Generation of a Hypomorphic Model of Propionic Acidemia Amenable to Gene Therapy Testing

Propionic acidemia (PA) is a recessive genetic disease that results in an inability to metabolize certain amino acids and odd-chain fatty acids. Current treatment involves restricting consumption of these substrates or liver transplantation. Deletion of the Pcca gene in mice mimics the most severe f...

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Detalhes bibliográficos
Main Authors: Guenzel, Adam J, Hofherr, Sean E, Hillestad, Matthew, Barry, Mary, Weaver, Eric, Venezia, Sarah, Kraus, Jan P, Matern, Dietrich, Barry, Michael A
Formato: Artigo
Idioma:Inglês
Publicado em: Nature Publishing Group 2013
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Original Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC3708067/
https://ncbi.nlm.nih.gov/pubmed/23648696
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2013.68
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