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Adeno‐associated virus serotype 1‐based gene therapy for FTD caused by GRN mutations

OBJECTIVE: Dominant loss‐of‐function mutations in the gene encoding the lysosomal protein, progranulin, cause 5‐10% of frontotemporal dementia cases. As progranulin undergoes secretion and endocytosis, a small number of progranulin‐expressing cells can potentially supply the protein to the entire ce...

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Detalles Bibliográficos
Autores principales:	Hinderer, Christian, Miller, Rod, Dyer, Cecilia, Johansson, Julia, Bell, Peter, Buza, Elizabeth, Wilson, James M.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	John Wiley and Sons Inc. 2020
Materias:	Research Articles
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7545603/ https://www.ncbi.nlm.nih.gov/pubmed/32937039 http://dx.doi.org/10.1002/acn3.51165

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