Reducing intron retention
    1.
    发明授权

    公开(公告)号:US10538764B2

    公开(公告)日:2020-01-21

    申请号:US15619984

    申请日:2017-06-12

    Abstract: Disclosed herein are methods, compositions, polynucleic acid polymers, assays, and kits for inducing processing of a partially processed mRNA transcript to remove a retained intron to produce a fully processed mRNA transcript that encodes a full-length functional form of a protein. Also described herein are methods and compositions for treating a disease or condition characterized by impaired production of a full-length functional form of a protein or for treating a disease or condition characterized by a defective splicing in a subject.

    REDUCING INTRON RETENTION
    5.
    发明申请
    REDUCING INTRON RETENTION 有权
    减少内在的保留

    公开(公告)号:US20160244762A1

    公开(公告)日:2016-08-25

    申请号:US15148303

    申请日:2016-05-06

    Abstract: Disclosed herein are methods, compositions, polynucleic acid polymers, assays, and kits for inducing processing of a partially processed mRNA transcript to remove a retained intron to produce a fully processed mRNA transcript that encodes a full-length functional form of a protein. Also described herein are methods and compositions for treating a disease or condition characterized by impaired production of a full-length functional form of a protein or for treating a disease or condition characterized by a defective splicing in a subject.

    Abstract translation: 本文公开了用于诱导加工部分处理的mRNA转录物以除去保留的内含子以产生编码蛋白质的全长功能形式的完全加工的mRNA转录物的方法,组合物,聚核酸聚合物,测定和试剂盒。 本文还描述了用于治疗疾病或病症的方法和组合物,其特征在于蛋白质的全长功能形式的产生受损或用于治疗特征在于受试者中拼接缺陷的疾病或病症。

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