ArticleMolecular therapy. Nucleic acids2013
Site-specific Genome Editing in PBMCs With PLGA Nanoparticle-delivered PNAs Confers HIV-1 Resistance in Humanized Mice.
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 26 papers.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
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Who cites it
26 citing papers in PubMed, 44 citations in OpenAlex.
- Multiple Charged Purine (MCP) PNA as a Simple Mode for Cellular Uptake.ACS polymers Au · 2026Article
- Advances in peptide nucleic acid for targeting RNA and genomic DNA.Cell reports. Physical science · 2026Article
- In pursuit of an HIV cure: from stem cell transplants to gene therapies.Frontiers in genome editing · 2025Review
- Next generation triplex-forming PNAs for site-specific genome editing of the F508del CFTR mutation.Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society · 2025Article
- Navigating Latency-Inducing Viral Infections: Therapeutic Targeting and Nanoparticle Utilization.Biomaterials research · 2024Review
- DNA recognition and induced genome modification by a hydroxymethyl-γ tail-clamp peptide nucleic acid.Cell reports. Physical science · 2023Article
- Cationic Calix[4]arene Vectors to Efficiently Deliver AntimiRNA Peptide Nucleic Acids (PNAs) and miRNA Mimics.Pharmaceutics · 2023Article
- In vivo correction of cystic fibrosis mediated by PNA nanoparticles.Science advances · 2022Article
- Antispacer peptide nucleic acids for sequence-specific CRISPR-Cas9 modulation.Nucleic acids research · 2022Article
- Surface conjugation of antibodies improves nanoparticle uptake in bronchial epithelial cells.PloS one · 2022Article
- Multifunctional Delivery Systems for Peptide Nucleic Acids.Pharmaceuticals (Basel, Switzerland) · 2020Review
- New Directions in Pulmonary Gene Therapy.Human gene therapy · 2020Review
- Peptide Nucleic Acids and Gene Editing: Perspectives on Structure and Repair.Molecules (Basel, Switzerland) · 2020Review
- Poly(Lactic-co-Glycolic Acid) Nanoparticle Delivery of Peptide Nucleic Acids In Vivo.Methods in molecular biology (Clifton, N.J.) · 2020Article
- Optimizing biodegradable nanoparticle size for tissue-specific delivery.Journal of controlled release : official journal of the Controlled Release Society · 2019Article
- In utero nanoparticle delivery for site-specific genome editing.Nature communications · 2018Article
- Peptide Nucleic Acids as a Tool for Site-Specific Gene Editing.Molecules (Basel, Switzerland) · 2018Review
- A novel mode for transcription inhibition mediated by PNA-induced R-loops with a model in vitro system.Biochimica et biophysica acta. Gene regulatory mechanisms · 2018Article
- Therapeutic Peptide Nucleic Acids: Principles, Limitations, and Opportunities.The Yale journal of biology and medicine · 2017Review
- Nanotechnology for delivery of peptide nucleic acids (PNAs).Journal of controlled release : official journal of the Controlled Release Society · 2016Review
Corrections and comments
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Authors and funding
10 authors at 3 institutions in 1 country.
Funding
Abstract
Biodegradable poly (lactic-co-glycolic acid) (PLGA) nanoparticles (NPs) encapsulating triplex-forming peptide nucleic acids (PNAs) and donor DNAs for recombination-mediated editing of the CCR5 gene were synthesized for delivery into human peripheral blood mononuclear cells (PBMCs). NPs containing the CCR5-targeting molecules efficiently entered PBMCs with low cytotoxicity. Deep sequencing revealed that a single treatment with the formulation resulted in a targeting frequency of 0.97% in the CCR5 gene and a low off-target frequency of 0.004% in the CCR2 gene, a 216-fold difference. NP-treated PBMCs efficiently engrafted immunodeficient NOD-scid IL-2rγ(-/-) mice, and the targeted CCR5 modification was detected in splenic lymphocytes 4 weeks posttransplantation. After infection with an R5-tropic strain of HIV-1, humanized mice with CCR5-NP-treated PBMCs displayed significantly higher levels of CD4(+) T cells and significantly reduced plasma viral RNA loads compared with control mice engrafted with mock-treated PBMCs. This work demonstrates the feasibility of PLGA-NP-encapsulated PNA-based gene-editing molecules for the targeted modification of CCR5 in human PBMCs as a platform for conferring HIV-1 resistance.Molecular Therapy-Nucleic Acids (2013) 2, e135; doi:10.1038/mtna.2013.59; published online 19 November 2013.
Identifiers
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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.