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1. O138 PHASE 1 CLINICAL TRIAL OF LIVER DIRECTED GENE THERAPY WITH rAAV5-PBGD IN ACUTE INTERMITTENT PORPHYRIA: PRELIMINARY SAFETY DATA.

Author

D'Avola, D., López-Franco, E., Harper, P., Fontanellas, A., Grosios, N., Sangro, B., Henrichson, A., Salmon, F., Fuertes, C., Abecia, S., Pañeda, A., Paz, M., Cornet, M.E., Municio, M.D.M., Troconiz, I., Kaeppel, C., Juan, R., von Kalle, C., Schmidt, M., and Petry, H.

Subjects
*CLINICAL trials, *GENE therapy, *PORPHYRIA, *DEAMINASES, *HOMOZYGOSITY, *CHOLESTASIS, *THERAPEUTICS
Published
2014
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