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SIRNA-HYDROPHILIC POLYMER CONJUGATES FOR INTRACELLULAR DELIVERY OF SIRNA AND METHOD THEREOF

机译:用于siRNA细胞内递送的siRNA-亲水性聚合物缀合物及其方法

摘要

Disclosed are hybrid conjugates formed by covalently bonding siRNA (smallinterfering RNA) molecules to hydrophilic polymers for improving stability ofthe siRNA molecules effective for gene therapy in vivo, and polyelectrolytecomplex micelles formed by ionic interactions between the conjugates andmultifunctional cationic compounds. The siRNA-hydrophilic polymer conjugatesand polyelectrolyte complex micelles derived therefrom can be advantageouslyused for improving stability of the siRNA molecules in vivo. Consequently, thedelivery of siRNA molecules for therapeutic applications into cells can befacilitated, and the siRNA is still active even though a small dose of thesiRNA is used.
机译:公开了通过共价键合siRNA(小干扰RNA)分子与亲水性聚合物的结合,以提高其稳定性体内有效进行基因治疗的siRNA分子和聚电解质复合物与胶束之间的离子相互作用形成的复杂胶束多功能阳离子化合物。 siRNA-亲水性聚合物结合物和由此衍生的聚电解质复合胶束可以是有利的用于改善siRNA分子在体内的稳定性。因此,可以将用于治疗应用的siRNA分子递送到细胞中即使小剂量的siRNA仍能保持活性使用siRNA。

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