The invention discloses a
nucleic acid inhibitor of a targeted
thyroid stimulating
hormone receptor and application. The
nucleic acid inhibitor has high targeting property, and can accurately recognize TSHR and avoid off-target effect; the inhibition effect is strong, and the TSHR function can be obviously blocked under the micro-mole or even nano-mole concentration; as a
nucleic acid molecule, the
nucleic acid molecule does not cause obvious immune response and is suitable for long-term treatment; good biological stability: after
chemical modification, excellent serum stability and tissue penetrating ability are achieved; the compound is suitable for various administration routes, is convenient for clinical transformation, and shows a wide clinical application prospect in treatment of various TSHR-mediated autoimmune and neoplastic diseases such as Graves diseases,
thyroid-related eye diseases, TSHR activated
mutation-driven
thyroid cancer and the like. Especially for moderate and severe TED patients lacking effective specific
targeted therapy means at present, the nucleic
acid inhibitor is expected to become the first precise therapy for directly intervening the TSHR pathogenic core link.