ArticleNucleic acids research2009
Targeted correction of a thalassemia-associated beta-globin mutation induced by pseudo-complementary peptide nucleic acids.
Article in Nucleic acids research, 2009. 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, 52 citations in OpenAlex.
- 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
- Recognition of mismatched sites in double-stranded DNA by a pair of partially noncomplementary peptide nucleic acids.Chemistry letters · 2024Article
- Recent Advancements in Development and Therapeutic Applications of Genome-Targeting Triplex-Forming Oligonucleotides and Peptide Nucleic Acids.Pharmaceutics · 2023Review
- Article
- Correction of the aprt Gene Using Repair-Polypurine Reverse Hoogsteen Hairpins in Mammalian Cells.Molecular therapy. Nucleic acids · 2020Article
- 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
- Applications of PNA-laden nanoparticles for hematological disorders.Cellular and molecular life sciences : CMLS · 2019Review
- Incorporating uracil and 5-halouracils into short peptide nucleic acids for enhanced recognition of A-U pairs in dsRNAs.Nucleic acids research · 2018Article
- Peptide Nucleic Acids as a Tool for Site-Specific Gene Editing.Molecules (Basel, Switzerland) · 2018Review
- Polypurine reverse-Hoogsteen (PPRH) oligonucleotides can form triplexes with their target sequences even under conditions where they fold into G-quadruplexes.Scientific reports · 2017Article
- Oligonucleotide Therapies: The Past and the Present.Human gene therapy · 2015Review
- Generation and Characterization of a Transgenic Mouse Carrying a Functional Human β -Globin Gene with the IVSI-6 Thalassemia Mutation.BioMed research international · 2015Article
- Chemical modifications of artificial restriction DNA cutter (ARCUT) to promote its in vivo and in vitro applications.Artificial DNA, PNA & XNA · 2014Review
- Repair of single-point mutations by polypurine reverse Hoogsteen hairpins.Human gene therapy methods · 2014Article
- Targeted genome modification via triple helix formation.Methods in molecular biology (Clifton, N.J.) · 2014Article
- Improved bioactivity of G-rich triplex-forming oligonucleotides containing modified guanine bases.Artificial DNA, PNA & XNA · 2014Article
- Single-stranded γPNAs for in vivo site-specific genome editing via Watson-Crick recognition.Current gene therapy · 2014Article
- Local delivery of gene-modifying triplex-forming molecules to the epidermis.The Journal of investigative dermatology · 2013Article
- Targeted gene modification of hematopoietic progenitor cells in mice following systemic administration of a PNA-peptide conjugate.Molecular therapy : the journal of the American Society of Gene Therapy · 2012Article
Corrections and comments
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Authors and funding
9 authors at 3 institutions in 2 countries.
Funding
Abstract
Beta-thalassemia is a genetic disorder caused by mutations in the beta-globin gene. Triplex-forming oligonucleotides and triplex-forming peptide nucleic acids (PNAs) have been shown to stimulate recombination in mammalian cells via site-specific binding and creation of altered helical structures that provoke DNA repair. However, the use of these molecules for gene targeting requires homopurine tracts to facilitate triple helix formation. Alternatively, to achieve binding to mixed-sequence target sites for the induced gene correction, we have used pseudo-complementary PNAs (pcPNAs). Due to steric hindrance, pcPNAs are unable to form pcPNA-pcPNA duplexes but can bind to complementary DNA sequences via double duplex-invasion complexes. We demonstrate here that pcPNAs, when co-transfected with donor DNA fragments, can promote single base pair modification at the start of the second intron of the beta-globin gene. This was detected by the restoration of proper splicing of transcripts produced from a green fluorescent protein-beta globin fusion gene. We also demonstrate that pcPNAs are effective in stimulating recombination in human fibroblast cells in a manner dependent on the nucleotide excision repair factor, XPA. These results suggest that pcPNAs can be effective tools to induce heritable, site-specific modification of disease-related genes in human cells without purine sequence restriction.
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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.