Pahenu1 is a mouse model for tetrahydrobiopterin-responsive phenylalanine hydroxylase deficiency and promotes analysis of the pharmacological chaperone mechanism in vivo.
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S. Gersting | R. Fingerhut | A. Roscher | K. Kemter | M. Staudigl | Dunja D Messing | M. Danecka | F. Lagler | A. Muntau | A. Eichinger | Katharina A Domdey | C. Zsifkovits | H. Glossmann | Dunja D. Messing