GEALL, Andrew John,DOPPALAPUDI, Venkata Ramana,CHU, David Sai-Ho,COCHRAN, Michael Caramian,HOOD, Michael,DARIMONT, Beatrice Diana,BURKE, Rob,SHI, Yunyu,MARELIUS, Gulin Erdogan,MALECOVA, Barbora
申请号：
AU2018378812
公开号：
AU2018378812A1
申请日:
2018.12.06
申请国别(地区):
AU
年份:
2020
代理人:
摘要：
This application disclose a polynucleic acid molecule conjugate comprising an antibody or binding fragment thereof conjugated to a polynucleic acid molecule that hybridizes to a target sequence of an atrogene； wherein the polynucleic acid molecule comprises at least one 2' modified nucleotide, at least one modified intemucleotide linkage, or at least one inverted abasic moiety； and wherein the polynucleic acid molecule conjugate mediates RNA interference against the atrogene, thereby treating muscle atrophy or myotonic dystrophy in a subject. In a certain embodiment, atrogene comprises atrogin-1 gene (FBXO32), MuRF1 gene (TRIA63), FOXO1, FOXO3, or MSTN.
