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

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

Arginase deficiency is associated with prominent neuromotor features, including spastic diplegia, clonus, and hyperreflexia; intellectual disability and progressive neurological decline are other signs. In a constitutive murine model, we recently described leukodystrophy as a significant component o...

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Bibliographic Details
Main Authors: Suhail Khoja, Xiao-Bo Liu, Brian Truong, Matthew Nitzahn, Jenna Lambert, Adam Eliav, Eram Nasser, Emma Randolph, Kristine E. Burke, Rebecca White, Xuling Zhu, Paolo G.V. Martini, Itzhak Nissim, Stephen D. Cederbaum, Gerald S. Lipshutz
Format: Article
Language:English
Published: Elsevier 2022-06-01
Series:Molecular Therapy: Nucleic Acids
Subjects:
MT: Oligonucleotides: Therapies and Applications
arginase deficiency
dysmyelination
lipid nanoparticle
messenger RNA
urea cycle
Online Access:http://www.sciencedirect.com/science/article/pii/S2162253122000889

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http://www.sciencedirect.com/science/article/pii/S2162253122000889

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