Hematopoietic-Stem-Cell-Based Gene Therapy for HIV Disease

被引：103

作者：

Kiem, Hans-Peter ^{[2
,4
,5
]}

Jerome, Keith R. ^{[3
,6
]}

Deeks, Steven G. ^{[7
]}

McCune, Joseph M. ^{[1
]}

机构：

[1] Univ Calif San Francisco, Dept Med, Div Expt Med, San Francisco, CA 94110 USA

[2] Fred Hutchinson Canc Res Ctr, Div Clin Res, Seattle, WA 98109 USA

[3] Fred Hutchinson Canc Res Ctr, Vaccine & Infect Dis Div, Seattle, WA 98109 USA

[4] Univ Washington, Dept Med, Seattle, WA 98109 USA

[5] Univ Washington, Dept Pathol, Seattle, WA 98109 USA

[6] Univ Washington, Dept Lab Med, Seattle, WA 98109 USA

[7] Univ Calif San Francisco, HIV AIDS Program, San Francisco, CA 94110 USA

来源：

CELL STEM CELL | 2012年 / 10卷 / 02期

关键词：

BLOOD PROGENITOR CELLS; MARROW REPOPULATING CELLS; IN-VIVO; T-CELLS; MULTILINEAGE HEMATOPOIESIS; EFFICIENT TRANSDUCTION; LENTIVIRAL VECTOR; MURINE MARROW; SELF-RENEWAL; IMMUNODEFICIENCY;

D O I：

10.1016/j.stem.2011.12.015

中图分类号：

Q813 [细胞工程];

学科分类号：

摘要：

Although combination antiretroviral therapy can dramatically reduce the circulating viral load in those infected with HIV, replication-competent virus persists. To eliminate the need for indefinite treatment, there is growing interest in creating a functional HIV-resistant immune system through the use of gene-modified hematopoietic stem cells (HSCs). Proof of concept for this approach has been provided in the instance of an HIV-infected adult transplanted with allogeneic stem cells from a donor lacking the HIV coreceptor, CCR5. Here, we review this and other strategies for HSC-based gene therapy for HIV disease. © 2012 Elsevier Inc.

引用

页码：137 / 147

页数：11

共 99 条

[1] Novel interfering bifunctional molecules against the CCR5 coreceptor are efficient inhibitors of HIV-1 infection [J].

Abad, JL ;

González, MA ;

del Real, G ;

Mira, E ;

Mañes, S ;

Serrano, F ;

Bernad, A .

MOLECULAR THERAPY, 2003, 8 (03) :475-484

[2] Gene Therapy for Immunodeficiency Due to Adenosine Deaminase Deficiency. [J].

Aiuti, Alessandro ;

Cattaneo, Federica ;

Galimberti, Stefania ;

Benninghoff, Ulrike ;

Cassani, Barbara ;

Callegaro, Luciano ;

Scaramuzza, Samantha ;

Andolfi, Grazia ;

Mirolo, Massimiliano ;

Brigida, Immacolata ;

Tabucchi, Antonella ;

Carlucci, Filippo ;

Eibl, Martha ;

Aker, Memet ;

Slavin, Shimon ;

Al-Mousa, Hamoud ;

Al Ghonaium, Abdulaziz ;

Ferster, Alina ;

Duppenthaler, Andrea ;

Notarangelo, Luigi ;

Wintergerst, Uwe ;

Buckley, Rebecca H. ;

Bregni, Marco ;

Marktel, Sarah ;

Valsecchi, Maria Grazia ;

Rossi, Paolo ;

Ciceri, Fabio ;

Miniero, Roberto ;

Bordignon, Claudio ;

Roncarolo, Maria-Grazia .

NEW ENGLAND JOURNAL OF MEDICINE, 2009, 360 (05) :447-458

[3] Evidence for the cure of HIV infection by CCR5Δ32/Δ32 stem cell transplantation [J].

Allers, Kristina ;

Huetter, Gero ;

Hofmann, Joerg ;

Loddenkemper, Christoph ;

Rieger, Kathrin ;

Thiel, Eckhard ;

Schneider, Thomas .

BLOOD, 2011, 117 (10) :2791-2799

[4] 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 [J].

Amado, RG ;

Mitsuyasu, RT ;

Rosenblatt, JD ;

Ngok, FK ;

Bakker, A ;

Cole, S ;

Chorn, N ;

Lin, LS ;

Bristol, G ;

Boyd, MP ;

Macpherson, JL ;

Fanning, GC ;

Todd, AV ;

Ely, JA ;

Zack, JA ;

Symonds, GP .

HUMAN GENE THERAPY, 2004, 15 (03) :251-262

[5] Human immunodeficiency virus type 1 restriction by human-rhesus chimeric tripartite motif 5α (TRIM 5α) in CD34+ cell-derived macrophages in vitro and in T cells in vivo in severe combined immunodeficient (SCID-hu) mice transplanted with human fetal tissue [J].

Anderson, Joseph ;

Akkina, Ramesh .

HUMAN GENE THERAPY, 2008, 19 (03) :217-228

[6] Successful Targeting and Disruption of an Integrated Reporter Lentivirus Using the Engineered Homing Endonuclease Y2 I-AniI [J].

Aubert, Martine ;

Ryu, Byoung Y. ;

Banks, Lindsey ;

Rawlings, David J. ;

Scharenberg, Andrew M. ;

Jerome, Keith R. .

PLOS ONE, 2011, 6 (02)

[7] GENE-THERAPY - INTRACELLULAR IMMUNIZATION [J].

BALTIMORE, D .

NATURE, 1988, 335 (6189) :395-396

[8] Lentiviral transduction of Tar Decoy and CCR5 ribozyme into CD34+ progenitor cells and derivation of HIV-1 resistant T cells and macrophages [J].

Banerjea A. ;

Li M.-J. ;

Remling L. ;

Rossi J. ;

Akkina R. .

AIDS Research and Therapy, 1 (1)

[9] Concise Review: Managing Genotoxicity in the Therapeutic Modification of Stem Cells [J].

Baum, Christopher ;

Modlich, Ute ;

Goehring, Gudrun ;

Schlegelberger, Brigitte .

STEM CELLS, 2011, 29 (10) :1479-1484

[10] Efficient and stable MGMT-mediated selection of long-term repopulating stem cells in nonhuman primates [J].

Beard, Brian C. ;

Trobridge, Grant D. ;

Ironside, Christina ;

McCune, Jeannine S. ;

Adair, Jennifer E. ;

Kiem, Hans-Peter .

JOURNAL OF CLINICAL INVESTIGATION, 2010, 120 (07) :2345-2354

← 1 2 3 4 5 6 7 8 9 10 →