评论:通过SKI信使RNA监视通路操作的FOCAD缺失导致儿童肝硬化综合征[1]

R. Traspas
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引用次数: 0

摘要

肝病正在成为一个主要的健康问题,据估计是全球第五大常见死因[2]。全球疾病负担研究(Global Burden of Disease Study)的一项系统综述发现,2017年肝硬化导致132万人死亡,占全球死亡总数的2%以上[3]。肝硬化通常在晚年才被诊断出来;它是不可逆转的,在患者的余生中一直是慢性的;它可能会危及生命,因为它可能会演变成肝细胞癌或导致肝功能衰竭。尽管进行了广泛的研究,但迄今为止,没有一种用于治疗这种疾病的测试化合物被证明对人类患者是安全有效的。目前可用的治疗方法通常是姑息性的,重点是减缓疾病,肝移植仍然是治疗失代偿性肝硬化患者的金标准策略。因此,有一个专利需要鉴定和验证新的靶点来治疗,逆转,并最终治愈这种肝脏疾病。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Commentary: Loss of FOCAD, Operating via the SKI Messenger RNA Surveillance Pathway, Causes a Pediatric Syndrome with Liver Cirrhosis [1]
Liver disease is becoming a major health concern and is estimated to be the fifth most common cause of death worldwide [2]. A systematic review from the Global Burden of Disease Study identified 1.32 million deaths due to liver cirrhosis in 2017, accounting for more than two percent of the total global deaths [3]. Liver cirrhosis is usually diagnosed late in life; it is irreversible, staying chronic for the rest of the patient ́s life; and it can be life-threatening, as it may evolve into hepatocellular carcinoma or lead to liver failure. Despite extensive research, to date none of the tested compounds for treating this disorder have proven to be safe and effective in human patients. Current available therapies are generally palliative and focused on slowing down the disease, with liver transplant remaining as the gold standard strategy to treat patients with decompensated cirrhosis. Therefore, there is a patent need for the identification and validation of new targets to treat, reverse and, ultimately, cure this hepatic disorder.
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