ArticleNucleic acids research2011
Efficient gene targeting mediated by a lentiviral vector-associated meganuclease.
Article in Nucleic acids research, 2011. 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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Who cites it
25 citing papers in PubMed, 42 citations in OpenAlex.
- Engineering a streamlined virus-like particle for programmable tissue-specific gene delivery.Nature communications · 2025Article
- Customizable virus-like particles deliver CRISPR-Cas9 ribonucleoprotein for effective ocular neovascular and Huntington's disease gene therapy.Nature nanotechnology · 2025Article
- Cell-targeted gene modification by delivery of CRISPR-Cas9 ribonucleoprotein complexes in pseudotyped lentivirus-derived nanoparticles.Molecular therapy. Nucleic acids · 2024Article
- Targeted delivery of CRISPR-Cas9 and transgenes enables complex immune cell engineering.Cell reports · 2021Article
- Gene Editing in Pluripotent Stem Cells and Their Derived Organoids.Stem cells international · 2021Review
- Review
- Highly efficient 'hit-and-run' genome editing with unconcentrated lentivectors carrying Vpr.Prot.Cas9 protein produced from RRE-containing transcripts.Nucleic acids research · 2020Article
- Ex Vivo COL7A1 Correction for Recessive Dystrophic Epidermolysis Bullosa Using CRISPR/Cas9 and Homology-Directed Repair.Molecular therapy. Nucleic acids · 2018Article
- Genome engineering: a new approach to gene therapy for neuromuscular disorders.Nature reviews. Neurology · 2017Review
- In Vivo Delivery Systems for Therapeutic Genome Editing.International journal of molecular sciences · 2016Review
- CRISPR/Cas9: An inexpensive, efficient loss of function tool to screen human disease genes in Xenopus.Developmental biology · 2015Article
- CRISPR-Cas9: A Revolutionary Tool for Cancer Modelling.International journal of molecular sciences · 2015Review
- Genome editing strategies: potential tools for eradicating HIV-1/AIDS.Journal of neurovirology · 2015Review
- Improved cell-penetrating zinc-finger nuclease proteins for precision genome engineering.Molecular therapy. Nucleic acids · 2015Article
- Lentiviral protein delivery of meganucleases in human cells mediates gene targeting and alleviates toxicity.Gene therapy · 2014Article
- Targeted genome editing by lentiviral protein transduction of zinc-finger and TAL-effector nucleases.eLife · 2014Article
- DNA transposition by protein transduction of the piggyBac transposase from lentiviral Gag precursors.Nucleic acids research · 2014Article
- Emerging gene editing strategies for Duchenne muscular dystrophy targeting stem cells.Frontiers in physiology · 2014Review
- Highly efficient gene knockout in mice and zebrafish with RNA-guided endonucleases.Genome research · 2014Article
- Driving DNA transposition by lentiviral protein transduction.Mobile genetic elements · 2014Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
5 authors at 4 institutions in 2 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Gene targeting can be achieved with lentiviral vectors delivering donor sequences along with a nuclease that creates a locus-specific double-strand break (DSB). Therapeutic applications of this system would require an appropriate control of the amount of endonuclease delivered to the target cells, and potentially toxic sustained expression must be avoided. Here, we show that the nuclease can be transferred into cells as a protein associated with a lentiviral vector particle. I-SceI, a prototypic meganuclease from yeast, was incorporated into the virions as a fusion with Vpr, an HIV accessory protein. Integration-deficient lentiviral vectors containing the donor sequences and the I-SceI fusion protein were tested in reporter cells in which targeting events were scored by the repair of a puromycin resistance gene. Molecular analysis of the targeted locus indicated a 2-fold higher frequency of the expected recombination event when the nuclease was delivered as a protein rather than encoded by a separate vector. In both systems, a proportion of clones displayed multiple integrated copies of the donor sequences, either as tandems at the targeted locus or at unrelated loci. These integration patterns were dependent upon the mode of meganuclease delivery, suggesting distinct recombination processes.
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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.