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Circuit-specific gene therapy reverses core symptoms in a primate Parkinson's disease model.
Chen Y, Hong Z, Wang J, Liu K, Liu J, Lin J, Feng S, Zhang T, Shan L, Liu T, Guo P, Lin Y, Li T, Chen Q, Jiang X, Li A, Li X, Li Y, Wilde JJ, Bao J, Dai J, Lu Z
Cell. 2023 Nov 22;186(24):5394-5410.e18. doi: 10.1016/j.cell.2023.10.004. Epub 2023 Nov 2.
PubMed Article

Plasmids from Article

ID Plasmid Purpose
213968AAV8R-12 CapsidAAV packaging vector carrying AAV2 rep gene and Cap8R modified AAV8 capsid coding sequence
213969pAAV-pCALM1-sFLEx-HA-SpCas9-miniU6-sgRNAShank3AAV vector for encoding SpCas9 driven by pCALM1 promoter targeting Shank3 locus in the presence of Cre recombinase
213970pAAV-G88P7-rM3Ds-2A-EYFPAAV vector with G88P7 promoter in order to induce robust gene expression in MSNs

Antibodies from Article