Inhibition of Ras-guanine nucleotide-releasing factor 1 (Ras-GRF1) signaling in the striatum reverts motor symptoms associated with L-dopa-induced dyskinesia.

S. Fasano, E. Bezard, A. D'Antoni, V. Francardo, M. Indrigo, L. Qin, S. Dovero, M. Cerovic, M. A. Cenci, R. Brambilla
Proceedings of the National Academy of Sciences. 2010-11-29; 107(50): 21824-21829
DOI: 10.1073/pnas.1012071107

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1. Proc Natl Acad Sci U S A. 2010 Dec 14;107(50):21824-9. doi:
10.1073/pnas.1012071107. Epub 2010 Nov 29.

Inhibition of Ras-guanine nucleotide-releasing factor 1 (Ras-GRF1) signaling in
the striatum reverts motor symptoms associated with L-dopa-induced dyskinesia.

Fasano S(1), Bezard E, D’Antoni A, Francardo V, Indrigo M, Qin L, Doveró S,
Cerovic M, Cenci MA, Brambilla R.

Author information:
(1)Basal Ganglia Pathophysiology Unit, Department of Experimental Medical
Science, Lund University, BMC F11, Lund 221 84, Sweden.

L-dopa-induced dyskinesia (LID) is a common debilitating complication of dopamine
replacement therapy in Parkinson’s disease. Recent evidence suggests that LID may
be linked causally to a hyperactivation of the Ras-ERK signaling cascade in the
basal ganglia. We set out to determine whether specific targeting of Ras-guanine
nucleotide-releasing factor 1 (Ras-GRF1), a brain-specific activator of the
Ras-ERK pathway, may provide a therapy for LID. On the rodent abnormal
involuntary movements scale, Ras-GRF1-deficient mice were significantly resistant
to the development of dyskinesia during chronic L-dopa treatment. Furthermore, in
a nonhuman primate model of LID, lentiviral vectors expressing dominant negative
forms of Ras-GRF1 caused a dramatic reversion of dyskinesia severity leaving
intact the therapeutic effect of L-dopa. These data reveal the central role of
Ras-GRF1 in governing striatal adaptations to dopamine replacement therapy and
validate a viable treatment for LID based on intracellular signaling modulation.

DOI: 10.1073/pnas.1012071107
PMCID: PMC3003069
PMID: 21115823 [Indexed for MEDLINE]

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