Journal
NEUROSCIENCE LETTERS
Volume 395, Issue 3, Pages 201-205Publisher
ELSEVIER IRELAND LTD
DOI: 10.1016/j.neulet.2005.10.098
Keywords
RNAi; shRNA; TorsinA; DYT1
Categories
Funding
- NINDS NIH HHS [NS37409, NS28384] Funding Source: Medline
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Early onset generalized dystonia is a dominantly inherited movement disorder caused by neuronal dysfunction without an apparent loss of neurons. The same single mutation (GAG deletion) causes most cases and results in loss of a glutamic acid (E) in the carboxy terminal region of torsinA (Delta 302/303). To model the neuronal involvement, adult rat primary sensory dorsal root ganglion neurons in culture were infected with lentivirus vectors expressing human wild-type or mutant torsinA. Expression of the mutant protein resulted in formation of torsinA-positive perinuclear inclusions. When the cells were co-infected with lentivirus vectors expressing the mutant torsinA message and a shRNA selectively targeting this message, inclusion formation was blocked. Vector-delivered siRNAs have the potential to decrease the adverse effects of this mutant protein in neurons without affecting wild-type protein. (c) 2005 Elsevier Ireland Ltd. All rights reserved.
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