Patient selection may affect gene therapy success. Dominant negative effects observed for ornithine transcarbamylase in mouse and human hepatocytes.

We have achieved significant improvement of ornithine transcarbamylase deficiency (OTCD) in a mouse model through adenoviral-mediated gene transfer of the human ornithine transcarbamylase cDNA. Substantial reduction in orotic aciduria was observed within 24 h of treatment. Metabolic correction was l...

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Detalhes bibliográficos

Publicado no:	J Clin Invest
Principais autores:	Morsy, M A, Zhao, J Z, Ngo, T T, Warman, A W, O'Brien, W E, Graham, F L, Caskey, C T
Formato:	Artigo
Idioma:	Inglês
Publicado em:	American Society for Clinical Investigation 1996
Assuntos:	Research Article
Acesso em linha:	https://ncbi.nlm.nih.govhttps://pmc.ncbi.nlm.nih.gov/articles/PMC507121/ https://ncbi.nlm.nih.govhttps://pubmed.ncbi.nlm.nih.gov/8609240/ https://ncbi.nlm.nih.govhttps://doi.org/10.1172/JCI118482
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