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Am J Pediatr Hematol Oncol

Specifically modified nanocarriers delivering targeted drugs can clear damaged mitochondria by initiating the mitochondrial autophagy program, effectively restoring mitochondrial homeostasis, reducing the neurotoxicity caused by oxidative stress, and rescuing the motor dysfunction in animal models

41,42 Our findings establish lentiviral U7-snRNA-mediated exon skipping as a promising, mechanism-based therapeutic approach for NF1 -mutant BC and support its further development for genetically defined, treatment-refractory tumors