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| Detail |
|---|
| 1. Inadequate gene transfer to the target cell – issues related to dose, vector, mode of delivery |
| 2. Elimination by the immune system – need for immunosuppression in some cases |
| 3. Liver sequestration |
| 4. Off-target effects |
| 5. Episomal degradation – long term gene expression desirable in heart failure patients |
| 6. Limited scale up capacity of gene therapy medicinal products |
