Sustained Enzymatic Correction by rAAV-Mediated Liver Gene Therapy Protects Against Induced Motor Neuropathy in Acute Porphyria Mice

Acute intermittent porphyria (AIP) is characterized by a hereditary deficiency of hepatic porphobilinogen deaminase (PBGD) activity. Clinical features are acute neurovisceral attacks accompanied by overproduction of porphyrin precursors in the liver. Recurrent life-threatening attacks can be cured o...

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Главные авторы: Unzu, Carmen, Sampedro, Ana, Mauleón, Itsaso, Alegre, Manuel, Beattie, Stuart G, de Salamanca, Rafael Enríquez, Snapper, Jolanda, Twisk, Jaap, Petry, Harald, González-Aseguinolaza, Gloria, Artieda, Julio, Rodríguez-Pena, María Sol, Prieto, Jesús, Fontanellas, Antonio
Формат: Artigo
Язык:Inglês
Опубликовано: Nature Publishing Group 2011
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Original Article
Online-ссылка:https://ncbi.nlm.nih.gov/pmc/articles/PMC3034840/
https://ncbi.nlm.nih.gov/pubmed/20877347
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2010.210
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