KMID : 1197720110040010001
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´ëÇÑÆÄŲ½¼º´ ¹× ÀÌ»ó¿îµ¿Áúȯ ÇÐȸÁö 2011 Volume.4 No. 1 p.1 ~ p.7
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Rationale for Therapeutic Silencing of Alpha-Synuclein in Parkinson¡¯s Disease
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Maraganore Demetrius M.
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Abstract
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The purpose of this paper is to provide the rationale for therapeutic silencing of the alpha-synuclein gene (SNCA) in Parkinson¡¯s disease (PD). The paper reviews the public health significance of PD; the causal links between rare SNCA variants and familial PD; the association of common SNCA variants and PD susceptibility; the association of SNCA variants also with age at onset and motor and cognitive outcomes in PD; therapeutic strategies targeting SNCA in PD; and preliminary findings and considerations on small interfering RNA-based therapies and PD.
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KEYWORD
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Parkinson¡¯s disease, Alpha-synuclein, RNA-based therapies
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