首页> 外国专利> MC4R AGONIST EFFICACY IN SUBJECTS WITH MC4R DEFICIENCIES AND IMPAIRED NFAT SIGNALING

MC4R AGONIST EFFICACY IN SUBJECTS WITH MC4R DEFICIENCIES AND IMPAIRED NFAT SIGNALING

机译:MC4R缺陷和NFAT信号受损的受试者的MC4R促效药

摘要

The invention relates to a Melanocortin-4 receptor (MC4R) agonist that exhibits greater induction of NFAT signaling compared to a-MSH for use in the treatment and/or prevention of a medical condition associated with MC4R deficiency in a subject having an MC4R deficiency associated with impaired Nuclear factor of activated T-cells (NFAT) signaling. The present invention further relates to an in vitro method for the diagnosis, prognosis and/or assessment of likelihood of whether a subject with, or at risk of having and/or developing, a medical condition associated with MC4R deficiency, will respond to treatment with an MC4R agonist that exhibits greater induction of NFAT signaling compared to a-MSH, the method comprising (i) providing a sample from said subject, and (ii) determining whether the subject has an MC4R deficiency associated with impaired NFAT signaling by assessing said sample, (iii) wherein the presence of an MC4R deficiency associated with impaired NFAT signaling is indicative that treatment with an MC4R agonist that exhibits greater induction of NFAT signaling compared to a-MSH will be effective in said subject.
机译:本发明涉及与用于治疗和/或预防与MC4R缺乏症相关的受试者的与MC4R缺乏症有关的医学状况相比,与α-MSH相比表现出更大诱导NFAT信号传导的黑皮质素-4受体(MC4R)激动剂。激活的T细胞(NFAT)信号的核因子受损。本发明进一步涉及一种体外方法,用于诊断,预后和/或评估患有MC4R缺乏症的医学病症,或具有患和/或患上与MC4R缺乏症有关的医学病症的受试者是否会对用MC4R治疗产生反应的可能性。与a-MSH相比,对NFAT信号具有更大诱导作用的MC4R激动剂,该方法包括(i)提供来自所述受试者的样品,以及(ii)通过评估所述样品来确定受试者是否患有与NFAT信号受损相关的MC4R缺乏(iii)其中与受损的NFAT信号传导相关的MC4R缺乏症的存在表明,与α-MSH相比,显示出更大的NFAT信号传导诱导作用的MC4R激动剂治疗在所述受试者中是有效的。

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