Table 1 Comparison of gene therapy results obtained by the canalostomy injection method in the current study and two published studies.
This study | Crispino et al. | Isgrig et al. | |
|---|---|---|---|
Viral subtype | AAV1 | BAAV | AAV8 |
Target gene | Kcne1 | Gjb6 | Whirlin |
Promoter used | CBA | CMV | CMV |
Virus injection time | P0–P2 | P4 | P4 |
Injection route | PSCC | LSCC | PSCC |
Repeated with different batches of viral solution | Yes | Unclear | Yes |
Dose | Low-dosage group: 0.5–1.0 µL; high-dosage group: 1.5–2.0 µL | 1.0 µL | 0.98 µL |
Targeted cells | Marginal cells and vestibular dark cells | Non-sensory cells of the sensory epithelium and fibrocytes in the supra-strial zone | Cochlear and vestibular hair cells |
Ectopic expression of therapeutic gene | Yes and extensive | No | No |
Expression of GFP | Yes | Yes | Yes |
Hearing improvement | 0–30 dB SPL at 8–18 kHz in the low-dosage group; 40–70 dB SPL at 8–18 kHz in the high-dosage group | Slight improvement (0–10 dB SPL) at 32 kHz in Cx30∆/∆ mice; no improvement in Cx30−/− mice | 0–40 dB SPL at 8 kHz |
Vestibular function assessment | Apparent improvement in vestibular function as measured by circling, rotarod, and swimming test | Unclear | Apparent improvement in vestibular function as measured by circling, rotarod, swimming, and vestibular-evoked potential test |
Long-term treatment effect | At least for 5 months | Unclear | Less than 4 months |
Intracellular trafficking | At the apical membrane of marginal cells and vestibular dark cells | In the cell membrane of supporting cells | At the top of cilia bundles of hair cells |
Secondary outcomes | Improvement in birth and litter survival rates | Unclear | Unclear |
