Targeted mRNA Therapy for Ornithine Transcarbamylase Deficiency
In this issue of Molecular Therapy, Prieve et al. describe a novel, targeted mRNA
delivery system that corrects a rare liver disease in a murine model of ornithine
transcarbamylase deficiency having a single enzyme defect. This non-viral delivery
method allows multi-dose, systemic administration for treatment of inherited metabolic
diseases.
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