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Items: 1 to 20 of 126


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.


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.


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.


[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.


Pharmacological read-through of nonsense ARSB mutations as a potential therapeutic approach for mucopolysaccharidosis VI.

Bartolomeo R, Polishchuk EV, Volpi N, Polishchuk RS, Auricchio A.

J Inherit Metab Dis. 2013 Mar;36(2):363-71. doi: 10.1007/s10545-012-9521-y. Epub 2012 Sep 13.


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.


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.


Prevalence of anti-adeno-associated virus serotype 8 neutralizing antibodies and arylsulfatase B cross-reactive immunologic material in mucopolysaccharidosis VI patient candidates for a gene therapy trial.

Ferla R, Claudiani P, Savarese M, Kozarsky K, Parini R, Scarpa M, Donati MA, Sorge G, Hopwood JJ, Parenti G, Fecarotta S, Nigro V, Sivri HS, Van Der Ploeg A, Andria G, Brunetti-Pierri N, Auricchio A.

Hum Gene Ther. 2015 Mar;26(3):145-52. doi: 10.1089/hum.2014.109.


Cardiac and ocular pathologies in a mouse model of mucopolysaccharidosis type VI.

Strauch OF, Stypmann J, Reinheckel T, Martinez E, Haverkamp W, Peters C.

Pediatr Res. 2003 Nov;54(5):701-8. Epub 2003 Aug 6.


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.


Mucopolysaccharidosis VI.

Valayannopoulos V, Nicely H, Harmatz P, Turbeville S.

Orphanet J Rare Dis. 2010 Apr 12;5:5. doi: 10.1186/1750-1172-5-5. Review.


Haploidentical stem cell transplantation in two children with mucopolysaccharidosis VI: clinical and biochemical outcome.

Jester S, Larsson J, Eklund EA, Papadopoulou D, Månsson JE, Békássy AN, Turkiewicz D, Toporski J, Øra I.

Orphanet J Rare Dis. 2013 Sep 5;8:134. doi: 10.1186/1750-1172-8-134.


Phenotypic rescue after adeno-associated virus-mediated delivery of 4-sulfatase to the retinal pigment epithelium of feline mucopolysaccharidosis VI.

Ho TT, Maguire AM, Aguirre GD, Surace EM, Anand V, Zeng Y, Salvetti A, Hopwood JJ, Haskins ME, Bennett J.

J Gene Med. 2002 Nov-Dec;4(6):613-21.


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.


A Clinical Trial of Gene Therapy for Mucopolysaccharidosis VI, a Severe Lysosomal Storage Disorder (MeuSIX).

[No authors listed]

Hum Gene Ther Clin Dev. 2015 Jun;26(2):86-8. doi: 10.1089/humc.2015.2525. No abstract available.


Novel mutations of the arylsulphatase B (ARSB) gene in Indian patients with mucopolysaccharidosis type VI.

Uttarilli A, Ranganath P, Jain SJ, Prasad CK, Sinha A, Verma IC, Phadke SR, Puri RD, Danda S, Muranjan MN, Jevalikar G, Nagarajaram HA, Dalal AB.

Indian J Med Res. 2015 Oct;142(4):414-25. doi: 10.4103/0971-5916.169201.


Replacement therapy in Mucopolysaccharidosis type VI: advantages of early onset of therapy.

Auclair D, Hopwood JJ, Brooks DA, Lemontt JF, Crawley AC.

Mol Genet Metab. 2003 Mar;78(3):163-74.


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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