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Items: 1 to 20 of 104


Novel oligodendroglial alpha synuclein viral vector models of multiple system atrophy: studies in rodents and nonhuman primates.

Mandel RJ, Marmion DJ, Kirik D, Chu Y, Heindel C, McCown T, Gray SJ, Kordower JH.

Acta Neuropathol Commun. 2017 Jun 16;5(1):47. doi: 10.1186/s40478-017-0451-7.


Type 3 Adenylyl Cyclase and Somatostatin Receptor 3 Expression Persists in Aged Rat Neocortical and Hippocampal Neuronal Cilia.

Guadiana SM, Parker AK, Filho GF, Sequeira A, Semple-Rowland S, Shaw G, Mandel RJ, Foster TC, Kumar A, Sarkisian MR.

Front Aging Neurosci. 2016 May 31;8:127. doi: 10.3389/fnagi.2016.00127. eCollection 2016.


Neuroprotective potential of pleiotrophin overexpression in the striatonigral pathway compared with overexpression in both the striatonigral and nigrostriatal pathways.

Gombash SE, Manfredsson FP, Mandel RJ, Collier TJ, Fischer DL, Kemp CJ, Kuhn NM, Wohlgenant SL, Fleming SM, Sortwell CE.

Gene Ther. 2014 Jul;21(7):682-93. doi: 10.1038/gt.2014.42. Epub 2014 May 8.


Atraumatic acute carpal tunnel syndrome in a patient taking dabigatran.

Sibley PA, Mandel RJ.

Orthopedics. 2012 Aug 1;35(8):e1286-9. doi: 10.3928/01477447-20120725-37.


Regulated protein expression for in vivo gene therapy for neurological disorders: progress, strategies, and issues.

Manfredsson FP, Bloom DC, Mandel RJ.

Neurobiol Dis. 2012 Nov;48(2):212-21. doi: 10.1016/j.nbd.2012.03.001. Epub 2012 Mar 9. Review.


Striatal pleiotrophin overexpression provides functional and morphological neuroprotection in the 6-hydroxydopamine model.

Gombash SE, Lipton JW, Collier TJ, Madhavan L, Steece-Collier K, Cole-Strauss A, Terpstra BT, Spieles-Engemann AL, Daley BF, Wohlgenant SL, Thompson VB, Manfredsson FP, Mandel RJ, Sortwell CE.

Mol Ther. 2012 Mar;20(3):544-54. doi: 10.1038/mt.2011.216. Epub 2011 Oct 18.


Transgenic mice expressing caspase-6-derived N-terminal fragments of mutant huntingtin develop neurologic abnormalities with predominant cytoplasmic inclusion pathology composed largely of a smaller proteolytic derivative.

Tebbenkamp AT, Green C, Xu G, Denovan-Wright EM, Rising AC, Fromholt SE, Brown HH, Swing D, Mandel RJ, Tessarollo L, Borchelt DR.

Hum Mol Genet. 2011 Jul 15;20(14):2770-82. doi: 10.1093/hmg/ddr176. Epub 2011 Apr 22.


The development of flexible lentiviral vectors for gene transfer in the CNS.

Manfredsson FP, Mandel RJ.

Exp Neurol. 2011 Jun;229(2):201-6. doi: 10.1016/j.expneurol.2011.03.013. Epub 2011 Apr 1.


AAV6-mediated gene silencing fALS short.

Mandel RJ, Lowenstein PR, Byrne BJ.

Mol Ther. 2011 Feb;19(2):231-3. doi: 10.1038/mt.2010.306. No abstract available.


Longitudinal behavioral, cross-sectional transcriptional and histopathological characterization of a knock-in mouse model of Huntington's disease with 140 CAG repeats.

Rising AC, Xu J, Carlson A, Napoli VV, Denovan-Wright EM, Mandel RJ.

Exp Neurol. 2011 Apr;228(2):173-82. doi: 10.1016/j.expneurol.2010.12.017. Epub 2010 Dec 28.


α-Synuclein expression in rat substantia nigra suppresses phospholipase D2 toxicity and nigral neurodegeneration.

Gorbatyuk OS, Li S, Nguyen FN, Manfredsson FP, Kondrikova G, Sullivan LF, Meyers C, Chen W, Mandel RJ, Muzyczka N.

Mol Ther. 2010 Oct;18(10):1758-68. doi: 10.1038/mt.2010.137. Epub 2010 Jul 27.


In vivo RNAi-mediated alpha-synuclein silencing induces nigrostriatal degeneration.

Gorbatyuk OS, Li S, Nash K, Gorbatyuk M, Lewin AS, Sullivan LF, Mandel RJ, Chen W, Meyers C, Manfredsson FP, Muzyczka N.

Mol Ther. 2010 Aug;18(8):1450-7. doi: 10.1038/mt.2010.115. Epub 2010 Jun 15.


Development of gene therapy for neurological disorders.

Manfredsson FP, Mandel RJ.

Discov Med. 2010 Mar;9(46):204-11. Review.


Differential transduction following basal ganglia administration of distinct pseudotyped AAV capsid serotypes in nonhuman primates.

Dodiya HB, Bjorklund T, Stansell J 3rd, Mandel RJ, Kirik D, Kordower JH.

Mol Ther. 2010 Mar;18(3):579-87. doi: 10.1038/mt.2009.216. Epub 2009 Sep 22.


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.


Optimization of continuous in vivo DOPA production and studies on ectopic DA synthesis using rAAV5 vectors in Parkinsonian rats.

Björklund T, Hall H, Breysse N, Soneson C, Carlsson T, Mandel RJ, Carta M, Kirik D.

J Neurochem. 2009 Oct;111(2):355-67. doi: 10.1111/j.1471-4159.2009.06340.x. Epub 2009 Aug 17.


Nigrostriatal rAAV-mediated GDNF overexpression induces robust weight loss in a rat model of age-related obesity.

Manfredsson FP, Tumer N, Erdos B, Landa T, Broxson CS, Sullivan LF, Rising AC, Foust KD, Zhang Y, Muzyczka N, Gorbatyuk OS, Scarpace PJ, Mandel RJ.

Mol Ther. 2009 Jun;17(6):980-91. doi: 10.1038/mt.2009.45. Epub 2009 Mar 10.


AAV9: a potential blood-brain barrier buster.

Manfredsson FP, Rising AC, Mandel RJ.

Mol Ther. 2009 Mar;17(3):403-5. doi: 10.1038/mt.2009.15. No abstract available.

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