BHANOT, SANJAY,GEARY, RICHARD S.,MCKAY, ROBERT,MONIA, BRETT, P.,SETH, PUNIT, P.,SIWKOWSKI, ANDREW, M.,SWAYZE, ERIC, E.,WANCEWICZ, EDWARD
申请号：
DK07811878
公开号：
DK2019692T3
申请日:
2007.05.07
申请国别(地区):
DK
年份:
2014
代理人:
摘要：
The present disclosure describes short antisense compounds, including such compounds comprising chemically-modified high-affinity monomers 8-16 monomers in length. Certain such short antisense compound are useful for the reduction of target nucleic acids and/or proteins in cells, tissues, and animals with increased potency and improved therapeutic index. Thus, provided herein are short antisense compounds comprising high-affinity nucleotide modifications useful for reducing a target RNA in vivo. Such short antisense compounds are effective at lower doses than previously described antisense compounds, allowing for a reduction in toxicity and cost of treatment. In addition, the described short antisense compounds have greater potential for oral dosing.
