TABLE 2.
Clinical trials involving AAV vectors
| Condition | Gene product(s) | Phase |
|---|---|---|
| CF | CFTR | I/II |
| Canavan's disease | Aspartoacylase | I |
| Parkinson's disease | GAD65, GAD65, AADC, neurturin | I |
| Alzheimer's disease | Beta nerve growth factor | I |
| Alpha-1-antitrypsin deficiency | AAT | I |
| Arthritis | TNFR:Fc | I |
| Leber congenital amaurosis | RPE65 | I |
| Hemophilia B | Factor IX | I |
| Late infantile neuronal lipofuscinosis | CLN2 | I |
| Muscular dystrophy | Minidystrophin, sarcoglycan | I |
| Heart failure | SERCA-2a | I |
| Prostate cancer | Granulocyte-macrophage colony-stimulating factory | I/II/III |
| Epilepsy | Neuropeptide Y | I |