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Description

Following an oral sucrose challenge, hCitrinmRNAtreated double KO mice further showed hepatic citrulline and plasma ammonium concentrations lower than those of double KO mice treated with a control mRNA, with concentrations of these metabolites maintained at levels similar to those measured in wildtype mice [118]

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Nervous system of patients with epilepsy suffers severe damage because phagocytes rupture after penetrating the spinal cord tissue and invade the spinal fluid which further enters CSF

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CRISPR Therapeutics is pushing beyond rare diseases and into cardiometabolic territory with its in vivo editing platformdebuting first-in-human data for CTX310 (targeting ANGPTL3) and preclinical results for CTX340 (targeting AGT) at AHA 2025

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