Early postnatal administration of an AAV9 gene therapy is safe and efficacious in CLN3 disease

CLN3 disease, caused by biallelic mutations in the CLN3 gene, is a rare pediatric neurodegenerative disease that has no cure or disease modifying treatment. The development of effective treatments has been hindered by a lack of etiological knowledge, but gene replacement has emerged as a promising t...

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Detalles Bibliográficos
Autores principales: Johnson, Tyler B., Brudvig, Jon J., Likhite, Shibi, Pratt, Melissa A., White, Katherine A., Cain, Jacob T., Booth, Clarissa D., Timm, Derek J., Davis, Samantha S., Meyerink, Brandon, Pineda, Ricardo, Dennys-Rivers, Cassandra, Kaspar, Brian K., Meyer, Kathrin, Weimer, Jill M.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Frontiers Media S.A. 2023
Materias:
Genetics
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10080320/
https://www.ncbi.nlm.nih.gov/pubmed/37035740
http://dx.doi.org/10.3389/fgene.2023.1118649

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