Grossman, Tamar R.,Mccaleb, Michael L.,Watt, Andrew T.,Freier, Susan M.
申请号：
AU2020201763
公开号：
AU2020201763A1
申请日:
2020.03.11
申请国别(地区):
AU
年份:
2020
代理人:
摘要：
#$%^&*AU2020201763A120200402.pdf#####ABSTRACT The present embodiments provide methods, compounds, and compositions for treating, preventing, or ameliorating a disease associated with dysregulation of the complement alternative pathway by administering a Complement Factor B (CFB) specific inhibitor to a subject. The inhibitor comprises a modified oligonucleotide consisting of 16 to 30 linked nucleosides and having a nucleobase sequence complementary to an equal length portion of nucleobases 2574-2626 of a CFB nucleic acid having the nucleobase sequence of SEQ ID NO: 1, wherein the modified oligonucleotide has a gap segment, and a 5'and a 3'wing segment.
