载药红细胞技术的最新进展和临床应用
Ongoing Developments and Clinical Progress in Drug-Loaded Red Blood Cell Technologies.
机构信息
Department of Biomolecular Sciences, Università degli Studi di Urbino Carlo Bo, 61029, Urbino, PU, Italy.
EryDel SpA, 20091, Bresso, MI, Italy.
出版信息
BioDrugs. 2020 Jun;34(3):265-272. doi: 10.1007/s40259-020-00415-0.
Abstract
Engineered red blood cells (RBCs) appear to be a promising method for therapeutic drug and protein delivery. With a number of agents in clinical trials (e.g., dexamethasone 21-phosphate in ataxia telangiectasia, asparaginase in pancreatic cancer/acute lymphoblastic leukemia, thymidine phosphorylase in mitochondrial neurogastrointestinal encephalomyopathy, RTX-134 in phenylketonuria, etc.), this leading article summarizes the ongoing efforts in developing these agents, focuses on the clinical progress, and provides a brief background into engineered RBCs and the different ways in which they can be exploited for therapeutic/diagnostic purposes. References to available data on safety, efficacy, and tolerability are reported. Due to the continuous progress in this field, the information is updated as of January 2020 from databases, websites, and press releases of the involved companies and information that is in the public domain.
摘要
工程化红细胞（RBC）似乎是一种很有前途的治疗性药物和蛋白质递药方法。目前有许多药物正在临床试验中（例如，共济失调毛细血管扩张症中的地塞米松 21-磷酸、胰腺癌/急性淋巴细胞白血病中的门冬酰胺酶、线粒体神经胃肠脑肌病中的胸苷磷酸化酶、苯丙酮尿症中的 RTX-134 等），本文综述了正在开发这些药物的努力，重点介绍了临床进展，并简要介绍了工程化 RBC 及其用于治疗/诊断目的的不同方式。报告了有关安全性、有效性和耐受性的可用数据的参考文献。由于该领域的持续进展，本文中的信息截至 2020 年 1 月，是从相关公司的数据库、网站和新闻稿以及公开信息中更新的。
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本文引用的文献
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