Gene therapy offers hope for rare, deadly heart disease in young men

A team at the Centro Nacional de Investigaciones Cardiovasculares (CNIC) has developed an innovative gene-therapy strategy that could transform the treatment of arrhythmogenic right ventricular cardiomyopathy type 5 (ARVC5), a rare and highly penetrant inherited cardiac disorder with an elevated risk of sudden cardiac death. This disease is particularly devastating in young men and lacks a cure, with current treatments focusing on palliative care.

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