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Neonatal gene therapy with a gamma retroviral vector in mucopolysaccharidosis VI cats.
Ponder KP, O'Malley TM, Wang P, O'Donnell PA, Traas AM, Knox VW, Aguirre GA, Ellinwood NM, Metcalf JA, Wang B, Parkinson-Lawrence EJ, Sleeper MM, Brooks DA, Hopwood JJ, Haskins ME. Ponder KP, et al. Among authors: hopwood jj. Mol Ther. 2012 May;20(5):898-907. doi: 10.1038/mt.2012.9. Epub 2012 Mar 6. Mol Ther. 2012. PMID: 22395531 Free PMC article.
Long-term amelioration of feline Mucopolysaccharidosis VI after AAV-mediated liver gene transfer.
Cotugno G, Annunziata P, Tessitore A, O'Malley T, Capalbo A, Faella A, Bartolomeo R, O'Donnell P, Wang P, Russo F, Sleeper MM, Knox VW, Fernandez S, Levanduski L, Hopwood J, De Leonibus E, Haskins M, Auricchio A. Cotugno G, et al. Mol Ther. 2011 Mar;19(3):461-9. doi: 10.1038/mt.2010.257. Epub 2010 Nov 30. Mol Ther. 2011. PMID: 21119624 Free PMC article.
Laronidase treatment of mucopolysaccharidosis I.
Wraith EJ, Hopwood JJ, Fuller M, Meikle PJ, Brooks DA. Wraith EJ, et al. Among authors: hopwood jj. BioDrugs. 2005;19(1):1-7. doi: 10.2165/00063030-200519010-00001. BioDrugs. 2005. PMID: 15691212 Review.
408 results