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...

Descrizione completa

Salvato in:
Dettagli Bibliografici
Pubblicato in:Sci Transl Med
Autori principali: 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.
Natura: Artigo
Lingua:Inglês
Pubblicazione: 2020
Soggetti:
Article
Accesso online: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
Tags: Aggiungi Tag
Nessun Tag, puoi essere il primo ad aggiungerne! !

Documenti analoghi