申请人:WAVE LIFE SCIENCES LTD.
公开号:US10450568B2
公开(公告)日:2019-10-22
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.
除其他外,本公开涉及设计的寡核苷酸、组合物及其方法。在一些实施方案中,所提供的寡核苷酸组合物能改变转录本的剪接。在一些实施方案中,所提供的寡核苷酸组合物具有低毒性。在一些实施方案中,所提供的寡核苷酸组合物提供了改进的蛋白质结合概况。在一些实施方案中,所提供的寡核苷酸组合物具有更好的传递性。在一些实施方案中,所提供的寡核苷酸组合物改善了吸收。在一些实施方案中,本公开提供了使用所提供的寡核苷酸组合物治疗疾病的方法。