REDUCING INTRON RETENTION
    11.
    发明申请

    公开(公告)号:US20200231969A1

    公开(公告)日:2020-07-23

    申请号:US16696635

    申请日:2019-11-26

    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.

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