Stem cell-based therapies for HIV/AIDS

作者：Pernet Olivier; Yadav Swati Seth; An Dong Sung

来源：Advanced Drug Delivery Reviews, 2016, 103: 187-201.

DOI：10.1016/j.addr.2016.04.027

• HIV
• Hematopoietic stem progenitor cells
• Anti-HIV genes
• RNA interference
• Genome editing technologies
• Zinc finger nucleases
• TALEN
• CRISPR/Cas9

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.

• 出版日期2016-8-1