Preprint Review Version 1 Preserved in Portico This version is not peer-reviewed

Recent Advancements in Genome-Targeting Nucleic Acids and Their Potential for Therapeutic Applications

Version 1 : Received: 13 September 2023 / Approved: 14 September 2023 / Online: 14 September 2023 (11:37:06 CEST)

A peer-reviewed article of this Preprint also exists.

Mikame, Y.; Yamayoshi, A. Recent Advancements in Development and Therapeutic Applications of Genome-Targeting Triplex-Forming Oligonucleotides and Peptide Nucleic Acids. Pharmaceutics 2023, 15, 2515. Mikame, Y.; Yamayoshi, A. Recent Advancements in Development and Therapeutic Applications of Genome-Targeting Triplex-Forming Oligonucleotides and Peptide Nucleic Acids. Pharmaceutics 2023, 15, 2515.

Abstract

Recent developments in artificial nucleic acid and drug delivery systems (DDS) present possibilities for the symbiotic engineering of oligonucleotide therapeutics, such as antisense oligonucleotides (ASOs), and small interfering RNA (siRNA). These technologies can also be applied for triplex-forming oligonucleotides (TFOs) to develop symbiotic genome-targeting tools and a new class of oligonucleotide drugs. Many reports have described the functionalization of TFOs and the enhancement of TFO activities. In particular, the tethering of crosslinking groups to TFOs enables irreversible triplex formation and is frequently exploited for antigene and genome-editing experiments. These crosslinkable TFOs have shown considerable potential for therapeutic application. In this review, we revisit these crosslinking techniques and explore the combination of these techniques with recent artificial oligonucleotide and DDS technologies.

Keywords

oligonucleotide therapeutic; triplex-forming oligonucleotide; peptide nucleic acid; antigene; genome editing

Subject

Medicine and Pharmacology, Medicine and Pharmacology

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