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Article

α-Synuclein antisense oligonucleotides as a disease-modifying therapy for Parkinson’s disease

7 Mar 2021

Abstract excerpt

Parkinson's disease (PD) is a prevalent neurodegenerative disease with no approved disease-modifying therapies. Multiplications, mutations, and single nucleotide polymorphisms in the SNCA gene, encoding α-synuclein (aSyn) protein, either cause or increase risk for PD. Intracellular accumulations of aSyn are pathological hallmarks of PD. Taken together, reduction of aSyn production may provide a disease-modifying...

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PMID: 33682798
DOI: 10.1172/jci.insight.135633

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