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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 (
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