Gene therapy reverses heart failure in mouse model of Barth syndrome

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A gene therapy delivery vector (adeno-associated virus) being taken up in the heart, as shown in green at increasing magnification. Credit: (Adapted from Prendiville TW et al., PLoS One 2015 May 29, 10.1371/journal.pone.0128105) Barth syndrome is a rare metabolic disease in boys caused by mutation of a gene called […]