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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| Main Authors: | , , , , , , , , , , , , , , |
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| Format: | Book |
| Published: |
Elsevier,
2022-06-01T00:00:00Z.
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| Subjects: | |
| Online Access: | Connect to this object online. |
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