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DEVELOPMENT OF NOVEL GENE THERAPEUTICS INFLAMMATION-INDUCED BONE LOSS

机译:新型基因治疗炎症性骨丢失的研究进展

摘要

Aspects of the disclosure relate to compositions and methods for reding inflammation and/or inhibiting bone loss (e.g., bone loss induced by inflammation). In some embodiments, the disclosure provides isolated nucleic acids and expression constructs (e.g., rAAVs, etc.) that encode one or more of the following transgenes: inhibitory nucleic acids targeting Schnurri 3 (SHN3), inhibitory nucleic acids targeting Cathepsin K (CTSK), inhibitory nucleic acids targeting sclerostin (SOST), inhibitory nucleic acids targeting receptor activator of NF-κβ (RANK), inhibitory nucleic acids targeting receptor activator of NF-κβ ligand (RANKL), soluble TNF-α Receptor 2 (sTNFR2), and soluble IL-1 Receptor Antagonist (sIL1Rα). In some embodiments, compositions described by the disclosure are useful for treating diseases associated with inflammation, for example rheumatoid arthritis (RA).
机译:本发明的方面涉及用于减轻炎症和/或抑制骨丢失(例如,炎症诱导的骨丢失)的组合物和方法。在一些实施例中,本发明提供了编码以下一个或多个转基因的分离核酸和表达构建体(例如RAAV等):靶向Schnurri 3的抑制性核酸(SHN3)、靶向组织蛋白酶K的抑制性核酸(CTSK)、靶向硬化蛋白的抑制性核酸(SOST),抑制性核酸靶向NF-κβ受体激活剂(RANK)、抑制性核酸靶向NF-κβ受体激活剂配体(RANKL)、可溶性TNF-α受体2(sTNFR2)和可溶性IL-1受体拮抗剂(sIL1Rα)。在一些实施例中,本发明所述组合物可用于治疗与炎症相关的疾病,例如类风湿性关节炎(RA)。

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