Adeno-associated virus vectors for human gene therapy

Table 1 Properties and applications of the commonly used adeno-associated virus serotypes and variants

AAV serotypes and variants	Target tissues	Preclinical applications	Clinical applications	Ref.
AAV1	Muscle, heart	Charcot-Marie-Tooth Neuropathy, congestive heart failure, Duchenne muscular dystrophy	Glybera, a drug for lipoprotein lipase deficiency, congenital heart failure	[1,2,11,15-17,19-21]
AAV2	Liver, eye	In vitro assays, various animal model studies	Cystic fibrosis; hemophilia B, Leber’s congenital amourosis, Parkinson’s disease; Canavan disease	[11,26-36]
AAV3A, 3B	Liver cancer	Liver cancer		[40]
AAV4	Ependyma, astrocyte, retinal pigmented epithelium	Mucopolysaccharidosis type VII, Familial amyotrophic lateral sclerosis, RPE65-deficient vision loss	RPE65-deficient disease	[42,47]
AAV5	Sensor neuron, airway epithelia, Dentritic cells	Globoid cell leukodystrophy; human immunodeficiency	-	[49-52]
AAV6	Airway epithelia, skeletal muscle, Dendritic cells, pancreatic beta cells	Duchenne muscular dystrophy	-	[53-56]
AAV7	Skeletal muscle, liver, central nervous system	-	-	[11,58-60]
AAV8	Liver, skeletal and cardiac muscle	Hemophilia A, familial hypercholesterolemia, glycogen storage disease type II	Hemophilia B	[11,62-66,143]
AAV9	Cardiac muscle, central nervous system	Heart failure, central nervous system disorders	Spinal muscular atrophy	[70-75]
AAVShH10	Müller cells	Retinitis pigmentosa	-	[76,77]
AAV7m8	Vitreous humor	Retinoschisis, Leber’s congenital amaurosis	-	[79,80]
AAVDJ	Liver, kidney, cervix, retina, oveary, skin, fibroblast, lung	-	-	[81]

AAV: Adeno-associated virus.