Effect of hCitrin-mRNA Therapy on Sucrose Aversion and Blood Ammonia in the CTLN2 Mouse Model
(A) Effect of hCitrin-mRNA therapy on sucrose aversion in CTLN2 mice. Preference index: intake of 20% sucrose versus water that was measured before (left) or after (right) mRNA therapy. (B) Effect of hCitrin-mRNA therapy on hepatic citrulline (left) and blood ammonia (right) in CTLN2 mice following sucrose challenge. CTLN2 mice were injected intravenously (i.v.) with LNPs containing either a non-specific control mRNA (Ctrl) or the hCitrin-mRNACoV (0.5 mg/kg). Twenty-four hours following mRNA injection, the mice were challenged by oral administration of 50% sucrose, followed by collection of the liver for citrulline analysis and plasma for ammonium analysis (Materials and Methods). The data are expressed as mean ± SEM (n = 3–5 per group). Student’s t test was performed to assess statistical relevance between the groups. *p < 0.05; NS, not significant.