Copyright ©The Author(s) 2015.
World J Med Genet. Aug 27, 2015; 5(3): 28-45
Published online Aug 27, 2015. doi: 10.5496/wjmg.v5.i3.28
Table 1 Properties and applications of the commonly used adeno-associated virus serotypes and variants
AAV serotypes and variantsTarget tissuesPreclinical applicationsClinical applicationsRef.
AAV1Muscle, heartCharcot-Marie-Tooth Neuropathy, congestive heart failure, Duchenne muscular dystrophyGlybera, a drug for lipoprotein lipase deficiency, congenital heart failure[1,2,11,15-17,19-21]
AAV2Liver, eyeIn vitro assays, various animal model studiesCystic fibrosis; hemophilia B, Leber’s congenital amourosis, Parkinson’s disease; Canavan disease[11,26-36]
AAV3A, 3BLiver cancerLiver cancer[40]
AAV4Ependyma, astrocyte, retinal pigmented epitheliumMucopolysaccharidosis type VII, Familial amyotrophic lateral sclerosis, RPE65-deficient vision lossRPE65-deficient disease[42,47]
AAV5Sensor neuron, airway epithelia, Dentritic cellsGloboid cell leukodystrophy; human immunodeficiency-[49-52]
AAV6Airway epithelia, skeletal muscle, Dendritic cells, pancreatic beta cellsDuchenne muscular dystrophy-[53-56]
AAV7Skeletal muscle, liver, central nervous system--[11,58-60]
AAV8Liver, skeletal and cardiac muscleHemophilia A, familial hypercholesterolemia, glycogen storage disease type IIHemophilia B[11,62-66,143]
AAV9Cardiac muscle, central nervous systemHeart failure, central nervous system disordersSpinal muscular atrophy[70-75]
AAVShH10Müller cellsRetinitis pigmentosa-[76,77]
AAV7m8Vitreous humorRetinoschisis, Leber’s congenital amaurosis-[79,80]
AAVDJLiver, kidney, cervix, retina, oveary, skin, fibroblast, lung--[81]