Comparison of Adeno-Associated Virus Pseudotype 1, 2, and 8 Vectors Administered by Intramuscular Injection in the Treatment of Murine Phenylketonuria
Tóm tắt
Từ khóa
Tài liệu tham khảo
Blau N., 2001, The Metabolic and Molecular Bases of Inherited Disease, 1725
Donlon J., 2008, Hyperphenylalaninemia: Phenylalanine hydroxylase deficiencies. In The Metabolic and Molecular Bases of Inherited Diseases
Harding C.O., 2006, PKU and BH4: Advances in Phenylketonuria and Tetrahydrobiopterin
Laipis P.J., 2004, Mol. Ther., 9, s334
Scavelli, R. (2006). Tetrahydrobiopterin metabolism in the mouse: Feeding studies and generation of a new mutant model. Ph.D. thesis. University of Zürich, Zürich, Switzerland.
Scriver, C.R., and Kaufman, S. (2001). Hyperphenylalaninemia: Phenylalanine hydroxylase deficiency. InThe Metabolic and Molecular Bases of Inherited Disease. C.R. Scriver, A.L. Beaudet, W.S. Sly, D. Valle, and B. Vogelstein, eds. (McGraw-Hill, New York)pp. 1667–1724.
Ye G.J., 2009, Mol. Ther., 17, S317