Adeno-associated Virus-mediated Rescue of Neonatal Lethality in Argininosuccinate Synthetase-deficient Mice

Viral vectors based on adeno-associated virus (AAV) are showing exciting promise in gene therapy trials targeting the adult liver. A major challenge in extending this promise to the pediatric liver is the loss of episomal vector genomes that accompanies hepatocellular proliferation during liver grow...

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Dettagli Bibliografici
Autori principali: Kok, Cindy Y, Cunningham, Sharon C, Carpenter, Kevin H, Dane, Allison P, Siew, Susan M, Logan, Grant J, Kuchel, Philip W, Alexander, Ian E
Natura: Artigo
Lingua:Inglês
Pubblicazione: Nature Publishing Group 2013
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Original Article
Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC3808136/
https://ncbi.nlm.nih.gov/pubmed/23817206
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2013.139
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