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Article
Taking charge of siRNA delivery
Delivery of siRNA into cells is achieved by neutralizing the negative charge of the phosphate backbone in a reversible manner.
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Article
Correction: Corrigendum: Taking charge of siRNA delivery
Nat. Biotechnol. 32, 1197–1198 (2014); published online 9 December 2014; corrected after print 29 April 2015 In the version of this article initially published, in two instances molecules were misidentified in...
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Article
Open AccessHeavily and fully modified RNAs guide efficient SpyCas9-mediated genome editing
RNA-based drugs depend on chemical modifications to increase potency and to decrease immunogenicity in vivo. Chemical modification will likely improve the guide RNAs involved in CRISPR-Cas9-based therapeutics ...
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Article
RNAi modulation of placental sFLT1 for the treatment of preeclampsia
Preeclampsia, a hypertension condition with no approved therapies, is treated in monkeys by gene silencing in the placenta.
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Article
A divalent siRNA chemical scaffold for potent and sustained modulation of gene expression throughout the central nervous system
Sustained silencing of gene expression throughout the brain using small interfering RNAs (siRNAs) has not been achieved. Here we describe an siRNA architecture, divalent siRNA (di-siRNA), that supports potent,...
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Article
Open AccessChemical engineering of therapeutic siRNAs for allele-specific gene silencing in Huntington’s disease models
Small interfering RNAs are a new class of drugs, exhibiting sequence-driven, potent, and sustained silencing of gene expression in vivo. We recently demonstrated that siRNA chemical architectures can be optimi...