Gene Therapy Corrects Brain and Behavioral Pathologies in CLN6-Batten Disease

CLN6-Batten disease, a form of neuronal ceroid lipofuscinosis is a rare lysosomal storage disorder presenting with gradual declines in motor, visual, and cognitive abilities and early death by 12–15 years of age. We developed a self-complementary adeno-associated virus serotype 9 (scAAV9) vector exp...

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Publicado no:Mol Ther
Main Authors: Cain, Jacob T., Likhite, Shibi, White, Katherine A., Timm, Derek J., Davis, Samantha S., Johnson, Tyler B., Dennys-Rivers, Cassandra N., Rinaldi, Federica, Motti, Dario, Corcoran, Sarah, Morales, Pablo, Pierson, Christopher, Hughes, Stephanie M., Lee, Stella Y., Kaspar, Brian K., Meyer, Kathrin, Weimer, Jill M.
Formato: Artigo
Idioma:Inglês
Publicado em: American Society of Gene & Cell Therapy 2019
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Original Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC6822284/
https://ncbi.nlm.nih.gov/pubmed/31331814
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymthe.2019.06.015
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