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Genetic suppression features <i>ABHD18</i> as a Barth syndrome therapeutic target
Nature, Published online: 03 September 2025; doi:10.1038/s41586-025-09373-5The enzyme ABHD18 is shown to have a key role in cardiolipin metabolism in mitochondria, offering a potential route for a small-molecule treatment of the rare genetic disease Barth syndrome.
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