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2. Gene therapy ameliorates cardiovascular disease in dogs with mucopolysaccharidosis VII. Sleeper MM; Fornasari B; Ellinwood NM; Weil MA; Melniczek J; O'Malley TM; Sammarco CD; Xu L; Ponder KP; Haskins ME Circulation; 2004 Aug; 110(7):815-20. PubMed ID: 15289379 [TBL] [Abstract][Full Text] [Related]
3. Expression in blood cells may contribute to biochemical and pathological improvements after neonatal intravenous gene therapy for mucopolysaccharidosis VII in dogs. Wang B; O'Malley TM; Xu L; Vite C; Wang P; O'Donnell PA; Ellinwood NM; Haskins ME; Ponder KP Mol Genet Metab; 2006 Jan; 87(1):8-21. PubMed ID: 16275036 [TBL] [Abstract][Full Text] [Related]
4. The effect of neonatal gene therapy on skeletal manifestations in mucopolysaccharidosis VII dogs after a decade. Xing EM; Knox VW; O'Donnell PA; Sikura T; Liu Y; Wu S; Casal ML; Haskins ME; Ponder KP Mol Genet Metab; 2013 Jun; 109(2):183-93. PubMed ID: 23628461 [TBL] [Abstract][Full Text] [Related]
5. Evaluation of pathological manifestations of disease in mucopolysaccharidosis VII mice after neonatal hepatic gene therapy. Xu L; Mango RL; Sands MS; Haskins ME; Ellinwood NM; Ponder KP Mol Ther; 2002 Dec; 6(6):745-58. PubMed ID: 12498771 [TBL] [Abstract][Full Text] [Related]
6. The effect of Tlr4 and/or C3 deficiency and of neonatal gene therapy on skeletal disease in mucopolysaccharidosis VII mice. Xing EM; Wu S; Ponder KP Mol Genet Metab; 2015 Feb; 114(2):209-16. PubMed ID: 25559179 [TBL] [Abstract][Full Text] [Related]
7. Therapeutic neonatal hepatic gene therapy in mucopolysaccharidosis VII dogs. Ponder KP; Melniczek JR; Xu L; Weil MA; O'Malley TM; O'Donnell PA; Knox VW; Aguirre GD; Mazrier H; Ellinwood NM; Sleeper M; Maguire AM; Volk SW; Mango RL; Zweigle J; Wolfe JH; Haskins ME Proc Natl Acad Sci U S A; 2002 Oct; 99(20):13102-7. PubMed ID: 12232044 [TBL] [Abstract][Full Text] [Related]
8. Transduction of hepatocytes after neonatal delivery of a Moloney murine leukemia virus based retroviral vector results in long-term expression of beta-glucuronidase in mucopolysaccharidosis VII dogs. Xu L; Haskins ME; Melniczek JR; Gao C; Weil MA; O'Malley TM; O'Donnell PA; Mazrier H; Ellinwood NM; Zweigle J; Wolfe JH; Ponder KP Mol Ther; 2002 Feb; 5(2):141-53. PubMed ID: 11829521 [TBL] [Abstract][Full Text] [Related]
9. Effect of neonatal gene therapy on lumbar spine disease in mucopolysaccharidosis VII dogs. Smith LJ; Martin JT; O'Donnell P; Wang P; Elliott DM; Haskins ME; Ponder KP Mol Genet Metab; 2012 Sep; 107(1-2):145-52. PubMed ID: 22510705 [TBL] [Abstract][Full Text] [Related]
10. The effect of neonatal gene therapy with a gamma retroviral vector on cardiac valve disease in mucopolysaccharidosis VII dogs after a decade. Bigg PW; Sleeper MM; O'Donnell PA; Liu Y; Wu S; Casal ML; Haskins ME; Ponder KP Mol Genet Metab; 2013 Nov; 110(3):311-8. PubMed ID: 23860311 [TBL] [Abstract][Full Text] [Related]
11. Neonatal intramuscular injection with recombinant adeno-associated virus results in prolonged beta-glucuronidase expression in situ and correction of liver pathology in mucopolysaccharidosis type VII mice. Daly TM; Okuyama T; Vogler C; Haskins ME; Muzyczka N; Sands MS Hum Gene Ther; 1999 Jan; 10(1):85-94. PubMed ID: 10022533 [TBL] [Abstract][Full Text] [Related]
12. Skeletal response to lentiviral mediated gene therapy in a mouse model of MPS VII. Macsai CE; Derrick-Roberts AL; Ding X; Zarrinkalam KH; McIntyre C; Anderson PH; Anson DS; Byers S Mol Genet Metab; 2012 Jun; 106(2):202-13. PubMed ID: 22525091 [TBL] [Abstract][Full Text] [Related]
13. Murine mucopolysaccharidosis type VII: the impact of therapies on the clinical course and pathology in a murine model of lysosomal storage disease. Vogler C; Sands MS; Galvin N; Levy B; Thorpe C; Barker J; Sly WS J Inherit Metab Dis; 1998 Aug; 21(5):575-86. PubMed ID: 9728337 [TBL] [Abstract][Full Text] [Related]
14. Evaluation of AAV-mediated Gene Therapy for Central Nervous System Disease in Canine Mucopolysaccharidosis VII. Gurda BL; De Guilhem De Lataillade A; Bell P; Zhu Y; Yu H; Wang P; Bagel J; Vite CH; Sikora T; Hinderer C; Calcedo R; Yox AD; Steet RA; Ruane T; O'Donnell P; Gao G; Wilson JM; Casal M; Ponder KP; Haskins ME Mol Ther; 2016 Feb; 24(2):206-216. PubMed ID: 26447927 [TBL] [Abstract][Full Text] [Related]
15. Delivery of a retroviral vector expressing human beta-glucuronidase to the liver and spleen decreases lysosomal storage in mucopolysaccharidosis VII mice. Gao C; Sands MS; Haskins ME; Ponder KP Mol Ther; 2000 Sep; 2(3):233-44. PubMed ID: 10985954 [TBL] [Abstract][Full Text] [Related]
16. Cloning of the canine beta-glucuronidase cDNA, mutation identification in canine MPS VII, and retroviral vector-mediated correction of MPS VII cells. Ray J; Bouvet A; DeSanto C; Fyfe JC; Xu D; Wolfe JH; Aguirre GD; Patterson DF; Haskins ME; Henthorn PS Genomics; 1998 Mar; 48(2):248-53. PubMed ID: 9521879 [TBL] [Abstract][Full Text] [Related]
17. Intracranial injection of recombinant adeno-associated virus improves cognitive function in a murine model of mucopolysaccharidosis type VII. Frisella WA; O'Connor LH; Vogler CA; Roberts M; Walkley S; Levy B; Daly TM; Sands MS Mol Ther; 2001 Mar; 3(3):351-8. PubMed ID: 11273777 [TBL] [Abstract][Full Text] [Related]
18. Radiographic evaluation of bones and joints in mucopolysaccharidosis I and VII dogs after neonatal gene therapy. Herati RS; Knox VW; O'Donnell P; D'Angelo M; Haskins ME; Ponder KP Mol Genet Metab; 2008 Nov; 95(3):142-51. PubMed ID: 18707908 [TBL] [Abstract][Full Text] [Related]
19. Comparison of ventricular and intravenous lentiviral-mediated gene therapy for murine MPS VII. Bielicki J; McIntyre C; Anson DS Mol Genet Metab; 2010 Dec; 101(4):370-82. PubMed ID: 20864369 [TBL] [Abstract][Full Text] [Related]
20. Widespread correction of lysosomal storage following intrahepatic injection of a recombinant adeno-associated virus in the adult MPS VII mouse. Sferra TJ; Backstrom K; Wang C; Rennard R; Miller M; Hu Y Mol Ther; 2004 Sep; 10(3):478-91. PubMed ID: 15336648 [TBL] [Abstract][Full Text] [Related] [Next] [New Search]