首页> 外国专利> USE OF ALGINATE OLIGOMERS IN THE TREATMENT OF CYSTIC FIBROSIS AND OTHER CONDITIONS ASSOCIATED WITH DEFECTIVE CFTR ION CHANNEL FUNCTION

USE OF ALGINATE OLIGOMERS IN THE TREATMENT OF CYSTIC FIBROSIS AND OTHER CONDITIONS ASSOCIATED WITH DEFECTIVE CFTR ION CHANNEL FUNCTION

机译:藻酸盐低聚物在囊性纤维化及CFTR离子通道功能减退相关的其他疾病中的应用

摘要

The invention provides a method for the treatment of a condition in a human patient arising from or associated with a defective cystic fibrosis transmembrane conductance regulator(CFTR) ion channel and/or abnormal mucus which is attached to underlying epithelium, said method comprising administering an alginate oligomer,wherein at least 30% of the monomer residues of the alginate oligomer are G residues,to the patient in an amount sufficient to achieve a local concentration of the alginate oligomer of 1to 6% w/v at at least part of a mucosal surface with a defective CFTR ion channel and/or said abnormal mucus in the patient, thereby to result in at least partial detachment of mucus from said mucosal surface. In certain embodiments said condition is cystic fibrosis (CF), non- compound CFTR gene mutation heterozygosity, abnormal mucus clearance in the respiratory tract and/or breathing difficulties resulting from chronic particulate inhalation, COPD, chronic bronchitis, emphysema, bronchiectasis, asthma or chronic sinusitis, or a complication thereof.
机译:本发明提供了一种用于治疗人类患者的病症的方法,所述病症是由附着于下面的上皮的囊性纤维化跨膜电导调节剂(CFTR)离子通道和/或异常粘液引起的或与之相关的,所述方法包括给予藻酸盐藻酸盐低聚物的至少30%的单体残基是G残基的低聚物,其量足以使至少部分粘膜表面的藻酸盐低聚物局部浓度达到1至6%w / v。 CFTR离子通道有缺陷和/或患者中所述粘液异常,从而导致粘液从所述粘膜表面至少部分脱离。在某些实施方案中,所述病症为囊性纤维化(CF),非复合CFTR基因突变杂合性,呼吸道中的粘液清除异常和/或由于慢性微粒吸入,COPD,慢性支气管炎,肺气肿,支气管扩张,哮喘或慢性引起的呼吸困难鼻窦炎或其并发症。

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