Gene therapy for cystic fibrosis
| Problems | Solutions |
|---|---|
| Inefficient gene delivery | Improved vectors and delivery devices |
| Inflammatory response to vector | Engineering and formulation of viral vectors and liposomes |
| Lack of cell specific targeting | Viral subtypes |
| Receptor mediated cell targeting | |
| Transient expression | Episomal maintenance |
| Safe site integration | |
| Transfect stem cells | |
| Non-physiological expression | Genomic context vectors |
| Cell specific expression |