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White Matter Pathology as a Barrier to Gangliosidosis Gene Therapy

The gangliosidoses are a family of neurodegenerative lysosomal storage diseases that have recently seen promising advances in gene therapy. White matter deficits are well established components of gangliosidosis pathology that are now receiving more attention because they are partially refractory to...

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Detalles Bibliográficos
Autores principales: Maguire, Anne S., Martin, Douglas R.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Frontiers Media S.A. 2021
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8397537/
https://www.ncbi.nlm.nih.gov/pubmed/34456684
http://dx.doi.org/10.3389/fncel.2021.682106