Slowing Late Infantile Batten Disease by Direct Brain Parenchymal Administration of a rh.10 Adeno-Associated Virus Expressing CLN2

Late infantile Batten disease (CLN2 disease) is a rare, autosomal recessive, neurodegenerative lysosomal storage disease caused by mutations in the CLN2 gene encoding tripeptidyl peptidase 1 (TPP1). We tested intraparenchymal delivery of AAVrh.10hCLN2, a nonhuman serotype rh.10 adeno-associated viru...

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Detalhes bibliográficos
Publicado no:Sci Transl Med
Main Authors: Sondhi, Dolan, Kaminsky, Stephen M., Hackett, Neil R., Pagovich, Odelya E., Rosenberg, Jonathan B., De, Bishnu P., Chen, Alvin, Van de Graaf, Benjamin, Mezey, Jason G., Mammen, Grace W., Mancenido, Denesy, Xu, Fang, Kosofsky, Barry, Yohay, Kaleb, Worgall, Stefan, Kaner, Robert J., Souwedaine, Mark, Greenwald, Bruce M., Kaplitt, Michael, Dyke, Jonathan P., Ballon, Douglas J., Heier, Linda A., Kiss, Szilard, Crystal, Ronald G.
Formato: Artigo
Idioma:Inglês
Publicado em: 2020
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Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC8056991/
https://ncbi.nlm.nih.gov/pubmed/33268510
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1126/scitranslmed.abb5413
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