RNAi blocks DYT1 mutant torsinA inclusions in neurons.
Neurosci Lett
; 395(3): 201-5, 2006 Mar 13.
Article
en En
| MEDLINE
| ID: mdl-16332410
ABSTRACT
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 (Delta302/303). 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.
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Colección:
01-internacional
Banco de datos:
MEDLINE
Asunto principal:
Chaperonas Moleculares
/
Interferencia de ARN
/
Neuronas
Límite:
Animals
/
Humans
Idioma:
En
Revista:
Neurosci Lett
Año:
2006
Tipo del documento:
Article
País de afiliación:
Estados Unidos