Preclinical Dose-Finding Study With a Liver-Tropic, Recombinant AAV-2/8 Vector in the Mouse Model of Galactosialidosis

Galactosialidosis (GS) is a lysosomal storage disease linked to deficiency of the protective protein/cathepsin A (PPCA). Similarly to GS patients, Ppca-null mice develop a systemic disease of the reticuloendothelial system, affecting most visceral organs and the nervous system. Symptoms include seve...

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Autori principali: Hu, Huimin, Gomero, Elida, Bonten, Erik, Gray, John T, Allay, Jim, Wu, Yanan, Wu, Jianrong, Calabrese, Christopher, Nienhuis, Arthur, d'Azzo, Alessandra
Natura: Artigo
Lingua:Inglês
Pubblicazione: Nature Publishing Group 2012
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Original Article
Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC3277225/
https://ncbi.nlm.nih.gov/pubmed/22008912
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2011.227
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