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Developments in siRNA Modification and Ligand Conjugated Delivery To Enhance RNA Interference Ability
Author(s) -
Maguregui Ander,
Abe Hiroshi
Publication year - 2020
Publication title -
chembiochem
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.05
H-Index - 126
eISSN - 1439-7633
pISSN - 1439-4227
DOI - 10.1002/cbic.202000009
Subject(s) - conjugated system , small interfering rna , rna interference , rna , chemistry , drug delivery , ligand (biochemistry) , conjugate , combinatorial chemistry , nanotechnology , biophysics , gene silencing , computational biology , biochemistry , biology , materials science , receptor , polymer , gene , mathematical analysis , mathematics , organic chemistry
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.