao Hangoc; Timothy B Campbell; Michael Goodman; Wen Tao; Karen Pollok; Edward F Srour; Hal E Broxmeyer
Journal:  Exp Hematol       Date:  2008-08       Impact factor: 3.084

8.  Hematopoietic chimerism and central tolerance created by peripheral-tolerance induction without myeloablative conditioning.

Authors:  Edward Seung; John P Mordes; Aldo A Rossini; Dale L Greiner
Journal:  J Clin Invest       Date:  2003-09       Impact factor: 14.808
  8 in total

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