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Peptide Nucleic Acids and Gene Editing: Perspectives on Structure and Repair

1
Department of Genetics, Yale University School of Medicine, New Haven, CT 06520, USA
2
Department of Therapeutic Radiology, Yale University School of Medicine, New Haven, CT 06520, USA
3
Department of Biomedical Engineering, Yale University, New Haven, CT 06511, USA
*
Author to whom correspondence should be addressed.
Academic Editor: Eylon Yavin
Molecules 2020, 25(3), 735; https://doi.org/10.3390/molecules25030735
Received: 16 January 2020 / Revised: 5 February 2020 / Accepted: 6 February 2020 / Published: 8 February 2020
(This article belongs to the Special Issue Peptide Nucleic Acids: Applications in Biomedical Sciences)
Unusual nucleic acid structures are salient triggers of endogenous repair and can occur in sequence-specific contexts. Peptide nucleic acids (PNAs) rely on these principles to achieve non-enzymatic gene editing. By forming high-affinity heterotriplex structures within the genome, PNAs have been used to correct multiple human disease-relevant mutations with low off-target effects. Advances in molecular design, chemical modification, and delivery have enabled systemic in vivo application of PNAs resulting in detectable editing in preclinical mouse models. In a model of β-thalassemia, treated animals demonstrated clinically relevant protein restoration and disease phenotype amelioration, suggesting a potential for curative therapeutic application of PNAs to monogenic disorders. This review discusses the rationale and advances of PNA technologies and their application to gene editing with an emphasis on structural biochemistry and repair. View Full-Text
Keywords: peptide nucleic acids; PNA; triplex; gene editing; structure; recombination; repair; nanoparticles; β-thalassemia; cystic fibrosis peptide nucleic acids; PNA; triplex; gene editing; structure; recombination; repair; nanoparticles; β-thalassemia; cystic fibrosis
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Economos, N.G.; Oyaghire, S.; Quijano, E.; Ricciardi, A.S.; Saltzman, W.M.; Glazer, P.M. Peptide Nucleic Acids and Gene Editing: Perspectives on Structure and Repair. Molecules 2020, 25, 735.

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