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公开(公告)号:US20240287511A1
公开(公告)日:2024-08-29
申请号:US18290183
申请日:2022-05-13
Inventor: Koutaro Yokote , Yasuo Ouchi , Hisaya Kato , Koji Eto , Yoshiro Maezawa
IPC: C12N15/113 , A61P43/00 , C12N15/86
CPC classification number: C12N15/113 , A61P43/00 , C12N15/86 , C12N2310/11 , C12N2310/3233 , C12N2740/15043
Abstract: The present invention is to provide an antisense oligomer or a pharmaceutically acceptable salt thereof capable of treating Werner syndrome without direct repair of a mutated gene. The present invention provides an antisense oligomer or a pharmaceutically acceptable salt thereof which consists of a base sequence complementary to the base sequence of the following (i) or (ii): (i) a contiguous base sequence of 15 or more bases in the base sequence shown in SEQ ID NO: 1; or (ii) a contiguous base sequence of 15 or more bases in the base sequence shown in SEQ ID NO: 1, having deletion, substitution, or insertion of one or more bases; and which is capable of causing skipping of the 27th exon in a human WRN gene.
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公开(公告)号:US20230030814A1
公开(公告)日:2023-02-02
申请号:US17783097
申请日:2020-12-11
Inventor: Seiji Ohtori , Yasuhiro Shiga , Kentaro Kosaka , Koji Eto
Abstract: The problem that differences among lots in components and amounts of growth factors and the like in platelet-rich plasmas derived from an autologous plasma and the freeze-dried preparations thereof are large, and the effects of the lots are not kept constant is solved.
Specifically, a freeze-dried preparation comprising megakaryocytes and platelets induced to differentiate from stem cells, a pharmaceutical composition comprising the freeze-dried preparation, and the like are provided.
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