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Second, translational tools should focus on dual-targeted nanocarriers designed to reach cerebellum and myocardium—using TAT peptides for blood–brain barrier penetration and CTP peptides for cardiomyocyte targeting—loaded with mitochondria-directed ferroptosis modulators capable of activating GPX4 while supporting iron–sulfur cluster synthesis, and released in response to GAA-repeat-dependent triggers to avoid off-target iron perturbation
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doi: 10.1200/JCO.2009.23.8329
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