[1] BULCHA TJ,WANG Y,MA H,et al.Viral vector platforms within the gene therapy landscape[J].Signal Transduct Target Ther,2021,6(1):53
[2] LI X,LE Y,ZHANG Z,et al.Viral vector-based gene therapy[J].Int J Mol Sci,2023,24(9):7736
[3] 李跃萍,宋丽萍,邱曙东.慢病毒载体在肿瘤基因治疗中的应用[J].现代肿瘤医学,2006,14(12):1614
LI YP,SONG LP,QIU SD.The appliance of lentivirus vector in cancer theray[J].J Mod Oncol,2006,14(12):1614
[4] NAVALE MS,BHOSALE MK,MOHITE MM,et al.Hemgenix as first gene therapy for treatment of haemophilia B[J].Haemophilia,2022,2(1):89
[5] ABREU JN,WALDROP AM.Overview of gene therapy in spinal muscular atrophy and Duchenne muscular dystrophy[J].Pediatr Pulmonol,2020,56(4):710
[6] 张丽娟,邵海涛,王跃秀,等.帕金森病研究进展[J].生命科学,2014,26(6):560
ZHANG LJ,SHAO HT,WANG YX,et al.Progress in Parkinson’s disease[J].Chin Bull Life Sci,2014,26(6):560
[7] BARTUS RT,BAUMANN TL,SIFFERT J,et al.Safety/feasibility of targeting the substantia nigra with AAV2-neurturin in Parkinson patients[J].Neurology,2013,80(18):1698
[8] WANG ZY,LISOWSKI L,FINEGOLD MJ,et al.AAV vectors containing rDNA homology display increased chromosomal integration and transgene persistence[J].Mol Ther,2012,20(10):1902
[9] WANG D,LI S,GESSLER DJ,et al.A rationally engineered capsid variant of AAV9 for systemic CNS-directed and peripheral tissue-detargeted gene delivery in neonates[J].Mol Ther Methods Clin Dev,2018,9:234
[10] MEZZINA M,MERTEN OW.Adeno-associated viruses[J].Methods Mol Biol,2011,737:211
[11] HILDEGARD B,LUCA P,OLIVER C,et al.Recent developments in adeno-associated virus vector technology[J].J Gene Med,2008,10(7):717
[12] GALIBERT L,HYVONEN A,ERIKSSON RA,et al.Functional roles of the membrane-associated AAV protein MAAP[J].Sci Rep,2021,11(1):21698
[13] FU Q,POLANCO A,LEE YS,et al.Critical challenges and advances in recombinant adeno-associated virus(rAAV)biomanufacturing[J].Biotechnol Bioeng,2023,120(9):2601
[14] JOHNSON FB,OZER HL,HOGGAN MD.Structural proteins of adenovirus-associated virus type 3[J].J Virol,1971,8(6):860
[15] ROSE JA,JR MAIZEL JV,INMAN JK,et al.Structural proteins of adenovirus-associated viruses[J].J Virol,1971,8(5):766
[16] BULLER RM,ROSE JA.Characterization of adenovirus-associated virus-induced polypeptides in KB cells[J].J Virol,1978,25(1):331
[17] MIETZSCH M,JOSE A,CHIPMAN P,et al.Completion of the AAV structural atlas:serotype capsid structures reveals clade-specific features[J].Viruses,2021,13(1):101
[18] URABE M,DING C,KOTIN RM.Insect cells as a factory to produce adeno-associated virus type 2 vectors[J].Hum Gene Ther,2002,13(16):1935
[19] KASHIWAKURA Y,TAMAYOSE K,IWABUCHI K,et al.Hepatocyte growth factor receptor is a coreceptor for adeno-associated virus type 2 infection[J].J Virol,2005,79(1):609
[20] QING K,MAH C,HANSEN J,et al.Human fibroblast growth factor receptor 1 is a co-receptor for infection by adeno-associated virus 2[J].Nat Med,1999,5(1):71
[21] SANLIOGLU S,BENSON PK,YANG J,et al.Endocytosis and nuclear trafficking of adeno-associated virus type 2 are controlled by rac1 and phosphatidylinositol-3 kinase activation[J].J Virol,2000,74(19):9184
[22] SUMMERFORD C,BARTLETT JS,SAMULSKI RJ.AlphaVbeta5 integrin:a co-receptor for adeno-associated virus type 2 infection[J].Nat Med,1999,5(1):78
[23] KERN A,SCHMIDT K,LEDER C,et al.Identification of a heparin-binding motif on adeno-associated virus type 2 capsids[J].J Virol,2003,77(20):11072
[24] GIROD A,WOBUS CE,ZADORI Z,et al.The VP1 capsid protein of adeno-associated virus type 2 is carrying a phospholipase A2 domain required for virus infectivity[J].J Gen Virol,2002,83(5):973
[25] STAHNKE S,LUX K,UHRIG S,et al.Intrinsic phospholipase A2 activity of adeno-associated virus is involved in endosomal escape of incoming particles[J].Virology,2011,409(1):77
[26] BLEKER S,SONNTAG F,KLEINSCHMIDT JA.Mutational analysis of narrow pores at the fivefold symmetry axes of adeno-associated virus type 2 capsids reveals a dual role in genome packaging and activation of phospholipase A2 activity[J].J Virol,2005,79(4):2528
[27] POPA WR,PORWAL M,KANN M,et al.Impact of VP1-specific protein sequence motifs on adeno-associated virus type 2 intracellular trafficking and nuclear entry[J].J Virol,2012,86(17):9163
[28] ZHONG L,LI B,JAYANDHARAN G,et al.Tyrosine-phosphorylation of AAV2 vectors and its consequences on viral intracellular trafficking and transgene expression[J].Virology,2008,381(2):194
[29] SEN D,BALAKRISHNAN B,GABRIEL N,et al.Improved adeno-associated virus(AAV)serotype 1 and 5 vectors for gene therapy[J].Sci Rep,2013,3:1832
[30] SEN D,GADKARI RA,SUDHA G,et al.Targeted modifications in adeno-associated virus serotype 8 capsid improves its hepatic gene transfer efficiency in vivo[J].Hum Gene Ther Methods,2013,24(2):104
[31] LI B,MA W,LING C,et al.Site-directed mutagenesis of surface-exposed lysine residues leads to improved transduction by AAV2,but not AAV8,vectors in murine hepatocytes in vivo[J].Hum Gene Ther Methods,2015,26(6):211
[32] PIPE S,LEEBEEK FWG,FERREIRA V,et al.Clinical considerations for capsid choice in the development of liver-targeted AAV-based gene transfer[J].Mol Ther Methods Clin Dev,2019,15:170
[33] FARRAHA M,KIZANA E.Assessing recombinant AAV shedding after cardiac gene therapy[J].Methods Mol Biol,2022,2573:333
[34] LEBORGNE C,BARBON E,ALEXANDER JM,et al.IgG-cleaving endopeptidase enables in vivo gene therapy in the presence of anti-AAV neutralizing antibodies[J].Nat Med,2020,26(7):1096
[35] KORNEYENKOV MA,ZAMYATNIN AA JR.Next step in gene delivery:modern approaches and further perspectives of AAV tropism modification[J].Pharmaceutics,2021,13(5):750
[36] MITCHELL AD,O’DONNELL J,HARE TJ,et al.Serotype-specific detection of adeno-associated virus during laboratory preparation[J].J Virol Methods,2006,136(1-2):277
[37] VAN K,MOHIUDDIN Y,MCCLUNG S,et al.Adeno-associated virus capsid serotype identification:analytical methods development and application[J].J Virol Methods,2009,159(2):167
[38] JIN XY,LIU L,NASS S,et al.Direct liquid chromatography/mass spectrometry analysis for complete characterization of recombinant adeno-associated virus capsid proteins[J].Hum Gene Ther Methods,2017,28(5):255
[39] ZAREI M,WANG P,JONVEAUX J,et al.A novel protocol for in-depth analysis of recombinant adeno-associated virus capsid proteins using UHPLC-MS/MS[J].Rapid Commun Mass Spectrom,2022,36(6):9247
[40] QIN X,LI X,CHEN LS,et al.Characterization of adeno-associated virus capsid proteins by microflow liquid chromatography coupled with mass spectrometry[J].Appl Biochem Biotechnol,2024,196(3):1623
[41] LIU AP,PATEL SK,XING T,et al.Characterization of adeno-associated virus capsid proteins using hydrophilic interaction chromatography coupled with mass spectrometry[J].J Pharm Biomed Anal,2020,189(10):113481
[42] SMITH J,GUAPO F,STRASSER L,et al.Development of a rapid adeno-associated virus(AAV)identity testing platform through comprehensive mass analysis of full-length AAV capsid proteins[J].J Proteome Res,2023,23(1):161
[43] LANSHOEFT C,HEUDI O,CIANFERANI S.SMART Digest™ compared with pellet digestion for analysis of human immunoglobulin G1 in rat serum by liquid chromatography tandem mass spectrometry[J].Anal Biochem,2016,501(15):23
[44] DICK LW JR,MAHON D,QIU D,et al.Peptide mapping of therapeutic monoclonal antibodies:improvements for increased speed and fewer artifacts[J].J Chromatogr B Analyt Technol Biomed Life Sci,2009,877(3),230
[45] TOOLE EN,DUFRESNE C,RAY S,et al.Rapid highly-efficient digestion and peptide mapping of adeno-associated viruses[J].Anal Chem,2021,93(30):10403
[46] GUAPO F,STRASSER L,MILLAN S,et al.Fast and efficient digestion of adeno associated virus(AAV)capsid proteins for liquid chromatography mass spectrometry(LC-MS)based peptide mapping and post translational modification analysis(PTMs)[J].J Pharm Biomed Anal,2022,207(5):114427
[47] WU ZJ,WANG HX,TUSTIAN A,et al.Development of a two-dimensional liquid chromatography-mass spectrometry platform for simultaneous multi-attribute characterization of adeno-associated viruses[J].Anal Chem,2022,94(7):3219
[48] GILES AR,SIMS JJ,TURNER KB,et al.Deamidation of amino acids on the surface of adeno-associated virus capsids leads to charge heterogeneity and altered vector function[J].Mol Ther,2018,26(12):2848
[49] WANG D,TAO PWL,GAO G.Adeno-associated virus vector as a platform for gene therapy delivery[J].Nat Rev Drug Discov,2019,18(5):358
[50] NONNENMACHER M,WEBER T.Intracellular transport of recombinant adeno-associated virus vectors[J].Gene Ther,2012,19(6):649
[51] MARY B,MAURYA S,ARUMUGAM S,et al.Post-translational modifications in capsid proteins of recombinant adeno-associated virus(AAV)1-rh10 serotypes[J].FEBS J,2019,286(24):4964
[52] BENNETT A,PATEL S,MIETZSCH M,et al.Thermal stability as a determinant of AAV serotype identity[J].Mol Ther Methods Clin Dev,2017,6:171
[53] LINS-AUSTIN B,PATEL S,MIETZSCH M,et al.Adeno-associated virus(AAV)capsid stability and liposome remodeling during endo/lysosomal pH trafficking[J].Viruses,2020,12(6):668
[54] XU Y,GUO P,ZHANG J,et al.Effects of thermally induced configuration changes on rAAV genome’s enzymatic accessibility[J].Mol Ther Methods Clin Dev,2020,18:328
[55] SUMMERFORD C,SAMULSKI RJ.Membrane-associated heparan sulfate proteoglycan is a receptor for adeno-associated virus type 2 virions[J].J Virol,1998,72(2):1438
[56] BARTLETT JS,KLEINSCHMIDT J,BOUCHER RC,et al.Targeted adeno-associated virus vector transduction of nonpermissive cells mediated by a bispecific F(ab’gamma)2 antibody[J].Nat Biotechnol,1999,17(2):181
[57] MULLER OJ,KAUL F,WEITZMAN MD,et al.Random peptide libraries displayed on adeno-associated virus to select for targeted gene therapy vectors[J].Nat Biotechnol,2003,21(9):1040
[58] PERABO L,BUNING H,KOFLER DM,et al.In vitro selection of viral vectors with modified tropism:the adeno-associated virus display[J].Mol Ther,2003,8(1):151
[59] PONNAZHAGAN S,MAHENDRA G,KUMAR S,et al.Conjugate-based targeting of recombinant adeno-associated virus type 2 vectors by using avidin-linked ligands[J].J Virol,2002,76(24):12900
[60] RIED MU,GIROD A,LEIKE K,et al.Adeno-associated virus capsids displaying immunoglobulin-binding domains permit antibody-mediated vector retargeting to specific cell surface receptors[J].J Virol,2002,76(9):4559
[61] HAUCK B,CHEN L,XIAO WD.Generation and characterization of chimeric recombinant AAV vectors[J].Mol Ther,2003,7(3):419
[62] RABINOWITZ JE,BOWLES DE,FAUST SM,et al.Cross-dressing the virion:the transcapsidation of adeno-associated virus serotypes functionally defines subgroups[J].J Virol,2004,78(9):4421
[63] WISTUBA A,KERN A,WEGER S,et al.Subcellular compartmentalization of adeno-associated virus type 2 assembly[J].J Virol,1997,71(2):1341
[64] WISTUBA A,WEGER S,KERN A,et al.Intermediates of adeno-associated virus type 2 assembly:identification of soluble complexes containing Rep and Cap proteins[J].J Virol,1995,69(9):5311
[65] GRIMM D,KERN A,PAWLITA M,et al.Titration of AAV-2 particles via a novel capsid ELISA:packaging of genomes can limit production of recombinant AAV-2[J].Gene Ther,1999,6(7):1322
[66] GRIMM D,KERN A,RITTNER K,et al.Novel tools for production and purification of recombinant adeno-associated virus vectors[J].Hum Gene Ther,1998,9(18):2745
[67] WOBUS CE,HUGLE-DORR B,GIROD A,et al.Monoclonal antibodies against the adeno-associated virus type 2(AAV-2)capsid:epitope mapping and identification of capsid domains involved in AAV-2-cell interaction and neutralization of AAV-2 infection[J].J Virol,2000,74(19):9281
[68] GEORG-FRIES B,BIEDERLASK S,WOLF J,et al.Analysis of proteins,helper dependence,and seroepidemiology of a new human parvovirus[J].Virology,1984,134(1):64
[69] KUCK D,KERN A,KLEINSCHMIDT JA.Development of AAV serotype-specific ELISAs using novel monoclonal antibodies[J].J Virol Methods,2007,140(1-2):17
[70] AEBISCHER MK,BOUVAREL T,BARROZO E,et al.Boosting the separation of adeno-associated virus capsid proteins by liquid chromatography and capillary[J].Int J Mol Sci,2023,24(10):8503
[71] OYAMA H,ISHII K,MARUNO T,et al.Characterization of adeno-associated virus capsid proteins with two types of VP3-Related components by capillary gel electrophoresis and mass spectrometry[J].Hum Gene Ther,2021,32(21-22):1403
[72] WU J,HEGER C.Establishment of a platform imaged capillary isoelectric focusing(icIEF)characterization method for adeno-associated virus(AAV)capsid proteins[J].Green Anal Chem,2022,3:100027
[73] HE XZ,POWERS TW,HUANG S,et al.Development of an icIEF assay for monitoring AAV capsid proteins and application to gene therapy products[J].Mol Ther Methods Clin Dev,2023,29:133
[74] BARNES LF,DRAPER BE,KURIAN J,et al.Analysis of AAV-extracted DNA by charge detection mass spectrometry reveals genome truncations[J].Anal Chem,2023,95(9):4310
