Fully Stabilized Assimetric siRNA Compounds: an Optimal Scaffold for Conjugate Mediated Delivery
- 詳細技術說明
- This invention discloses hydrophobically-modified siRNA, featuring an advanced stabilization pattern, "hsiRNA-ASP". These siRNA compounds having the following properties: (1) fully chemically stabili
- *Abstract
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This invention discloses hydrophobically-modified siRNA, featuring an advanced stabilization pattern, "hsiRNA-ASP". These siRNA compounds having the following properties: (1) fully chemically stabilized (i.e., no unmodified 2'-OH residues); (2) asymmetry; (3) 11-16 base pair duplexes; (4) alternating patten of chemicically-modified nucleotides (e.g., 2'-flour and 2'-methoxy modifications); (5) single-stranded, fully phosphorothioated tails of 5-8 bases. siRNAs with these structural properties show a dramatic enhancement in potency (5-10) fold in unassisted delivery. Also embodied are hsiRNA-ASPs conjugated to targeting agents including, but not limited to, cholesterol. Further, alteration of hsiRNA-ASP PS content has a major impact on tissue distribution and oligonucleotide uptake making these compounds very attractive therapeutic agents.
- *IP Issue Date
- None
- *IP Type
- Other Patent
- 國家
- Not Available
- 申請號碼
- None
- 國家/地區
- 美國
