Skip to main content
This website uses cookies to ensure you get the best experience. By continuing to use this site, you agree to the use of cookies.

Please note: Your browser does not support the features used on Addgene's website. You may not be able to create an account or request plasmids through this website until you upgrade your browser. Learn more

Please note: Your browser does not fully support some of the features used on Addgene's website. If you run into any problems registering, depositing, or ordering please contact us at [email protected]. Learn more

Addgene
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. PubMed Article

Plasmids from Article

ID Plasmid Purpose
165450pAAV-3xsgRNA_Opn1mw-RHO-Cas9N-IntN-polyAExpress N-terminal part of split dCas9-VPR and 3 sgRNAs targeting murine Opn1mw promoter
166692pAAV-RHO-IntC-dCas9C-VPR-polyAExpress C-terminal part of split dCas9-VPR
166693PB-TRE-dCas9-VPR-pA-3xsgRNA-EF1a-Tet.O-T2A-PuroR-polyAExpress 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.

Antibodies from Article