ArticleNucleic acids research2020
Highly efficient 'hit-and-run' genome editing with unconcentrated lentivectors carrying Vpr.Prot.Cas9 protein produced from RRE-containing transcripts.
Article in Nucleic acids research, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 33 papers.
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Who cites it
33 citing papers in PubMed, 48 citations in OpenAlex.
- Optimized lentivirus-derived virus-like particles for efficient delivery of Cas9-based genome editors.Nucleic acids research · 2026Article
- Genome-wide screening reveals producer-cell modifications that improve virus-like particle production and delivery potency.Nature communications · 2026Article
- Bipolar CD4-targeted dual-DARPin-55/57 lipid nanoparticle enables efficient CRISPR/Cas-mediated HIV-1 DNA excision and reactivation blockade in latent CD4 T cell lines.Materials today. Bio · 2026Article
- Genome-wide screening reveals producer-cell modifications that improve virus-like particle production and delivery potency.bioRxiv : the preprint server for biology · 2026Article
- Review
- Targeted delivery of genome editors in vivo.Nature biotechnology · 2026Review
- Base and Prime Editing for Inherited Retinal Diseases: Delivery Platforms, Safety, Efficacy, and Translational Perspectives.Pharmaceutics · 2025Review
- Directed evolution of engineered virus-like particles with improved production and transduction efficiencies.Nature biotechnology · 2025Article
- An innovative approach using CRISPR-ribonucleoprotein packaged in virus-like particles to generate genetically engineered mouse models.Nature communications · 2025Article
- Mechanistic Insights into the Tools for Intracellular Protein Delivery.Chem & bio engineering · 2025Review
- Packaged delivery of CRISPR-Cas9 ribonucleoproteins accelerates genome editing.Nucleic acids research · 2025Article
- Synergestic Protein-Green synthesized Nanoparticles Nanosystems: A Sustainable and Safe Approach for Cancer Theranostics.Nanotheranostics · 2025Review
- Cell-targeted gene modification by delivery of CRISPR-Cas9 ribonucleoprotein complexes in pseudotyped lentivirus-derived nanoparticles.Molecular therapy. Nucleic acids · 2024Article
- In vivo human T cell engineering with enveloped delivery vehicles.Nature biotechnology · 2024Article
- Viral and nonviral nanocarriers forNano research · 2024Article
- Engineered virus-like particles for transient delivery of prime editor ribonucleoprotein complexes in vivo.Nature biotechnology · 2024Article
- An engineered baculoviral protein and DNA co-delivery system for CRISPR-based mammalian genome editing.Nucleic acids research · 2024Article
- Targeted nonviral delivery of genome editors in vivo.Proceedings of the National Academy of Sciences of the United States of America · 2024Review
- The RRE-Rev Module Has No Effect on the Packaging Efficiency of Cas9 and Gag Proteins into NanoMEDIC Virus-like Particles.Doklady biological sciences : proceedings of the Academy of Sciences of the USSR, Biological sciences sections · 2023Article
- Gene editing innovations and their applications in cardiomyopathy research.Disease models & mechanisms · 2023Review
Corrections and comments
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Authors and funding
2 authors at 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
The application of gene-editing technology is currently limited by the lack of safe and efficient methods to deliver RNA-guided endonucleases to target cells. We engineered lentivirus-based nanoparticles to co-package the U6-sgRNA template and the CRISPR-associated protein 9 (Cas9) fused with a virion-targeted protein Vpr (Vpr.Prot.Cas9), for simultaneous delivery to cells. Equal spatiotemporal control of the vpr.prot.cas9 and gag/pol gene expression (the presence of Rev responsive element, RRE) greatly enhanced the encapsidation of the fusion protein and resulted in the production of highly efficient lentivector nanoparticles. Transduction of the unconcentrated, Vpr.Prot.Cas9-containing vectors led to >98% disruption of the EGFP gene in reporter HEK293-EGFP cells with minimal cytotoxicity. Furthermore, we detected indels in the targeted endogenous loci at frequencies of up to 100% in cell lines derived from lymphocytes and monocytes and up to 15% in primary CD4+ T cells by high-throughput sequencing. This approach may provide a platform for the efficient, dose-controlled and tissue-specific delivery of genome editing enzymes to cells and it may be suitable for simultaneous endogenous gene disruption and a transgene delivery.
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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.