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Early clinical studies in Barth syndrome, primary mitochondrial myopathies, and age-related macular degeneration have shown favorable safety, tolerability, and signals of efficacy (Thompson et al., 2024)

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Intermittent PI3K inhibition reshapes the immune TME in PTEN-null prostate cancer, converts cold tumors to hot tumors, suppresses T regs while preserving CD8 + T-cell function, and maintains inflammatory states posttreatment

Expression of interferon-stimulated genes in insulitic pancreatic islets of patients recently diagnosed with type 1 diabetes