| abstract |
The present invention relates to mRNA therapy for the treatment of Fabry disease. The mRNA for use in the present invention encodes a fusion protein comprising human alpha-galactosidase A (GLA), an isoform thereof, a functional fragment thereof, and GLA when administered in vivo. The mRNA of the invention is preferably encapsulated in lipid nanoparticles (LNP) to achieve efficient delivery to cells and / or tissues in the subject when administered to the subject. The mRNA therapy of the invention increases and / or restores deprived levels of GLA expression and / or activity in a subject. The mRNA therapies of the invention further reduce the levels of toxic metabolites associated with depleted GLA activity in the subject, namely Gb3 and Lyso-Gb3. 【Selection chart】 None |