Functional features defining the efficacy of cholesterol-conjugated, self-deliverable, chemically modified siRNAs.

ABSTRACT: Progress in oligonucleotide chemistry has produced a shift in the nature of siRNA used, from formulated, minimally modified siRNAs, to unformulated, heavily modified siRNA conjugates. The introduction of extensive chemical modifications is essential for conjugate-mediated delivery. Modifications have a significant impact on siRNA efficacy through interference with recognition and processing by RNAi enzymatic machinery, severely restricting the sequence space available for siRNA design. Many algorithms available publicly can successfully predict the activity of non-modified siRNAs, but the efficiency of the algorithms for designing heavily modified siRNAs has never been systematically evaluated experimentally. Here we screened 356 cholesterol-conjugated siRNAs with extensive modifications a

SUBMITTER: Shmushkovich T 

PROVIDER: S-EPMC6237813 | biostudies-literature | 2018 Nov

REPOSITORIES: biostudies-literature