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Article

<i>SNCA</i> -targeted epigenome therapy for Parkinson’s disease alleviates pathological and behavioral perturbations in a mouse model

2025-06-17

Abstract excerpt

Alpha-synuclein (SNCA) overexpression is implicated in Parkinson’s disease (PD) pathogenesis, making SNCA downregulation a promising therapeutic strategy. We developed a SNCA -targeted epigenome therapy using an all-in-one lentiviral vector (LV) carrying deactivated CRISPR/(d)Cas9, gRNA targeted at SNCA -intron1, and either the catalytic domain of DNA-methyltransferase3A (DNMT3A), or a synthetic repressor molec...

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Identifiers and source

Literature Corpus work
3e63113c-0ca8-55fb-a557-52e59dcd1300
DOI
10.1101/2025.06.16.659948
Open publication

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<i>SNCA</i> -targeted epigenome therapy for Parkinson’s disease alleviates pathological and behavioral perturbations in a mouse modelDOI 10.1101/2025.06.16.659948
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