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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Detaylı Bibliyografya
Yayımlandı:Sci Transl Med
Asıl Yazarlar: 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.
Materyal Türü: Artigo
Dil:Inglês
Baskı/Yayın Bilgisi: 2020
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Article
Online Erişim: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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