ArticleNature communications2016
In vivo correction of anaemia in β-thalassemic mice by γPNA-mediated gene editing with nanoparticle delivery.
Article in Nature communications, 2016. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 104 papers.
What it found
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Who cites it
104 citing papers in PubMed, 181 citations in OpenAlex.
- Sequence-specific targeting of VEGFA genomic DNA to inhibit tumor growth.NPJ precision oncology · 2026Article
- Multiple Charged Purine (MCP) PNA as a Simple Mode for Cellular Uptake.ACS polymers Au · 2026Article
- Engineered cargo-free nanoparticle decoys for phagocytic modulation of macrophages.Journal of controlled release : official journal of the Controlled Release Society · 2026Article
- Engineered miR-122 inhibitors preserve endothelial mitochondrial function and prevent vascular dysfunction in obesity-associated prediabetes.Molecular therapy. Nucleic acids · 2026Article
- Prenatal Intraportal Delivery of Polymeric Nanoparticles to Fetal Rhesus Monkeys (Human gene therapy · 2026Article
- Article
- Advances in peptide nucleic acid for targeting RNA and genomic DNA.Cell reports. Physical science · 2026Article
- Sequence-Directed Transcriptional Silencing Using Gamma Peptide Nucleic Acids.Methods in molecular biology (Clifton, N.J.) · 2026Article
- Fmoc-Based Assembly of PNA Oligomers: Manual and Microwave-Assisted Automated Synthesis.Methods in molecular biology (Clifton, N.J.) · 2026Article
- Cooperative Mismatch Discrimination by PNA and DNAzyme Enables High-Fidelity Cleavage of Plasmid DNA.Biochemistry · 2025Article
- Gene editing using gamma modified PNA: HBB gene as a model.Journal, genetic engineering & biotechnology · 2025Article
- Investigation of the protein corona and biodistribution profile of polymeric nanoparticles for intra-amniotic delivery.Biomaterials · 2025Article
- Systemic in utero gene editing as a treatment for cystic fibrosis.Proceedings of the National Academy of Sciences of the United States of America · 2025Article
- Fusions of Catalytically Inactive RusA to FokI Nuclease Coupled with PNA Enable Programmable Site-Specific Double-Stranded DNA Breaks.ACS omega · 2025Article
- Transcription regulation by RNA-induced structural strain in duplex DNA.Nucleic acids research · 2025Article
- Advanced delivery systems for gene editing: A comprehensive review from the GenE-HumDi COST Action Working Group.Molecular therapy. Nucleic acids · 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 therapeutic potential of sγPNA-141: Pharmacodynamics and mechanistic insights during ischemic stroke recovery.Molecular therapy. Nucleic acids · 2024Article
- Peptide Nucleic Acid-Mediated Regulation of CRISPR-Cas9 Specificity.Nucleic acid therapeutics · 2024Article
- Enhancing RNA inhibitory activity using clamp-G-modified nucleobases.Cell reports. Physical science · 2024Article
44 more citing papers are in PubMed but not listed here.
Corrections and comments
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Authors and funding
22 authors at 3 institutions in 1 country.
Funding
Abstract
The blood disorder, β-thalassaemia, is considered an attractive target for gene correction. Site-specific triplex formation has been shown to induce DNA repair and thereby catalyse genome editing. Here we report that triplex-forming peptide nucleic acids (PNAs) substituted at the γ position plus stimulation of the stem cell factor (SCF)/c-Kit pathway yielded high levels of gene editing in haematopoietic stem cells (HSCs) in a mouse model of human β-thalassaemia. Injection of thalassemic mice with SCF plus nanoparticles containing γPNAs and donor DNAs ameliorated the disease phenotype, with sustained elevation of blood haemoglobin levels into the normal range, reduced reticulocytosis, reversal of splenomegaly and up to 7% β-globin gene correction in HSCs, with extremely low off-target effects. The combination of nanoparticle delivery, next generation γPNAs and SCF treatment may offer a minimally invasive treatment for genetic disorders of the blood that can be achieved safely and simply by intravenous administration.
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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.