title: Recent advances in RNAi-based strategies for therapy and prevention of HIV-1/AIDS.
abstract: RNA interference (RNAi) provides a powerful tool to silence specific gene expression and has been widely used to suppress host factors such as CCR5 and/or viral genes involved in HIV-1 replication. Newer nuclease-based gene-editing technologies, such as zinc finger nucleases (ZFN), transcription activator-like effector nucleases (TALEN) and the clustered regularly interspaced short palindromic repeats (CRISPR)/Cas9 system, also provide powerful tools to ablate specific genes. Because of differences in co-receptor usage and the high mutability of the HIV-1 genome, a combination of host factors and viral genes needs to be suppressed for effective prevention and treatment of HIV-1 infection. Whereas the continued presence of small interfering/short hairpin RNA (si/shRNA) mediators is needed for RNAi to be effective, the continued expression of nucleases in the gene-editing systems is undesirable. Thus, RNAi provides the only practical way for expression of multiple silencers in infected and uninfected cells, which is needed for effective prevention/treatment of infection. There have been several advances in the RNAi field in terms of si/shRNA design, targeted delivery to HIV-1 susceptible cells, and testing for efficacy in preclinical humanized mouse models. Here, we comprehensively review the latest advances in RNAi technology towards prevention and treatment of HIV-1.
authors.affiliation: Center of Emphasis in Infectious Disease, Department of Biomedical Sciences, Paul L. Foster School of Medicine, Texas Tech University Health Sciences Center, El Paso, TX 79905, USA. Electronic address: Manjunath.swamy@ttuhsc.edu.
authors.forename: Manjunath N
authors.initials: MN
authors.lastname: Swamy
authors.affiliation: Center of Emphasis in Infectious Disease, Department of Biomedical Sciences, Paul L. Foster School of Medicine, Texas Tech University Health Sciences Center, El Paso, TX 79905, USA.
authors.forename: Haoquan
authors.initials: H
authors.lastname: Wu
authors.affiliation: Center of Emphasis in Infectious Disease, Department of Biomedical Sciences, Paul L. Foster School of Medicine, Texas Tech University Health Sciences Center, El Paso, TX 79905, USA. Electronic address: Premlata.shankar@ttuhsc.edu.
authors.forename: Premlata
authors.initials: P
authors.lastname: Shankar
mesh_terms: D000163:Acquired Immunodeficiency Syndrome / Q000235:genetics / Q000517:prevention & control* / Q000628:therapy* / Q000821:virology; D000818:Animals; D064112:Clustered Regularly Interspaced Short Palindromic Repeats; D000072669:Gene Editing; D015658:HIV Infections / Q000235:genetics / Q000517:prevention & control* / Q000628:therapy* / Q000821:virology; D015497:HIV-1; D006801:Humans; D034622:RNA Interference; D034741:RNA, Small Interfering / Q000235:genetics; D000067492:RNAi Therapeutics / Q000379:methods*
chemical_list: D034741:RNA, Small Interfering
keywords: AgoshRNA; CCR5; HIV-1; Humanized mice; Lentiviral vector; RNAi; shRNA-miR; si/shRNA