公开(公告)号：US20180216108A1

公开(公告)日：2018-08-02

申请号：US15746220

申请日：2016-07-22

申请人： Chandra Vargeese ,  Meena ,  Nenad Svrzikapa ,  Susovan Mohapatra ,  Christopher J. Francis ,  Gregory L. Verdine ,  Anna Sokolovska

发明人： Chandra Vargeese ,  Meena ,  Nenad Svrzikapa ,  Susovan Mohapatra ,  Christopher J. Francis ,  Gregory L. Verdine ,  Anna Sokolovska

IPC分类号： C12N15/113 ,  A61P25/28

摘要： Among other things, the present disclosure relates to chirally controlled oligonucleotides of select designs, chirally controlled oligonucleotide compositions, and methods of making and using the same. In some embodiments, a provided chirally controlled oligonucleotide composition provides different cleavage patterns of a nucleic acid polymer than a reference oligonucleotide composition. In some embodiments, a provided chirally controlled oligonucleotide composition provides single site cleavage within a complementary sequence of a nucleic acid polymer. In some embodiments, a chirally controlled oligonucleotide composition has any sequence of bases, and/or pattern or base modifications, sugar modifications, backbone modifications and/or stereochemistry, or combination of these elements, described herein.

公开(公告)号：US20180216107A1

公开(公告)日：2018-08-02

申请号：US15746199

申请日：2016-07-22

申请人： Maria David Frank-Kamenetsky ,  Hailin Yang ,  Aaron Morris ,  Chandra Vargeese ,  Christopher J. Francis

发明人： Maria David Frank-Kamenetsky ,  Hailin Yang ,  Aaron Morris ,  Chandra Vargeese ,  Christopher J. Francis

IPC分类号： C12N15/113 ,  C12N15/115

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 up-take. In some embodiments, the present disclosure provides methods for treatment of diseases using provided oligonucleotide compositions.