公开(公告)号：EP3353300A1

公开(公告)日：2018-08-01

申请号：EP16770006.1

申请日：2016-09-21

申请人： Oommen, Oommen P ,  Varghese, Oommen P

发明人： Oommen, Oommen P ,  Varghese, Oommen P

IPC分类号： C12N15/11 ,  C12N15/113 ,  A61K31/713

CPC分类号： C12N15/113 ,  C12N15/111 ,  C12N2310/14 ,  C12N2310/141 ,  C12N2310/20 ,  C12N2310/32 ,  C12N2310/531 ,  C12N2310/533 ,  C12N2320/50

摘要： Double-stranded nucleic acid molecules (100, 200, 300, 400, 500 and 600) are provided comprising a sense strand (110, 210, 310, 410, 510 and 610) and an antisense strand (120, 220, 320, 420, 520 and 620). The sense strand has a 3′-overhang (115, 215, 315, 415, 515 and 615) of from four to eight nucleotides. The double-stranded nucleic acid molecules include double-stranded RNA (dsRNA), double-stranded DNA (dsDNA), double-stranded DNA-RNA (sense-antisense) hybrid and double-stranded RNA-DNA (sense-antisense) hybrid. The double-stranded nucleic acid molecules have beneficial properties in terms of unaided cellular delivery (e.g. in the absence of a cationic agent), cellular delivery and release of the antisense strand in a cell as a single-stranded molecule and the delivery of the antisense strand into the nucleus of a cell as a single-stranded molecule. Related compositions, cells, uses and methods are also provided.