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对基因治疗的结果监测和中间终点

Deni Kukavica1, Alessandro Trancuccio2, Gabriel Brooks3

  • 1Molecular Cardiology Unit, IRCCS Istituti Clinici Scientifici Maugeri, Pavia, Italy; Department of Molecular Medicine, University of Pavia, Pavia, Italy. Electronic address: https://x.com/DeniKukavica.

The Canadian journal of cardiology
|December 4, 2025
PubMed
概括

设计有效的基因疗法临床试验需要仔细的结果监测和终点选择. 这对于评估治疗疗效和确保患者安全至关重要,特别是考虑到这些疗法的不可逆转性质.

关键词:
心肌病性心脏病 - 心肌病性心脏病道病变是一种道病变.基因治疗的基因疗法遗传性心律失常综合征遗传性心律失常综合征结果结果结果结果.试验 试验 试验 试验

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