ArticleGene therapy2013
Systemic delivery of triplex-forming PNA and donor DNA by nanoparticles mediates site-specific genome editing of human hematopoietic cells in vivo.
Article in Gene therapy, 2013. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 52 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
52 citing papers in PubMed, 77 citations in OpenAlex.
- Fmoc-Based Assembly of PNA Oligomers: Manual and Microwave-Assisted Automated Synthesis.Methods in molecular biology (Clifton, N.J.) · 2026Article
- Gene editing using gamma modified PNA: HBB gene as a model.Journal, genetic engineering & biotechnology · 2025Article
- Recent Cutting-Edge Technologies for the Delivery of Peptide Nucleic Acid.Chemistry (Weinheim an der Bergstrasse, Germany) · 2025Review
- 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
- Exploring the DNAHeliyon · 2024Article
- The Development of Non-natural Type Nucleoside to Stabilize Triplex DNA Formation against CG and TA Inversion Site.Current medicinal chemistry · 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
- Genome Editing for Cystic Fibrosis.Cells · 2023Review
- Nanoparticle-mediated genome editing in single-cell embryos via peptide nucleic acids.Bioengineering & translational medicine · 2023Article
- Spotlight on Genetic Kidney Diseases: A Call for Drug Delivery and Nanomedicine Solutions.ACS nano · 2023Review
- Review
- 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
- Review
- Surface conjugation of antibodies improves nanoparticle uptake in bronchial epithelial cells.PloS one · 2022Article
- Nanoparticles targeting hematopoietic stem and progenitor cells: Multimodal carriers for the treatment of hematological diseases.Frontiers in genome editing · 2022Review
- Peptide nucleic acid-dependent artifact can lead to false-positive triplex gene editing signals.Proceedings of the National Academy of Sciences of the United States of America · 2021Article
- A narrative review of in utero gene therapy: advances, challenges, and future considerations.Translational pediatrics · 2021Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
12 authors at 3 institutions in 1 country.
Funding
Abstract
In vivo delivery is a major barrier to the use of molecular tools for gene modification. Here we demonstrate site-specific gene editing of human cells in vivo in hematopoietic stem cell-engrafted NOD.Cg-Prkdc(scid)IL2rγ(tm1Wjl) (abbreviated NOD-scid IL2rγ(null)) mice, using biodegradable nanoparticles loaded with triplex-forming peptide nucleic acids (PNAs) and single-stranded donor DNA molecules. In vitro screening showed greater efficacy of nanoparticles containing PNAs/DNAs together over PNA-alone or DNA-alone. Intravenous injection of particles containing PNAs/DNAs produced modification of the human CCR5 gene in hematolymphoid cells in the mice, with modification confirmed at the genomic DNA, mRNA and functional levels. Deep sequencing revealed in vivo modification of the CCR5 gene at frequencies of 0.43% in hematopoietic cells in the spleen and 0.05% in the bone marrow: off-target modification in the partially homologous CCR2 gene was two orders of magnitude lower. We also induced specific modification in the β-globin gene using nanoparticles carrying β-globin-targeted PNAs/DNAs, demonstrating this method's versatility. In vivo testing in an enhanced green fluorescent protein-β-globin reporter mouse showed greater activity of nanoparticles containing PNAs/DNAs together over DNA only. Direct in vivo gene modification, such as we demonstrate here, would allow for gene therapy in systemic diseases or in cells that cannot be manipulated ex vivo.
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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.