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
