Adeno-Associated Virus Serotype 8 Gene Transfer Rescues a Neonatal Lethal Murine Model of Propionic Acidemia

Propionic acidemia (PA) is an autosomal recessive disorder of metabolism caused by a deficiency of propionyl-coenzyme A carboxylase (PCC). Despite optimal dietary and cofactor therapy, PA patients still suffer from lethal metabolic instability and experience multisystemic complications. A murine mod...

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Bibliographische Detailangaben
Hauptverfasser: Chandler, Randy J., Chandrasekaran, Suma, Carrillo-Carrasco, Nuria, Senac, Julien S., Hofherr, Sean E., Barry, Michael A., Venditti, Charles P.
Format: Artigo
Sprache:Inglês
Veröffentlicht: Mary Ann Liebert, Inc. 2011
Schlagworte:
Brief Reports
Online Zugang:https://ncbi.nlm.nih.gov/pmc/articles/PMC3073074/
https://ncbi.nlm.nih.gov/pubmed/20950151
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hum.2010.164
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