1Naumer M,Popa-Wagner R,Kleinschmidt JA.Impact of capsid modifications by selected peptide ligands on recombinant adenoassociated virus serotype 2-mediated gene transduction[J].J Gen Virol,2012,93(Pt 10):2131-2141.
2Ellis BL,Hirsch ML,Barker JC,et al.A survey of ex vivo/in vitro transduction efficiency of mammalian primary cells and cell lines with Nine natural adeno-associated virus(AAV1-9)and one engineered adeno-associated virus serotype[J].Virol J,2013,10:74.
3Mueller C,Tang Q,Gruntman A,et al.Sustained miRNA-mediated knockdown of mutant AAT with simultaneous augmentation of wildtype AAT has minimal effect on global liver miRNA profiles[J].Mol Ther,2012,20(3):590-600.
4Macloughlin RJ,Higgins BD,Devaney J,et al.Aerosol-mediated delivery of AAV2/6-Ikappa Balpha attenuates lipopolysaccharideinduced acute lung injury in rats[J].Hum Gene Ther,2015,26(1):36-46.
5Mao H,Gorbatyuk MS,Rossmiller B,et al.Long-term rescue of retinal structure and function by rhodopsin RNA replacement with a single adeno-associated viral vector in P23H RHO transgenic mice[J].Hum Gene Ther,2012,23(4):356-366.
6Mingozzi F,High KA.Therapeutic in vivo gene transfer for genetic disease using AAV:progress and challenges[J].Nat Rev Genet,2011,12(5):341-355.
8Junge N,Mingozzi F,Ott M,et al.Adeno-associated Virus Vector Based Gene Therapy for Monogenetic Metabolic Diseases of the Liver[J].J Pediatr Gastroenterol Nutr,2015,60(4):433-440.