Potential complications of gene therapy
| General . | AAV-FIX specific* . | Potential solutions . | References . |
|---|---|---|---|
| Gene silencing | — | — | — |
| Genotoxicity | — | — | — |
| Immunotoxicity | Neutralizing antibodies that block delivery | Current; exclude subjects with neutralizing antibodies | — |
| Plasmapheresis? | 23 | ||
| Pharmacologic reduction of antibodies? | 24, 25 | ||
| T-cell response to capsid | Prednisolone | 2 | |
| Phenotoxicity | — | — | |
| Vertical transmission | Risk of germline transmission | Barrier birth control until semen is negative by PCR | 14 |
| Horizontal transmission | — | — | — |
| General . | AAV-FIX specific* . | Potential solutions . | References . |
|---|---|---|---|
| Gene silencing | — | — | — |
| Genotoxicity | — | — | — |
| Immunotoxicity | Neutralizing antibodies that block delivery | Current; exclude subjects with neutralizing antibodies | — |
| Plasmapheresis? | 23 | ||
| Pharmacologic reduction of antibodies? | 24, 25 | ||
| T-cell response to capsid | Prednisolone | 2 | |
| Phenotoxicity | — | — | |
| Vertical transmission | Risk of germline transmission | Barrier birth control until semen is negative by PCR | 14 |
| Horizontal transmission | — | — | — |
— indicates not applicable.
Based on observations in clinical trials.