SINGLE-STRANDED RNAI AGENTS CONTAINING AN INTERNAL, NON-NUCLEIC ACID SPACER

The instant disclosure features single-stranded RNA molecules comprising one or more internal, non-nucleotide spacers. A non-nucleotide spacer covalently links nucleotide portions of the molecule. The single-stranded RNA molecules function as guide or antisense strands that are capable of inhibiting...

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Bibliographic Details
Main Authors Zhao Lihong, Willingham Aarron T, Lim Lee, Chorn Guillaume
Format Patent
LanguageEnglish
Published 22.03.2018
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Summary:The instant disclosure features single-stranded RNA molecules comprising one or more internal, non-nucleotide spacers. A non-nucleotide spacer covalently links nucleotide portions of the molecule. The single-stranded RNA molecules function as guide or antisense strands that are capable of inhibiting gene expression via an RNA interference mechanism and, thus, represent single-stranded RNAi agents. The single-stranded RNAi molecules can be used in methods for a variety of therapeutic, diagnostic, target validation, genomic discovery, genetic engineering, and pharmacogenomic applications.
Bibliography:Application Number: US201715809777