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Updated: Jun 7, 2025

Gene Transfer for Ischemic Heart Failure in a Preclinical Model
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心肌病基因治疗临床试验设计中的挑战 心肌病基因治疗临床试验设计

Tejus Satish1, Kimberly N Hong2, Juan Pablo Kaski3

  • 1University of Texas Southwestern Medical Center, Dallas, Texas, USA.

JACC. Heart failure
|November 15, 2024
PubMed
概括

基因疗法为罕见的遗传性心脏病 (孟德尔心肌病) 提供了希望. 本综述探讨了临床试验中的挑战,并提出了开发有效基因疗法的解决方案.

关键词:
心肌病性心脏病 - 心肌病性心脏病挑战 挑战 挑战 挑战 挑战临床试验临床试验临床试验临床试验临床试验基因治疗的基因疗法罕见病是一种罕见的疾病.

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