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COMPOUNDS AND METHODS FOR PEPTIDE RIBONUCLEIC ACID CONDENSATE PARTICLES FOR RNA THERAPEUTICS

机译:用于RNA疗法的肽核糖核酸冷凝颗粒的化合物和方法

摘要

PROBLEM TO BE SOLVED: To provide compounds comprising small particles which contain an active nucleic acid agent for intercellular delivery as a therapy, and methods for producing such compounds; in particular, compounds and production methods for delivering double-stranded RNA to cells to generate the RNAi response.SOLUTION: Provided are compounds comprising condensed particles having diameters less than 1000 nm. The particles comprise one or more double stranded ribonucleic acids (dsRNAs) and one or more peptides. The compounds, compositions and production methods are useful for modulating gene expression by RNA interference.
机译:解决的问题:提供包含小颗粒的化合物,其包含用于细胞间递送的活性核酸剂作为治疗剂,以及制备此类化合物的方法;特别是,用于将双链RNA传递到细胞以产生RNAi反应的化合物和生产方法。解决方案:提供了包含直径小于1000 nm的浓缩颗粒的化合物。颗粒包含一种或多种双链核糖核酸(dsRNA)和一种或多种肽。该化合物,组合物和生产方法可用于通过RNA干扰调节基因表达。

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