Stem cell-based therapies for HIV/AIDS

被引:30
|
作者
Pernet, Olivier [1 ,2 ]
Yadav, Swati Seth [1 ,2 ]
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
关键词
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.
引用
收藏
页码:187 / 201
页数:15
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