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9. Liver-directed gene therapy: a retroviral vector with a complete LTR and the ApoE enhancer-alpha 1-antitrypsin promoter dramatically increases expression of human alpha 1-antitrypsin in vivo. Okuyama T, Huber RM, Bowling W, Pearline R, Kennedy SC, Flye MW, Ponder KP. Hum Gene Ther; 1996 Mar 20; 7(5):637-45. PubMed ID: 8845389 [Abstract] [Full Text] [Related]
10. Mucopolysaccharidosis I cats mount a cytotoxic T lymphocyte response after neonatal gene therapy that can be blocked with CTLA4-Ig. Ponder KP, Wang B, Wang P, Ma X, Herati R, Wang B, Cullen K, O'Donnell P, Ellinwood NM, Traas A, Primeau TM, Haskins ME. Mol Ther; 2006 Jul 20; 14(1):5-13. PubMed ID: 16698321 [Abstract] [Full Text] [Related]
11. Neonatal gene therapy of MPS I mice by intravenous injection of a lentiviral vector. Kobayashi H, Carbonaro D, Pepper K, Petersen D, Ge S, Jackson H, Shimada H, Moats R, Kohn DB. Mol Ther; 2005 May 20; 11(5):776-89. PubMed ID: 15851016 [Abstract] [Full Text] [Related]
17. Limited transgene immune response and long-term expression of human alpha-L-iduronidase in young adult mice with mucopolysaccharidosis type I by liver-directed gene therapy. Di Domenico C, Di Napoli D, Gonzalez Y Reyero E, Lombardo A, Naldini L, Di Natale P. Hum Gene Ther; 2006 Nov 13; 17(11):1112-21. PubMed ID: 17044753 [Abstract] [Full Text] [Related]