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Molecules 2018, 23(3), 632; https://doi.org/10.3390/molecules23030632

Peptide Nucleic Acids as a Tool for Site-Specific Gene Editing

1
Department of Biomedical Engineering, Yale University, New Haven, CT 06511, USA
2
Department of Genetics, Yale University School of Medicine, New Haven, CT 06520, USA
3
Department of Therapeutic Radiology, Yale University School of Medicine, New Haven, CT 06520, USA
*
Author to whom correspondence should be addressed.
Received: 21 February 2018 / Revised: 8 March 2018 / Accepted: 10 March 2018 / Published: 11 March 2018
(This article belongs to the Special Issue Molecular Properties and the Applications of Peptide Nucleic Acids)
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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. View Full-Text
Keywords: peptide nucleic acids; PNA; gene editing; nanoparticles; β-thalassemia; sickle cell disease; cystic fibrosis; CCR5; PLGA; Duchenne muscular dystrophy; triplex peptide nucleic acids; PNA; gene editing; nanoparticles; β-thalassemia; sickle cell disease; cystic fibrosis; CCR5; PLGA; Duchenne muscular dystrophy; triplex
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This is an open access article distributed under the Creative Commons Attribution License which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited. (CC BY 4.0).
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Ricciardi, A.S.; Quijano, E.; Putman, R.; Saltzman, W.M.; Glazer, P.M. Peptide Nucleic Acids as a Tool for Site-Specific Gene Editing. Molecules 2018, 23, 632.

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