טוען...

Hepatocytes from wild-type or heterozygous donors are equally effective in achieving successful therapeutic liver repopulation in murine phenylketonuria (PKU)

Successful restoration of phenylalanine (Phe) clearance following liver-directed gene therapy in murine phenylketonuria (PKU) is likely dependent upon both the number of cells successfully transduced and the amount of phenylalanine hydroxylase (PAH) activity expressed per cell. At low levels of tran...

תיאור מלא

שמור ב:
מידע ביבליוגרפי
Main Authors: Hamman, Kelly J., Winn, Shelley, Harding, Cary O.
פורמט: Artigo
שפה:Inglês
יצא לאור: 2011
נושאים:
גישה מקוונת:https://ncbi.nlm.nih.gov/pmc/articles/PMC3219060/
https://ncbi.nlm.nih.gov/pubmed/21917493
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymgme.2011.07.027
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