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...

Täydet tiedot

Tallennettuna:
Bibliografiset tiedot
Päätekijät: Morsy, M A, Zhao, J Z, Ngo, T T, Warman, A W, O'Brien, W E, Graham, F L, Caskey, C T
Aineistotyyppi: Artigo
Kieli:Inglês
Julkaistu: 1996
Aiheet:
Research Article
Linkit:https://ncbi.nlm.nih.gov/pmc/articles/PMC507121/
https://ncbi.nlm.nih.gov/pubmed/8609240
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