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促红细胞生成素受体(EpoR)激动剂被用于治疗广泛的疾病。

Erythropoietin receptor (EpoR) agonism is used to treat a wide range of disease.

机构信息

Department of Physiology, Faculty of Medicine, University of Valencia, Research Foundation of the University Clinic Hospital of Valencia/INCLIVA, Valencia, Spain.

出版信息

Mol Med. 2013 Apr 30;19(1):62-4. doi: 10.2119/molmed.2013.00025.

Abstract

The erythropoietin receptor (EpoR) was discovered and described in red blood cells (RBCs), stimulating its proliferation and survival. The target in humans for EpoR agonists drugs appears clear-to treat anemia. However, there is evidence of the pleitropic actions of erythropoietin (Epo). For that reason, rhEpo therapy was suggested as a reliable approach for treating a broad range of pathologies, including heart and cardiovascular diseases, neurodegenerative disorders (Parkinson's and Alzheimer's disease), spinal cord injury, stroke, diabetic retinopathy and rare diseases (Friedreich ataxia). Unfortunately, the side effects of rhEpo are also evident. A new generation of nonhematopoietic EpoR agonists drugs (asialoEpo, Cepo and ARA 290) have been investigated and further developed. These EpoR agonists, without the erythropoietic activity of Epo, while preserving its tissue-protective properties, will provide better outcomes in ongoing clinical trials. Nonhematopoietic EpoR agonists represent safer and more effective surrogates for the treatment of several diseases such as brain and peripheral nerve injury, diabetic complications, renal ischemia, rare diseases, myocardial infarction, chronic heart disease and others.

摘要

促红细胞生成素受体 (EpoR) 在红细胞 (RBC) 中被发现并描述,刺激其增殖和存活。EpoR 激动剂药物在人类中的靶点似乎很明确——治疗贫血。然而,促红细胞生成素 (Epo) 的多种作用有证据表明。因此,rhEpo 治疗被认为是治疗广泛病理疾病的可靠方法,包括心脏和心血管疾病、神经退行性疾病(帕金森病和阿尔茨海默病)、脊髓损伤、中风、糖尿病性视网膜病变和罕见疾病(弗里德里希共济失调)。不幸的是,rhEpo 的副作用也很明显。新一代非造血性 EpoR 激动剂药物(去唾液酸 Epo、Cepo 和 ARA 290)已被研究并进一步开发。这些 EpoR 激动剂没有 Epo 的促红细胞生成活性,但保留了其组织保护特性,将在正在进行的临床试验中提供更好的结果。非造血性 EpoR 激动剂代表了治疗多种疾病(如脑和周围神经损伤、糖尿病并发症、肾缺血、罕见疾病、心肌梗死、慢性心脏病等)更安全、更有效的替代品。

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