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Results: 1 to 20 of 107

1.

Therapeutic efficacy of bone marrow transplant, intracranial AAV-mediated gene therapy, or both in the mouse model of MPS IIIB.

Heldermon CD, Ohlemiller KK, Herzog ED, Vogler C, Qin E, Wozniak DF, Tan Y, Orrock JL, Sands MS.

Mol Ther. 2010 May;18(5):873-80. doi: 10.1038/mt.2010.17. Epub 2010 Feb 23.

PMID:
20179679
[PubMed - indexed for MEDLINE]
Free PMC Article
2.

Disease correction by combined neonatal intracranial AAV and systemic lentiviral gene therapy in Sanfilippo Syndrome type B mice.

Heldermon CD, Qin EY, Ohlemiller KK, Herzog ED, Brown JR, Vogler C, Hou W, Orrock JL, Crawford BE, Sands MS.

Gene Ther. 2013 Sep;20(9):913-21. doi: 10.1038/gt.2013.14. Epub 2013 Mar 28.

PMID:
23535899
[PubMed - indexed for MEDLINE]
Free PMC Article
3.

Synergistic effects of central nervous system-directed gene therapy and bone marrow transplantation in the murine model of infantile neuronal ceroid lipofuscinosis.

Macauley SL, Roberts MS, Wong AM, McSloy F, Reddy AS, Cooper JD, Sands MS.

Ann Neurol. 2012 Jun;71(6):797-804. doi: 10.1002/ana.23545. Epub 2012 Feb 24.

PMID:
22368049
[PubMed - indexed for MEDLINE]
Free PMC Article
4.

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]
Free Article
5.

Significantly increased lifespan and improved behavioral performances by rAAV gene delivery in adult mucopolysaccharidosis IIIB mice.

Fu H, Kang L, Jennings JS, Moy SS, Perez A, Dirosario J, McCarty DM, Muenzer J.

Gene Ther. 2007 Jul;14(14):1065-77. Epub 2007 Apr 26.

PMID:
17460717
[PubMed - indexed for MEDLINE]
6.
7.

Bone marrow transplantation augments the effect of brain- and spinal cord-directed adeno-associated virus 2/5 gene therapy by altering inflammation in the murine model of globoid-cell leukodystrophy.

Reddy AS, Kim JH, Hawkins-Salsbury JA, Macauley SL, Tracy ET, Vogler CA, Han X, Song SK, Wozniak DF, Fowler SC, Klein RS, Sands MS.

J Neurosci. 2011 Jul 6;31(27):9945-57. doi: 10.1523/JNEUROSCI.1802-11.2011.

PMID:
21734286
[PubMed - indexed for MEDLINE]
Free PMC Article
8.

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]
Free Article
9.

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.

PMID:
21386820
[PubMed - indexed for MEDLINE]
Free PMC Article
10.

Improved behavior and neuropathology in the mouse model of Sanfilippo type IIIB disease after adeno-associated virus-mediated gene transfer in the striatum.

Cressant A, Desmaris N, Verot L, Bréjot T, Froissart R, Vanier MT, Maire I, Heard JM.

J Neurosci. 2004 Nov 10;24(45):10229-39.

PMID:
15537895
[PubMed - indexed for MEDLINE]
Free Article
11.

Central nervous system-directed AAV2/5-mediated gene therapy synergizes with bone marrow transplantation in the murine model of globoid-cell leukodystrophy.

Lin D, Donsante A, Macauley S, Levy B, Vogler C, Sands MS.

Mol Ther. 2007 Jan;15(1):44-52.

PMID:
17164774
[PubMed - indexed for MEDLINE]
12.

Retrovirally transduced bone marrow has a therapeutic effect on brain in the mouse model of mucopolysaccharidosis IIIB.

Zheng Y, Ryazantsev S, Ohmi K, Zhao HZ, Rozengurt N, Kohn DB, Neufeld EF.

Mol Genet Metab. 2004 Aug;82(4):286-95.

PMID:
15308126
[PubMed - indexed for MEDLINE]
13.

Accelerated clinical disease and pathology in mucopolysaccharidosis type IIIB and GalNAc transferase double knockout mice.

Mohammed EE, Snella EM, Rutz-Mendicino MM, Echevarria FD, Awedikian R, Whitley EM, Ellinwood NM.

Mol Genet Metab. 2012 Sep;107(1-2):129-35. doi: 10.1016/j.ymgme.2012.07.017. Epub 2012 Jul 25.

PMID:
22867887
[PubMed - indexed for MEDLINE]
14.

Liver production of sulfamidase reverses peripheral and ameliorates CNS pathology in mucopolysaccharidosis IIIA mice.

Ruzo A, Garcia M, Ribera A, Villacampa P, Haurigot V, Marcó S, Ayuso E, Anguela XM, Roca C, Agudo J, Ramos D, Ruberte J, Bosch F.

Mol Ther. 2012 Feb;20(2):254-66. doi: 10.1038/mt.2011.220. Epub 2011 Oct 18.

PMID:
22008915
[PubMed - indexed for MEDLINE]
Free PMC Article
15.

CNS-directed gene therapy for lysosomal storage diseases.

Sands MS, Haskins ME.

Acta Paediatr Suppl. 2008 Apr;97(457):22-7. doi: 10.1111/j.1651-2227.2008.00660.x. Review.

PMID:
18339183
[PubMed - indexed for MEDLINE]
Free PMC Article
16.

Safe, efficient, and reproducible gene therapy of the brain in the dog models of Sanfilippo and Hurler syndromes.

Ellinwood NM, Ausseil J, Desmaris N, Bigou S, Liu S, Jens JK, Snella EM, Mohammed EE, Thomson CB, Raoul S, Joussemet B, Roux F, Chérel Y, Lajat Y, Piraud M, Benchaouir R, Hermening S, Petry H, Froissart R, Tardieu M, Ciron C, Moullier P, Parkes J, Kline KL, Maire I, Vanier MT, Heard JM, Colle MA.

Mol Ther. 2011 Feb;19(2):251-9. doi: 10.1038/mt.2010.265. Epub 2010 Dec 7.

PMID:
21139569
[PubMed - indexed for MEDLINE]
Free PMC Article
17.

Intracranial gene delivery of LV-NAGLU vector corrects neuropathology in murine MPS IIIB.

Di Domenico C, Villani GR, Di Napoli D, Nusco E, Calì G, Nitsch L, Di Natale P.

Am J Med Genet A. 2009 Jun;149A(6):1209-18. doi: 10.1002/ajmg.a.32861.

PMID:
19449420
[PubMed - indexed for MEDLINE]
18.

AAV-mediated intravitreal gene therapy reduces lysosomal storage in the retinal pigmented epithelium and improves retinal function in adult MPS VII mice.

Hennig AK, Ogilvie JM, Ohlemiller KK, Timmers AM, Hauswirth WW, Sands MS.

Mol Ther. 2004 Jul;10(1):106-16.

PMID:
15233947
[PubMed - indexed for MEDLINE]
19.

CNS-targeted AAV5 gene transfer results in global dispersal of vector and prevention of morphological and function deterioration in CNS of globoid cell leukodystrophy mouse model.

Lin DS, Hsiao CD, Liau I, Lin SP, Chiang MF, Chuang CK, Wang TJ, Wu TY, Jian YR, Huang SF, Liu HL.

Mol Genet Metab. 2011 Aug;103(4):367-77. doi: 10.1016/j.ymgme.2011.05.005. Epub 2011 May 12.

PMID:
21620749
[PubMed - indexed for MEDLINE]
20.

Murine mucopolysaccharidosis type VII: the impact of therapies on the clinical course and pathology in a murine model of lysosomal storage disease.

Vogler C, Sands MS, Galvin N, Levy B, Thorpe C, Barker J, Sly WS.

J Inherit Metab Dis. 1998 Aug;21(5):575-86. Review.

PMID:
9728337
[PubMed - indexed for MEDLINE]

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