ArticleNature communications2018
In utero nanoparticle delivery for site-specific genome editing.
Article in Nature communications, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 98 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.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
98 citing papers in PubMed, 174 citations in OpenAlex.
- Focused Ultrasound is Poised to Take Non-Viral Gene Therapies to the Blood-Brain Barrier - and Beyond.Cell biomaterials · 2026Article
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- Considerations for early life genetic therapies in cystic fibrosis.American journal of physiology. Lung cellular and molecular physiology · 2026Review
- Engineered miR-122 inhibitors preserve endothelial mitochondrial function and prevent vascular dysfunction in obesity-associated prediabetes.Molecular therapy. Nucleic acids · 2026Article
- Gamma-FIT-PNAs as sensitive RNA probes.RSC chemical biology · 2026Article
- 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
- Prenatal Delivery of HIF-1α siRNA Using Transferrin-Modified Lipid Nanoparticles Alleviates Hypoxia-Induced Neurodevelopmental Abnormalities via PTEN/PI3K/AKT Signaling.International journal of nanomedicine · 2026Article
- Gene editing using gamma modified PNA: HBB gene as a model.Journal, genetic engineering & biotechnology · 2025Article
- Artificial Uterus and Artificial Embryos: Unsolved Tasks.Reproductive sciences (Thousand Oaks, Calif.) · 2025Review
- In utero therapy for spinal muscular atrophy: closer to clinical translation.Brain : a journal of neurology · 2025Review
- 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
- 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
- Synthetic-polymer-assisted antisense oligonucleotide delivery: targeted approaches for precision disease treatment.Beilstein journal of nanotechnology · 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
- Nanomedicine for Maternal and Fetal Health.Small (Weinheim an der Bergstrasse, Germany) · 2024Review
- Enhancing RNA inhibitory activity using clamp-G-modified nucleobases.Cell reports. Physical science · 2024Article
- Embryo and fetal gene editing: Technical challenges and progress toward clinical applications.Molecular therapy. Methods & clinical development · 2024Review
38 more citing papers are in PubMed but not listed here.
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
16 authors at 4 institutions in 1 country.
Funding
Abstract
Genetic diseases can be diagnosed early during pregnancy, but many monogenic disorders continue to cause considerable neonatal and pediatric morbidity and mortality. Early intervention through intrauterine gene editing, however, could correct the genetic defect, potentially allowing for normal organ development, functional disease improvement, or cure. Here we demonstrate safe intravenous and intra-amniotic administration of polymeric nanoparticles to fetal mouse tissues at selected gestational ages with no effect on survival or postnatal growth. In utero introduction of nanoparticles containing peptide nucleic acids (PNAs) and donor DNAs corrects a disease-causing mutation in the β-globin gene in a mouse model of human β-thalassemia, yielding sustained postnatal elevation of blood hemoglobin levels into the normal range, reduced reticulocyte counts, reversal of splenomegaly, and improved survival, with no detected off-target mutations in partially homologous loci. This work may provide the basis for a safe and versatile method of fetal gene editing for human monogenic disorders.
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What OpenQuestion holds
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.