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

• Published in: Molecules (Basel, Switzerland) Vol. 23; no. 3; p. 632
• Main Authors: Ricciardi, Adele, Quijano, Elias, Putman, Rachael, Saltzman, W., Glazer, Peter
• Format: Journal Article
• Language: English
• Published: Switzerland MDPI AG 11.03.2018; MDPI
• Subjects: CCR5; cystic fibrosis; Duchenne muscular dystrophy; gene editing; Genome editing; Genomes; nanoparticles; peptide nucleic acids; PLGA; PNA; Review; sickle cell disease; triplex; β-thalassemia; PNA; PLGA; nanoparticles; Duchenne muscular dystrophy; cystic fibrosis; CCR5; gene editing; triplex; β-thalassemia; peptide nucleic acids; sickle cell disease

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...