ArticleMolecular therapy : the journal of the American Society of Gene Therapy2014
Optimized lentiviral vectors for HIV gene therapy: multiplexed expression of small RNAs and inclusion of MGMT(P140K) drug resistance gene.
Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2014. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 14 papers, 1 of them a synthesis that pooled it.
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Who cites it
14 citing papers in PubMed, 1 synthesis or guideline pooled it, 31 citations in OpenAlex.
- Development of siRNA therapeutics to combat microbial infections: a bibliometric analysis.Frontiers in cellular and infection microbiology · 2025Pooled it
- Identifying active and inhibitor-resistant MGMT variants for gene therapy.American journal of human genetics · 2025Article
- Therapeutic Aptamers: Evolving to Find their Clinical Niche.Current medicinal chemistry · 2020Article
- Genetic Strategies for HIV Treatment and Prevention.Molecular therapy. Nucleic acids · 2018Review
- Incorporation of aptamers in the terminal loop of shRNAs yields an effective and novel combinatorial targeting strategy.Nucleic acids research · 2018Article
- RNA Interference Therapies for an HIV-1 Functional Cure.Viruses · 2017Review
- Stem cell-based therapies for HIV/AIDS.Advanced drug delivery reviews · 2016Review
- Recent advances in RNAi-based strategies for therapy and prevention of HIV-1/AIDS.Advanced drug delivery reviews · 2016Review
- Improvements and Limitations of Humanized Mouse Models for HIV Research: NIH/NIAID "Meet the Experts" 2015 Workshop Summary.AIDS research and human retroviruses · 2016Article
- Article
- Foamy viral vector integration sites in SCID-repopulating cells after MGMTP140K-mediated in vivo selection.Gene therapy · 2015Article
- The therapeutic application of CRISPR/Cas9 technologies for HIV.Expert opinion on biological therapy · 2015Review
- Stem cell gene therapy for HIV: strategies to inhibit viral entry and replication.Current HIV/AIDS reports · 2015Review
- Enhanced genetic modification of adult growth factor mobilized peripheral blood hematopoietic stem and progenitor cells with rapamycin.Stem cells translational medicine · 2014Article
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Authors and funding
8 authors at 2 institutions in 1 country.
Funding
Abstract
Gene therapy with hematopoietic stem and progenitor cells is a promising approach to engineering immunity to human immunodeficiency virus (HIV) that may lead to a functional cure for acquired immunodeficiency syndrome (AIDS). In support of this approach, we created lentiviral vectors with an engineered polycistronic platform derived from the endogenous MCM7 gene to express a diverse set of small antiviral RNAs and a drug resistance MGMT(P140K) marker. Multiple strategies for simultaneous expression of up to five RNA transgenes were tested. The placement and orientation of each transgene and its promoter were important determinants for optimal gene expression. Antiviral RNA expression from the MCM7 platform with a U1 promoter was sufficient to provide protection from R5-tropic HIV in macrophages and resulted in reduced hematopoietic toxicity compared with constructs expressing RNA from independent RNA polymerase III promoters. The addition of an HIV entry inhibitor and nucleolar TAR RNA decoy did not enhance antiviral potency over constructs that targeted only viral RNA transcripts. We also demonstrated selective enrichment of gene-modified cells in vivo using a humanized mouse model. The use of these less toxic, potent anti-HIV vectors expressing a drug selection marker is likely to enhance the in vivo efficacy of our stem cell gene therapy approach in treating HIV/AIDS.
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Registered trials
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