Intermittent lipid nanoparticle mRNA administration prevents cortical dysmyelination associated with arginase deficiency

Suhail Khoja(University of California, Los Angeles), Xiao-Bo Liu(University of California, Los Angeles), Brian Truong(University of California, Los Angeles), Matthew Nitzahn, Jenna Lambert(University of California, Los Angeles), Adam Eliav(University of California, Los Angeles), Eram Nasser(University of California, Los Angeles), Emma Randolph(University of California, Los Angeles), Kristine Burke(Moderna Therapeutics (United States)), Rebecca White(Moderna Therapeutics (United States)), Xuling Zhu(Moderna Therapeutics (United States)), Paolo G.V. Martini(Moderna Therapeutics (United States)), Itzhak Nissim(Children's Hospital of Philadelphia), Stephen D. Cederbaum(University of California, Los Angeles), Gerald S. Lipshutz(University of California, Los Angeles)
Molecular Therapy — Nucleic Acids
April 27, 2022
10.1016/j.omtn.2022.04.012
Cited by 24Open Access
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Abstract

mRNA administration in arginase deficiency prevents the associated leukodystrophy and restores normal oligodendrocyte function.

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