All-in-one AAV-delivered epigenome-editing platform: proof-of-concept and therapeutic implications for neurodegenerative disorders.
Kantor B, Odonovan B, Rittiner J, Hodgson D, Lindner N, Guerrero S, Dong W, Zhang A, Chiba-Falek O
bioRxiv [Preprint]. 2024 May 19:2023.04.14.536951. doi: 10.1101/2023.04.14.536951.
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Plasmids from Article
| ID | Plasmid | Purpose |
|---|---|---|
| 223138 | pBK1289-AAV-dCjCas9 | AAV backbone for dCjCas9 (endonuclease dead Cas9 from Campylobacter jejuni) with Cj-gRNA scaffold |
| 223139 | pBK1290-AAV-dSaCas9 | AAV backbone for dSaCas9 (endonuclease dead Cas9 from Staphylococcus aureus) and Sa-gRNA Scaffold |
| 223140 | pBK1295-AAV-EFSNC-dCjCas9-HP1a(72-177) | Expression of truncated HP1a with dCjCas9 and empty gRNA scaffold |
| 223141 | pBK1296-AAV-EFSNC-dCjCas9-HP1aNH(115-177) | Expression of truncated HP1a with dCjCas9 and empty gRNA scaffold |
| 223142 | pBK1297-AAV-EFSNC-dCjCas9-HP1b(79-173) | Expression of truncated HP1b with dCjCas9 and empty gRNA scaffold |
| 223143 | pBK1298-AAV-EFSNC-dCjCas9-HP1bNH(111-173) | Expression of truncated HP1b with dCjCas9 and empty gRNA scaffold |
| 223144 | pBK1299-AAV-EFSNC-dCjCas9-MBD1(529-592) | Expression of truncated MBD1 with dCjCas9 and empty gRNA scaffold |
| 223145 | pBK1300-AAV-EFSNC-dCjCas9-MBD2(176-231) | Expression of truncated MBD2 with dCjCas9 and empty gRNA scaffold |
| 223146 | pBK1301-AAV-EFSNC-dCjCas9-NIPP1(143-224) | Expression of truncated NIPP1 with dCjCas9 and empty gRNA scaffold |
| 223147 | pBK1302-AAV-EFSNC-dCjCas9-KRAB | Expression of KRAB with dCjCas9 and empty gRNA scaffold |
| 223148 | pBK1303-AAV-EFSNC-dCjCas9-MECP2(204-310) | Expression of truncated MECP2 with dCjCas9 and empty gRNA scaffold |
| 223149 | pBK1304-AAV-EFSNC-dCjCas9-KRAB-MECP2 | Expression of KRAB and truncated MECP2 with dCjCas9 and empty gRNA scaffold |
| 223150 | pBK1305-AAV-EFSNC-dSaCas9-HP1a(72-177) | Expression of truncated HP1a with dSaCas9 and empty gRNA scaffold |
| 223151 | pBK1306-AAV-EFSNC-dSaCas9-HP1aNH(115-177) | Expression of truncated HP1a with dSaCas9 and empty gRNA scaffold |
| 223152 | pBK1307-AAV-EFSNC-dSaCas9-HP1b(79-173) | Expression of truncated HP1b with dSaCas9 and empty gRNA scaffold |
| 223153 | pBK1308-AAV-EFSNC-dSaCas9-HP1bNH(111-173) | Expression of truncated HP1b with dSaCas9 and empty gRNA scaffold |
| 223154 | pBK1309-AAV-EFSNC-dSaCas9-MBD1(529-592) | Expression of truncated MBD1 with dSaCas9 and empty gRNA scaffold |
| 223155 | pBK1310-AAV-EFSNC-dSaCas9-MBD2(176-231) | Expression of truncated MBD2 with dSaCas9 and empty gRNA scaffold |
| 223156 | pBK1311-AAV-EFSNC-dSaCas9-NIPP1(143-224) | Expression of truncated NIPP1 with dSaCas9 and empty gRNA scaffold |
| 223157 | pBK1312-AAV-EFSNC-dSaCas9-KRAB | Expression of KRAB with dSaCas9 and empty gRNA scaffold |
| 223158 | pBK1313-AAV-EFSNC-dSaCas9-MECP2(204-310) | Expression of truncated MECP2 with dSaCas9 and empty gRNA scaffold |
| 223159 | pBK1314-AAV-EFSNC-dSaCas9-KRAB-MECP2 | Expression of KRAB and truncated MECP2 with dSaCas9 and empty gRNA scaffold |
| 223160 | pBK1340-LV-DUAL-dGFP-NanoLuc-Puro | Expression of destabilized GFP (dGFP) and NanoLuc for shortened in vivo half-life |
| 223161 | pBK1861-AAV-EFSNC-dSaCas9-KRAB-MECP2(APOE-gRNA1) | Expression of KRAB and truncated MECP2 with dSaCas9 and gRNA1 targeting human/mouse APOE |
| 223162 | pBK1876-AAV-EFSNC-dSaCas9-KRAB-MECP2(APOE-gRNA2) | Expression of KRAB and truncated MECP2 with dSaCas9 and gRNA2 targeting human/mouse APOE |
| 223163 | pBK2043-AAV-2xSp1-2xNFkB-EFSNC-dSaCas9 | 2nd gen. AAV backbone for dSaCas9 (endonuclease dead Cas9 from Staphylococcus aureus) and Sa-gRNA Scaffold |
| 223164 | pBK2044-AAV-2xSp1-2xNFkB-EFSNC-dSaCas9-KRAB-MECP2 | 2nd gen. vector for expression of KRAB and truncated MECP2 with dSaCas9 and empty gRNA scaffold |
| 223165 | pBK2045-AAV-2xSp1-2xNFkB-EFSNC-dSaCas9-KRAB-MECP2(CMV-gRNA1) | 2nd gen. vector for expression of KRAB and truncated MECP2 with dSaCas9 and gRNA1 targeting CMV |
| 223166 | pBK2046-AAV-2xSp1-2xNFkB-EFSNC-dSaCas9-KRAB-MECP2(APOE-gRNA1) | 2nd gen. vector for expression of KRAB and truncated MECP2 with dSaCas9 and gRNA1 targeting human/mouse APOE |
| 223167 | pBK2047-AAV-2xSp1-2xNFkB-EFSNC-dSaCas9-KRAB-MECP2(APOE-gRNA2) | 2nd gen. vector for expression of KRAB and truncated MECP2 with dSaCas9 and gRNA2 targeting human/mouse APOE |