AAV8-mediated Gene Therapy Prevents Induced Biochemical Attacks of Acute Intermittent Porphyria and Improves Neuromotor Function

Acute intermittent porphyria (AIP), an autosomal dominant hepatic porphyria due to half-normal hydroxymethylbilane synthase (HMB-synthase) activity, is manifested by life-threatening acute neurological attacks that are precipitated by factors that induce heme biosynthesis. The acute attacks are curr...

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Autori principali: Yasuda, Makiko, Bishop, David F, Fowkes, Mary, Cheng, Seng H, Gan, Lin, Desnick, Robert J
Natura: Artigo
Lingua:Inglês
Pubblicazione: Nature Publishing Group 2009
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Original Articles
Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC2839207/
https://ncbi.nlm.nih.gov/pubmed/19861948
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2009.250
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