Table 2.

Summary of AAV-mediated gene therapies in clinical application in recent years.

AAV serotypes	Disease	Gene transferred	Reference
AAV1	Pompe disease, pulmonary emphysema, LGMD2D, LPLD, cystic fibrosis, Alzheimer’s disease	hGAA, M-AAT, alpha-sarcoglycan, LPL(S447X), IL10, anti-Abeta single-chain antibody	[32], [33, 34], [35], [36], [37], [38]
AAV2	Choroideremia, LCA, PD	CHM, RPE65, AADC	[39], [40], [41]
AAV3	Liver cancer	Pyruvate dehydrogenase E1 alpha subunit	[14]
AAV4	Huntington’s disease, ALS	BDNF and noggin, IGF-1 or VEGF-165	[42], [43]
AAV5	Cone-rod dystrophy, polyglutamine disease	RPGRIP1, Hsp40	[44], [45]
AAV6	Heart failure, cardiac transplantation	S100A1, HO-1	[46], [47]
AAV7	GSD-1a	G6Pase	[48]
AAV8	Hemophilia B, muscular dystrophies,	hFIXco, VEGF-C/D	[49], [50]
AAV9	Alzheimer’s disease, heart failure, salivary hypofunction	IgVL5D3/TLK1B/, SERCA/MiR378, tousled-like kinase 1B	[51], [52], [53], [54]
AAVrh.10	Friedreich’s ataxia, JNCL	Frataxin, CLN3	[55], [56]

Abbreviations: GAA = lysosomal acid alpha-glucosidase, AAT = α-1 antitrypsin, LGMD2D = limb-girdle muscular dystrophy type 2D, CHM = choroideremia, LCA = Leber congenital amaurosis, PD = Parkinson’s disease, AADC = aromatic amino acid decarboxylase, RPE65 = retinal pigment epithelium-specific protein 65 kDa, hFIXco = codon-optimized human factor IX, LPLD = lipoprotein lipase deficiency, TLK1B = tousled-like kinase 1B, SERCA = sarcoplasmic reticulum Ca(2+)-ATPase, BDNF = brain-derived neurotrophic factor, GSD-1a = glycogen storage disease type 1a, JNCL = juvenile neuronal ceroid lipofuscinosis, PNS = peripheral nervous system.