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Items: 1 to 20 of 44


Gene transfer corrects acute GM2 gangliosidosis--potential therapeutic contribution of perivascular enzyme flow.

Cachón-González MB, Wang SZ, McNair R, Bradley J, Lunn D, Ziegler R, Cheng SH, Cox TM.

Mol Ther. 2012 Aug;20(8):1489-500. doi: 10.1038/mt.2012.44. Epub 2012 Mar 27.


Depot formation of doxycycline impairs Tet-regulated gene expression in vivo.

Anders K, Buschow C, Charo J, Blankenstein T.

Transgenic Res. 2012 Oct;21(5):1099-107. doi: 10.1007/s11248-011-9580-0. Epub 2011 Dec 14.


GM2 gangliosidosis in a UK study of children with progressive neurodegeneration: 73 cases reviewed.

Smith NJ, Winstone AM, Stellitano L, Cox TM, Verity CM.

Dev Med Child Neurol. 2012 Feb;54(2):176-82. doi: 10.1111/j.1469-8749.2011.04160.x. Epub 2011 Nov 24.


Natural history of infantile G(M2) gangliosidosis.

Bley AE, Giannikopoulos OA, Hayden D, Kubilus K, Tifft CJ, Eichler FS.

Pediatrics. 2011 Nov;128(5):e1233-41. doi: 10.1542/peds.2011-0078. Epub 2011 Oct 24.


Temporal and cell-specific deletion establishes that neuronal Npc1 deficiency is sufficient to mediate neurodegeneration.

Yu T, Shakkottai VG, Chung C, Lieberman AP.

Hum Mol Genet. 2011 Nov 15;20(22):4440-51. doi: 10.1093/hmg/ddr372. Epub 2011 Aug 19.


Adeno-associated virus-mediated expression of β-hexosaminidase prevents neuronal loss in the Sandhoff mouse brain.

Sargeant TJ, Wang S, Bradley J, Smith NJ, Raha AA, McNair R, Ziegler RJ, Cheng SH, Cox TM, Cachón-González MB.

Hum Mol Genet. 2011 Nov 15;20(22):4371-80. doi: 10.1093/hmg/ddr364. Epub 2011 Aug 18.


Anatomically defined neuron-based rescue of neurodegenerative Niemann-Pick type C disorder.

Lopez ME, Klein AD, Dimbil UJ, Scott MP.

J Neurosci. 2011 Mar 23;31(12):4367-78. doi: 10.1523/JNEUROSCI.5981-10.2011.


Tight Long-term dynamic doxycycline responsive nigrostriatal GDNF using a single rAAV vector.

Manfredsson FP, Burger C, Rising AC, Zuobi-Hasona K, Sullivan LF, Lewin AS, Huang J, Piercefield E, Muzyczka N, Mandel RJ.

Mol Ther. 2009 Nov;17(11):1857-67. doi: 10.1038/mt.2009.196. Epub 2009 Aug 25.


Direct and indirect connections with upper limb motoneurons from the primate reticulospinal tract.

Riddle CN, Edgley SA, Baker SN.

J Neurosci. 2009 Apr 15;29(15):4993-9. doi: 10.1523/JNEUROSCI.3720-08.2009.


Developmental changes of glycosphingolipids and expression of glycogenes in mouse brains.

Ngamukote S, Yanagisawa M, Ariga T, Ando S, Yu RK.

J Neurochem. 2007 Dec;103(6):2327-41. Epub 2007 Sep 19.


Silencing and un-silencing of tetracycline-controlled genes in neurons.

Zhu P, Aller MI, Baron U, Cambridge S, Bausen M, Herb J, Sawinski J, Cetin A, Osten P, Nelson ML, Kügler S, Seeburg PH, Sprengel R, Hasan MT.

PLoS One. 2007 Jun 20;2(6):e533.


The spinal pathophysiology of spasticity--from a basic science point of view.

Nielsen JB, Crone C, Hultborn H.

Acta Physiol (Oxf). 2007 Feb;189(2):171-80. Review.


Effective gene therapy in an authentic model of Tay-Sachs-related diseases.

Cachón-González MB, Wang SZ, Lynch A, Ziegler R, Cheng SH, Cox TM.

Proc Natl Acad Sci U S A. 2006 Jul 5;103(27):10373-8. Epub 2006 Jun 26.


Development of a single-cassette system for spatiotemporal gene regulation in mice.

Miyazaki S, Miyazaki T, Tashiro F, Yamato E, Miyazaki J.

Biochem Biophys Res Commun. 2005 Dec 16;338(2):1083-8. Epub 2005 Oct 21.


Functional recovery of glycine receptors in spastic murine model of startle disease.

Molon A, Di Giovanni S, Hathout Y, Natale J, Hoffman EP.

Neurobiol Dis. 2006 Feb;21(2):291-304. Epub 2005 Sep 22.


Tau suppression in a neurodegenerative mouse model improves memory function.

Santacruz K, Lewis J, Spires T, Paulson J, Kotilinek L, Ingelsson M, Guimaraes A, DeTure M, Ramsden M, McGowan E, Forster C, Yue M, Orne J, Janus C, Mariash A, Kuskowski M, Hyman B, Hutton M, Ashe KH.

Science. 2005 Jul 15;309(5733):476-81.


An efficient system to establish multiple embryonic stem cell lines carrying an inducible expression unit.

Masui S, Shimosato D, Toyooka Y, Yagi R, Takahashi K, Niwa H.

Nucleic Acids Res. 2005 Mar 1;33(4):e43.


A genetic switch for epilepsy in adult mice.

Krestel HE, Shimshek DR, Jensen V, Nevian T, Kim J, Geng Y, Bast T, Depaulis A, Schonig K, Schwenk F, Bujard H, Hvalby Ø, Sprengel R, Seeburg PH.

J Neurosci. 2004 Nov 17;24(46):10568-78.


Reciprocal inhibition and corticospinal transmission in the arm and leg in patients with autosomal dominant pure spastic paraparesis (ADPSP).

Crone C, Petersen NT, Nielsen JE, Hansen NL, Nielsen JB.

Brain. 2004 Dec;127(Pt 12):2693-702. Epub 2004 Oct 27.


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