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Items: 1 to 20 of 116

1.

Cell- and gene-based therapeutic approaches for neurological deficits in mucopolysaccharidoses.

Pan D.

Curr Pharm Biotechnol. 2011 Jun;12(6):884-96. doi: 1389-2010/11 $58.00+.00. Review.

2.

Gene therapy for neurologic manifestations of mucopolysaccharidoses.

Wolf DA, Banerjee S, Hackett PB, Whitley CB, McIvor RS, Low WC.

Expert Opin Drug Deliv. 2015 Feb;12(2):283-96. doi: 10.1517/17425247.2015.966682. Epub 2014 Dec 16. Review.

3.

Targeting of the CNS in MPS-IH using a nonviral transferrin-alpha-L-iduronidase fusion gene product.

Osborn MJ, McElmurry RT, Peacock B, Tolar J, Blazar BR.

Mol Ther. 2008 Aug;16(8):1459-66. doi: 10.1038/mt.2008.119. Epub 2008 Jun 3.

4.

Current and potential therapeutic strategies for mucopolysaccharidoses.

Noh H, Lee JI.

J Clin Pharm Ther. 2014 Jun;39(3):215-24. doi: 10.1111/jcpt.12136. Epub 2014 Feb 25. Review.

PMID:
24612142
5.

Characterization of an immunodeficient mouse model of mucopolysaccharidosis type I suitable for preclinical testing of human stem cell and gene therapy.

Garcia-Rivera MF, Colvin-Wanshura LE, Nelson MS, Nan Z, Khan SA, Rogers TB, Maitra I, Low WC, Gupta P.

Brain Res Bull. 2007 Nov 1;74(6):429-38. Epub 2007 Aug 6. Erratum in: Brain Res Bull. 2008 Aug 15;76(6):640-1.

6.

The mucopolysaccharidoses: a success of molecular medicine.

Clarke LA.

Expert Rev Mol Med. 2008 Jan 18;10:e1. doi: 10.1017/S1462399408000550. Review.

PMID:
18201392
7.

Putative biological mechanisms of efficiency of substrate reduction therapies for mucopolysaccharidoses.

Banecka-Majkutewicz Z, Jakóbkiewicz-Banecka J, Gabig-Cimińska M, Węgrzyn A, Węgrzyn G.

Arch Immunol Ther Exp (Warsz). 2012 Dec;60(6):461-8. doi: 10.1007/s00005-012-0195-9. Epub 2012 Sep 5. Review.

PMID:
22949095
8.

Correction of neurological disease of mucopolysaccharidosis IIIB in adult mice by rAAV9 trans-blood-brain barrier gene delivery.

Fu H, Dirosario J, Killedar S, Zaraspe K, McCarty DM.

Mol Ther. 2011 Jun;19(6):1025-33. doi: 10.1038/mt.2011.34. Epub 2011 Mar 8.

9.

Therapies for neurological disease in the mucopolysaccharidoses.

Anson DS, McIntyre C, Byers S.

Curr Gene Ther. 2011 Apr;11(2):132-43. Review.

PMID:
21291356
10.

[Neurologic manifestations in mucopolysaccharidoses].

Héron B.

Arch Pediatr. 2014 Jun;21 Suppl 1:S14-21. doi: 10.1016/S0929-693X(14)72254-7. French.

PMID:
25063379
11.

Substrate reduction therapies for mucopolysaccharidoses.

Jakóbkiewicz-Banecka J, Piotrowska E, Gabig-Cimińska M, Borysiewicz E, Słomińska-Wojewódzka M, Narajczyk M, Węgrzyn A, Węgrzyn G.

Curr Pharm Biotechnol. 2011 Nov;12(11):1860-5. Review.

PMID:
21902626
12.

Gene therapy for mucopolysaccharidosis.

Ponder KP, Haskins ME.

Expert Opin Biol Ther. 2007 Sep;7(9):1333-45. Review.

13.

Hepatic storage of glycosaminoglycans in feline and canine models of mucopolysaccharidoses I, VI, and VII.

Haskins ME, Otis EJ, Hayden JE, Jezyk PF, Stramm L.

Vet Pathol. 1992 Mar;29(2):112-9.

14.

Large animal models of neurological disorders for gene therapy.

Gagliardi C, Bunnell BA.

ILAR J. 2009;50(2):128-43. Review.

15.

Gene expression-targeted isoflavone therapy.

Węgrzyn A.

IUBMB Life. 2012 Apr;64(4):307-15. doi: 10.1002/iub.1007. Epub 2012 Feb 23. Review.

16.

Different serum enzyme levels are required to rescue the various systemic features of the mucopolysaccharidoses.

Cotugno G, Tessitore A, Capalbo A, Annunziata P, Strisciuglio C, Faella A, Aurilio M, Di Tommaso M, Russo F, Mancini A, De Leonibus E, Aloj L, Auricchio A.

Hum Gene Ther. 2010 May;21(5):555-69. doi: 10.1089/hum.2009.189.

PMID:
20021231
17.

Gene delivery strategies for the treatment of mucopolysaccharidoses.

Baldo G, Giugliani R, Matte U.

Expert Opin Drug Deliv. 2014 Mar;11(3):449-59. doi: 10.1517/17425247.2014.880689. Epub 2014 Jan 23. Review.

PMID:
24450877
18.

Quantitative estimation of lysosomal storage in mucopolysaccharidoses by electron microscopy analysis.

Narajczyk M, Moskot M, Konieczna A.

Acta Biochim Pol. 2012;59(4):693-6. Epub 2012 Dec 11.

19.

Genistein-mediated inhibition of glycosaminoglycan synthesis as a basis for gene expression-targeted isoflavone therapy for mucopolysaccharidoses.

Piotrowska E, Jakóbkiewicz-Banecka J, Barańska S, Tylki-Szymańska A, Czartoryska B, Wegrzyn A, Wegrzyn G.

Eur J Hum Genet. 2006 Jul;14(7):846-52. Epub 2006 May 3.

20.

The final frontier -- crossing the blood-brain barrier.

Sly WS, Vogler C.

EMBO Mol Med. 2013 May;5(5):655-7. doi: 10.1002/emmm.201302668. No abstract available.

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