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274 related items for PubMed ID: 16319949
1. Complete correction of hyperphenylalaninemia following liver-directed, recombinant AAV2/8 vector-mediated gene therapy in murine phenylketonuria. Harding CO, Gillingham MB, Hamman K, Clark H, Goebel-Daghighi E, Bird A, Koeberl DD. Gene Ther; 2006 Mar; 13(5):457-62. PubMed ID: 16319949 [Abstract] [Full Text] [Related]
2. Administration-route and gender-independent long-term therapeutic correction of phenylketonuria (PKU) in a mouse model by recombinant adeno-associated virus 8 pseudotyped vector-mediated gene transfer. Ding Z, Georgiev P, Thöny B. Gene Ther; 2006 Apr; 13(7):587-93. PubMed ID: 16319947 [Abstract] [Full Text] [Related]
3. Long-term correction of hyperphenylalaninemia by AAV-mediated gene transfer leads to behavioral recovery in phenylketonuria mice. Mochizuki S, Mizukami H, Ogura T, Kure S, Ichinohe A, Kojima K, Matsubara Y, Kobayahi E, Okada T, Hoshika A, Ozawa K, Kume A. Gene Ther; 2004 Jul; 11(13):1081-6. PubMed ID: 15057263 [Abstract] [Full Text] [Related]
4. Long-term enzymatic and phenotypic correction in the phenylketonuria mouse model by adeno-associated virus vector-mediated gene transfer. Oh HJ, Park ES, Kang S, Jo I, Jung SC. Pediatr Res; 2004 Aug; 56(2):278-84. PubMed ID: 15181195 [Abstract] [Full Text] [Related]
13. Expression of phenylalanine hydroxylase (PAH) in erythrogenic bone marrow does not correct hyperphenylalaninemia in Pah(enu2) mice. Harding CO, Neff M, Jones K, Wild K, Wolff JA. J Gene Med; 2003 Nov; 5(11):984-93. PubMed ID: 14601136 [Abstract] [Full Text] [Related]
14. Treatment of a metabolic liver disease by in vivo genome base editing in adult mice. Villiger L, Grisch-Chan HM, Lindsay H, Ringnalda F, Pogliano CB, Allegri G, Fingerhut R, Häberle J, Matos J, Robinson MD, Thöny B, Schwank G. Nat Med; 2018 Oct; 24(10):1519-1525. PubMed ID: 30297904 [Abstract] [Full Text] [Related]
15. Reversal of hypopigmentation in phenylketonuria mice by adenovirus-mediated gene transfer. Nagasaki Y, Matsubara Y, Takano H, Fujii K, Senoo M, Akanuma J, Takahashi K, Kure S, Hara M, Kanegae Y, Saito I, Narisawa K. Pediatr Res; 1999 Apr; 45(4 Pt 1):465-73. PubMed ID: 10203136 [Abstract] [Full Text] [Related]
16. Reversal of metabolic and neurological symptoms of phenylketonuric mice treated with a PAH containing helper-dependent adenoviral vector. Cerreto M, Mehdawy B, Ombrone D, Nisticò R, Ruoppolo M, Usiello A, Daniele A, Pastore L, Salvatore F. Curr Gene Ther; 2012 Feb 01; 12(1):48-56. PubMed ID: 22348550 [Abstract] [Full Text] [Related]