Oligonucleotide compositions and methods thereof

The present disclosure relates to oligonucleotide compositions and methods thereof, in particular to designed oligonucleotides, compositions and methods thereof. In some embodiments, provided oligonucleotide compositions provide altered transcript splicing. In some embodiments, provided oligonucleot...

Ausführliche Beschreibung

Gespeichert in:
Bibliographische Detailangaben
Hauptverfasser: YANG HAILIN, FRANCIS CHRISTOPHER J, VARGHESE, CHANDRA, FRANK-KAMENETSKY MARIA D, MORRIS AARON
Format: Patent
Sprache:chi ; eng
Schlagworte:
Online-Zugang:Volltext bestellen
Tags: Tag hinzufügen
Keine Tags, Fügen Sie den ersten Tag hinzu!
Beschreibung
Zusammenfassung:The present disclosure relates to oligonucleotide compositions and methods thereof, in particular to designed oligonucleotides, compositions and methods thereof. In some embodiments, provided oligonucleotide compositions provide altered transcript splicing. 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 of treating disease using provided oligonucleotide compositions. 本公开内容涉及寡核苷酸组合物及其方法,尤其涉及设计的寡核苷酸、组合物及其方法。在一些实施方案中,提供的寡核苷酸组合物提供了改变的转录物剪接。在一些实施方案中,提供的寡核苷酸组合物具有低毒性。在一些实施方案中,提供的寡核苷酸组合物提供了改善的蛋白质结合谱。在一些实施方案中,提供的寡核苷酸组合物具有改善的递送。在一些实施方案中,提供的寡核苷酸组合物具有改善的摄取。在一些实施方案中,本公开内容提供了使用提供的寡核苷酸组合物治疗疾病的方法。