AAV9 Gene Therapy Increases Lifespan and Treats Pathological and Behavioral Abnormalities in a Mouse Model of CLN8-Batten Disease

CLN8 disease is a rare form of neuronal ceroid lipofuscinosis caused by biallelic mutations in the CLN8 gene, which encodes a transmembrane endoplasmic reticulum protein involved in trafficking of lysosomal enzymes. CLN8 disease patients present with myoclonus, tonic-clonic seizures, and progressive...

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Detalhes bibliográficos
Publicado no:Mol Ther
Main Authors: Johnson, Tyler B., White, Katherine A., Brudvig, Jon J., Cain, Jacob T., Langin, Logan, Pratt, Melissa A., Booth, Clarissa D., Timm, Derek J., Davis, Samantha S., Meyerink, Brandon, Likhite, Shibi, Meyer, Kathrin, Weimer, Jill M.
Formato: Artigo
Idioma:Inglês
Publicado em: American Society of Gene & Cell Therapy 2021
Assuntos:
Original Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC7791144/
https://ncbi.nlm.nih.gov/pubmed/33010819
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymthe.2020.09.033
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