Long-term correction of hyperphenylalaninemia by AAV-mediated gene transfer leads to behavioral recovery in phenylketonuria mice

Author:

Mochizuki S,Mizukami H,Ogura T,Kure S,Ichinohe A,Kojima K,Matsubara Y,Kobayahi E,Okada T,Hoshika A,Ozawa K,Kume A

Publisher

Springer Science and Business Media LLC

Subject

Genetics,Molecular Biology,Molecular Medicine

Reference29 articles.

1. Scriver CR, Kaufman S . Hyperphenylalaninemia: phenylalanine hydroxylase deficiency. In: Scriver CR, Beaudet AL, Sly WS, Valle D (eds) The Metabolic and Molecular Basis of Inherited Diseases. McGraw-Hill: New York, 2001, pp 1667–1724.

2. Liu T-J, Kay MA, Darlington GJ, Woo SLC . Reconstitution of enzymatic activity in hepatocytes of phenylalanine hydroxylase-deficient mice. Somat Cell Mol Genet 1992; 18: 89–96.

3. Eisensmith RC, Woo SLC . Gene therapy for phenylketonuria. Eur J Pediatr 1996; 155 (Suppl 1): S16–S19.

4. Fang B et al. Gene therapy for phenylketonuria: phenotypic correction in a genetically deficient mouse model by adenovirus-mediated hepatic gene transfer. Gene Therapy 1994; 1: 247–254.

5. Nagasaki Y et al. Reversal of hypopigmentation in phenylketonuria mice by adenovirus-mediated gene transfer. Pediatr Res 1999; 45: 465–473.

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