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...

Descripción completa

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

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8884910/
https://www.ncbi.nlm.nih.gov/pubmed/35025759
http://dx.doi.org/10.1172/JCI146286

Ejemplares similares