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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...
Autores principales: | , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Frontiers Media S.A.
2021
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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 |