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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Pubblicato in:Mol Ther
Autori principali: 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.
Natura: Artigo
Lingua:Inglês
Pubblicazione: American Society of Gene & Cell Therapy 2021
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Original Article
Accesso online: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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