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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 Ther. 2026 Mar 9. doi: 10.1038/s41434-026-00605-5. (Link opens in a new window) PubMed (Link opens in a new window) Article

Plasmids from Article

ID Plasmid Purpose
246091pEMS2244For preclinical gene therapy: ss-AAV expression of N-term FLAG-tagged human PAX6 under control of the smCBA promoter; contains WPRE.
246092pEMS2393For preclinical gene therapy: ss-AAV expression of N-term FLAG-tagged human PAX6 under control of Ple331 (PAX6 MiniPromoter); contains WPRE.

Antibodies from Article