Abstract:Synthetic nucleic acids could be designed in sequence-specific structure to trigger endogenous repair systems. Depending on this concept, peptide nucleic acids (or PNAs) could achieve non-enzymatic gene editing i.e. without the use of nucleases unlike CRISPR and TALENs. PNAs could bind to the DNA to form highly specific hetero-triplex structures, so PNAs have been used severally in the last decades to induce correction of different human disease-causing mutations with low off-targets. Systemic in-vivo and in-u… Show more
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