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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.

Mol Ther. 2012 May;20(5):898-907. doi: 10.1038/mt.2012.9. Epub 2012 Mar 6.


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.

Mol Ther. 2011 Mar;19(3):461-9. doi: 10.1038/mt.2010.257. Epub 2010 Nov 30.


Evaluation of fibroblast-mediated gene therapy in a feline model of mucopolysaccharidosis type VI.

Yogalingam G, Crawley A, Hopwood JJ, Anson DS.

Biochim Biophys Acta. 1999 Feb 24;1453(2):284-96.


Feline mucopolysaccharidosis type VI. Characterization of recombinant N-acetylgalactosamine 4-sulfatase and identification of a mutation causing the disease.

Yogalingam G, Litjens T, Bielicki J, Crawley AC, Muller V, Anson DS, Hopwood JJ.

J Biol Chem. 1996 Nov 1;271(44):27259-65.


Feline mucopolysaccharidosis VI: purification and characterization of the resident arylsulfatase B activity.

Vine DT, McGovern MM, Haskins ME, Desnick RJ.

Am J Hum Genet. 1981 Nov;33(6):916-27.


Biochemical, pathological, and skeletal improvement of mucopolysaccharidosis VI after gene transfer to liver but not to muscle.

Tessitore A, Faella A, O'Malley T, Cotugno G, Doria M, Kunieda T, Matarese G, Haskins M, Auricchio A.

Mol Ther. 2008 Jan;16(1):30-7. Epub 2007 Oct 23.


Neonatal retroviral vector-mediated hepatic gene therapy reduces bone, joint, and cartilage disease in mucopolysaccharidosis VII mice and dogs.

Mango RL, Xu L, Sands MS, Vogler C, Seiler G, Schwarz T, Haskins ME, Ponder KP.

Mol Genet Metab. 2004 May;82(1):4-19.


Pharmacodynamics, pharmacokinetics and biodistribution of recombinant human N-acetylgalactosamine 4-sulfatase after 6months of therapy in cats using different IV infusion durations.

Ruane T, Haskins M, Cheng A, Wang P, Aguirre G, Knox VW 4th, Qi Y, Tompkins T, O'Neill CA.

Mol Genet Metab. 2016 Feb;117(2):157-63. doi: 10.1016/j.ymgme.2015.10.006. Epub 2015 Oct 21.


Intrathecal recombinant human 4-sulfatase reduces accumulation of glycosaminoglycans in dura of mucopolysaccharidosis VI cats.

Auclair D, Finnie J, Walkley SU, White J, Nielsen T, Fuller M, Cheng A, O'Neill CA, Hopwood JJ.

Pediatr Res. 2012 Jan;71(1):39-45. doi: 10.1038/pr.2011.13.


Effect of enzyme replacement therapy on bone formation in a feline model of mucopolysaccharidosis type VI.

Byers S, Nuttall JD, Crawley AC, Hopwood JJ, Smith K, Fazzalari NL.

Bone. 1997 Nov;21(5):425-31.


Lentiviral-mediated correction of MPS VI cells and gene transfer to joint tissues.

Byers S, Rothe M, Lalic J, Koldej R, Anson DS.

Mol Genet Metab. 2009 Jun;97(2):102-8. doi: 10.1016/j.ymgme.2009.02.008. Epub 2009 Feb 27.


Long-term intra-articular administration of recombinant human N-acetylgalactosamine-4-sulfatase in feline mucopolysaccharidosis VI.

Auclair D, Hopwood JJ, Lemontt JF, Chen L, Byers S.

Mol Genet Metab. 2007 Aug;91(4):352-61. Epub 2007 Jun 1.


[Analysis of clinical features and arylsulfatase B gene mutation in thirteen Chinese children with mucopolysaccharidosis type VI].

Zheng J, Huang Y, Zhao X, Sheng H, Cheng J, Zhou Z, Li X, Mao X, Liu L.

Zhonghua Er Ke Za Zhi. 2014 Jun;52(6):403-8. Chinese.


Gene therapy for mucopolysaccharidosis type VI is effective in cats without pre-existing immunity to AAV8.

Ferla R, O'Malley T, Calcedo R, O'Donnell P, Wang P, Cotugno G, Claudiani P, Wilson JM, Haskins M, Auricchio A.

Hum Gene Ther. 2013 Feb;24(2):163-9. doi: 10.1089/hum.2012.179. Epub 2013 Jan 30.


Autologous transplantation of retrovirally transduced bone marrow or neonatal blood cells into cats can lead to long-term engraftment in the absence of myeloablation.

Simonaro CM, Haskins ME, Abkowitz JL, Brooks DA, Hopwood JJ, Zhang J, Schuchman EH.

Gene Ther. 1999 Jan;6(1):107-13.

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