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Striatal and nigral pathology in a lentiviral rat model of Machado-Joseph disease
Alves, Sandro; Régulier, Etienne; Nascimento-Ferreira, Isabel; Hassig, Raymonde; Dufour, Noelle; Koeppen, Arnulf; Carvalho, Ana Luísa; Simões, SérgioMachado-Joseph disease (MJD) is a fatal, dominant neurodegenerative disorder. MJD results from polyglutamine repeat expansion in the MJD-1 gene, conferring a toxic gain of function to the ataxin-3 protein. In this study, we aimed at overexpressing ataxin-3 in the rat brain using lentiviral vectors (LV), to generate an in vivo MJD genetic model and, to study the disorder in defined brain regions: substantia nigr...
Allele-specific RNA silencing of mutant ataxin-3 mediates neuroprotection in a ...
Alves, Sandro; Nascimento-Ferreira, Isabel; Auregan, Gwennaëlle; Hassig, Raymonde; Dufour, Noëlle; Brouillet, Emmanuel; Lima, Maria C. Pedroso deRecent studies have demonstrated that RNAi is a promising approach for treating autosomal dominant disorders. However, discrimination between wild-type and mutant transcripts is essential, to preserve wild-type expression and function. A single nucleotide polymorphism (SNP) is present in more than 70% of patients with Machado-Joseph disease (MJD). We investigated whether this SNP could be used to inactivate mut...
Long-term lentiviral-mediated expression of ciliary neurotrophic factor in the ...
Zala, Diana; Bensadoun, Jean-Charles; Pereira de Almeida, Luis; Leavitt, Blair R.; Gutekunst, Claire-Anne; Aebischer, Patrick; Hayden, Michael R.Ciliary neurotrophic factor (CNTF) has been shown to prevent behavioral deficits and striatal degeneration in neurotoxic models of Huntington's disease (HD), but its effect in a genetic model has not been evaluated. Lentiviral vectors expressing the human CNTF or LacZ reporter gene were therefore injected in the striatum of wild-type (WT) and transgenic mice expressing full-length huntingtin with 72 CAG repeats...
Comparative study of GDNF delivery systems for the CNS: polymer rods, encapsula...
Bensadoun, Jean-Charles; Pereira de Almeida, Luis; Fine, Eric G.; Tseng, Jack L.; Déglon, Nicole; Aebischer, PatrickGlial cell line-derived neurotrophic factor (GDNF) holds great promise for the treatment of Parkinson's disease. In humans, its intracerebroventricular administration leads to limiting side effects. Direct parenchymal delivery using mechanical means, or cell and gene therapy represent potential alternatives. In the present study, a representative of each of these three approaches, i.e. polymer rods, genetically...
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