ArticleChemistry & biology2011
Targeted disruption of the CCR5 gene in human hematopoietic stem cells stimulated by peptide nucleic acids.
Article in Chemistry & biology, 2011. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 37 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
37 citing papers in PubMed, 59 citations in OpenAlex.
- Advances in peptide nucleic acid for targeting RNA and genomic DNA.Cell reports. Physical science · 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
- In pursuit of an HIV cure: from stem cell transplants to gene therapies.Frontiers in genome editing · 2025Review
- Recent Advancements in Development and Therapeutic Applications of Genome-Targeting Triplex-Forming Oligonucleotides and Peptide Nucleic Acids.Pharmaceutics · 2023Review
- DNA recognition and induced genome modification by a hydroxymethyl-γ tail-clamp peptide nucleic acid.Cell reports. Physical science · 2023Article
- 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
- Knowledge From London and Berlin: Finding Threads to a Functional HIV Cure.Frontiers in immunology · 2021Review
- Multifunctional Delivery Systems for Peptide Nucleic Acids.Pharmaceuticals (Basel, Switzerland) · 2020Review
- New Directions in Pulmonary Gene Therapy.Human gene therapy · 2020Review
- 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
- Antisense peptide nucleic acids as a potential anti-infective agent.Journal of microbiology (Seoul, Korea) · 2019Review
- Peptide Nucleic Acids as a Tool for Site-Specific Gene Editing.Molecules (Basel, Switzerland) · 2018Review
- Therapeutic Peptide Nucleic Acids: Principles, Limitations, and Opportunities.The Yale journal of biology and medicine · 2017Review
- Design of Tail-Clamp Peptide Nucleic Acid Tethered with Azobenzene Linker for Sequence-Specific Detection of Homopurine DNA.Molecules (Basel, Switzerland) · 2017Article
- Nanotechnology for delivery of peptide nucleic acids (PNAs).Journal of controlled release : official journal of the Controlled Release Society · 2016Review
- In vivo correction of anaemia in β-thalassemic mice by γPNA-mediated gene editing with nanoparticle delivery.Nature communications · 2016Article
- Cell-based gene therapy against HIV.Gene therapy · 2015Review
- The therapeutic application of CRISPR/Cas9 technologies for HIV.Expert opinion on biological therapy · 2015Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
11 authors at 4 institutions in 1 country.
Funding
Abstract
Peptide nucleic acids (PNAs) bind duplex DNA in a sequence-specific manner, creating triplex structures that can provoke DNA repair and produce genome modification. CCR5 encodes a chemokine receptor required for HIV-1 entry into human cells, and individuals carrying mutations in this gene are resistant to HIV-1 infection. Transfection of human cells with PNAs targeted to the CCR5 gene, plus donor DNAs designed to introduce stop codons mimicking the naturally occurring CCR5-delta32 mutation, produced 2.46% targeted gene modification. CCR5 modification was confirmed at the DNA, RNA, and protein levels and was shown to confer resistance to infection with HIV-1. Targeting of CCR5 was achieved in human CD34(+) hematopoietic stem cells (HSCs) with subsequent engraftment into mice and persistence of the gene modification more than four months posttransplantation. This work suggests a therapeutic strategy for CCR5 knockout in HSCs from HIV-1-infected individuals, rendering cells resistant to HIV-1 and preserving immune system function.
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.