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Development of a translatable gene augmentation therapy for CNGB1-retinitis pigmentosa.
Occelli LM, Zobel L, Stoddard J, Wagner J, Pasmanter N, Querubin J, Renner LM, Reynaga R, Winkler PA, Sun K, Marinho LFLP, O'Riordan CR, Frederick A, Lauer A, Tsang SH, Hauswirth WW, McGill TJ, Neuringer M, Michalakis S, Petersen-Jones SM. Occelli LM, et al. Among authors: querubin j. Mol Ther. 2023 Jul 5;31(7):2028-2041. doi: 10.1016/j.ymthe.2023.04.005. Epub 2023 Apr 13. Mol Ther. 2023. PMID: 37056049 Free article.
Gene Therapy in a Large Animal Model of PDE6A-Retinitis Pigmentosa.
Mowat FM, Occelli LM, Bartoe JT, Gervais KJ, Bruewer AR, Querubin J, Dinculescu A, Boye SL, Hauswirth WW, Petersen-Jones SM. Mowat FM, et al. Among authors: querubin j. Front Neurosci. 2017 Jun 20;11:342. doi: 10.3389/fnins.2017.00342. eCollection 2017. Front Neurosci. 2017. PMID: 28676737 Free PMC article.
Residual rod function in CNGB1 mutant dogs.
Petersen-Jones SM, Pasmanter N, Occelli LM, Querubin JR, Winkler PA. Petersen-Jones SM, et al. Among authors: querubin jr. Doc Ophthalmol. 2022 Dec;145(3):237-246. doi: 10.1007/s10633-022-09899-3. Epub 2022 Sep 15. Doc Ophthalmol. 2022. PMID: 36107278
A large animal model of RDH5-associated retinopathy recapitulates important features of the human phenotype.
Occelli LM, Daruwalla A, De Silva SR, Winkler PA, Sun K, Pasmanter N, Minella A, Querubin J, Lyons LA; 99 Lives Consortium; Robson AG, Heon E, Michaelides M, Webster AR, Palczewski K, Vincent A, Mahroo OA, Kiser PD, Petersen-Jones SM. Occelli LM, et al. Among authors: querubin j. Hum Mol Genet. 2022 Apr 22;31(8):1263-1277. doi: 10.1093/hmg/ddab316. Hum Mol Genet. 2022. PMID: 34726233 Free PMC article.
Early-Onset Progressive Degeneration of the Area Centralis in RPE65-Deficient Dogs.
Mowat FM, Gervais KJ, Occelli LM, Annear MJ, Querubin J, Bainbridge JW, Smith AJ, Ali RR, Petersen-Jones SM. Mowat FM, et al. Among authors: querubin j. Invest Ophthalmol Vis Sci. 2017 Jun 1;58(7):3268-3277. doi: 10.1167/iovs.17-21930. Invest Ophthalmol Vis Sci. 2017. PMID: 28662231 Free article.
16 results