Table 1 AAV in clinical trials
From: Improving clinical efficacy of adeno associated vectors by rational capsid bioengineering
AAV serotype | Disease | Administration route | Trial Number (www.clinicaltrial.gov) |
|---|---|---|---|
rh.10 | Late Infantile Neuronal Ceroid Lipofuscinosis | Intracranial | NCT01161576 |
1 | Pompe disease | Intradiaphragmatic | NCT00976352 |
2 | Leber Congenital Amaurosis | Subretinal | NCT00643747; NCT00516477; NCT00999609; NCT00749957 |
2 | Retinal disease (MERTK mutation) | Subretinal | NCT01482195 |
2.8 | Hemophilia B | Intramuscular, hepatic, intravenous | NCT00515710; NCT01687608; NCT00979238; NCT01620801 |
2 | Idiopathic Parkinson’s Disease | Intracranial | NCT00985517 |
1,2 | Alpha-1 Antitrypsin Deficiency | Intramuscular | NCT00430768; NCT01054339; NCT00377416 |
1 | Lipoprotein Lipase Deficiency | Intramuscular | NCT00891306; NCT01109498 |
2.5 | Duchenne Muscular Dystrophy | Intramuscular | NCT00428935 |
2 | Cystic Fibrosis | Intranasal, endobronchial | NCT00004533 |
2 | Rheumatoid arthritis | Intraartiular | NCT00617032, NCT00126724 |
2 | Age-related macular degeneration | Intravitreal | NCT01024998 |
1,6 | Severe heart failure | Intracoronary | NCT00454818; NCT00534703 |