|
|
 Previous Article | Table of Contents | Next Article 
Blood, 15 November 2000, Vol. 96, No. 10, pp. 3392-3398
GENE THERAPY
High-level transgene expression in human hematopoietic
progenitors and differentiated blood lineages after transduction with
improved lentiviral vectors
Patrick Salmon,
Vincent Kindler,
Odile Ducrey,
Bernard Chapuis,
Rudolf H. Zubler, and
Didier Trono
From the Department of Genetics and Microbiology and
Division of Hematology, Faculty of Medicine, Geneva,
Switzerland.
Recent experiments point to the great value of lentiviral vectors
for the transduction of human hematopoietic stem cells (hHSCs). Vectors
used so far, however, have been poorly satisfying in terms of either
biosafety or efficiency of transgene expression. Herein is described
the results obtained with human immunodeficiency virus-based vectors
optimized in both of these aspects. It is thus shown that vectors
containing the EF1 and, to a lesser extent, the phosphoglycerate
kinase (PGK) promoter, govern high-level gene expression in human
hematopoietic progenitors as well as derived hematopoietic
lineages of therapeutic relevance, such as erythrocytes,
granulocytes, monocytes, dendritic cells, and megakaryocytes.
EF1 promoter-containing lentiviral vectors can also induce strong
transgene expression in primary T lymphocytes isolated from peripheral
blood. A self-inactivating design did not affect the performance of
EF1 promoter-based vectors but significantly reduced expression from
the PGK promoter. This negative effect could nevertheless be largely
rescued by inserting the post-transcriptional regulatory element of
woodchuck hepatitis virus upstream of the vector 3' long terminal
repeat. These results have important practical implications for the
genetic treatment of lymphohematologic disorders as well as for the
study of hematopoiesis via the lentivector-mediated modification of hHSCs.
 CiteULike  Connotea  Del.icio.us  Digg  Reddit  Technorati What's this?
This article has been cited by other articles:
|
|
|
|
 
P. Laje, D. Shang, W. Cao, M. Niiya, M. Endo, A. Radu, N. DeRogatis, F. Scheiflinger, P. W. Zoltick, A. W. Flake, et al.
Correction of murine ADAMTS13 deficiency by hematopoietic progenitor cell-mediated gene therapy
Blood,
March 5, 2009;
113(10):
2172 - 2180.
[Abstract]
[Full Text]
[PDF]
|
|
|
|
|
|
|
T. I. A. Roach, R. A. Rebres, I. D. C. Fraser, D. L. DeCamp, K.-M. Lin, P. C. Sternweis, M. I. Simon, and W. E. Seaman
Signaling and Cross-talk by C5a and UDP in Macrophages Selectively Use PLC{beta}3 to Regulate Intracellular Free Calcium
J. Biol. Chem.,
June 20, 2008;
283(25):
17351 - 17361.
[Abstract]
[Full Text]
[PDF]
|
|
|
|
|
|
|
R. L. Skalsky, J. Hu, and R. Renne
Analysis of Viral cis Elements Conferring Kaposi's Sarcoma-Associated Herpesvirus Episome Partitioning and Maintenance
J. Virol.,
September 15, 2007;
81(18):
9825 - 9837.
[Abstract]
[Full Text]
[PDF]
|
|
|
|
|
|
|
N. Satta, S. Dunoyer-Geindre, G. Reber, R. J. Fish, F. Boehlen, E. K. O. Kruithof, and P. de Moerloose
The role of TLR2 in the inflammatory activation of mouse fibroblasts by human antiphospholipid antibodies
Blood,
February 15, 2007;
109(4):
1507 - 1514.
[Abstract]
[Full Text]
[PDF]
|
|
|
|
|
|
|
V. Kindler
Postnatal stem cell survival: does the niche, a rare harbor where to resist the ebb tide of differentiation, also provide lineage-specific instructions?
J. Leukoc. Biol.,
October 1, 2005;
78(4):
836 - 844.
[Abstract]
[Full Text]
[PDF]
|
|
|
|
|
|
|
I. Bot, J. Guo, M. Van Eck, P. J. Van Santbrink, P. H. E. Groot, R. B. Hildebrand, J. Seppen, T. J. C. Van Berkel, and E. A. L. Biessen
Lentiviral shRNA silencing of murine bone marrow cell CCR2 leads to persistent knockdown of CCR2 function in vivo
Blood,
August 15, 2005;
106(4):
1147 - 1153.
[Abstract]
[Full Text]
[PDF]
|
|
|
|
|
|
|
I. Dunand-Sauthier, M. Hohl, F. Thorel, P. Jaquier-Gubler, S. G. Clarkson, and O. D. Scharer
The Spacer Region of XPG Mediates Recruitment to Nucleotide Excision Repair Complexes and Determines Substrate Specificity
J. Biol. Chem.,
February 25, 2005;
280(8):
7030 - 7037.
[Abstract]
[Full Text]
[PDF]
|
|
|
|
|
|
|
F. Thorel, A. Constantinou, I. Dunand-Sauthier, T. Nouspikel, P. Lalle, A. Raams, N. G. J. Jaspers, W. Vermeulen, M. K. K. Shivji, R. D. Wood, et al.
Definition of a Short Region of XPG Necessary for TFIIH Interaction and Stable Recruitment to Sites of UV Damage
Mol. Cell. Biol.,
December 15, 2004;
24(24):
10670 - 10680.
[Abstract]
[Full Text]
[PDF]
|
|
|
|
|
|
|
C. V. Borlongan, M. Hadman, C. Davis Sanberg, and P. R. Sanberg
Central Nervous System Entry of Peripherally Injected Umbilical Cord Blood Cells Is Not Required for Neuroprotection in Stroke
Stroke,
October 1, 2004;
35(10):
2385 - 2389.
[Abstract]
[Full Text]
[PDF]
|
|
|
|
|
|
|
A. C. Logan, D. L. Haas, T. Kafri, and D. B. Kohn
Integrated Self-Inactivating Lentiviral Vectors Produce Full-Length Genomic Transcripts Competent for Encapsidation and Integration
J. Virol.,
August 15, 2004;
78(16):
8421 - 8436.
[Abstract]
[Full Text]
[PDF]
|
|
|
|
|
|
|
Y. Soda, K. Tani, Y. Bai, M. Saiki, M. Chen, K. Izawa, S. Kobayashi, S. Takahashi, K. Uchimaru, T. Kuwabara, et al.
A novel maxizyme vector targeting a bcr-abl fusion gene induced specific cell death in Philadelphia chromosome-positive acute lymphoblastic leukemia
Blood,
July 15, 2004;
104(2):
356 - 363.
[Abstract]
[Full Text]
[PDF]
|
|
|
|
|
|
|
T. Schomber, C. P. Kalberer, A. Wodnar-Filipowicz, and R. C. Skoda
Gene silencing by lentivirus-mediated delivery of siRNA in human CD34+ cells
Blood,
June 15, 2004;
103(12):
4511 - 4513.
[Abstract]
[Full Text]
[PDF]
|
|
|
|
|
|
|
F. Matheux and J. Villard
Cellular and Gene Therapy for Major Histocompatibility Complex Class II Deficiency
Physiology,
June 1, 2004;
19(3):
154 - 158.
[Abstract]
[Full Text]
[PDF]
|
|
|
|
|
|
|
M. Bros, X.-L. Ross, A. Pautz, A. B. Reske-Kunz, and R. Ross
The Human Fascin Gene Promoter Is Highly Active in Mature Dendritic Cells Due to a Stage-Specific Enhancer
J. Immunol.,
August 15, 2003;
171(4):
1825 - 1834.
[Abstract]
[Full Text]
[PDF]
|
|
|
|
|
|
|
E. Verhoeyen, V. Dardalhon, O. Ducrey-Rundquist, D. Trono, N. Taylor, and F.-L. Cosset
IL-7 surface-engineered lentiviral vectors promote survival and efficient gene transfer in resting primary T lymphocytes
Blood,
March 15, 2003;
101(6):
2167 - 2174.
[Abstract]
[Full Text]
[PDF]
|
|
|
|
|
|
|
F. Bovia, P. Salmon, T. Matthes, K. Kvell, T. H. Nguyen, C. Werner-Favre, M. Barnet, M. Nagy, F. Leuba, J.-F. Arrighi, et al.
Efficient transduction of primary human B lymphocytes and nondividing myeloma B cells with HIV-1-derived lentiviral vectors
Blood,
March 1, 2003;
101(5):
1727 - 1733.
[Abstract]
[Full Text]
[PDF]
|
|
|
|
|
|
|
N.-B. Woods, A. Muessig, M. Schmidt, J. Flygare, K. Olsson, P. Salmon, D. Trono, C. von Kalle, and S. Karlsson
Lentiviral vector transduction of NOD/SCID repopulating cells results in multiple vector integrations per transduced cell: risk of insertional mutagenesis
Blood,
February 15, 2003;
101(4):
1284 - 1289.
[Abstract]
[Full Text]
[PDF]
|
|
|
|
|
|
|
J. Roesler, S. Brenner, A. A. Bukovsky, N. Whiting-Theobald, T. Dull, M. Kelly, C. I. Civin, and H. L. Malech
Third-generation, self-inactivating gp91phox lentivector corrects the oxidase defect in NOD/SCID mouse-repopulating peripheral blood-mobilized CD34+ cells from patients with X-linked chronic granulomatous disease
Blood,
December 15, 2002;
100(13):
4381 - 4390.
[Abstract]
[Full Text]
[PDF]
|
|
|
|
|
|
|
I. Hamaguchi, A. Ooka, A. Brun, J. Richter, N. Dahl, and S. Karlsson
Gene transfer improves erythroid development in ribosomal protein S19-deficient Diamond-Blackfan anemia
Blood,
September 26, 2002;
100(8):
2724 - 2731.
[Abstract]
[Full Text]
[PDF]
|
|
|
|
|
|
|
O. Ducrey-Rundquist, M. Guyader, and D. Trono
Modalities of Interleukin-7-Induced Human Immunodeficiency Virus Permissiveness in Quiescent T Lymphocytes
J. Virol.,
August 12, 2002;
76(18):
9103 - 9111.
[Abstract]
[Full Text]
[PDF]
|
|
|
|
|
|
|
T. VandenDriessche, L. Thorrez, L. Naldini, A. Follenzi, L. Moons, Z. Berneman, D. Collen, and M. K. L. Chuah
Lentiviral vectors containing the human immunodeficiency virus type-1 central polypurine tract can efficiently transduce nondividing hepatocytes and antigen-presenting cells in vivo
Blood,
July 18, 2002;
100(3):
813 - 822.
[Abstract]
[Full Text]
[PDF]
|
|
|
|
|
|
|
V. Sandrin, B. Boson, P. Salmon, W. Gay, D. Negre, R. Le Grand, D. Trono, and F.-L. Cosset
Lentiviral vectors pseudotyped with a modified RD114 envelope glycoprotein show increased stability in sera and augmented transduction of primary lymphocytes and CD34+ cells derived from human and nonhuman primates
Blood,
July 18, 2002;
100(3):
823 - 832.
[Abstract]
[Full Text]
[PDF]
|
|
|
|
|
|
|
A.-K. Zaiss, S. Son, and L.-J. Chang
RNA 3' Readthrough of Oncoretrovirus and Lentivirus: Implications for Vector Safety and Efficacy
J. Virol.,
June 14, 2002;
76(14):
7209 - 7219.
[Abstract]
[Full Text]
[PDF]
|
|
|
|
|
|
|
N. Srinivasakumar, M. Zaboikin, T. Zaboikina, and F. Schuening
Evaluation of Tat-Encoding Bicistronic Human Immunodeficiency Virus Type 1 Gene Transfer Vectors in Primary Canine Bone Marrow Mononuclear Cells
J. Virol.,
June 14, 2002;
76(14):
7334 - 7342.
[Abstract]
[Full Text]
[PDF]
|
|
|
|
|
|
|
M. Maurice, E. Verhoeyen, P. Salmon, D. Trono, S. J. Russell, and F.-L. Cosset
Efficient gene transfer into human primary blood lymphocytes by surface-engineered lentiviral vectors that display a T cell-activating polypeptide
Blood,
April 1, 2002;
99(7):
2342 - 2350.
[Abstract]
[Full Text]
[PDF]
|
|
|
|
|
|
|
Y. Cui, J. Golob, E. Kelleher, Z. Ye, D. Pardoll, and L. Cheng
Targeting transgene expression to antigen-presenting cells derived from lentivirus-transduced engrafting human hematopoietic stem/progenitor cells
Blood,
January 15, 2002;
99(2):
399 - 408.
[Abstract]
[Full Text]
[PDF]
|
|
|
|
|
|
|
A. L. Feldman, H. R. Alexander, S. M. Hewitt, D. Lorang, C. E. Thiruvathukal, E. M. Turner, and S. K. Libutti
Effect of Retroviral Endostatin Gene Transfer on Subcutaneous and Intraperitoneal Growth of Murine Tumors
J Natl Cancer Inst,
July 4, 2001;
93(13):
1014 - 1020.
[Abstract]
[Full Text]
[PDF]
|
|
|
|
|
