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    ANTI-ADAM33 OLIGONUCLEOTIDES AND RELATED METHODS
    2.
    发明申请
    ANTI-ADAM33 OLIGONUCLEOTIDES AND RELATED METHODS 审中-公开

    公开(公告)号：US20190002885A1

    公开(公告)日：2019-01-03

    申请号：US16001265

    申请日：2018-06-06

    Applicant: UNIVERSITY OF MASSACHUSETTS ,  UNIVERSITY OF SOUTHAMPTON

    Inventor： Jonathan Watts ,  Hans Michael Haitchi ,  Hannah Pendergraff

    IPC: C12N15/113

    Abstract: The present disclosure provides antisense compounds, methods, and compositions for silencing ADAM33 mRNA. The present disclosure provides antisense compounds, methods, and compositions for the treatment, prevention, or amelioration of diseases, disorders, and conditions associated with ADAM33 in a subject in need thereof. Also contemplated are antisense compounds and methods for the preparation of a medicament for the treatment, prevention, or amelioration of a disease, disorder, or condition associated with ADAM33.

    OLIGONUCLEOTIDES TARGETING FRATAXIN AND RELATED METHODS
    4.
    发明申请
    OLIGONUCLEOTIDES TARGETING FRATAXIN AND RELATED METHODS 有权

    公开(公告)号：US20210285002A1

    公开(公告)日：2021-09-16

    申请号：US17175156

    申请日：2021-02-12

    Applicant: UNIVERSITY OF MASSACHUSETTS

    Inventor： Feng Wang ,  Jonathan Watts

    IPC: C12N15/113

    Abstract: The present disclosure provides antisense compounds, methods, and compositions for increasing FXN gene expression. The present disclosure provides antisense compounds, methods, and compositions for the treatment, prevention, or amelioration of diseases, disorders, and conditions associated with FXN in a subject in need thereof. Also contemplated are antisense compounds and methods for the preparation of a medicament for the treatment, prevention, or amelioration of a disease, disorder, or condition associated with FXN.

    THERAPEUTIC TREATMENT FOR FRAGILE X-ASSOCIATED DISORDER
    5.
    发明申请
    THERAPEUTIC TREATMENT FOR FRAGILE X-ASSOCIATED DISORDER 有权

    公开(公告)号：US20240417729A1

    公开(公告)日：2024-12-19

    申请号：US18751096

    申请日：2024-06-21

    Applicant: University of Massachusetts

    Inventor： Joel D. Richter ,  Sneha Shah ,  Jonathan Watts

    IPC: C12N15/113

    Abstract: Provided herein, in various embodiments, are methods of treating a fragile X-associated disorder (e.g., fragile X syndrome), comprising administering to a subject in need thereof, a therapeutically effective amount of an agent that decreases expression of an aberrant fragile X messenger ribonucleoprotein 1 (FMR1) gene product (e.g., FMR1-217). Also provided herein, in various embodiments, are compositions (e.g., polynucleotides such as antisense oligonucleotides or pharmaceutical compositions) for decreasing expression of an aberrant FMR1 gene product.

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