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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. Xu L, et al. Among authors: weil ma. Mol Ther. 2002 Feb;5(2):141-53. doi: 10.1006/mthe.2002.0527. Mol Ther. 2002. PMID: 11829521 Free article.
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. Ponder KP, et al. Among authors: weil ma. Proc Natl Acad Sci U S A. 2002 Oct 1;99(20):13102-7. doi: 10.1073/pnas.192353499. Epub 2002 Sep 13. Proc Natl Acad Sci U S A. 2002. PMID: 12232044 Free PMC article.
Bone marrow transplantation for feline mucopolysaccharidosis I.
Ellinwood NM, Colle MA, Weil MA, Casal ML, Vite CH, Wiemelt S, Hasson CW, O'Malley TM, He X, Prociuk U, Verot L, Melniczek JR, Lannon A, Aguirre GD, Knox VW, Evans SM, Vanier MT, Schuchman EH, Walkley SU, Haskins ME. Ellinwood NM, et al. Among authors: weil ma. Mol Genet Metab. 2007 Jul;91(3):239-50. doi: 10.1016/j.ymgme.2007.03.001. Epub 2007 May 7. Mol Genet Metab. 2007. PMID: 17482862 Free PMC article.
Enzyme replacement therapy in feline mucopolysaccharidosis I.
Kakkis ED, Schuchman E, He X, Wan Q, Kania S, Wiemelt S, Hasson CW, O'Malley T, Weil MA, Aguirre GA, Brown DE, Haskins ME. Kakkis ED, et al. Among authors: weil ma. Mol Genet Metab. 2001 Mar;72(3):199-208. doi: 10.1006/mgme.2000.3140. Mol Genet Metab. 2001. PMID: 11243725