Cas9 nickase-mediated contraction of CAG/CTG repeats at multiple disease loci
Alvaro Murillo, Melanie Alpaugh, Meghan Larin, Emma L. Randall, Laura Heraty, Ruban Rex Durairaj, Alys N. Aston, Alysha S. Taylor, Alex Mas Monteys, Nina Stöberl, Aeverie E. R. Heuchan, Pascale Aeschlimann, Soumyasree Bhattacharyya, Nicholas D. Allen, Jack Puymirat, Beverly L. Davidson, Francesca Cicchetti, Mariah Lelos, Vincent Dion
bioRxiv
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Article
Plasmids from Article
| ID | Plasmid | Purpose |
|---|---|---|
| 216732 | pLV(gRNA)-CMV-eGFP-U6(sgCTG) | Contains a eGFP gene along with a U6 promoter driving the sgCTG (target sequence: (CUG)6). Used with the HD iPSC-derived astrocytes |
| 216733 | pLenti-U6-sgCTG-CAG-Cas9D10A nickase-Blast | Expresses SpCas9-D10A nickase under a CAG promoter together with a blasticidin resistance gene, along with a U6 promoter driving the sgCTG (target sequence: (CUG)6). Suitable for lentivirus packaging. |
| 216734 | pAAV-U6-sgCTG-CMV-GFP | AAV plasmid containing sgCTG (target sequence: (CUG)6) driven by a U6 promoter and eGFP under the control of the CMV promoter. Used for in vivo experiments. |
| 216735 | pX551-miniCMV-SpCas9D10A nickase | Expresses SpCas9-D10A nickase from a miniCMV promoter. For AAV packaging. Derived from pX551-miniCMV-SpCas9, which was a gift from Alex Hewitt (Addgene #107031) |
| 216736 | pAAV-nEF-SpCas9D10A nickase | Expresses SpCas9-D10A nickase from a nEF promoter. For AAV packaging. Derived from pAAV-nEF-SpCas9 (Addgene #87115) |
| 216737 | pAAV-CMV-SpCas9D10A nickase | Expresses SpCas9-D10A nickase from a CMVd1 promoter. For AAV packaging. Derived from pAAV-CMV-SpCas9 (Addgene #113034) |