Porphobilinogen deaminase over-expression in hepatocytes, but not in erythrocytes, prevents accumulation of toxic porphyrin precursors in a mouse model of acute intermittent porphyria
Keywords:
Acute intermittent porphyria
Acute attack
Porphyrin precursor excretion
Motor coordination
Bone marrow transplant
Liver gene transfer
Citation:
Unzu C, Sampedro A, Mauleon I, Vanrell L, Dubrot J, de Salamanca RE, et al. Porphobilinogen deaminase over-expression in hepatocytes, but not in erythrocytes, prevents accumulation of toxic porphyrin precursors in a mouse model of acute intermittent porphyria. J Hepatol 2010 Mar;52(3):417-424.
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