Elizabeth Simpson Lab: First pilot study of intravenous rAAV-PAX6 gene therapy increases retinal-ganglion-cell-layer thickness and Notch1 transcription in a mouse model of aniridia
Djaksigulova D, Kaad SG, Korecki AJ, Lam SL, Lengyell TC, Simpson EM
Gene Therapy, 2026.
Plasmids from Article
| ID | Plasmid | Purpose |
|---|---|---|
| 246091 | pEMS2244 | For preclinical gene therapy: ss-AAV expression of N-term FLAG-tagged human PAX6 under control of the smCBA promoter; contains WPRE. |
| 246092 | pEMS2393 | For preclinical gene therapy: ss-AAV expression of N-term FLAG-tagged human PAX6 under control of Ple331 (PAX6 MiniPromoter); contains WPRE. |