Peptide Nucleic Acids as a Tool for Site-Specific Gene Editing
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...
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| Published in | Molecules (Basel, Switzerland) Vol. 23; no. 3; p. 632 |
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| Main Authors | , , , , |
| Format | Journal Article |
| Language | English |
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11.03.2018
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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. |
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| AbstractList | 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. 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.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. |
| Author | Quijano, Elias Saltzman, W. Putman, Rachael Ricciardi, Adele Glazer, Peter |
| AuthorAffiliation | 3 Department of Therapeutic Radiology, Yale University School of Medicine, New Haven, CT 06520, USA 2 Department of Genetics, Yale University School of Medicine, New Haven, CT 06520, USA; elias.quijano@yale.edu 1 Department of Biomedical Engineering, Yale University, New Haven, CT 06511, USA; adele.ricciardi@yale.edu (A.S.R.); rachael.putman@yale.edu (R.P.); mark.saltzman@yale.edu (W.M.S.) |
| AuthorAffiliation_xml | – name: 1 Department of Biomedical Engineering, Yale University, New Haven, CT 06511, USA; adele.ricciardi@yale.edu (A.S.R.); rachael.putman@yale.edu (R.P.); mark.saltzman@yale.edu (W.M.S.) – name: 2 Department of Genetics, Yale University School of Medicine, New Haven, CT 06520, USA; elias.quijano@yale.edu – name: 3 Department of Therapeutic Radiology, Yale University School of Medicine, New Haven, CT 06520, USA |
| Author_xml | – sequence: 1 givenname: Adele orcidid: 0000-0003-2637-8522 surname: Ricciardi fullname: Ricciardi, Adele – sequence: 2 givenname: Elias surname: Quijano fullname: Quijano, Elias – sequence: 3 givenname: Rachael surname: Putman fullname: Putman, Rachael – sequence: 4 givenname: W. surname: Saltzman fullname: Saltzman, W. – sequence: 5 givenname: Peter orcidid: 0000-0003-4525-5560 surname: Glazer fullname: Glazer, Peter |
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| Keywords | PNA PLGA nanoparticles Duchenne muscular dystrophy cystic fibrosis CCR5 gene editing triplex β-thalassemia peptide nucleic acids sickle cell disease |
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| SubjectTerms | CCR5 cystic fibrosis Duchenne muscular dystrophy gene editing Genome editing Genomes nanoparticles peptide nucleic acids PLGA PNA Review sickle cell disease triplex β-thalassemia |
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| Title | Peptide Nucleic Acids as a Tool for Site-Specific Gene Editing |
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