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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Detalles Bibliográficos
Autores principales: Khoja, Suhail, Liu, Xiao-Bo, Truong, Brian, Nitzahn, Matthew, Lambert, Jenna, Eliav, Adam, Nasser, Eram, Randolph, Emma, Burke, Kristine E., White, Rebecca, Zhu, Xuling, Martini, Paolo G.V., Nissim, Itzhak, Cederbaum, Stephen D., Lipshutz, Gerald S.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2022
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9156989/
https://www.ncbi.nlm.nih.gov/pubmed/35694211
http://dx.doi.org/10.1016/j.omtn.2022.04.012

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