ArticleMolecular therapy : the journal of the American Society of Gene Therapy2017
Eliminating HIV-1 Packaging Sequences from Lentiviral Vector Proviruses Enhances Safety and Expedites Gene Transfer for Gene Therapy.
Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 25 papers.
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25 citing papers in PubMed, 40 citations in OpenAlex.
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- The Era of Gene Therapy: The Advancement of Lentiviral Vectors and Their Pseudotyping.Viruses · 2025Review
- Advances in HIV Gene Therapy.International journal of molecular sciences · 2024Review
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- Lentiviral Vectors for Ocular Gene Therapy.Pharmaceutics · 2022Review
- Development of SARS-CoV-2 packaged RNA reference material for nucleic acid testing.Analytical and bioanalytical chemistry · 2022Article
- Lentiviral Vectors for T Cell Engineering: Clinical Applications, Bioprocessing and Future Perspectives.Viruses · 2021Review
- HIV-1 sequences in lentiviral vector genomes can be substantially reduced without compromising transduction efficiency.Scientific reports · 2021Article
- Gene Therapy for Lysosomal Storage Disorders: Ongoing Studies and Clinical Development.Biomolecules · 2021Review
- The TOP vector: a new high-titer lentiviral construct for delivery of sgRNAs and transgenes to primary T cells.Molecular therapy. Methods & clinical development · 2021Article
- Re-structuring lentiviral vectors to express genomic RNA via cap-dependent translation.Molecular therapy. Methods & clinical development · 2021Article
- Gene therapy for inherited metabolic diseases.Journal of mother and child · 2020Review
- Large-Scale Production of Lentiviral Vectors: Current Perspectives and Challenges.Pharmaceutics · 2020Review
- Nanoparticles presenting clusters of CD4 expose a universal vulnerability of HIV-1 by mimicking target cells.Proceedings of the National Academy of Sciences of the United States of America · 2020Article
- Use of Heterologous Vesiculovirus G Proteins Circumvents the Humoral Anti-envelope Immunity in Lentivector-Based In Vivo Gene Delivery.Molecular therapy. Nucleic acids · 2019Article
- Production of lentiviral vectors using novel, enzymatically produced, linear DNA.Gene therapy · 2019Article
- Foamy Virus Vectors Transduce Visceral Organs and Hippocampal Structures following In Vivo Delivery to Neonatal Mice.Molecular therapy. Nucleic acids · 2018Article
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11 authors at 5 institutions in 4 countries.
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Abstract
Lentiviral vector genomic RNA requires sequences that partially overlap wild-type HIV-1 gag and env genes for packaging into vector particles. These HIV-1 packaging sequences constitute 19.6% of the wild-type HIV-1 genome and contain functional cis elements that potentially compromise clinical safety. Here, we describe the development of a novel lentiviral vector (LTR1) with a unique genomic structure designed to prevent transfer of HIV-1 packaging sequences to patient cells, thus reducing the total HIV-1 content to just 4.8% of the wild-type genome. This has been achieved by reconfiguring the vector to mediate reverse-transcription with a single strand transfer, instead of the usual two, and in which HIV-1 packaging sequences are not copied. We show that LTR1 vectors offer improved safety in their resistance to remobilization in HIV-1 particles and reduced frequency of splicing into human genes. Following intravenous luciferase vector administration to neonatal mice, LTR1 sustained a higher level of liver transgene expression than an equivalent dose of a standard lentivirus. LTR1 vectors produce reverse-transcription products earlier and start to express transgenes significantly quicker than standard lentiviruses after transduction. Finally, we show that LTR1 is an effective lentiviral gene therapy vector as demonstrated by correction of a mouse hemophilia B model.
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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.