| 主权项 |
1. A method of expressing a protein product in a subject with a metabolic disorder, the method comprising:
administering an isolated cell to the subject, the isolated cell comprising an exogenous transgene encoding a protein, wherein the exogenous transgene is integrated site-specifically using a zinc finger nuclease into an endogenous safe harbor gene and further wherein the protein is a functional version of a protein that is aberrantly expressed in the metabolic disorder and further wherein the metabolic disorder is selected from the group consisting of methylmalonic acidemia, propionic acidemia, glycogen storage diseases type 1, familial hypercholesterolemia (FH) is a common genetic and metabolic disease, urea cycle disorders, Crigler Najjar Syndrome (CNS), Gilbert syndrome, hepatorenal tyrosinemia, primary hyperoxaluria. transthyretin gene (TTR)-mediated amyloidosis (ATTR), Wilson's disease, phenylketonuria (PKU), and familial lipoprotein lipase deficiency (LPLD). |
