精准医学的综合患者数字和仿生双胞胎:一个前景
Mark T Miedel1, Mark E Schurdak2, Andrew M Stern2
1Department of Pharmacology and Chemical Biology, Organ Pathobiology and Therapeutics Institute, University of Pittsburgh, Pittsburgh, Pennsylvania.
Seminars in liver disease
|July 4, 2025
概括
使用患者数字双胞胎 (PDT) 和仿生双胞胎 (PBT) 的新精密医疗平台可以改善治疗代谢功能障碍相关脂肪性肝病 (MASLD) 等复杂疾病的药物开发. 这种方法模拟个体患者以预测治疗反应,克服当前方法的局限性.
科学领域:
- 生物医学工程 生物医学工程
- 计算生物学 计算生物学
- 翻译医学是一种翻译医学.
背景情况:
- 复杂的疾病,如代谢功能障碍相关的脂肪性肝病 (MASLD),表现出显著的患者异质性,使药物开发和治疗策略复杂化.
- 目前对MASLD的治疗方法由于患者反应不可预测和药物成本高,所以成功程度有限.
- 现有的药物开发范式与异质疾病的多面性质作斗争,导致需要新的战略.
研究的目的:
- 引入一个新的精准医学平台,整合患者数字双胞胎 (PDT) 和患者仿生双胞胎 (PBT).
- 应对开发有效药物开发和患者治疗策略的挑战,以应对像MASLD这样复杂,异构的疾病.
- 利用计算建模和患者衍生器官来预测治疗反应.
主要方法:
- 患者数字双胞胎 (PDT) 的生成使用clinomics和multi-omics数据用于计算患者建模.
- 从患者衍生的有机体或诱导的多能干干细胞制造患者仿生双胞胎 (PBTs).
- 将PDT与PBT集成,以创建模拟患者病理生理学的实验模型,用于预测测试.
主要成果:
- 集成的PDT-PBT平台可以对治疗反应进行个性化预测.
- 这种方法有可能克服目前用于异质疾病的药物开发方法的局限性.
- 该平台允许在反映个体患者状况的仿生模型中测试治疗预测.
结论:
- 患者数字双胞胎和仿生双胞胎的整合为精准医学提供了一个强大的新平台.
- 这种创新方法对推动药物开发和为像MASLD这样的复杂疾病量身定制治疗策略具有重大前景.
- 虽然挑战仍然存在,但这种范式转变有可能彻底改变以高患者异质性为特征的疾病的治疗.
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