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Nucleoside Agents for reducing the harmful activity of genes containing Extended nucleotide repeats.

机译:核苷类试剂,用于减少包含扩展核苷酸重复序列的基因的有害活性。

摘要

p THE ASPECTS OF THE INVENTION INCLUDE MEacute; ALL FOR THE REDUCTION OF THE HARMFUL ACTIVITY OF AN OBJECTIVE GENE CONTAINING A REPETITION OF NUCLEOacute; TIDOS (NR, BY THEIR CENTURIES IN ENGLISH) IN A CEacute; LULA THROUGH THE CONTACT OF THE CEacute; LULA WITH AN EFFECTIVE AMOUNT OF A NUCLEOAcute AGENT, ASIacute; AS ALSO COMPOSITIONS USED IN SUCH MEacute; ALL. HARMFUL ACTIVITY (FOR EXAMPLE,TOXICITY AND / OR DYSFUNCTIONALITY OF PRODUCTS CODED BY THIS) OF AN OBJECTIVE GENE CONTAINING MUTANT EXTENDED NR MAY BE REDUCED IN A VARIETY OF DIFFERENT FORMS, FOR EXAMPLE, THROUGH REDUCTION; AND (IN SOME CASES, REDUCE THEMSELVES; INCLUDES SELECTIVE) THE PRODUCTION, NON-ACTIVITY OF THE EXPRESSION PRODUCTS, TOXIC (FOR EXAMPLE, RNA OR PROTEINATION) CODED BY THE OBJECTIVE GENE. KITS AND COMPOSITIONS ARE ALSO PROVIDED TO PUT ON THE MEacute; ALL OBJECT. /p
机译:>本发明的方面包括宏。都是为了减少含有核糖核酸的目标基因的有害活性;泰多斯(NR,以他们的英语为母语) LULA通过CEACATE的联系方式; LULA有效量的NucleoAcute代理商,ASIacute;以及如此微不足道的组合物;所有。包含突变扩展NR的目标基因的有害活性(例如,本编码的产品的毒性和/或功能失调)可以通过各种形式减少,例如,通过减少;并且(在某些情况下,要减少其自身;包括选择性地)由目的基因编码的表达产物的产生,无活性,有毒(例如,RNA或蛋白)。还提供了套件和组成部分。所有对象。

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