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Items: 1 to 20 of 84

1.

Gene therapy for late infantile neuronal ceroid lipofuscinosis: neurosurgical considerations.

Souweidane MM, Fraser JF, Arkin LM, Sondhi D, Hackett NR, Kaminsky SM, Heier L, Kosofsky BE, Worgall S, Crystal RG, Kaplitt MG.

J Neurosurg Pediatr. 2010 Aug;6(2):115-22. doi: 10.3171/2010.4.PEDS09507.

2.

Oxidative stress in Niemann-Pick disease, type C.

Fu R, Yanjanin NM, Bianconi S, Pavan WJ, Porter FD.

Mol Genet Metab. 2010 Oct-Nov;101(2-3):214-8. doi: 10.1016/j.ymgme.2010.06.018. Epub 2010 Jul 27.

3.

Common and uncommon pathogenic cascades in lysosomal storage diseases.

Vitner EB, Platt FM, Futerman AH.

J Biol Chem. 2010 Jul 2;285(27):20423-7. doi: 10.1074/jbc.R110.134452. Epub 2010 Apr 29. Review.

4.

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.

5.

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
6.

Molecular signatures of disease brain endothelia provide new sites for CNS-directed enzyme therapy.

Chen YH, Chang M, Davidson BL.

Nat Med. 2009 Oct;15(10):1215-8. doi: 10.1038/nm.2025. Epub 2009 Sep 13.

7.

Metabolic adaptations to interrupted glycosaminoglycan recycling.

Woloszynek JC, Kovacs A, Ohlemiller KK, Roberts M, Sands MS.

J Biol Chem. 2009 Oct 23;284(43):29684-91. doi: 10.1074/jbc.M109.020818. Epub 2009 Aug 21.

8.

Treatment of late infantile neuronal ceroid lipofuscinosis by CNS administration of a serotype 2 adeno-associated virus expressing CLN2 cDNA.

Worgall S, Sondhi D, Hackett NR, Kosofsky B, Kekatpure MV, Neyzi N, Dyke JP, Ballon D, Heier L, Greenwald BM, Christos P, Mazumdar M, Souweidane MM, Kaplitt MG, Crystal RG.

Hum Gene Ther. 2008 May;19(5):463-74. doi: 10.1089/hum.2008.022.

PMID:
18473686
9.

Lentiviral-transduced human mesenchymal stem cells persistently express therapeutic levels of enzyme in a xenotransplantation model of human disease.

Meyerrose TE, Roberts M, Ohlemiller KK, Vogler CA, Wirthlin L, Nolta JA, Sands MS.

Stem Cells. 2008 Jul;26(7):1713-22. doi: 10.1634/stemcells.2008-0008. Epub 2008 Apr 24.

10.

The role of AMPK in psychosine mediated effects on oligodendrocytes and astrocytes: implication for Krabbe disease.

Giri S, Khan M, Nath N, Singh I, Singh AK.

J Neurochem. 2008 Jun;105(5):1820-33. doi: 10.1111/j.1471-4159.2008.05279.x. Epub 2008 Feb 4.

11.

Mitochondrial Ca2+ homeostasis in lysosomal storage diseases.

Kiselyov K, Muallem S.

Cell Calcium. 2008 Jul;44(1):103-11. doi: 10.1016/j.ceca.2007.12.005. Epub 2008 Feb 1. Review.

12.

N-butyldeoxygalactonojirimycin reduces brain ganglioside and GM2 content in neonatal Sandhoff disease mice.

Baek RC, Kasperzyk JL, Platt FM, Seyfried TN.

Neurochem Int. 2008 May;52(6):1125-33. doi: 10.1016/j.neuint.2007.12.001. Epub 2007 Dec 8.

PMID:
18207611
13.

ER and oxidative stresses are common mediators of apoptosis in both neurodegenerative and non-neurodegenerative lysosomal storage disorders and are alleviated by chemical chaperones.

Wei H, Kim SJ, Zhang Z, Tsai PC, Wisniewski KE, Mukherjee AB.

Hum Mol Genet. 2008 Feb 15;17(4):469-77. Epub 2007 Nov 7.

PMID:
17989065
14.

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.

15.

Lysosomal dysfunction results in altered energy balance.

Woloszynek JC, Coleman T, Semenkovich CF, Sands MS.

J Biol Chem. 2007 Dec 7;282(49):35765-71. Epub 2007 Oct 2.

17.

Combination brain and systemic injections of AAV provide maximal functional and survival benefits in the Niemann-Pick mouse.

Passini MA, Bu J, Fidler JA, Ziegler RJ, Foley JW, Dodge JC, Yang WW, Clarke J, Taksir TV, Griffiths DA, Zhao MA, O'Riordan CR, Schuchman EH, Shihabuddin LS, Cheng SH.

Proc Natl Acad Sci U S A. 2007 May 29;104(22):9505-10. Epub 2007 May 21.

18.

Stem cells act through multiple mechanisms to benefit mice with neurodegenerative metabolic disease.

Lee JP, Jeyakumar M, Gonzalez R, Takahashi H, Lee PJ, Baek RC, Clark D, Rose H, Fu G, Clarke J, McKercher S, Meerloo J, Muller FJ, Park KI, Butters TD, Dwek RA, Schwartz P, Tong G, Wenger D, Lipton SA, Seyfried TN, Platt FM, Snyder EY.

Nat Med. 2007 Apr;13(4):439-47. Epub 2007 Mar 11.

PMID:
17351625
19.
20.

Cytokines, neurotrophins, and oxidative stress in brain disease from mucopolysaccharidosis IIIB.

Villani GR, Gargiulo N, Faraonio R, Castaldo S, Gonzalez Y Reyero E, Di Natale P.

J Neurosci Res. 2007 Feb 15;85(3):612-22.

PMID:
17139681

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