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Prednisolone reduces the interferon response to AAV in cynomolgus macaques and may increase liver gene expression
Ornithine transcarbamylase deficiency is a rare X-linked genetic urea cycle disorder leading to episodes of acute hyperammonemia, adverse cognitive and neurological effects, hospitalizations, and in some cases death. DTX301, a non-replicating, recombinant self-complimentary adeno-associated virus ve...
Autores principales: | Wang, Lili, Warzecha, Claude C., Kistner, Alexander, Chichester, Jessica A., Bell, Peter, Buza, Elizabeth L., He, Zhenning, Pampena, M. Betina, Couthouis, Julien, Sethi, Sunjay, McKeever, Kathleen, Betts, Michael R., Kakkis, Emil, Wilson, James M., Wadsworth, Samuel, Sullivan, Barbara A. |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
American Society of Gene & Cell Therapy
2022
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8841522/ https://www.ncbi.nlm.nih.gov/pubmed/35211641 http://dx.doi.org/10.1016/j.omtm.2022.01.007 |
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