OLIGONUCLEOTIDES TARGETING FRATAXIN AND RELATED METHODS

    公开(公告)号:US20210285002A1

    公开(公告)日:2021-09-16

    申请号:US17175156

    申请日:2021-02-12

    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.

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