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Downregulation of SNCA Expression by Targeted Editing of DNA Methylation: A Potential Strategy for Precision Therapy in PD.

Kantor B, Tagliafierro L, Gu J, Zamora ME, Ilich E, Grenier C, Huang ZY, Murphy S, Chiba-Falek O
Mol Ther. 2018 Nov 7;26(11):2638-2649. doi: 10.1016/j.ymthe.2018.08.019. Epub 2018 Aug 29. (Link opens in a new window) PubMed (Link opens in a new window) Article

Plasmids from Article

ID Plasmid Purpose
239792pBK1838pAAV-CMV-SNCAintron1-AlphaSyn[A53T]-WPRE-SV40pA (PD-INDUCER)
239793pBK1575pLV-U6-[empty]-EFSNC-dSaCas9-[empty linker]-P2A-Puro-WPRE
239794pBK1579pLV-U6-[empty]-EFSNC-dSaCas9-DNMT3a-P2A-Puro-WPRE
239796pBK1576pLV-U6-[empty]-EFSNC-dSaCas9-KRAB-MeCp2-P2A-Puro-WPRE (no gRNA vector)
239797pBK1841pLV-U6-[SNCAintron1 gRNA 1]-EFSNC-dSaCas9-KRAB-MeCp2-P2A-Puro-WPRE (THERAPEUTIC VECTOR)
239839pBK546pLV-U6-[hSNCA gRNA 4]-EFSNC-dSpCas9-DNMT3a-P2A-PURO-WPRE
239840pBK18377. BK1837- pLV-CMV-SNCAintron1-AlphaSyn-d2EGFP-P2A-NLuc-SV40-Puro-WPRE

Antibodies from Article