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RNA interference mediated inhibition of TGF-beta and TGF-beta receptor gene expression using short interfering nucleic acid (siNA)

机译:RNA干扰介导的使用短干扰核酸(siNA)的TGF-β和TGF-β受体基因表达的抑制

摘要

This invention relates to compounds, compositions, and methods useful for modulating TGF-beta and/or TGF-betaR gene expression using short interfering nucleic acid (siNA) molecules. This invention also relates to compounds, compositions, and methods useful for modulating the expression and activity of other genes involved in pathways of TGF-beta and/or TGF-betaR gene expression and/or activity by RNA interference (RNAi) using small nucleic acid molecules. In particular, the instant invention features small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules and methods used to modulate the expression of TGF-beta and/or TGF-betaR genes. Such small nucleic acid molecules are useful, for example, for treating, preventing, inhibiting, or reducing inflammatory, respiratory, autoimmune, and/or proliferative diseases, disorders, conditions, or traits in a cell, subject or organism and any other disease, condition, trait or indication that can respond to the level of TGF-beta and/or TGF-betaR in a cell or tissue; or alternately in providing long term hematopeitic reconstitution in a subject or organism.
机译:本发明涉及用于使用短干扰核酸(siNA)分子调节TGF-β和/或TGF-βR基因表达的化合物,组合物和方法。本发明还涉及可用于利用小核酸通过RNA干扰(RNAi)来调节与TGF-β和/或TGF-βR基因表达途径和/或活性有关的其他基因的表达和活性的化合物,组合物和方法。分子。特别地,本发明的特征在于小的核酸分子,例如短干扰核酸(siNA),短干扰RNA(siRNA),双链RNA(dsRNA),微小RNA(miRNA)和短发夹RNA(shRNA)。 )用于调节TGF-beta和/或TGF-betaR基因表达的分子和方法。此类小核酸分子可用于,例如,治疗,预防,抑制或减少细胞,受试者或生物体中的炎性,呼吸道,自身免疫和/或增生性疾病,病症,病状或性状以及任何其他疾病,可以对细胞或组织中TGF-β和/或TGF-βR的水平产生反应的病状,性状或适应症;或在受试者或生物体中提供长期造血重建。

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