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Long-Term Sex-Biased Correction of Circulating Propionic Acidemia Disease Markers by Adeno-Associated Virus Vectors

Propionic academia (PA) occurs because of mutations in the PCCA or PCCB genes encoding the two subunits of propionyl-CoA carboxylase, a pivotal enzyme in the breakdown of certain amino acids and odd-chain fatty acids. There is no cure for PA, but dietary protein restriction and liver transplantation...

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Detalhes bibliográficos
Publicado no:Hum Gene Ther
Main Authors: Guenzel, Adam J., Collard, Renata, Kraus, Jan P., Matern, Dietrich, Barry, Michael A.
Formato: Artigo
Idioma:Inglês
Publicado em: Mary Ann Liebert, Inc. 2015
Assuntos:
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC4367234/
https://ncbi.nlm.nih.gov/pubmed/25654275
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hum.2014.126
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