Liver-Directed Adeno-Associated Virus Serotype 8 Gene Transfer Rescues a Lethal Murine Model of Citrullinemia Type 1

Citrullinemia type 1 (CTLN1) is an autosomal recessive disorder of metabolism caused by a deficiency of argininosuccinate synthetase. Despite optimal management, CTLN1 patients still suffer from lethal metabolic instability and experience life threatening episodes of acute hyperammonemia. A murine m...

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Autori principali: Chandler, Randy J., Tarasenko, Tatiana N., Cusmano-Ozog, Kristina, Sun, Qin, Sutton, V. Reid, Venditti, Charles P., McGuire, Peter J.
Natura: Artigo
Lingua:Inglês
Pubblicazione: 2013
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Article
Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC3855546/
https://ncbi.nlm.nih.gov/pubmed/24131980
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/gt.2013.53
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