Kiyoshi Tachikawa,Joseph E. Payne,Padmanabh Chivukula
申请号:
US16109231
公开号:
US20180362985A1
申请日:
2018.08.22
申请国别(地区):
US
年份:
2018
代理人:
摘要:
This invention provides UNA oligomers for gene silencing with reduced off-target effects. The UNA oligomers can have a first strand and a second strand, each of the strands being 19-29 monomers in length, the monomers being UNA monomers and various nucleic acid monomers. Embodiments include pharmaceutical compositions and methods for treating or preventing TTR-related amyloidosis with reduced off-target effects by administering a UNA oligomer to a subject.