Table 1.
Group name | Tmc1 genotype | Viral vector name | Titer (vg/ml) |
---|---|---|---|
WT control | +/+ | None | n/a |
AAV2/9 eGFP | +/+ | AAV2/9.miSafe.eGFP | 3.9 × 1013 |
AAV2/2 eGFP | +/+ | AAV2/2.miSafe.eGFP | 4.2 × 1012 |
AAV2/2 eGFP w/WPRE | +/+ | AAV2/2.miSafe.eGFP.WPRE | 5.8 × 1012 |
Bth/+ control | Bth/+ | None | n/a |
AAV2/9 replacement alone | Bth/+ | AAV2/9.miSafe.mTmc1 | 1.8× 1013 |
AAV2/9 RNAi + replacement | Bth/+ | AAV2/9.miTmc1D11.mTmc1 | 2.1 × 1013 |
AAV2/2 RNAi + replacement | Bth/+ | AAV2/2.miTmc1D11.mTmc1 | 4.0 × 1012 |
AAV2/2 RNAi + replacement w/WPRE | Bth/+ | AAV2/2.miTmc1D11.mTmc1.WPRE | 4.8 × 1012 |
Bth/+ + AAV2/2 eGFP | Bth/+ | AAV2/2.miSafe.eGFP | 4.2 × 1012 |
For readability, a shorthand description of each experimental group is used throughout this study.