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

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

Khoja, S., Liu, X. B., Truong, B., Nitzahn, M., Lambert, J., Eliav, A., Nasser, E., Randolph, E., Burke, K. E., White, R., Zhu, X., Martini, P. G., Nissim, I., Cederbaum, S. D., & Lipshutz, G. S. (2022). Intermittent lipid nanoparticle mRNA administration prevents cortical dysmyelination associated with arginase deficiency. Molecular Therapy – Nucleic Acids, 28, 859–874. https://doi.org/10.1016/j.omtn.2022.04.012

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