公开(公告)号：EP2609198B8

公开(公告)日：2018-03-28

申请号：EP11820420.5

申请日：2011-08-19

申请人： Sirna Therapeutics, Inc.

发明人： LIM, Lee ,  WILLINGHAM, Aarron ,  ZHAO, Lihong ,  CHORN, Guillaume

IPC分类号： C12N15/11 ,  C12N15/113

CPC分类号： C12N15/113 ,  C12N15/111 ,  C12N15/1138 ,  C12N2310/14 ,  C12N2310/141 ,  C12N2310/318 ,  C12N2310/3183 ,  C12N2310/332 ,  C12N2310/344 ,  C12N2310/351 ,  C12N2310/3515 ,  C12N2310/3519 ,  C12N2310/322 ,  C12N2310/3533 ,  C12N2310/321 ,  C12N2310/3521

摘要： 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.

公开(公告)号：EP2609198B1

公开(公告)日：2018-02-21

申请号：EP11820420.5

申请日：2011-08-19

申请人： Sirna Therapeutics, Inc.

发明人： LIM, Lee ,  WILLINGHAM, Aarron ,  ZHAO, Lihong

IPC分类号： C12N15/11 ,  C12N15/113

CPC分类号： C12N15/113 ,  C12N15/111 ,  C12N15/1138 ,  C12N2310/14 ,  C12N2310/141 ,  C12N2310/318 ,  C12N2310/3183 ,  C12N2310/332 ,  C12N2310/344 ,  C12N2310/351 ,  C12N2310/3515 ,  C12N2310/3519 ,  C12N2310/322 ,  C12N2310/3533 ,  C12N2310/321 ,  C12N2310/3521

摘要： 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.