| abstract |
The present invention relates to mRNA therapeutics for the treatment of type II citrullineemia ("CTLN2"). The mRNA used in the present invention, when administered in vivo, encodes a fusion protein comprising human citrin, an isoform thereof, a functional fragment thereof, and citrin. The mRNA of the present invention is preferably encapsulated in lipid nanoparticles (LNP) to achieve efficient delivery to cells and / or tissues of the subject when it is administered. The mRNA therapy of the invention increases and / or restores insufficient levels of citrin expression and / or activity in a subject. The mRNA therapy of the present invention further reduces the level of biomarker associated with poor citrin activity in the subject, ie, ammonia and / or triglycerides. |