title: Stem cell-based therapies for HIV/AIDS.
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.
authors.affiliation: School of Nursing, University of California Los Angeles, 188 BSRB, 615 Charles E. Young Dr. South, Los Angeles, CA 90095, USA; UCLA AIDS Institute, 188 BSRB, 615 Charles E. Young Dr. South, Los Angeles, CA 90095, USA. Electronic address: opernet@ucla.edu.
authors.forename: Olivier
authors.initials: O
authors.lastname: Pernet
authors.affiliation: School of Nursing, University of California Los Angeles, 188 BSRB, 615 Charles E. Young Dr. South, Los Angeles, CA 90095, USA; UCLA AIDS Institute, 188 BSRB, 615 Charles E. Young Dr. South, Los Angeles, CA 90095, USA. Electronic address: swatisyadav@ucla.edu.
authors.forename: Swati Seth
authors.initials: SS
authors.lastname: Yadav
authors.affiliation: School of Nursing, University of California Los Angeles, 188 BSRB, 615 Charles E. Young Dr. South, Los Angeles, CA 90095, USA; UCLA AIDS Institute, 188 BSRB, 615 Charles E. Young Dr. South, Los Angeles, CA 90095, USA; Hematology-Oncology, The Department of Medicine, David Geffen School of Medicine at UCLA, 188 BSRB, 615 Charles E. Young Dr. South, Los Angeles, CA 90095, USA. Electronic address: an@ucla.edu.
authors.forename: Dong Sung
authors.initials: DS
authors.lastname: An
mesh_terms: D000163:Acquired Immunodeficiency Syndrome / Q000235:genetics / Q000276:immunology* / Q000473:pathology / Q000628:therapy*; D000818:Animals; D015316:Genetic Therapy / Q000379:methods; D015658:HIV Infections / Q000235:genetics / Q000276:immunology* / Q000628:therapy*; D018380:Hematopoietic Stem Cell Transplantation / Q000379:methods*; D006412:Hematopoietic Stem Cells / Q000166:cytology / Q000276:immunology* / Q000378:metabolism*; D006801:Humans
keywords: Anti-HIV genes; CRISPR/Cas9; Genome editing technologies; HIV; Hematopoietic stem progenitor cells; RNA interference; TALEN; Zinc finger nucleases