Complete correction of hyperphenylalaninemia following liver-directed, recombinant AAV2/8 vector-mediated gene therapy in murine phenylketonuria

Author:

Harding C O,Gillingham M B,Hamman K,Clark H,Goebel-Daghighi E,Bird A,Koeberl D D

Publisher

Springer Science and Business Media LLC

Subject

Genetics,Molecular Biology,Molecular Medicine

Reference33 articles.

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

2. Udenfriend S, Cooper JR . The enzymatic conversion of phenylalanine to tyrosine. J Biol Chem 1952; 194: 503–511.

3. Azen CG, Koch R, Friedman EG, Berlow S, Coldwell J, Krause W et al. Intellectual development in 12-year-old children treated for phenylketonuria. Am J Dis Child 1991; 145: 35–39.

4. Koch R, Hanley W, Levy H, Matalon K, Matalon R, Rouse B et al. The Maternal Phenylketonuria International Study: 1984–2002. Pediatrics 2003; 112: 1523–1529.

5. National Institutes of Health Consensus Development Conference Statement. Phenylketonuria: screening and management, October 16–18, 2000. Pediatrics 2001; 108: 972–982.

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