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Mutations identified in patients with defective lipoic acid metabolism affect genes involved in the synthesis of iron-sulfur clusters and genes coding for lipoic acid synthetase ( LIAS ), lipoyl transferase 1 ( LIPT1 ), and dihydrolipoamide dehydrogenase (E3 component of -ketoacid dehydrogenase complexes
they have each had decades of human trials, been FDA approved, and are approved for multiple medical uses
75,76 ASOs that employ this mechanism are splicing-switching oligonucleotides, which modulate functional target gene expression by skipping or including exons
A recent 2025 animal study found that this promising peptide dramatically reduced damage to the liver, kidneys, and lungs after ischemia-reperfusion a type of injury that can occur when blood flow is temporarily cut off like during a heart attack or stroke and then suddenly restored
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