Brainwide silencing of prion protein by AAV-mediated delivery of an engineered compact epigenetic editor.
Neumann EN, Bertozzi TM, Wu E, Serack F, Harvey JW, Brauer PP, Pirtle CP, Coffey A, Howard M, Kamath N, Lenz K, Guzman K, Raymond MH, Khalil AS, Deverman BE, Minikel EV, Vallabh SM, Weissman JS
Science. 2024 Jun 28;384(6703):ado7082. doi: 10.1126/science.ado7082. Epub 2024 Jun 28.
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Plasmids from Article
| ID | Plasmid | Purpose |
|---|---|---|
| 220834 | pENN226_CRISPRcharm | CRISPRcharm expression in mammalian cells |
| 220835 | pENN456_TALEcharm_Kv2_MmT8x | TALEcharm expression in mammalian cells |
| 220836 | pENN491_TALEcharm_MmT8x | TALEcharm Kv2 expression in mammalian cells; targets Prnp |
| 220837 | pENN496_miniCRISPRcharm_dSaCas9 | mini CRISPRcharm expression in mammalian cells |
| 220838 | pENN239_CRISPRcharm_Kv1 | CRISPRcharm Kv1 expression in mammalian cells |
| 220839 | pENN236_CRISPRcharm_Kv2 | CRISPRcharm Kv2 expression in mammalian cells |
| 220840 | pTMB301_ZFcharm_Kv1_Prnp_SPM | AAV genome expressing ZFcharm Kv1 targeting Prnp; single perfect match self-silencing binding site |
| 220841 | pTMB303_ZFcharm_Kv1_Prnp_SMM | AAV genome expressing ZFcharm Kv1 targeting Prnp; single mismatch self-silencing binding site |
| 220842 | pTMB304_ZFcharm_Kv1_Prnp_SCR | AAV genome expressing ZFcharm Kv1 targeting Prnp; scrambled self-silencing binding site |
| 220843 | pTMB340_ZFcharm_Kv1_Prnp_DPM | AAV genome expressing ZFcharm Kv1 targeting Prnp; double perfect match self-silencing binding site |
| 220844 | pTMB341_ZFcharm_Prnp_SCR | AAV genome expressing ZFcharm targeting Prnp; scrambled self-silencing binding site |
| 220845 | pTMB398_ZFcharm_Prnp_SMM | AAV genome expressing ZFcharm targeting Prnp; single mismatch self-silencing binding site |
| 220846 | pTMB399_ZFcharm_Prnp_SPM | AAV genome expressing ZFcharm targeting Prnp; single perfect match self-silencing binding site |
| 220847 | pTMB400_ZFcharm_Prnp_DPM | AAV genome expressing ZFcharm targeting Prnp; double perfect match self-silencing binding site |
| 220848 | pTMB352_ZFcharm_Kv1_split-inteins | Lentiviral vector expressing ZFcharm Kv2 fused to ZF3 and MmZFP_81187 via split Npu inteins; perfect match ZF3 binding site located upstream of pEFS |