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Targeted Brain Delivery of Dendrimer-4-Phenylbutyrate Ameliorates Neurological Deficits in a Long-Term ABCD1-Deficient Mouse Model of X-Linked Adrenoleukodystrophy

X-linked adrenoleukodystrophy (ALD) is a genetic disorder that presents neurologically as either a rapid and fatal cerebral demyelinating disease in childhood (childhood cerebral adrenoleukodystrophy; ccALD) or slow degeneration of the spinal cord in adulthood (adrenomyeloneuropathy; AMN). All forms...

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Detalles Bibliográficos
Autores principales: Nemeth, Christina L., Gӧk, Özgül, Tomlinson, Sophia N., Sharma, Anjali, Moser, Ann B., Kannan, Sujatha, Kannan, Rangaramanujam M., Fatemi, Ali
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Springer International Publishing 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9542479/
https://www.ncbi.nlm.nih.gov/pubmed/36207570
http://dx.doi.org/10.1007/s13311-022-01311-x