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6. In vivo assessment of mutations in OTC for dominant-negative effects following rAAV2/8-mediated gene delivery to the mouse liver. Ginn SL; Cunningham SC; Zheng M; Spinoulas A; Carpenter KH; Alexander IE Gene Ther; 2009 Jun; 16(6):820-3. PubMed ID: 19357713 [TBL] [Abstract][Full Text] [Related]
7. Correction of ornithine transcarbamylase deficiency in adult spf(ash) mice and in OTC-deficient human hepatocytes with recombinant adenoviruses bearing the CAG promoter. Kiwaki K; Kanegae Y; Saito I; Komaki S; Nakamura K; Miyazaki JI; Endo F; Matsuda I Hum Gene Ther; 1996 May; 7(7):821-30. PubMed ID: 8860834 [TBL] [Abstract][Full Text] [Related]
8. Patient selection may affect gene therapy success. Dominant negative effects observed for ornithine transcarbamylase in mouse and human hepatocytes. Morsy MA; Zhao JZ; Ngo TT; Warman AW; O'Brien WE; Graham FL; Caskey CT J Clin Invest; 1996 Feb; 97(3):826-32. PubMed ID: 8609240 [TBL] [Abstract][Full Text] [Related]
9. Retroviral-mediated gene transfer of human ornithine transcarbamylase into primary hepatocytes of spf and spf-ash mice. Grompe M; Jones SN; Loulseged H; Caskey CT Hum Gene Ther; 1992 Feb; 3(1):35-44. PubMed ID: 1562638 [TBL] [Abstract][Full Text] [Related]
10. Analysis of tumors arising in male B6C3F1 mice with and without AAV vector delivery to liver. Bell P; Moscioni AD; McCarter RJ; Wu D; Gao G; Hoang A; Sanmiguel JC; Sun X; Wivel NA; Raper SE; Furth EE; Batshaw ML; Wilson JM Mol Ther; 2006 Jul; 14(1):34-44. PubMed ID: 16682254 [TBL] [Abstract][Full Text] [Related]
11. Efficient adenoviral-mediated ornithine transcarbamylase expression in deficient mouse and human hepatocytes. Morsy MA; Alford EL; Bett A; Graham FL; Caskey CT J Clin Invest; 1993 Sep; 92(3):1580-6. PubMed ID: 8376608 [TBL] [Abstract][Full Text] [Related]
12. Correction of ureagenesis after gene transfer in an animal model and after liver transplantation in humans with ornithine transcarbamylase deficiency. Batshaw ML; Robinson MB; Ye X; Pabin C; Daikhin Y; Burton BK; Wilson JM; Yudkoff M Pediatr Res; 1999 Nov; 46(5):588-93. PubMed ID: 10541323 [TBL] [Abstract][Full Text] [Related]
13. The sparse fur mouse as a model for gene therapy in ornithine carbamoyltransferase deficiency. Batshaw ML; Yudkoff M; McLaughlin BA; Gorry E; Anegawa NJ; Smith IA; Hyman SL; Robinson MB Gene Ther; 1995 Dec; 2(10):743-9. PubMed ID: 8750014 [TBL] [Abstract][Full Text] [Related]
14. AAV-encoded OTC activity persisting to adulthood following delivery to newborn spf(ash) mice is insufficient to prevent shRNA-induced hyperammonaemia. Cunningham SC; Kok CY; Spinoulas A; Carpenter KH; Alexander IE Gene Ther; 2013 Dec; 20(12):1184-7. PubMed ID: 24108150 [TBL] [Abstract][Full Text] [Related]
15. A dual AAV system enables the Cas9-mediated correction of a metabolic liver disease in newborn mice. Yang Y; Wang L; Bell P; McMenamin D; He Z; White J; Yu H; Xu C; Morizono H; Musunuru K; Batshaw ML; Wilson JM Nat Biotechnol; 2016 Mar; 34(3):334-8. PubMed ID: 26829317 [TBL] [Abstract][Full Text] [Related]
16. Developing adenoviral-mediated in vivo gene therapy for ornithine transcarbamylase deficiency. Raper SE; Wilson JM; Yudkoff M; Robinson MB; Ye X; Batshaw ML J Inherit Metab Dis; 1998; 21 Suppl 1():119-37. PubMed ID: 9686350 [TBL] [Abstract][Full Text] [Related]
17. Gene therapy on trial. Marshall E Science; 2000 May; 288(5468):951-7. PubMed ID: 10841710 [No Abstract] [Full Text] [Related]
18. Transient depletion of CD4 lymphocyte improves efficacy of repeated administration of recombinant adenovirus in the ornithine transcarbamylase deficient sparse fur mouse. Ye X; Robinson MB; Pabin C; Batshaw ML; Wilson JM Gene Ther; 2000 Oct; 7(20):1761-7. PubMed ID: 11083498 [TBL] [Abstract][Full Text] [Related]
19. Cultured fibroblasts as a tool for improvement of molecular analysis in ornithine transcarbamylase (OTC) deficiency. Häberle J; Koch HG Hum Mutat; 2003 Jun; 21(6):649. PubMed ID: 12754713 [No Abstract] [Full Text] [Related]