机构:
Univ Calif Los Angeles, Sch Nursing, 188 BSRB,615 Charles E Young Dr South, Los Angeles, CA 90095 USA
Univ Calif Los Angeles, AIDS Inst, 188 BSRB,615 Charles E Young Dr South, Los Angeles, CA 90095 USAUniv Calif Los Angeles, Sch Nursing, 188 BSRB,615 Charles E Young Dr South, Los Angeles, CA 90095 USA
Pernet, Olivier
[1
,2
]
Yadav, Swati Seth
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h-index: 0
机构:
Univ Calif Los Angeles, Sch Nursing, 188 BSRB,615 Charles E Young Dr South, Los Angeles, CA 90095 USA
Univ Calif Los Angeles, AIDS Inst, 188 BSRB,615 Charles E Young Dr South, Los Angeles, CA 90095 USAUniv Calif Los Angeles, Sch Nursing, 188 BSRB,615 Charles E Young Dr South, Los Angeles, CA 90095 USA
Yadav, Swati Seth
[1
,2
]
An, Dong Sung
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机构:
Univ Calif Los Angeles, Sch Nursing, 188 BSRB,615 Charles E Young Dr South, Los Angeles, CA 90095 USA
Univ Calif Los Angeles, AIDS Inst, 188 BSRB,615 Charles E Young Dr South, Los Angeles, CA 90095 USA
Univ Calif Los Angeles, David Geffen Sch Med, Dept Med, Hematol Oncol, 188 BSRB,615 Charles E Young Dr South, Los Angeles, CA 90095 USAUniv Calif Los Angeles, Sch Nursing, 188 BSRB,615 Charles E Young Dr South, Los Angeles, CA 90095 USA
An, Dong Sung
[1
,2
,3
]
机构:
[1] Univ Calif Los Angeles, Sch Nursing, 188 BSRB,615 Charles E Young Dr South, Los Angeles, CA 90095 USA
[2] Univ Calif Los Angeles, AIDS Inst, 188 BSRB,615 Charles E Young Dr South, Los Angeles, CA 90095 USA
[3] Univ Calif Los Angeles, David Geffen Sch Med, Dept Med, Hematol Oncol, 188 BSRB,615 Charles E Young Dr South, Los Angeles, CA 90095 USA
HIV;
Hematopoietic stem progenitor cells;
Anti-HIV genes;
RNA interference;
Genome editing technologies;
Zinc finger nucleases;
TALEN;
CRISPR/Cas9;
ZINC-FINGER-NUCLEASES;
SHORT HAIRPIN RNA;
IMMUNODEFICIENCY-VIRUS TYPE-1;
HUMAN HEMATOPOIETIC STEM;
CD4(+) T-CELLS;
HIV LENTIVIRAL VECTOR;
HUMAN CD34(+) CELLS;
GENE-THERAPY;
CHEMOKINE RECEPTOR;
PROGENITOR CELLS;
D O I:
10.1016/j.addr.2016.04.027
中图分类号:
R9 [药学];
学科分类号:
1007 ;
摘要:
One of the current focuses in HIV/AIDS research is to develop a novel therapeutic strategy that can provide a lifelong remission of HIV/AIDS without daily drug treatment and, ultimately, a cure for HIV/AIDS. Hematopoietic stem cell-based anti-HIV gene therapy aims to reconstitute the patient immune system by transplantation of genetically engineered hematopoietic stem cells with anti-HIV genes. Hematopoietic stem cells can self-renew, proliferate and differentiate into mature immune cells. In theory, anti-HIV gene-modified hematopoietic stem cells can continuously provide HIV-resistant immune cells throughout the life of a patient. Therefore, hematopoietic stem cell-based anti-HIV gene therapy has a great potential to provide a life-long remission of HIV/AIDS by a single treatment. Here, we provide a comprehensive review of the recent progress of developing anti-HIV genes, genetic modification of hematopoietic stem progenitor cells, engraftment and reconstitution of anti-HIV gene modified immune cells, HIV inhibition in in vitro and in vivo animal models, and in human clinical trials. (C) 2016 Elsevier B.V. All rights reserved.
机构:
Buck Inst Res Aging, Paul F Glenn Ctr Biol Aging Res, 8001 Redwood Blvd, Novato, CA 94945 USABuck Inst Res Aging, Paul F Glenn Ctr Biol Aging Res, 8001 Redwood Blvd, Novato, CA 94945 USA
Neves, Joana
Sousa-Victor, Pedro
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机构:
Buck Inst Res Aging, Paul F Glenn Ctr Biol Aging Res, 8001 Redwood Blvd, Novato, CA 94945 USABuck Inst Res Aging, Paul F Glenn Ctr Biol Aging Res, 8001 Redwood Blvd, Novato, CA 94945 USA
Sousa-Victor, Pedro
Jasper, Heinrich
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h-index: 0
机构:
Buck Inst Res Aging, Paul F Glenn Ctr Biol Aging Res, 8001 Redwood Blvd, Novato, CA 94945 USA
Fritz Lipmann Inst, Leibniz Inst Aging, D-07745 Jena, GermanyBuck Inst Res Aging, Paul F Glenn Ctr Biol Aging Res, 8001 Redwood Blvd, Novato, CA 94945 USA
机构:
Colorado State Univ, Dept Microbiol Immunol & Pathol, Ft Collins, CO 80523 USAColorado State Univ, Dept Microbiol Immunol & Pathol, Ft Collins, CO 80523 USA
Akkina, R
Banerjea, A
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机构:Colorado State Univ, Dept Microbiol Immunol & Pathol, Ft Collins, CO 80523 USA
Banerjea, A
Bai, JR
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机构:Colorado State Univ, Dept Microbiol Immunol & Pathol, Ft Collins, CO 80523 USA
Bai, JR
Anderson, J
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机构:Colorado State Univ, Dept Microbiol Immunol & Pathol, Ft Collins, CO 80523 USA
Anderson, J
Li, MJ
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h-index: 0
机构:Colorado State Univ, Dept Microbiol Immunol & Pathol, Ft Collins, CO 80523 USA
Li, MJ
Rossi, J
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h-index: 0
机构:Colorado State Univ, Dept Microbiol Immunol & Pathol, Ft Collins, CO 80523 USA