BUTLER, David Charles Donnell,DIVAKARAMENON, Sethumadhavan,FRANCIS, Christopher J.,FRANK-KAMENETSKY, Maria David,IWAMOTO, Naoki,LU, Genliang,MARAPPAN, Subramanian,Meena,VARGEESE, Chandra,VERDINE, Greg
申请号：
EP17859275
公开号：
EP3523437A4
申请日:
2017.10.06
申请国别(地区):
EP
年份:
2020
代理人:
摘要：
Among other things, the present disclosure relates to designed oligonucleotides, compositions, and methods thereof. In some embodiments, provided oligonucleotide compositions provide altered splicing of a transcript. In some embodiments, provided oligonucleotide compositions have low toxicity. In some embodiments, provided oligonucleotide compositions provide improved protein binding profiles. In some embodiments, provided oligonucleotide compositions have improved delivery. In some embodiments, provided oligonucleotide compositions have improved uptake. In some embodiments, the present disclosure provides methods for treatment of diseases using provided oligonucleotide compositions.
