Precise Genome Modification Using Triplex Forming Oligonucleotides and Peptide Nucleic Acids

Abstract

Many genetic disorders are caused by single base pair mutations which lead to defective protein synthesis. In addition to gene replacement therapy, modification of genomic DNA sequences at specific sites has been employed to manipulate the function and expression of various genes, which are implicated in various genetic disorders. On this front, triplex technology has been used to alter the expression of different genes by correcting mutations site specifically via homologous recombination (HR) or targeted mutagenesis based mechanisms. In this chapter we will discuss the advances made in triplex technology involving triplex forming oligonucleotides (TFOs) and peptide nucleic acids (PNAs) for site specific genome editing.