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    METHODS FOR MODULATING RNA SPLICING
    1.
    发明公开
    METHODS FOR MODULATING RNA SPLICING 审中-公开

    公开(公告)号：EP4249472A3

    公开(公告)日：2023-12-13

    申请号：EP23173467.4

    申请日：2016-05-27

    申请人： PTC Therapeutics, Inc.

    发明人： NARYSHKIN, Nikolai, A. ,  DAKKA, Amal

    IPC分类号： A01N43/90 ,  A61K31/519 ,  C07D239/70

    摘要： In one aspect, described herein is a recognition element for splicing (REMS) that can be recognized by a compound provided herein. In another aspect, described herein are methods for modulating the amount of a product of a gene, wherein a precursor RNA transcript transcribed from the gene contains a REMS, and the methods utilizing a compound described herein. More particularly, described herein are methods for modulating the amount of an RNA transcript or protein product encoded by a gene, wherein a precursor RNA transcript transcribed from the gene comprises a REMS, and the methods utilizing a compound described herein. In another aspect, provided herein are artificial gene construct comprising a REMS, and uses of those artificial gene constructs to modulate functional protein production. In another aspect, provided herein are methods for altering endogenous genes to comprise a REMS, and the use of a compound described herein to modulate the functional protein produced from such altered endogenous genes.

    METHODS FOR MODULATING RNA SPLICING
    10.
    发明公开
    METHODS FOR MODULATING RNA SPLICING 审中-公开
    调节RNA拼接的方法

    公开(公告)号：EP3310169A1

    公开(公告)日：2018-04-25

    申请号：EP16804178.8

    申请日：2016-05-27

    申请人： PTC Therapeutics, Inc.

    发明人： NARYSHKIN, Nikolai ,  DAKKA, Amal

    IPC分类号： A01N43/90 ,  A61K31/519 ,  C07D239/70

    CPC分类号： A61K48/0025 ,  A61K31/519 ,  A61K31/711 ,  C07D239/70 ,  C12N15/11 ,  C12N15/85 ,  C12N2320/33 ,  Y02A50/465

    摘要： In one aspect, described herein is a recognition element for splicing modifier (REMS) that can be recognized by a compound provided herein. In another aspect, described herein are methods for modulating the amount of a product of a gene, wherein a precursor RNA transcript transcribed from the gene contains a REMS, and the methods utilizing a compound described herein. More particularly, described herein are methods for modulating the amount of an RNA transcript or protein product encoded by a gene, wherein a precursor RNA transcript transcribed from the gene comprises a REMS, and the methods utilizing a compound described herein. In another aspect, provided herein are artificial gene constructs comprising a REMS, and uses of those artificial gene constructs to modulate functional protein production. In another aspect, provided herein are methods for altering endogenous genes to comprise a REMS, and the use of a compound described herein to modulate the functional protein produced from such altered endogenous genes.