AAV-Mediated GALC Gene Therapy Rescues Alpha-Synucleinopathy in the Spinal Cord of a Leukodystrophic Lysosomal Storage Disease Mouse Model

Krabbe's disease (KD) is primarily a demyelinating disorder, but recent studies have identified the presence of neuronal protein aggregates in the brain, at least partially composed by alpha-synuclein (α-syn). The role of this protein aggregation in the pathogenesis of KD is largely unknown, bu...

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Detalles Bibliográficos
Autores principales: Marshall, Michael S., Issa, Yazan, Heller, Gregory, Nguyen, Duc, Bongarzone, Ernesto R.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Frontiers Media S.A. 2020
Materias:
Cellular Neuroscience
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7785790/
https://www.ncbi.nlm.nih.gov/pubmed/33424556
http://dx.doi.org/10.3389/fncel.2020.619712

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7785790/
https://www.ncbi.nlm.nih.gov/pubmed/33424556
http://dx.doi.org/10.3389/fncel.2020.619712

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