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基因治疗与心血管疾病

Gene Therapy and Cardiovascular Diseases.

作者信息

Lu Dongchao, Cushman Sarah, Thum Thomas, Bär Christian

机构信息

Institute of Molecular and Translational Therapeutic Strategies, Hannover Medical School, Hannover, Germany.

REBIRTH Center for Translational Regenerative Medicine, Hannover Medical School, Hannover, Germany.

出版信息

Adv Exp Med Biol. 2023;1396:235-254. doi: 10.1007/978-981-19-5642-3_16.

Abstract

Cardiovascular diseases (CVDs) are the leading causes of death globally and urgently require new novel therapeutic strategies. Gene therapy is the application of gene modulation technology to treat abnormal gene expression under disease conditions. Viral- and nonviral-based gene delivery systems are the foundation of gene modulation in target cells. Moreover, plasmid- or oligo-based gene modulation tools as well as new advancements in gene editing using CRISPR/Cas technology are currently being tested in a variety of clinical trials. Here, we summarized state-of-the-art gene therapy technologies as well as recent clinical trials and discuss the applications and lessons of gene therapy in CVDs.

摘要

心血管疾病(CVDs)是全球主要的死亡原因,迫切需要新的治疗策略。基因治疗是应用基因调控技术来治疗疾病状态下的异常基因表达。基于病毒和非病毒的基因递送系统是靶细胞中基因调控的基础。此外,基于质粒或寡核苷酸的基因调控工具以及使用CRISPR/Cas技术的基因编辑新进展目前正在各种临床试验中进行测试。在此,我们总结了最先进的基因治疗技术以及最近的临床试验,并讨论了基因治疗在心血管疾病中的应用和经验教训。

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