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| 001 | 276937 | ||
| 005 | 20240229155007.0 | ||
| 024 | 7 | _ | |a 10.1016/j.ymthe.2023.06.012 |2 doi |
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| 041 | _ | _ | |a English |
| 082 | _ | _ | |a 610 |
| 100 | 1 | _ | |a Toms, Maria |b 0 |
| 245 | _ | _ | |a Successful large gene augmentation of USH2A with non-viral episomal vectors. |
| 260 | _ | _ | |a New York, NY |c 2023 |b Nature Publ. Group |
| 336 | 7 | _ | |a article |2 DRIVER |
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| 500 | _ | _ | |a 2023 Sep 6;31(9):2755-2766 |
| 520 | _ | _ | |a USH2A mutations are a common cause of autosomal recessive retinitis pigmentosa (RP) and Usher syndrome, for which there are currently no approved treatments. Gene augmentation is a valuable therapeutic strategy for treating many inherited retinal diseases, however conventional adeno-associated virus (AAV) gene therapy cannot accommodate cDNAs exceeding 4.7kb, such as the 15.6kb-long USH2A coding sequence. In the present study, we adopted an alternative strategy to successfully generate scaffold/matrix attachment region (S/MAR) DNA plasmid vectors containing the full-length human USH2A coding sequence, a GFP reporter gene and a ubiquitous promoter (CMV or CAG), reaching a size of approximately 23kb. We assessed the vectors in transfected HEK-293 cells and USH2A patient-derived dermal fibroblasts, in addition to ush2au507 zebrafish microinjected with the vector at the one-cell stage. pS/MAR-USH2A vectors drove persistent transgene expression in patient fibroblasts with restoration of usherin. Twelve months of GFP expression was detected in the photoreceptor cells, with rescue of Usher 2 complex localisation in the photoreceptors of ush2au507 zebrafish retina injected with pS/MAR-USH2A. To our knowledge, this is the first reported vector which can be used to express full-length usherin with functional rescue. S/MAR DNA vectors have shown promise as a novel non-viral retinal gene therapy, warranting further translational development. |
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| 700 | 1 | _ | |a Toualbi, Lyes |b 1 |
| 700 | 1 | _ | |a Almeida, Patrick V |0 P:(DE-HGF)0 |b 2 |
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| 700 | 1 | _ | |a Moosajee, Mariya |b 4 |
| 773 | _ | _ | |a 10.1016/j.ymthe.2023.06.012 |g p. S1525001623003234 |0 PERI:(DE-600)2001818-6 |n 9 |p 2755-2766 |t Molecular therapy |v 31 |y 2023 |x 1525-0016 |
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