An engineered UGA suppressor tRNA gene for disease-agnostic AAV delivery.
Xu M, Liu H, Wang J, Vieira AFC, Zhou X, Liu N, Liang J, Du A, Chen X, Xing R, Yang Y, Gonzalez-Perez MP, Kumar V, Wang D
Nat Biotechnol. 2026 Jan 19. doi: 10.1038/s41587-025-02982-5.
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Plasmids from Article
| ID | Plasmid | Purpose |
|---|---|---|
| 263980 | pAAV.2x U6-UGA-sup-tRNAArg-R8.CB6-mCherry-RBG | AAV genome expressing 2x UGA suppressor tRNA driven by U6 promoter and mCherry |
| 263981 | pAAV. 2x human NFS-UGA-sup-tRNAArg-R8.CB6-mCherry. RBG | AAV genome expressing 2x UGA suppressor tRNA with human natural flanking sequence (NFS) and mCherry |
| 263982 | pAAV. 2x mouse NFS-UGA-sup-tRNAArg-R8.CB6-mCherry. RBG | AAV genome expressing 2x UGA suppressor tRNA with mouse natural flanking sequence (NFS) and mCherry |
| 263983 | pCMV-CymR-V5 | Mammalian expression plasmid for CymR |
| 263984 | pAAV. 2x human NFS-CuO-UGA-sup-tRNAArg-R8.CB6-mCherry. RBG (VG1) | AAV genome expressing 2x UGA suppressor tRNA with human natural flanking sequence (NFS) and CuO element and mCherry (vector genome design 1) |
| 263985 | pAAV. Human NFS-CuO/mouse NFS-CuO-UGA-sup-tRNAArg-R8.CB6-mCherry. RBG (VG2) | AAV genome expressing 2x UGA suppressor tRNA with mouse and mouse natural flanking sequence (NFS) and CuO element and mCherry (vector genome design 2) |
| 263986 | pAAV.2x human NFS-CuO-UGA-sup-tRNAArg-R8.CB6-mCherry. RBG (VG3) | AAV genome expressing 2x UGA suppressor tRNA with human natural flanking sequence (NFS) and CuO element and mCherry (vector genome design 3) |
| 263987 | pAAV.2x human NFS-CuO-UGA-sup-tRNAArg-R8.CB6-mCherry. RBG (VG4) | AAV genome expressing 2x UGA suppressor tRNA with human natural flanking sequence (NFS) and CuO element and mCherry (vector genome design 4) |
| 263989 | pAAV.CB6-CLuc-T2A-MeCP2 R168X-GLuc | Dual-luciferase PTC reporter with in-frame UGA premature termination codon to detect nonsense suppression; AAV genome expressing wild-type CLuc and GLuc with the T2A and Mecp2 R168X sequence context |
| 263990 | pAAV. Human NFS- tRT5.CB6-mCherry. RBG | AAV genome expressing UGA suppressor tRNA (tRT5, (Albers et al, Nature 2023)) with human natural flanking sequence (NFS) and mCherry |
| 263991 | pAAV. Human NFS-CuO tRT5.CB6-mCherry. RBG | AAV genome expressing CuO-UGA suppressor tRNA (tRT5, (Albers et al, Nature 2023)) with human natural flanking sequence (NFS) and mCherry |
| 263992 | pAAV. OptACE-tRNA-Arg-UGA-combo8.CB6-mCherry. RBG | AAV genome expressing the optimized UGA suppressor tRNA (OptACE-tRNA-Arg-UGA-combo8, (Porter et al, Nucleic Acids Res 2024)) and mCherry |
| 263993 | pAAV. CuO-OptACE-tRNA-Arg-UGA-combo8.CB6-mCherry. RBG | AAV genome expressing the optimized UGA suppressor tRNA (OptACE-tRNA-Arg-UGA-combo8, (Porter et al, Nucleic Acids Res 2024)) with a CuO insertion and mCherry |
| 263994 | pCMV-mIdua WT-Flag | Mammalian expression plasmid for wild-type mouse Idua-FLAG |
| 263995 | pCMV-mIdua W401X (TGA)-Flag | Mammalian expression plasmid for mouse Idua-FLAG with W401X (TGA) mutation |
| 263996 | pCMV-mIdua W401Q-Flag | Mammalian expression plasmid for mouse Idua-FLAG with W401Q mutation |
| 263997 | pCMV-mIdua W401R-Flag | Mammalian expression plasmid for mouse Idua-FLAG with W401R mutation |
| 263998 | pCMV-mIdua W401D-Flag | Mammalian expression plasmid for mouse Idua-FLAG with W401D mutation |
| 263999 | pLenti-CSCGW2.human NFS-CuO-UGA-sup-tRNAArg-R8.CMV-EGFP | Lentiviral plasmid expressing UGA suppressor tRNA with human natural flanking sequence (NFS) and CuO element and EGFP |