Exploring the Therapeutic Potential of Gene Therapy in Arrhythmogenic Right Ventricular Cardiomyopathy

被引：5

作者：

Mundisugih, Juan ^{[1
,2
,3
]}

Ravindran, Dhanya ^{[1
,3
]}

Kizana, Eddy ^{[1
,2
,3
]}

机构：

[1] Westmead Inst Med Res, Ctr Heart Res, Westmead, NSW 2145, Australia

[2] Westmead Hosp, Dept Cardiol, Westmead, NSW 2145, Australia

[3] Univ Sydney, Sydney Med Sch, Camperdown, NSW 2050, Australia

来源：

BIOMEDICINES | 2024年 / 12卷 / 06期

基金：

英国医学研究理事会;

关键词：

adeno-associated virus; gene therapy; arrhythmogenic right ventricular cardiomyopathy; genetic heart disease; biotechnology; AAV; DELIVERY; VECTORS; TRIAL;

D O I：

10.3390/biomedicines12061351

中图分类号：

Q5 [生物化学]; Q7 [分子生物学];

学科分类号：

071010 ; 081704 ;

摘要：

Right dominant arrhythmogenic cardiomyopathy, commonly known as Arrhythmogenic Right Ventricular Cardiomyopathy (ARVC), represents a formidable challenge in cardiovascular medicine, as conventional therapies are commonly ineffective in impeding disease progression and the development of end-stage heart failure. Recombinant adeno-associated virus (AAV)-mediated gene therapy presents a promising avenue for targeted therapeutic interventions, potentially revolutionising treatment approaches for ARVC patients. Encouraging results from preclinical studies have sparked optimism about the possibility of curing specific subtypes of ARVC in the near future. This narrative review delves into the dynamic landscape of genetic therapy for ARVC, elucidating its underlying mechanisms and developmental stages, and providing updates on forthcoming trials. Additionally, it examines the hurdles and complexities impeding the successful translation of ARVC genetic therapies into clinical practice. Despite notable scientific advancements, the journey towards implementing genetic therapies for ARVC patients in real-world clinical settings is still in its early phases.

引用

页数：14

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