ArticleMolecular therapy. Nucleic acids2013
Preclinical in vivo evaluation of the safety of a multi-shRNA-based gene therapy against HIV-1.
Article in Molecular therapy. Nucleic acids, 2013. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 27 papers.
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Who cites it
27 citing papers in PubMed.
- Influence of a 3' Terminal Ribozyme on AgoshRNA Biogenesis and Activity.Molecular therapy. Nucleic acids · 2019Article
- Baboon envelope LVs efficiently transduced human adult, fetal, and progenitor T cells and corrected SCID-X1 T-cell deficiency.Blood advances · 2019Article
- Sixth European Seminar in Virology on Virus⁻Host Interaction at Single Cell and Organism Level.Viruses · 2018Article
- Barriers and strategies to achieve a cure for HIV.The lancet. HIV · 2018Review
- APOBEC Enzymes as Targets for Virus and Cancer Therapy.Cell chemical biology · 2018Review
- RNA Interference Therapies for an HIV-1 Functional Cure.Viruses · 2017Review
- Combinatorial CRISPR-Cas9 and RNA Interference Attack on HIV-1 DNA and RNA Can Lead to Cross-Resistance.Antimicrobial agents and chemotherapy · 2017Article
- Dual-targeting nanoparticles with excellent gene transfection efficiency for gene therapy of peritoneal metastasis of colorectal cancer.Oncotarget · 2017Article
- Dicer-independent processing of small RNA duplexes: mechanistic insights and applications.Nucleic acids research · 2017Review
- Silencing of HIV-1 by AgoshRNA molecules.Gene therapy · 2017Article
- Multifunctional Nucleus-targeting Nanoparticles with Ultra-high Gene Transfection Efficiency forTheranostics · 2017Article
- Review
- Recent advances in RNAi-based strategies for therapy and prevention of HIV-1/AIDS.Advanced drug delivery reviews · 2016Review
- Stem cell-based therapies for HIV/AIDS.Advanced drug delivery reviews · 2016Review
- Recent advances of biomaterials in biotherapy.Regenerative biomaterials · 2016Review
- Development of Lentiviral Vectors Simultaneously Expressing Multiple siRNAs Against CCR5, vif and tat/rev Genes for an HIV-1 Gene Therapy Approach.Molecular therapy. Nucleic acids · 2016Article
- ABX464: a good drug candidate instead of a magic bullet.Retrovirology · 2015Article
- Bone Marrow Gene Therapy for HIV/AIDS.Viruses · 2015Review
- The impact of HIV-1 genetic diversity on the efficacy of a combinatorial RNAi-based gene therapy.Gene therapy · 2015Article
- New challenges in modern vaccinology.BMC immunology · 2015Review
Corrections and comments
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Authors and funding
12 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Highly active antiretroviral therapy (HAART) has significantly improved the quality of life and the life expectancy of HIV-infected individuals. Still, drug-induced side effects and emergence of drug-resistant viral variants remain important issues that justify the exploration of alternative therapeutic options. One strategy consists of a gene therapy based on RNA interference to induce the sequence-specific degradation of the HIV-1 RNA genome. We have selected four potent short hairpin RNA (shRNA) candidates targeting the viral capside, integrase, protease and tat/rev open-reading frames and screened the safety of them during human hematopoietic cell development, both in vitro and in vivo. Although the four shRNA candidates appeared to be safe in vitro, one shRNA candidate impaired the in vivo development of the human immune system in Balb/c Rag2(-/-)IL-2Rγc(-/-) (BRG) mice. The three remaining shRNA candidates were combined into one single lentiviral vector (LV), and safety of the shRNA combination during human hematopoietic cell development was confirmed. Overall, we demonstrate here the preclinical in vivo safety of a LV expressing three shRNAs against HIV-1, which is proposed for a future Phase I clinical trial.Molecular Therapy-Nucleic Acids (2013) 2, e120; doi:10.1038/mtna.2013.48; published online 3 September 2013.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.