Article
Single delivery of an adeno-associated viral construct to transfer the CASQ2 gene to knock-in mice affected by catecholaminergic polymorphic ventricular tachycardia is able to cure the disease from birth to advanced age.
Circulation - 24 Jun 2014
Denegri Marco, Bongianino Rossana, Lodola Francesco, Boncompagni Simona, De Giusti Verónica C, Avelino-Cruz José E, Liu Nian, Persampieri Simone, Curcio Antonio, Esposito Francesca, Pietrangelo Laura, Marty Isabelle, Villani Laura, Moyaho Alejandro, Baiardi Paola, Auricchio Alberto, Protasi Feliciano, Napolitano Carlo, Priori Silvia G
Abstract excerpt
BACKGROUND: Catecholaminergic polymorphic ventricular tachycardia is an inherited arrhythmogenic disorder characterized by sudden cardiac death in children. Drug therapy is still insufficient to provide full protection against cardiac arrest, and the use of implantable defibrillators in the pedia...
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