A gene therapy for inherited blindness using dCas9-VPR-mediated transcriptional activation.
Bohm S, Splith V, Riedmayr LM, Rotzer RD, Gasparoni G, Nordstrom KJV, Wagner JE, Hinrichsmeyer KS, Walter J, Wahl-Schott C, Fenske S, Biel M, Michalakis S, Becirovic E
Sci Adv. 2020 Aug 19;6(34):eaba5614. doi: 10.1126/sciadv.aba5614. eCollection 2020 Aug.
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Plasmids from Article
| ID | Plasmid | Purpose |
|---|---|---|
| 165450 | pAAV-3xsgRNA_Opn1mw-RHO-Cas9N-IntN-polyA | Express N-terminal part of split dCas9-VPR and 3 sgRNAs targeting murine Opn1mw promoter |
| 166692 | pAAV-RHO-IntC-dCas9C-VPR-polyA | Express C-terminal part of split dCas9-VPR |
| 166693 | PB-TRE-dCas9-VPR-pA-3xsgRNA-EF1a-Tet.O-T2A-PuroR-polyA | Express dCas9-VPR in a doxycyclin-inducable system and 3 sgRNAs targeting the murine Cnga1 promoter. Can be stably integrated into the genome via the PiggyBac Transposon system. |