OLIGONUCLEOTIDE COMPOSITIONS AND METHODS THEREOF

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 so...

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Bibliographische Detailangaben
Hauptverfasser: Butler, David Charles Donnell, Francis, Christopher J, Lu, Genliang, Meena, Verdine, Gregory L, Iwamoto, Naoki, Zhang, Jason Jingxin, Frank-Kamenetsky, Maria David, Vargeese, Chandra, Divakaramenon, Sethumadhavan, Marappan, Subramanian, Yang, Hailin
Format: Patent
Sprache:eng
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Zusammenfassung: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.