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基于人类的复杂的体外模型:它们对罕见疾病治疗的承诺和潜力

Surat Parvatam¹, Francesca Pistollato², Lindsay J Marshall³

¹Department of Research and Toxicology, Humane Society International/India, Hyderabad, India.

Frontiers in cell and developmental biology

|February 11, 2025

概括

复杂的体外模型 (CIVM) 为罕见疾病研究提供了有前途的患者特异性临床前系统. 这些以人类为基础的模型加速了对罕见遗传疾病的有效和负担得起的治疗方法的开发.

科学领域:

生物医学研究生物医学研究
罕见疾病治疗方法罕见疾病治疗方法
临床前建模临床前模型

背景情况:

罕见疾病影响全球人口的高达10%,已识别了7,000多种类型.
近95%的罕见病缺乏经批准的治疗方法,现有的治疗方法往往过于昂贵.
超过70%的罕见疾病是遗传性的,需要个性化的临床前模型来开发有效的治疗方法.

研究的目的:

审查复杂体外模型 (CIVM) 在罕见疾病研究中的应用.
探索CIVM在临床试验中的使用,以开发罕见疾病疗效模型.
提出利用CIVMs的策略,以提高罕见疾病治疗研究的翻译.

主要方法:

关于CIVM和罕见疾病的当前文献的叙事综述.
讨论CIVM,包括诱导多能干细胞 (iPSC),有机体和芯片上的器官.
分析CIVM在提供临床前疗效数据方面的能力.

主要成果:

CIVM正在成为用于罕见疾病研究的强大基于人体的临床前系统.
这些模型提供了关键的疗效数据,以推动药物开发进入临床试验.
在临床试验中,CIVM正在被用于建立罕见疾病的疗效模型.

结论:

CIVM为罕见疾病提供了个性化,有效和负担得起的治疗选择的途径.
建议优化CIVM的使用,以提高全球的研究可翻译性.
利用CIVM可以加速在高收入国家和低收入和中等收入国家开发罕见疾病治疗方法.

关键词:

在CIVM中,你会发现.囊性纤维化症是什么药物开发是药物的发展.这些是iPSCs.器官在芯片上的器官有机生物有机物罕见的疾病罕见的疾病

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