遗传性心律失常的基因和干细胞疗法

IF 12 1区 医学 Q1 PHARMACOLOGY & PHARMACY Pharmacology & Therapeutics Pub Date : 2024-01-30 DOI:10.1016/j.pharmthera.2024.108596
Zhong-He Zhang , Hector Barajas-Martinez , Hong Jiang , Cong-Xin Huang , Charles Antzelevitch , Hao Xia , Dan Hu
{"title":"遗传性心律失常的基因和干细胞疗法","authors":"Zhong-He Zhang ,&nbsp;Hector Barajas-Martinez ,&nbsp;Hong Jiang ,&nbsp;Cong-Xin Huang ,&nbsp;Charles Antzelevitch ,&nbsp;Hao Xia ,&nbsp;Dan Hu","doi":"10.1016/j.pharmthera.2024.108596","DOIUrl":null,"url":null,"abstract":"<div><p>Inherited cardiac arrhythmias are a group of genetic diseases predisposing to sudden cardiac arrest, mainly resulting from variants in genes encoding cardiac ion channels or proteins involved in their regulation. Currently available therapeutic options (pharmacotherapy, ablative therapy and device-based therapy) can not preclude the occurrence of arrhythmia events and/or provide complete protection. With growing understanding of the genetic background and molecular mechanisms of inherited cardiac arrhythmias, advancing insight of stem cell technology, and development of vectors and delivery strategies, gene therapy and stem cell therapy may be promising approaches for treatment of inherited cardiac arrhythmias. Recent years have witnessed impressive progress in the basic science aspects and there is a clear and urgent need to be translated into the clinical management of arrhythmic events. In this review, we present a succinct overview of gene and cell therapy strategies, and summarize the current status of gene and cell therapy. Finally, we discuss future directions for implementation of gene and cell therapy in the therapy of inherited cardiac arrhythmias.</p></div>","PeriodicalId":402,"journal":{"name":"Pharmacology & Therapeutics","volume":"256 ","pages":"Article 108596"},"PeriodicalIF":12.0000,"publicationDate":"2024-01-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"Gene and stem cell therapy for inherited cardiac arrhythmias\",\"authors\":\"Zhong-He Zhang ,&nbsp;Hector Barajas-Martinez ,&nbsp;Hong Jiang ,&nbsp;Cong-Xin Huang ,&nbsp;Charles Antzelevitch ,&nbsp;Hao Xia ,&nbsp;Dan Hu\",\"doi\":\"10.1016/j.pharmthera.2024.108596\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<div><p>Inherited cardiac arrhythmias are a group of genetic diseases predisposing to sudden cardiac arrest, mainly resulting from variants in genes encoding cardiac ion channels or proteins involved in their regulation. Currently available therapeutic options (pharmacotherapy, ablative therapy and device-based therapy) can not preclude the occurrence of arrhythmia events and/or provide complete protection. With growing understanding of the genetic background and molecular mechanisms of inherited cardiac arrhythmias, advancing insight of stem cell technology, and development of vectors and delivery strategies, gene therapy and stem cell therapy may be promising approaches for treatment of inherited cardiac arrhythmias. Recent years have witnessed impressive progress in the basic science aspects and there is a clear and urgent need to be translated into the clinical management of arrhythmic events. In this review, we present a succinct overview of gene and cell therapy strategies, and summarize the current status of gene and cell therapy. Finally, we discuss future directions for implementation of gene and cell therapy in the therapy of inherited cardiac arrhythmias.</p></div>\",\"PeriodicalId\":402,\"journal\":{\"name\":\"Pharmacology & Therapeutics\",\"volume\":\"256 \",\"pages\":\"Article 108596\"},\"PeriodicalIF\":12.0000,\"publicationDate\":\"2024-01-30\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Pharmacology & Therapeutics\",\"FirstCategoryId\":\"3\",\"ListUrlMain\":\"https://www.sciencedirect.com/science/article/pii/S0163725824000160\",\"RegionNum\":1,\"RegionCategory\":\"医学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q1\",\"JCRName\":\"PHARMACOLOGY & PHARMACY\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Pharmacology & Therapeutics","FirstCategoryId":"3","ListUrlMain":"https://www.sciencedirect.com/science/article/pii/S0163725824000160","RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q1","JCRName":"PHARMACOLOGY & PHARMACY","Score":null,"Total":0}
引用次数: 0

摘要

遗传性心律失常是一组易导致心脏骤停的遗传疾病,主要是由于编码心脏离子通道或参与其调节的蛋白质的基因发生变异所致。目前可用的治疗方案(药物治疗、消融治疗和基于设备的治疗)无法避免心律失常事件的发生和/或提供完全的保护。随着对遗传性心律失常的遗传背景和分子机制的了解不断加深,对干细胞技术的认识不断提高,以及载体和递送策略的发展,基因治疗和干细胞治疗可能成为治疗遗传性心律失常的有前途的方法。近年来,基础科学方面取得了令人瞩目的进展,但显然迫切需要将其转化为心律失常的临床治疗方法。在这篇综述中,我们简明扼要地概述了基因和细胞治疗策略,并总结了基因和细胞治疗的现状。最后,我们讨论了基因和细胞疗法在治疗遗传性心律失常方面的未来发展方向。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
查看原文
分享 分享
微信好友 朋友圈 QQ好友 复制链接
本刊更多论文
Gene and stem cell therapy for inherited cardiac arrhythmias

Inherited cardiac arrhythmias are a group of genetic diseases predisposing to sudden cardiac arrest, mainly resulting from variants in genes encoding cardiac ion channels or proteins involved in their regulation. Currently available therapeutic options (pharmacotherapy, ablative therapy and device-based therapy) can not preclude the occurrence of arrhythmia events and/or provide complete protection. With growing understanding of the genetic background and molecular mechanisms of inherited cardiac arrhythmias, advancing insight of stem cell technology, and development of vectors and delivery strategies, gene therapy and stem cell therapy may be promising approaches for treatment of inherited cardiac arrhythmias. Recent years have witnessed impressive progress in the basic science aspects and there is a clear and urgent need to be translated into the clinical management of arrhythmic events. In this review, we present a succinct overview of gene and cell therapy strategies, and summarize the current status of gene and cell therapy. Finally, we discuss future directions for implementation of gene and cell therapy in the therapy of inherited cardiac arrhythmias.

求助全文
通过发布文献求助,成功后即可免费获取论文全文。 去求助
来源期刊
CiteScore
23.00
自引率
0.70%
发文量
222
审稿时长
90 days
期刊介绍: Pharmacology & Therapeutics, in its 20th year, delivers lucid, critical, and authoritative reviews on current pharmacological topics.Articles, commissioned by the editor, follow specific author instructions.This journal maintains its scientific excellence and ranks among the top 10 most cited journals in pharmacology.
期刊最新文献
Pro-resolving lipid mediators and therapeutic innovations in resolution of inflammation. The pharmacodynamic and pharmacological mechanisms underlying nanovesicles of natural products: Developments and challenges. Exploring the landscape of post-translational modification in drug discovery. New drug discovery and development from natural products: Advances and strategies. Neurobiology of cancer: Adrenergic signaling and drug repurposing
×
引用
GB/T 7714-2015
复制
MLA
复制
APA
复制
导出至
BibTeX EndNote RefMan NoteFirst NoteExpress
×
×
提示
您的信息不完整,为了账户安全,请先补充。
现在去补充
×
提示
您因"违规操作"
具体请查看互助需知
我知道了
×
提示
现在去查看 取消
×
提示
确定
0
微信
客服QQ
Book学术公众号 扫码关注我们
反馈
×
意见反馈
请填写您的意见或建议
请填写您的手机或邮箱
已复制链接
已复制链接
快去分享给好友吧!
我知道了
×
扫码分享
扫码分享
Book学术官方微信
Book学术文献互助
Book学术文献互助群
群 号:481959085
Book学术
文献互助 智能选刊 最新文献 互助须知 联系我们:info@booksci.cn
Book学术提供免费学术资源搜索服务,方便国内外学者检索中英文文献。致力于提供最便捷和优质的服务体验。
Copyright © 2023 Book学术 All rights reserved.
ghs 京公网安备 11010802042870号 京ICP备2023020795号-1