Trends and challenges of AAV-delivered gene editing therapeutics for CNS disorders: Implications for neurodegenerative disease.

Mol Ther Nucleic Acids · 2025 · PMC12341529 · PMID 40799507

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highly significantno p-value reported

188 , 189 , 190 , 191 GWAS implicated the SNCA gene (encodes the α-synuclein protein) as a highly significant genetic risk factor for synucleinopathies, including PD 192 , 193 , 194 , 195 , 196 , 197 , 198 , 199 , 200 , 201 , 202 , 203 , 204 , 205 , 206 and DLB. 207 Moreover, accumulating evidence has suggested that elevated levels of wild-type α-synuclein are causative in the pathogenesis of synucleinopat

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