AAV-Mediated Artificial miRNA Reduces Pathogenic Polyglucosan Bodies and Neuroinflammation in Adult Polyglucosan Body and Lafora Disease Mouse Models

Adult polyglucosan body disease (APBD) and Lafora disease (LD) are autosomal recessive glycogen storage neurological disorders. APBD is caused by mutations in the glycogen branching enzyme (GBE1) gene and is characterized by progressive upper and lower motor neuron dysfunction and premature death. L...

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Detalles Bibliográficos
Autores principales: Gumusgoz, Emrah, Kasiri, Sahba, Guisso, Dikran R., Wu, Jun, Dear, Matthew, Verhalen, Brandy, Minassian, Berge A.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Springer International Publishing 2022
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9294094/
https://www.ncbi.nlm.nih.gov/pubmed/35347645
http://dx.doi.org/10.1007/s13311-022-01218-7

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