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.
Denegri M, Bongianino R, Lodola F, Boncompagni S, De Giusti VC, Avelino-Cruz JE, Liu N, Persampieri S, Curcio A, Esposito F, Pietrangelo L, Marty I, Villani L, Moyaho A, Baiardi P, Auricchio A, Protasi F, Napolitano C, Priori SG.
Denegri M, et al. Among authors: lodola f.
Circulation. 2014 Jun 24;129(25):2673-81. doi: 10.1161/CIRCULATIONAHA.113.006901. Epub 2014 Jun 2.
Circulation. 2014.
PMID: 24888331
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