ArticleMolecular therapy : the journal of the American Society of Gene Therapy2012
Targeted gene modification of hematopoietic progenitor cells in mice following systemic administration of a PNA-peptide conjugate.
Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2012. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 30 papers.
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Who cites it
30 citing papers in PubMed, 47 citations in OpenAlex.
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- Advances in peptide nucleic acid for targeting RNA and genomic DNA.Cell reports. Physical science · 2026Article
- Dually Decorated Palmitate-Containing Lipid Nanoparticles for the Targeted Delivery of siRNAs against HER2 and Hsp27 in HER2Journal of medicinal chemistry · 2025Article
- DNA recognition and induced genome modification by a hydroxymethyl-γ tail-clamp peptide nucleic acid.Cell reports. Physical science · 2023Article
- Cationic Calix[4]arene Vectors to Efficiently Deliver AntimiRNA Peptide Nucleic Acids (PNAs) and miRNA Mimics.Pharmaceutics · 2023Article
- Automated Flow Synthesis of Peptide-PNA Conjugates.ACS central science · 2022Article
- Multifunctional Delivery Systems for Peptide Nucleic Acids.Pharmaceuticals (Basel, Switzerland) · 2020Review
- Cell Penetrating Peptides, Novel Vectors for Gene Therapy.Pharmaceutics · 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
- Potential of Peptide Nucleic Acids in Future Therapeutic Applications.Advanced pharmaceutical bulletin · 2018Review
- 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
- Nanotechnology for delivery of peptide nucleic acids (PNAs).Journal of controlled release : official journal of the Controlled Release Society · 2016Review
- miR-155 Overexpression Promotes Genomic Instability by Reducing High-fidelity Polymerase Delta Expression and Activating Error-Prone DSB Repair.Molecular cancer research : MCR · 2016Article
- Genetic treatment of a molecular disorder: gene therapy approaches to sickle cell disease.Blood · 2016Review
- Delivery of therapeutic oligonucleotides with cell penetrating peptides.Advanced drug delivery reviews · 2015Review
- The TTSMI database: a catalog of triplex target DNA sites associated with genes and regulatory elements in the human genome.Nucleic acids research · 2015Article
- Targeted genome modification via triple helix formation.Methods in molecular biology (Clifton, N.J.) · 2014Article
- Single-stranded γPNAs for in vivo site-specific genome editing via Watson-Crick recognition.Current gene therapy · 2014Article
Corrections and comments
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Authors and funding
5 authors at 1 institution in 1 country.
Funding
Abstract
Hematopoietic stem cell (HSC) gene therapy offers promise for the development of new treatments for a variety of hematologic disorders. However, efficient in vivo modification of HSCs has proved challenging, thus imposing constraints on the therapeutic potential of this approach. Herein, we provide a gene-targeting strategy that allows site-specific in vivo gene modification in the HSCs of mice. Through conjugation of a triplex-forming peptide nucleic acid (PNA) to the transport peptide, antennapedia (Antp), we achieved successful in vivo chromosomal genomic modification of hematopoietic progenitor cells, while still retaining intact differentiation capabilities. Following systemic administration of PNA-Antp conjugates, sequence-specific gene modification was observed in multiple somatic tissues as well as within multiple compartments of the hematopoietic system, including erythroid, myeloid, and lymphoid cell lineages. As a true functional measure of gene targeting in a long-term renewable HSC, we also demonstrate preserved genomic modification in the bone marrow and spleen of primary recipient mice following transplantation of bone marrow from PNA-Antp-treated donor mice. Our approach offers a minimally invasive alternative to ex vivo gene therapy, by eliminating the need for the complex steps of stem cell mobilization and harvesting, ex vivo manipulation, and transplantation of stem cells. Therefore, our approach may provide new options for individualized therapies in the treatment of monogenic hematologic diseases such as sickle cell anemia and thalassemia.
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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.