Concepedia

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,

Molecular Therapy — Nucleic Acids · 2022 · 25 citations · 47 references

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Abstract

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

References

47

The metabolic and molecular bases of inherited disease

Ying-Tsong Chen · Medical Entomology and Zoology · 2001 · 6.9K citations

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Targeted Delivery of RNAi Therapeutics With Endogenous and Exogenous Ligand-Based Mechanisms

Akin Akinc, William Querbes, Soma De et al. · Molecular Therapy · 2010 · 1.2K citations · Full text

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TNFα promotes proliferation of oligodendrocyte progenitors and remyelination

Heather A. Arnett, Jeff Mason, Mike Marino et al. · Nature Neuroscience · 2001 · 993 citations

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A Novel Amino Lipid Series for mRNA Delivery: Improved Endosomal Escape and Sustained Pharmacology and Safety in Non-human Primates

Staci Sabnis, E. Sathyajith Kumarasinghe, Timothy Salerno et al. · Molecular Therapy · 2018 · 794 citations · Full text

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What Is the Optimal Value of the g-Ratio for Myelinated Fibers in the Rat CNS? A Theoretical Approach

Taylor Chomiak, Bin Hu · PLoS ONE · 2009 · 459 citations · Full text

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