Intermittent lipid nanoparticle mRNA administration prevents cortical dysmyelination associated with arginase deficiency
Suhail Khoja, Xiao-Bo Liu, Brian Truong, Matthew Nitzahn, Jenna Lambert, Adam Eliav, Eram Nasser, Emma Randolph, Kristine Burke, Rebecca White, Xuling Zhu, Paolo G.V. Martini, Itzhak Nissim, Stephen D. Cederbaum, Gerald S. Lipshutz
Abstract
mRNA administration in arginase deficiency prevents the associated leukodystrophy and restores normal oligodendrocyte function.
Topics & Concepts
ArginaseMyelinOligodendrocyteEndocrinologyInternal medicineLeukodystrophyBiologyCentral nervous systemChemistryMedicineBiochemistryAmino acidDiseaseArginineMetabolism and Genetic DisordersNeurogenetic and Muscular Disorders ResearchAmino Acid Enzymes and Metabolism