Gene-specific therapy for inherited arrhythmogenic diseases.

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Napolitano C, Bloise R, Priori SG

Gene-specific therapy for inherited arrhythmogenic diseases.

Pharmacol Ther. 2006 Apr;110(1):1-13. Epub 2005 Sep 15.

PubMed ID

16168489 [ View in PubMed

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Abstract

In the last few years, major advancement has been made in the understanding of the genetic basis of inherited arrhythmogenic diseases. Interestingly, the information obtained with the application of molecular genetics to these diseases is now influencing their clinical management, allowing gene-specific risk stratification and gene-specific management. The first attempt for a gene-specific therapy was made in 1995 with the use of mexiletine in long-QT syndrome (LQTS) patients with mutations in the SCN5A gene. Since then, several investigators have proposed novel therapeutic approaches based on the identification of the functional consequences of genetic mutations. In some instances, these novel therapies have already been introduced in clinical practice, and data are being collected to establish their long-term efficacy. In this review, we will summarize the current understanding of the molecular bases of inherited arrhythmias, with a specific focus toward discussing the most recent advancements toward the development of gene-specific therapies.

DrugBank Data that Cites this Article

Drug Targets

Drug	Target	Kind	Organism	Pharmacological Action	Actions
Mexiletine	Sodium channel protein type 5 subunit alpha	Protein	Humans	Yes	Inhibitor	Details