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AAV9/MFSD8 gene therapy is effective in preclinical models of neuronal ceroid lipofuscinosis type 7 disease

Neuronal ceroid lipofuscinosis type 7 (CLN7) disease is a lysosomal storage disease caused by mutations in the facilitator superfamily domain containing 8 (MFSD8) gene, which encodes a membrane-bound lysosomal protein, MFSD8. To test the effectiveness and safety of adeno-associated viral (AAV) gene...

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Detalles Bibliográficos
Autores principales:	Chen, Xin, Dong, Thomas, Hu, Yuhui, Shaffo, Frances C., Belur, Nandkishore R., Mazzulli, Joseph R., Gray, Steven J.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	American Society for Clinical Investigation 2022
Materias:	Research Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8884910/ https://www.ncbi.nlm.nih.gov/pubmed/35025759 http://dx.doi.org/10.1172/JCI146286

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