ReviewMolecules (Basel, Switzerland)2018
Peptide Nucleic Acids as a Tool for Site-Specific Gene Editing.
Review in Molecules (Basel, Switzerland), 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 35 papers.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
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
35 citing papers in PubMed, 67 citations in OpenAlex.
- Gene editing using gamma modified PNA: HBB gene as a model.Journal, genetic engineering & biotechnology · 2025Article
- Matrix-assisted Laser Desorption Ionization Mass Spectrometry for Detection of α/β-Chain Changes From Human Hemoglobin: An Exploratory Study.Analytical science advances · 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
- 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
- Fire Burn and Cauldron Bubble: What Is in Your Genome Editing Brew?Biochemistry · 2023Review
- Programmable site-specific DNA double-strand breaks via PNA-assisted prokaryotic Argonautes.Nucleic acids research · 2023Article
- Nanoparticle-mediated genome editing in single-cell embryos via peptide nucleic acids.Bioengineering & translational medicine · 2023Article
- Variability in genome-engineering source materials: consider your starting point.Synthetic biology (Oxford, England) · 2023Review
- Perspectives on conformationally constrained peptide nucleic acid (PNA): insights into the structural design, properties and applications.RSC chemical biology · 2022Review
- Surface conjugation of antibodies improves nanoparticle uptake in bronchial epithelial cells.PloS one · 2022Article
- Therapeutic Potential of Chemically Modified, Synthetic, Triplex Peptide Nucleic Acid-Based Oncomir Inhibitors for Cancer Therapy.Cancer research · 2021Article
- 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
- Cardiomyocyte Death and Genome-Edited Stem Cell Therapy for Ischemic Heart Disease.Stem cell reviews and reports · 2021Review
- Efficient CRISPR-Cas9-based genome editing of β-globin gene on erythroid cells from homozygous βMolecular therapy. Methods & clinical development · 2021Article
- Cell and Gene Therapy for Anemia: Hematopoietic Stem Cells and Gene Editing.International journal of molecular sciences · 2021Review
- A narrative review of in utero gene therapy: advances, challenges, and future considerations.Translational pediatrics · 2021Review
- Emerging technologies for cystic fibrosis transmembrane conductance regulator restoration in all people with CF.Pediatric pulmonology · 2021Review
- Knowledge From London and Berlin: Finding Threads to a Functional HIV Cure.Frontiers in immunology · 2021Review
- On the Corner of Models and Cure: Gene Editing in Cystic Fibrosis.Frontiers in pharmacology · 2021Review
- "Protein" no longer means what it used to.Current research in structural biology · 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
5 authors at 1 institution in 1 country.
Funding
Abstract
Peptide nucleic acids (PNAs) can bind duplex DNA in a sequence-targeted manner, forming a triplex structure capable of inducing DNA repair and producing specific genome modifications. Since the first description of PNA-mediated gene editing in cell free extracts, PNAs have been used to successfully correct human disease-causing mutations in cell culture and in vivo in preclinical mouse models. Gene correction via PNAs has resulted in clinically-relevant functional protein restoration and disease improvement, with low off-target genome effects, indicating a strong therapeutic potential for PNAs in the treatment or cure of genetic disorders. This review discusses the progress that has been made in developing PNAs as an effective, targeted agent for gene editing, with an emphasis on recent in vivo, nanoparticle-based strategies.
Indexed as
Identifiers
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.