Tag: citrullinemia

Prime editing therapy delivered by lipid nanoparticles correcting CPS1 gene mutation in liver cells
Drugs and Treatments

Gene-Editing Therapy Reverses Genetic Liver Disease in Mice: First In Vivo Success

Researchers have successfully reversed citrullinemia type I symptoms in mice using RNA-loaded lipid nanoparticles to deliver prime-editing machinery. This breakthrough…

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