VECTOR-BASED THERAPY FOR THYROID DISEASE
    2.
    发明申请

    公开(公告)号:WO2020243659A1

    公开(公告)日:2020-12-03

    申请号:PCT/US2020/035441

    申请日:2020-05-29

    Abstract: Disclosed herein are compositions and methods for continuous AAV-based delivery of blocking anti-TSHR antibodies to a subject having a thyroid disease. The present disclosure is based, at least in part, on the realization that blocking anti-TSHR antibodies may be delivered in a continuous manner using rAAV, e.g., rAAVS, to effectively block the stimulating effects of TSAbs or TSH on TSHR, thereby blocking or reducing the synthesis of thyroid hormone. By blocking or reducing the synthesis of thyroid hormone, the presently described methods and compositions for rAAV-based delivery of blocking anti-TSHR antibodies (e.g., K1-70) may be used to treat thyroid diseases, including Graves' disease, Graves' orbitopathy, and thyroid cancer without the requirement of repeated administrations.

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