| A regulatory toolbox of MiniPromoters to drive selective expression in the brain. | Portales-Casamar E, Swanson DJ, Liu L, de Leeuw CN, Banks KG, Ho Sui SJ, Fulton DL, Ali J, Amirabbasi M, Arenillas DJ, Babyak N, Black SF, Bonaguro RJ, Brauer E, Candido TR, Castellarin M, Chen J, Chen Y, Cheng JC, Chopra V, Docking TR, Dreolini L, D'Souza CA, Flynn EK, Glenn R, Hatakka K, Hearty TG, Imanian B, Jiang S, Khorasan-zadeh S, Komljenovic I, Laprise S, Liao NY, Lim JS, Lithwick S, Liu F, Liu J, Lu M, McConechy M, McLeod AJ, Milisavljevic M, Mis J, O'Connor K, Palma B, Palmquist DL, Schmouth JF, Swanson MI, Tam B, Ticoll A, Turner JL, Varhol R, Vermeulen J, Watkins RF, Wilson G, Wong BK, Wong SH, Wong TY, Yang GS, Ypsilanti AR, Jones SJ, Holt RA, Goldowitz D, Wasserman WW, Simpson EM | Proc Natl Acad Sci U S A. 2010 Sep 21. 107(38):16589-94. |
| Ferroportin-mediated iron export from vascular endothelial cells in retina and brain. | Baumann BH, Shu W, Song Y, Simpson EM, Lakhal-Littleton S, Dunaief JL | Exp Eye Res. 2019 Jul 16:107728. doi: 10.1016/j.exer.2019.107728. |
| Human MiniPromoters for ocular-rAAV expression in ON bipolar, cone, corneal, endothelial, Muller glial, and PAX6 cells. | Korecki AJ, Cueva-Vargas JL, Fornes O, Agostinone J, Farkas RA, Hickmott JW, Lam SL, Mathelier A, Zhou M, Wasserman WW, Di Polo A, Simpson EM | Gene Ther. 2021 Feb 2. pii: 10.1038/s41434-021-00227-z. doi: 10.1038/s41434-021-00227-z. |
| Intracerebroventricular Administration of AAV9-PHP.B SYN1-EmGFP Induces Widespread Transgene Expression in the Mouse and Monkey CNS. | Galvan A, Petkau TL, Hill AM, Korecki AJ, Lu G, Choi D, Rahman K, Simpson E, Leavitt BR, Smith Y | Hum Gene Ther. 2021 Apr 16. doi: 10.1089/hum.2020.301. |
| New MiniPromoter Ple345 (NEFL) Drives Strong and Specific Expression in Retinal Ganglion Cells of Mouse and Primate Retina. | Simpson E, Korecki AJ, Fornes O, McGill TJ, Cueva-Vargas JL, Agostinone J, Farkas RA, Hickmott JW, Lam SL, Mathelier A, Renner LM, Stoddard J, Zhou M, Di Polo A, Neuringer M, Wasserman WW | Hum Gene Ther. 2018 Jul 31. doi: 10.1089/hum.2018.118. |
| New MiniPromoter Ple389 (ADORA2A) drives selective expression in medium spiny neurons in mice and non-human primates. | de Moura Gomes A, L Petkau T, J Korecki A, Fornes O, Galvan A, Lu G, M Hill A, Ling Lam S, Yao A, A Farkas R, W Wasserman W, Smith Y, M Simpson E, R Leavitt B | Sci Rep. 2024 Nov 15;14(1):28194. doi: 10.1038/s41598-024-79004-y. |
| OnTarget: in silico design of MiniPromoters for targeted delivery of expression. | Fornes O, Av-Shalom TV, Korecki AJ, Farkas RA, Arenillas DJ, Mathelier A, Simpson EM, Wasserman WW | Nucleic Acids Res. 2023 Jul 5;51(W1):W379-W386. doi: 10.1093/nar/gkad375. |
| PAX6 MiniPromoters drive restricted expression from rAAV in the adult mouse retina. | Hickmott JW, Chen CY, Arenillas DJ, Korecki AJ, Lam SL, Molday LL, Bonaguro RJ, Zhou M, Chou AY, Mathelier A, Boye SL, Hauswirth WW, Molday RS, Wasserman WW, Simpson EM | Mol Ther Methods Clin Dev. 2016 Aug 10;3:16051. doi: 10.1038/mtm.2016.51. eCollection 2016. |
| rAAV-compatible MiniPromoters for restricted expression in the brain and eye. | de Leeuw CN, Korecki AJ, Berry GE, Hickmott JW, Lam SL, Lengyell TC, Bonaguro RJ, Borretta LJ, Chopra V, Chou AY, D'Souza CA, Kaspieva O, Laprise S, McInerny SC, Portales-Casamar E, Swanson-Newman MI, Wong K, Yang GS, Zhou M, Jones SJ, Holt RA, Asokan A, Goldowitz D, Wasserman WW, Simpson EM | Mol Brain. 2016 May 10;9(1):52. doi: 10.1186/s13041-016-0232-4. |
| rAAV-PHP.B escapes the mouse eye and causes lethality whereas rAAV9 can transduce aniridic corneal limbal stem cells without lethality. | Mirjalili Mohanna SZ, Korecki AJ, Simpson EM | Gene Ther. 2023 Apr 19. doi: 10.1038/s41434-023-00400-6. |
