首页> 外国专利> The composition, analogo of oligonucleotideo, oligonucleotideo sequenced joint, process for the preparation of nucleosideos 2 '- replaced, structure to modulate the activity of RNA.Processes for the production of a modular protein by a body, to treat an animal, to detect the presence or absence of the RNA.Oligonucleotideo or analogo of analogo of oligonucleotideo resistant to nuclease and process for treating a body having a disease

The composition, analogo of oligonucleotideo, oligonucleotideo sequenced joint, process for the preparation of nucleosideos 2 '- replaced, structure to modulate the activity of RNA.Processes for the production of a modular protein by a body, to treat an animal, to detect the presence or absence of the RNA.Oligonucleotideo or analogo of analogo of oligonucleotideo resistant to nuclease and process for treating a body having a disease

机译:寡核苷酸的组成,寡核苷酸的类似物,寡核苷酸测序的接头,制备被2'-取代的核苷的过程,调节RNA活性的结构。人体产生模块蛋白的过程,治疗动物,检测动物体内RNA的存在与否,寡核苷酸或对核酸酶具有抗性的寡核苷酸类似物的类似物以及治疗患有疾病的身体的方法

摘要

Compositions and methods for modulating the activity of RNA and DNA are disclosed. In accordance with preferred embodiments, antisense compositions are prepared comprising targeting and reactive portions. Reactive portions which act, alternatively, through phosphorodiester bond cleavage, through backbone sugar bond cleavage or through base modification are preferably employed. Groups which improve the pharmacodynamic and pharmacokinetic properties of the oligonucleotides are also useful in accordance with certain embodiments of this invention. Delivery of the reactive or non-reactive functionalities into the minor groove formed by the hybridization of the composition with the target RNA is also preferably accomplished. Therapeutics, diagnostics and research methods are also disclosed. Synthetic nucleosides and nucleoside fragments are also provided useful for elaboration of oligonucleotides and oligonucleotide analogs for such purposes.
机译:公开了用于调节RNA和DNA活性的组合物和方法。根据优选的实施方案,制备了包含靶向部分和反应部分的反义组合物。优选使用反应性部分,其通过磷酸二酯键裂解,通过主链糖键裂解或通过碱基修饰起作用。根据本发明的某些实施方案,改善寡核苷酸的药代动力学和药代动力学性质的基团也是有用的。还优选将反应性或非反应性官能团递送到通过组合物与靶RNA的杂交形成的小沟中。还公开了治疗,诊断和研究方法。还提供了合成的核苷和核苷片段,可用于为此目的加工寡核苷酸和寡核苷酸类似物。

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