| Literature DB >> 27151309 |
Olivier Pernet1, Swati Seth Yadav2, Dong Sung An3.
Abstract
One of the current focuses in HIV/AIDS research is to develop a novel therapeutic strategy that can provide a life-long 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.Entities:
Keywords: Anti-HIV genes; CRISPR/Cas9; Genome editing technologies; HIV; Hematopoietic stem progenitor cells; RNA interference; TALEN; Zinc finger nucleases
Mesh：
Year: 2016 PMID： 27151309 PMCID： PMC4935568 DOI： 10.1016/j.addr.2016.04.027
Source DB: PubMed Journal: Adv Drug Deliv Rev ISSN： 0169-409X Impact factor: 15.470