Developments in siRNA Modification and Ligand Conjugated Delivery To Enhance RNA Interference Ability.

Ander Maguregui, Hiroshi Abe

Journal: Chembiochem : a European journal of chemical biology 2021;21(13):1808-1815

PMID: 32181563

Abstract

There is great potential for siRNA in the treatment of diseases through the reduction of damaging protein translation by RNA interference. However, the delivery and cell uptake of siRNA pose a serious problem in its therapeutic application. Methods to overcome this issue include chemical modification of the siRNA duplex to improve pharmacokinetics, stability and efficacy, and conjugation to small ligand molecules to enable membrane penetration, targetability and potency. In this review, the most common modifications of siRNA will be discussed, along with ligand conjugates that are believed to be the most promising in advancing the field of targeted siRNA delivery.

Address: Bioorganic Chemistry Laboratory, Graduate School of Science, Nagoya University, Chikusa-ku, Nagoya, Aichi, 464-0813, Japan.; Bioorganic Chemistry Laboratory, Graduate School of Science, Nagoya University, Chikusa-Ku, Nagoya, Aichi, 464-8602, Japan.

DOI: 10.1002/cbic.202000009

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https://doi.org/10.1002/cbic.202000009

MeSH Terms: Cholesterol, Disulfides, Humans, Ligands, RNA Interference, RNA Stability, RNA, Small Interfering, Transfection