Inventors:
Lee Lim - San Francisco CA, US
Guillaume Chorn - Sheung Wan, HK
Aarron Willingham - Mountain View CA, US
Lihong Zhao - Walnut Creek CA, US
International Classification:
C12N 15/113
US Classification:
424450, 536 245, 514 44 A, 435375, 424400
Abstract:
Single-stranded RNA molecules comprise one or more internal, non-nucleotide spacers, covalently linked with nucleotide portions of the molecule are provided. 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.