PubMed

Lentiviral-mediated gene correction of mucopolysaccharidosis type IIIA.

Anson DS, McIntyre C, Thomas B, Koldej R, Ranieri E, Roberts A, Clements PR, Dunning K, Byers S.

Genet Vaccines Ther. 2007 Jan 16;5:1.PMID: 17227588 [PubMed]Related articlesFree article

Lentiviral-mediated gene therapy for murine mucopolysaccharidosis type IIIA.

McIntyre C, Derrick Roberts AL, Ranieri E, Clements PR, Byers S, Anson DS.

Mol Genet Metab. 2008 Apr;93(4):411-8. Epub 2008 Jan 8.PMID: 18248829 [PubMed - indexed for MEDLINE]Related articles

Improvement in behaviour after substrate deprivation therapy with rhodamine B in a mouse model of MPS IIIA.

Roberts AL, Rees MH, Klebe S, Fletcher JM, Byers S.

Mol Genet Metab. 2007 Sep-Oct;92(1-2):115-21. Epub 2007 Aug 6.PMID: 17681480 [PubMed - indexed for MEDLINE]Related articles

Recombinant human sulphamidase: expression, amplification, purification and characterization.

Bielicki J, Hopwood JJ, Melville EL, Anson DS.

Biochem J. 1998 Jan 1;329 ( Pt 1):145-50.PMID: 9405287 [PubMed - indexed for MEDLINE]Related articlesFree article

Inhibition of glycosaminoglycan synthesis using rhodamine B in a mouse model of mucopolysaccharidosis type IIIA.

Roberts AL, Thomas BJ, Wilkinson AS, Fletcher JM, Byers S.

Pediatr Res. 2006 Sep;60(3):309-14. Epub 2006 Jul 20.PMID: 16857766 [PubMed - indexed for MEDLINE]Related articles

Gastrointestinal pathology in a mouse model of mucopolysaccharidosis type IIIA.

Roberts AL, Howarth GS, Liaw WC, Moretta S, Kritas S, Lymn KA, Yazbeck R, Tran C, Fletcher JM, Butler RN, Byers S.

J Cell Physiol. 2009 May;219(2):259-64.PMID: 19170061 [PubMed - indexed for MEDLINE]Related articles

Functional correction of CNS lesions in an MPS-IIIA mouse model by intracerebral AAV-mediated delivery of sulfamidase and SUMF1 genes.

Fraldi A, Hemsley K, Crawley A, Lombardi A, Lau A, Sutherland L, Auricchio A, Ballabio A, Hopwood JJ.

Hum Mol Genet. 2007 Nov 15;16(22):2693-702. Epub 2007 Aug 27.PMID: 17725987 [PubMed - indexed for MEDLINE]Related articlesFree article

Gene therapy for a mucopolysaccharidosis type I murine model with lentiviral-IDUA vector.

Di Domenico C, Villani GR, Di Napoli D, Reyero EG, Lombardo A, Naldini L, Di Natale P.

Hum Gene Ther. 2005 Jan;16(1):81-90.PMID: 15703491 [PubMed - indexed for MEDLINE]Related articles

Sulphamidase.

Anson DS, Bielicki J.

Int J Biochem Cell Biol. 1999 Mar-Apr;31(3-4):363-7. Review.PMID: 10224661 [PubMed - indexed for MEDLINE]Related articles

Cloning of the sulphamidase gene and identification of mutations in Sanfilippo A syndrome.

Scott HS, Blanch L, Guo XH, Freeman C, Orsborn A, Baker E, Sutherland GR, Morris CP, Hopwood JJ.

Nat Genet. 1995 Dec;11(4):465-7.PMID: 7493035 [PubMed - indexed for MEDLINE]Related articles

Enzymatic correction and cross-correction of mucopolysaccharidosis type I fibroblasts by adeno-associated virus-mediated transduction of the alpha-L-iduronidase gene.

Hartung SD, Reddy RG, Whitley CB, McIvor RS.

Hum Gene Ther. 1999 Sep 1;10(13):2163-72.PMID: 10498248 [PubMed - indexed for MEDLINE]Related articles

In vitro gene therapy of mucopolysaccharidosis type I by lentiviral vectors.

Di Natale P, Di Domenico C, Villani GR, Lombardo A, Follenzi A, Naldini L.

Eur J Biochem. 2002 Jun;269(11):2764-71.PMID: 12047386 [PubMed - indexed for MEDLINE]Related articlesFree article

Correction of mucopolysaccharidosis type IIIb fibroblasts by lentiviral vector-mediated gene transfer.

Villani GR, Follenzi A, Vanacore B, Di Domenico C, Naldini L, Di Natale P.

Biochem J. 2002 Jun 15;364(Pt 3):747-53.PMID: 12049639 [PubMed - indexed for MEDLINE]Related articlesFree article

Neonatal gene therapy of MPS I mice by intravenous injection of a lentiviral vector.

Kobayashi H, Carbonaro D, Pepper K, Petersen D, Ge S, Jackson H, Shimada H, Moats R, Kohn DB.

Mol Ther. 2005 May;11(5):776-89.PMID: 15851016 [PubMed - indexed for MEDLINE]Related articles

Effect of high dose, repeated intra-CSF injection of sulphamidase on neuropathology in MPS IIIA mice.

Hemsley KM, Beard H, King BM, Hopwood JJ.

Genes Brain Behav. 2008 Jun 2. [Epub ahead of print]PMID: 18518922 [PubMed - as supplied by publisher]Related articles

Prediction of Sanfilippo phenotype severity from immunoquantification of heparan-N-sulfamidase in cultured fibroblasts from mucopolysaccharidosis type IIIA patients.

Perkins KJ, Muller V, Weber B, Hopwood JJ.

Mol Genet Metab. 2001 Aug;73(4):306-12.PMID: 11509012 [PubMed - indexed for MEDLINE]Related articles

Impairment of glycosaminoglycan synthesis in mucopolysaccharidosis type IIIA cells by using siRNA: a potential therapeutic approach for Sanfilippo disease.

Dziedzic D, Wegrzyn G, Jakóbkiewicz-Banecka J.

Eur J Hum Genet. 2010 Feb;18(2):200-5. Epub 2009 Aug 19.PMID: 19690584 [PubMed - indexed for MEDLINE]Related articles

Molecular defects in Sanfilippo syndrome type A.

Blanch L, Weber B, Guo XH, Scott HS, Hopwood JJ.

Hum Mol Genet. 1997 May;6(5):787-91.PMID: 9158154 [PubMed - indexed for MEDLINE]Related articlesFree article

Neurological correction of lysosomal storage in a mucopolysaccharidosis IIIB mouse model by adeno-associated virus-mediated gene delivery.

Fu H, Samulski RJ, McCown TJ, Picornell YJ, Fletcher D, Muenzer J.

Mol Ther. 2002 Jan;5(1):42-9.PMID: 11786044 [PubMed - indexed for MEDLINE]Related articlesFree article

Enzyme-replacement therapy from birth delays the development of behavior and learning problems in mucopolysaccharidosis type IIIA mice.

Gliddon BL, Hopwood JJ.

Pediatr Res. 2004 Jul;56(1):65-72. Epub 2004 May 5.PMID: 15128919 [PubMed - indexed for MEDLINE]Related articles