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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Wedi'i Gadw mewn:
Manylion Llyfryddiaeth
Cyhoeddwyd yn:Sci Transl Med
Prif Awduron: 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.
Fformat: Artigo
Iaith:Inglês
Cyhoeddwyd: 2020
Pynciau:
Article
Mynediad Ar-lein: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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