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Endosomal escape cell-penetrating peptides significantly enhance pharmacological effectiveness and CNS activity of systemically administered antisense oligonucleotides

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Molecular analysis revealed that, compared to controls, Usp2 OE mice exhibited enhanced induction of fibrotic (CCN2, FN1, COL1A1, and COL3A1) and ferroptotic (HMOX1) markers, alongside further suppression of GPX4 following UUO (Fig

Mounting studies have revealed that cell injury in HD primarily involves the loss of astrocytes, oligodendrocytes, and neurons, potentially affecting the cerebral cortex and striatum (Yu et al., 2003