Table 1 Clinical trials with AAV-based vectors [22].
From: The state of the art of adeno-associated virus-based vectors in gene therapy
| Category/Disease | Clinical trial | ||||
|---|---|---|---|---|---|
| Phase I O/C* | Phase I/II O/C | Phase II O/C | Phase III O/C | Total | |
| Infeccious | |||||
| HIV vaccine | 2/0 | 0/0 | 0/0 | 0/0 | 2 |
| Cancer | |||||
| Malignant melanoma | 1/0 | 0/0 | 0/0 | 0/0 | 1 |
| Prostate cancer | 0/1 | 2/1 | 0/0 | 4/0 | 8 |
| Hormone refractory prostate cancer | 1/0 | 0/0 | 0/0 | 0/0 | 1 |
| Metastatic prostate cancer | 0/0 | 0/0 | 0/0 | 1/0 | 1 |
| Genetic | |||||
| Lipoprotein lipase deficience | 0/0 | 1/0 | 0/0 | 0/0 | 1 |
| Haemophilia B | 1/2 | 0/0 | 0/0 | 0/0 | 3 |
| Early onset retinal degeneration | 3/0 | 0/0 | 0/0 | 0/0 | 3 |
| Cystic fibrosis | 2/3 | 1/1 | 0/2 | 0/0 | 9 |
| Muscular dystrophy | 1/0 | 0/0 | 0/0 | 0/0 | 1 |
| Canavan disease | 1/0 | 0/0 | 0/0 | 0/0 | 1 |
| Duchenne muscular dystrophy | 1/1 | 0/0 | 0/0 | 0/0 | 2 |
| Limb Girdle muscular dystrophy | 1/0 | 0/0 | 0/0 | 0/0 | 1 |
| Amyotrophic lateral sclerosis | 1**/0 | 0/0 | 0/0 | 0/0 | 1 |
| Inherited autosomal recessive alpha-1-antitrypsin deficiency | 2/0 | 0/0 | 0/0 | 0/0 | 2 |
| Late infantile neuronal ceroid lipofuscinosis | 1/0 | 0/0 | 0/0 | 0/0 | 1 |
| Neurological | |||||
| Parkinson's disease | 1/1 | 0/0 | 1/0 | 0/0 | 3 |
| Alzheimer's disease | 0/0 | 0/1 | 0/0 | 0/0 | 1 |
| Epilepsy | 1/0 | 0/0 | 0/0 | 0/0 | 1 |
| Cardiovascular | |||||
| Heart failure | 2/0 | 0/0 | 0/0 | 0/0 | 2 |
| Others | |||||
| Rheumatoid arthritis | 2/0 | 0/0 | 0/0 | 0/0 | 2 |
| TOTAL | 24/8 | 4/3 | 1/2 | 5/0 | 47 |