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Low adenosine anti-sense oligonucleotide compositions kit and method for treatment of airway disorders associated with bronchoconstriction lung inflammation allergyies and surfactant depletion

机译:低腺苷反义寡核苷酸组合物试剂盒和方法,用于治疗与支气管收缩性肺部炎症过敏和表面活性剂耗竭相关的气道疾病

摘要

An in vivo method of selectively delivering a nucleic acid to a target gene or mRNA comprises the topical admistration, e.g. to the respiratory system, of a subject of a therapeutic amount of an oligonucleotide that is antisense to the initiation codon region, coding region, 5' or 3' inton-exon junctions or regions within 2 to 10 nucleotides of the junctions of the target gene, or antisense to a mRNA complementary to the target gene in an amount effective to reach the target polynucleotide and reducing or inhibiting expression. Additionally, a method of treating and adenosine mediated effect is described also utilizing the delivery of antisense and in order to minimize triggering adenosine receptors by their metabolism, the administered oligos have a low content of or are essentially free of adenosine. The agent, composition and formulations are used for prophlactic, preventive and therapeutic treatment of ailments associated with impaired respiration, lung allergies and/or vasoconstriction, inflammation, allergies, allergic rhynitis, asthma, impeded respiration, lung pain, cystic fibrosis and bronchoconstriction.
机译:选择性地将核酸递送至靶基因或mRNA的体内方法包括局部给药。对受试者的呼吸系统而言,是治疗量的寡核苷酸,该寡核苷酸与靶密码子的起始密码子区域,编码区域,5'或3'内含子-外显子连接或2-10个核苷酸内的区域反义,或以与目标基因互补的mRNA的反义量有效达到目标多核苷酸并减少或抑制表达。另外,还描述了一种利用腺苷介导的治疗和腺苷介导的作用的方法,并且为了通过其代谢使触发性腺苷受体的触发作用最小化,所施用的寡核苷酸的腺苷含量低或基本不含。所述试剂,组合物和制剂用于与呼吸不良,肺过敏和/或血管收缩,炎症,过敏,过敏性鼻炎,哮喘,呼吸障碍,肺痛,囊性纤维化和支气管收缩有关的疾病的预防性,预防性和治疗性治疗。

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