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

• Main Authors: Suhail Khoja (Author), Xiao-Bo Liu (Author), Brian Truong (Author), Matthew Nitzahn (Author), Jenna Lambert (Author), Adam Eliav (Author), Eram Nasser (Author), Emma Randolph (Author), Kristine E. Burke (Author), Rebecca White (Author), Xuling Zhu (Author), Paolo G.V. Martini (Author), Itzhak Nissim (Author), Stephen D. Cederbaum (Author), Gerald S. Lipshutz (Author)
• Format: Book
• Published: Elsevier, 2022-06-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

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...