Articles Tagged With: neurogenetic
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Gene and Substrate Therapy for Neurogenetic Disease: A Combined Approach to Treat Mitochondrial Myopathy
A combined adenoviral-mediated gene therapy plus substrate therapy delivered to a mouse model of thymidine kinase 2 (TK2) deficiency, manifested most often as a fatal mitochondrial myopathy in infants and children, rescued TK2 activity and prolonged animal lifespan, thus indicating a promising therapeutic approach for affected patients.