反义寡核苷酸:慢性肾脏疾病的潜在治疗干预

Yalin Li, Yuqin Tan, Rui Zhang, Tao Wang, N. Na, T. Zheng, R. Veedu, Suxiang Chen
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摘要

慢性肾脏病(CKD)是一个全球性的公共卫生问题,给发达国家和发展中国家的医疗保健系统带来了越来越大的负担。CKD是一种进行性和不可逆转的疾病,影响着全球约10%的人口。进展为终末期肾病(ESRD)的患者需要昂贵的肾脏替代治疗,即透析或肾移植。目前的CKD治疗主要依赖于血管紧张素转换酶抑制剂(ACEis)和血管紧张素受体阻滞剂(ARBs)的使用。然而,这些治疗并不能阻止CKD向ESRD的进展。因此,迫切需要开发新的治疗方法。反义寡核苷酸(ASO)作为一种药物开发平台,近年来引起了人们的极大兴趣。到目前为止,已有八种基于ASO的药物获得美国食品和药物管理局的批准,用于治疗各种疾病。在此,我们综述了为鉴定CKD相关基因和/或同时开发ASOs作为治疗CKD的潜在疗法而开发的ASOs。
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Antisense Oligonucleotide: A Potential Therapeutic Intervention for Chronic Kidney Disease
Chronic kidney disease (CKD) is a global public health issue that places an increasing burden on the healthcare systems of both the developed and developing countries. CKD is a progressive and irreversible condition, affecting approximately 10% of the population worldwide. Patients that have progressed to end-stage renal disease (ESRD) require expensive renal replacement therapy, i.e., dialysis or kidney transplantation. Current CKD therapy largely relies on the use of angiotensin-converting enzyme inhibitors (ACEis) and angiotensin receptor blockers (ARBs). However, these treatments by no means halt the progression of CKD to ESRD. Therefore, the development of new therapies is urgently needed. Antisense oligonucleotide (ASO) has recently attracted considerable interest as a drug development platform. Thus far, eight ASO-based drugs have been granted approval by the US Food and Drug Administration for the treatment of various diseases. Herein, we review the ASOs developed for the identification of CKD-relevant genes and/or the simultaneous development of the ASOs as potential therapeutics towards treating CKD.
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