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2 results on '"Rachael Putman"'

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

Author

Adele S. Ricciardi, Elias Quijano, Rachael Putman, W. Mark Saltzman, and Peter M. Glazer

Subjects
peptide nucleic acids, PNA, gene editing, nanoparticles, β-thalassemia, sickle cell disease, cystic fibrosis, CCR5, PLGA, Duchenne muscular dystrophy, triplex, Organic chemistry, QD241-441
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.

Published
2018
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2. Peptide Nucleic Acids as a Tool for Site-Specific Gene Editing

Author

Rachael Putman, Adele S. Ricciardi, Elias Quijano, Peter M. Glazer, and William Mark Saltzman

Subjects
Duchenne muscular dystrophy, 0301 basic medicine, Dna duplex, DNA repair, Pharmaceutical Science, Peptide, Computational biology, Genome, Article, Analytical Chemistry, cystic fibrosis, lcsh:QD241-441, Mice, 03 medical and health sciences, 0302 clinical medicine, lcsh:Organic chemistry, Genome editing, In vivo, Drug Discovery, Animals, Humans, triplex, Genetic Predisposition to Disease, Physical and Theoretical Chemistry, Gene, chemistry.chemical_classification, peptide nucleic acids, gene editing, Chemistry, Organic Chemistry, PLGA, DNA, humanities, 3. Good health, Disease Models, Animal, 030104 developmental biology, Chemistry (miscellaneous), 030220 oncology & carcinogenesis, β-thalassemia, Mutagenesis, Site-Directed, Nucleic acid, Nucleic Acid Conformation, Molecular Medicine, nanoparticles, sickle cell disease, CCR5, PNA
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.

Published
2018
Full Text
View/download PDF

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