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Volumn 7, Issue 1, 2000, Pages 24-30

AAV vectors: Is clinical success on the horizon?

Author keywords

AAV; Adeno associated virus; Gene therapy; Viral vectors

Indexed keywords

VIRUS VECTOR;

EID: 0033978147     PISSN: 09697128     EISSN: None     Source Type: Journal    
DOI: 10.1038/sj.gt.3301109     Document Type: Review
Times cited : (271)

References (48)
  • 1
    • 0029762402 scopus 로고    scopus 로고
    • NIDDK workshop on AAV vectors: Gene transfer into quiescent cells
    • McKeon C, Samulski R. NIDDK Workshop on AAV vectors: gene transfer into quiescent cells. Hum Gene Ther 1996; 7: 1615-1619.
    • (1996) Hum Gene Ther , vol.7 , pp. 1615-1619
    • McKeon, C.1    Samulski, R.2
  • 2
    • 0023953618 scopus 로고
    • Gene transfer into hematopoietic progenitor cells mediated by an adeno-associated virus vector
    • LaFace D et al. Gene transfer into hematopoietic progenitor cells mediated by an adeno-associated virus vector. Virology 1988; 162: 483-486.
    • (1988) Virology , vol.162 , pp. 483-486
    • LaFace, D.1
  • 3
    • 0029843596 scopus 로고    scopus 로고
    • Efficient long term gene transfer into muscle tissue of immunocompetent mice by adeno-associated virus vector
    • Xiao X, Li J, Samulski R. Efficient long term gene transfer into muscle tissue of immunocompetent mice by adeno-associated virus vector. J Virol 1996; 70: 8098-8108.
    • (1996) J Virol , vol.70 , pp. 8098-8108
    • Xiao, X.1    Li, J.2    Samulski, R.3
  • 4
    • 0030451214 scopus 로고    scopus 로고
    • Gene delivery to skeletal muscle results in sustained expression and systemic delivery of a therapeutic protein
    • Kessler PD et al. Gene delivery to skeletal muscle results in sustained expression and systemic delivery of a therapeutic protein. Proc Natl Acad Sci USA 1996; 93: 14082-14087.
    • (1996) Proc Natl Acad Sci USA , vol.93 , pp. 14082-14087
    • Kessler, P.D.1
  • 5
    • 0033544010 scopus 로고    scopus 로고
    • Progress in adeno-associated virus type 2 vector production: Promises and prospects for clinical use
    • Grimm D, Kleinschmidt J. Progress in adeno-associated virus type 2 vector production: promises and prospects for clinical use. Hum Gene Ther 1999; 10: 2445-2450.
    • (1999) Hum Gene Ther , vol.10 , pp. 2445-2450
    • Grimm, D.1    Kleinschmidt, J.2
  • 6
    • 0033022953 scopus 로고    scopus 로고
    • Recombinant adeno-associated virus purification using novel methods improves infectious titer and yield
    • Zolotukhin S et al. Recombinant adeno-associated virus purification using novel methods improves infectious titer and yield. Gene Therapy 1999; 6: 973-985.
    • (1999) Gene Therapy , vol.6 , pp. 973-985
    • Zolotukhin, S.1
  • 7
    • 0033541640 scopus 로고    scopus 로고
    • Highly purified recombinant adeno-associated virus vectors are biologically active and free of detectable helper and wild-type virus
    • Clark K et al. Highly purified recombinant adeno-associated virus vectors are biologically active and free of detectable helper and wild-type virus. Hum Gene Ther 1999; 10: 1031-1039.
    • (1999) Hum Gene Ther , vol.10 , pp. 1031-1039
    • Clark, K.1
  • 8
    • 0032917854 scopus 로고    scopus 로고
    • Viral receptors and vector purification: New approaches for generating clinical-grade reagents
    • Summerford C, Samulski R. Viral receptors and vector purification: new approaches for generating clinical-grade reagents. Nature Med 1999; 5: 587-588.
    • (1999) Nature Med , vol.5 , pp. 587-588
    • Summerford, C.1    Samulski, R.2
  • 9
    • 0032773889 scopus 로고    scopus 로고
    • Titration of AAV-2 particles via a novel capsid ELISA: Packaging of genomes can limit production of recombinant AAV-2
    • Grimm D et al. Titration of AAV-2 particles via a novel capsid ELISA: packaging of genomes can limit production of recombinant AAV-2. Gene Therapy 1999; 6: 1322-1330.
    • (1999) Gene Therapy , vol.6 , pp. 1322-1330
    • Grimm, D.1
  • 10
    • 0000609138 scopus 로고    scopus 로고
    • Adeno-associated viral vectors
    • Friedman T (ed). Cold Spring Harbor Laboratory Press: Cold Spring Harbor
    • Samulski RJ, Sally M, Muzyczka N. Adeno-associated viral vectors. In: Friedman T (ed). The Development of Human Gene Therapy. Cold Spring Harbor Laboratory Press: Cold Spring Harbor, 1999, pp 131-172.
    • (1999) The Development of Human Gene Therapy , pp. 131-172
    • Samulski, R.J.1    Sally, M.2    Muzyczka, N.3
  • 11
    • 0033010884 scopus 로고    scopus 로고
    • Human fibroblast growth factor receptor 1 is a coreceptor for infection by adeno-associated virus 2
    • Qing K et al. Human fibroblast growth factor receptor 1 is a coreceptor for infection by adeno-associated virus 2. Nature Med 1999; 5: 71-77.
    • (1999) Nature Med , vol.5 , pp. 71-77
    • Qing, K.1
  • 12
    • 0342529670 scopus 로고    scopus 로고
    • Transduction and utility of the granulocyte-macrophage colony-stimulating factor gene into monocytes and dendritic cells by adeno-associated virus
    • in press
    • Liu Y et al. Transduction and utility of the granulocyte-macrophage colony-stimulating factor gene into monocytes and dendritic cells by adeno-associated virus. J Interfer Cytokine Res 1999 (in press).
    • (1999) J Interfer Cytokine Res
    • Liu, Y.1
  • 13
    • 0032212276 scopus 로고    scopus 로고
    • High-titer adeno-associated viral vectors from a Rep/Cap cell line and hybrid shuttle virus
    • Gao G et al. High-titer adeno-associated viral vectors from a Rep/Cap cell line and hybrid shuttle virus. Hum Gene Ther 1998; 9: 2353-2362.
    • (1998) Hum Gene Ther , vol.9 , pp. 2353-2362
    • Gao, G.1
  • 14
    • 0033054126 scopus 로고    scopus 로고
    • High-titer recombinant adeno-associated virus production utilizing a recombinant herpes simplex virus type 1 vector expressing AAV-2 Rep and Cap
    • Conway J et al. High-titer recombinant adeno-associated virus production utilizing a recombinant herpes simplex virus type 1 vector expressing AAV-2 Rep and Cap. Gene Therapy 1999; 6: 986-993.
    • (1999) Gene Therapy , vol.6 , pp. 986-993
    • Conway, J.1
  • 15
    • 0030776949 scopus 로고    scopus 로고
    • Vitro packaging of an infectious recombinant adeno-associated virus 2
    • Ding L, Lu S, Munshi N. In vitro packaging of an infectious recombinant adeno-associated virus 2. Gene Therapy 1997; 4: 1167-1172.
    • (1997) Gene Therapy , vol.4 , pp. 1167-1172
    • Ding, L.1    Lu, S.2    Munshi, N.3
  • 16
    • 2642704243 scopus 로고    scopus 로고
    • Vitro packaging of adeno-associated virus DNA
    • Zhou X, Muzyczka N. In vitro packaging of adeno-associated virus DNA. J Virol 1998; 72: 3241-3247.
    • (1998) J Virol , vol.72 , pp. 3241-3247
    • Zhou, X.1    Muzyczka, N.2
  • 17
    • 0031838721 scopus 로고    scopus 로고
    • Adeno-associated viral vector-mediated gene transfer of human blood coagulation factor IX into mouse liver
    • Nakai H et al. Adeno-associated viral vector-mediated gene transfer of human blood coagulation factor IX into mouse liver. Blood 1998; 91: 4600-4607.
    • (1998) Blood , vol.91 , pp. 4600-4607
    • Nakai, H.1
  • 18
    • 17444372024 scopus 로고    scopus 로고
    • Correction of hemophilia B in canine and murine models using recombinant adeno-associated viral vectors
    • Snyder RO et al. Correction of hemophilia B in canine and murine models using recombinant adeno-associated viral vectors. Nature Med 1999; 5: 64-70.
    • (1999) Nature Med , vol.5 , pp. 64-70
    • Snyder, R.O.1
  • 19
    • 0033616680 scopus 로고    scopus 로고
    • Sustained correction of bleeding disorder in hemophilia B mice by gene therapy
    • Wang L et al. Sustained correction of bleeding disorder in hemophilia B mice by gene therapy. Proc Natl Acad Sci USA 1999; 96: 3906-3910.
    • (1999) Proc Natl Acad Sci USA , vol.96 , pp. 3906-3910
    • Wang, L.1
  • 20
    • 13144305062 scopus 로고    scopus 로고
    • Sustained secretion of human alpha-1-antitrypsin from murine muscle transduced with adeno-associated virus vectors
    • Song S et al. Sustained secretion of human alpha-1-antitrypsin from murine muscle transduced with adeno-associated virus vectors. Proc Natl Acad Sci USA 1998; 95: 14384-14388.
    • (1998) Proc Natl Acad Sci USA , vol.95 , pp. 14384-14388
    • Song, S.1
  • 21
    • 0342529667 scopus 로고    scopus 로고
    • The use of adeno-associated virus to circumvent the maturation dependent viral transduction of myofibers
    • accepted
    • Pruchnic R et al. The use of adeno-associated virus to circumvent the maturation dependent viral transduction of myofibers. Hum Gene Ther 1999 (accepted).
    • (1999) Hum Gene Ther
    • Pruchnic, R.1
  • 22
    • 0032959311 scopus 로고    scopus 로고
    • Cloning and characterization of adeno-associated virus type 5
    • Chiorini J et al. Cloning and characterization of adeno-associated virus type 5. J Virol 1999; 73: 1309-1319.
    • (1999) J Virol , vol.73 , pp. 1309-1319
    • Chiorini, J.1
  • 23
    • 0343834934 scopus 로고    scopus 로고
    • Insertional mutagenesis of AAV2 capsid and the protection of recombinant virus
    • in press
    • Rabinowitz J, Xiao W, Samulski R. Insertional mutagenesis of AAV2 capsid and the protection of recombinant virus. Virology 2000: (in press).
    • (2000) Virology
    • Rabinowitz, J.1    Xiao, W.2    Samulski, R.3
  • 24
    • 0032870158 scopus 로고    scopus 로고
    • Genetic capsid modifications allow efficient retargeting of adeno-associated virus type 2
    • Girod A et al. Genetic capsid modifications allow efficient retargeting of adeno-associated virus type 2. Nature Med 1999; 5: 1052-1056.
    • (1999) Nature Med , vol.5 , pp. 1052-1056
    • Girod, A.1
  • 25
    • 0031954781 scopus 로고    scopus 로고
    • Recombinant human parvovirus B19 vectors: Erythroid cell-specific delivery and expression of transduced genes
    • Ponnazhagan S et al. Recombinant human parvovirus B19 vectors: erythroid cell-specific delivery and expression of transduced genes. J Virol 1998; 72: 5224-5230.
    • (1998) J Virol , vol.72 , pp. 5224-5230
    • Ponnazhagan, S.1
  • 26
    • 0034161486 scopus 로고    scopus 로고
    • Sustained expression of human factor VIII in mice using a parvovirus-based vector
    • in press
    • Chao H et al. Sustained expression of human factor VIII in mice using a parvovirus-based vector. Blood 2000 (in press).
    • (2000) Blood
    • Chao, H.1
  • 27
    • 0032544053 scopus 로고    scopus 로고
    • Efficient expression of CFTR function with adeno-associated vectors that carry shortened CFTR genes
    • Zhang L et al. Efficient expression of CFTR function with adeno-associated vectors that carry shortened CFTR genes. Prof Natl Acad Sci USA 1998; 95: 10158-10163.
    • (1998) Prof Natl Acad Sci USA , vol.95 , pp. 10158-10163
    • Zhang, L.1
  • 29
    • 0032862285 scopus 로고    scopus 로고
    • Concatamerization of adeno-associatcd virus circular genomes occurs through intermolecular recombination
    • Yang J et al. Concatamerization of adeno-associatcd virus circular genomes occurs through intermolecular recombination. J Virol 1999; 73: 9468-9477.
    • (1999) J Virol , vol.73 , pp. 9468-9477
    • Yang, J.1
  • 30
    • 0031058024 scopus 로고    scopus 로고
    • Recombinant adeno-associated virus for muscle directed gene therapy
    • Fisher KJ et al. Recombinant adeno-associated virus for muscle directed gene therapy. Nature Med 1997; 3: 306-312.
    • (1997) Nature Med , vol.3 , pp. 306-312
    • Fisher, K.J.1
  • 31
    • 0031969428 scopus 로고    scopus 로고
    • Transduction of dendritic cells by DNA viral vectors directs the immune response to transgene products in muscle fibers
    • Jooss K et al. Transduction of dendritic cells by DNA viral vectors directs the immune response to transgene products in muscle fibers. J Virol 1998; 72: 4212-4223.
    • (1998) J Virol , vol.72 , pp. 4212-4223
    • Jooss, K.1
  • 32
    • 0032977833 scopus 로고    scopus 로고
    • Induction of immunity to antigens expressed by recombinant adeno-associated virus depends on the route of administration
    • Brockstedt D et al. Induction of immunity to antigens expressed by recombinant adeno-associated virus depends on the route of administration. J Appl Biomaterials 1999; 92: 67-75.
    • (1999) J Appl Biomaterials , vol.92 , pp. 67-75
    • Brockstedt, D.1
  • 33
    • 0030819372 scopus 로고    scopus 로고
    • Genetic immunization with adeno-associated virus vectors expressing herpes simplex virus type 2 glycoproteins B and D
    • Manning W et al. Genetic immunization with adeno-associated virus vectors expressing herpes simplex virus type 2 glycoproteins B and D. J Virol 1997; 71: 7960-7962.
    • (1997) J Virol , vol.71 , pp. 7960-7962
    • Manning, W.1
  • 34
    • 0032826773 scopus 로고    scopus 로고
    • Latent adeno-associatcd virus infection elicits humoral but not cell-mediated immune responses in a nonhuman primate model
    • Hemandez Y et al. Latent adeno-associatcd virus infection elicits humoral but not cell-mediated immune responses in a nonhuman primate model. J Virol 1999; 73: 8549-8558.
    • (1999) J Virol , vol.73 , pp. 8549-8558
    • Hemandez, Y.1
  • 35
    • 0033178657 scopus 로고    scopus 로고
    • Adeno-associated virus vectors and hematology
    • Russell D, Kay M. Adeno-associated virus vectors and hematology. Blood 1999; 94: 864-874.
    • (1999) Blood , vol.94 , pp. 864-874
    • Russell, D.1    Kay, M.2
  • 36
    • 0032853699 scopus 로고    scopus 로고
    • Immune responses to adenovirus and adeno-associatcd virus in humans
    • Chirmule N et al. Immune responses to adenovirus and adeno-associatcd virus in humans. Gene Therapy 1999; 6: 1574-1583.
    • (1999) Gene Therapy , vol.6 , pp. 1574-1583
    • Chirmule, N.1
  • 37
    • 0032924185 scopus 로고    scopus 로고
    • Gene therapy vectors based on adeno-associated virus type 1
    • Xiao W et al. Gene therapy vectors based on adeno-associated virus type 1. J Virol 1999; 73: 3994-4003.
    • (1999) J Virol , vol.73 , pp. 3994-4003
    • Xiao, W.1
  • 38
    • 0031984786 scopus 로고    scopus 로고
    • Infectious clones and vectors derived from adeno-associated virus (AAV) serotypes other than AAV type 2
    • Rutledge E, Halbert C, Russell D. Infectious clones and vectors derived from adeno-associated virus (AAV) serotypes other than AAV type 2. J Virol 1998; 72: 309-319.
    • (1998) J Virol , vol.72 , pp. 309-319
    • Rutledge, E.1    Halbert, C.2    Russell, D.3
  • 39
    • 0030198916 scopus 로고    scopus 로고
    • Nucleotide sequencing and generation of an infectious clone of adeno-associated virus 3
    • Muramatsu S et al. Nucleotide sequencing and generation of an infectious clone of adeno-associated virus 3. Virology 1996; 221: 208-217.
    • (1996) Virology , vol.221 , pp. 208-217
    • Muramatsu, S.1
  • 40
    • 0032170592 scopus 로고    scopus 로고
    • Control of erythopoietin by doxycycline in mice after intramuscular injection of adeno-associated vector
    • Bohl D et al. Control of erythopoietin by doxycycline in mice after intramuscular injection of adeno-associated vector. Blood 1998; 92: 1512-1517.
    • (1998) Blood , vol.92 , pp. 1512-1517
    • Bohl, D.1
  • 41
    • 0031834034 scopus 로고    scopus 로고
    • Adeno-associated virus-mediated delivery of erythropoietin leads to sustained elevation of hematocrit in nonhuman primates
    • Zhou S et al. Adeno-associated virus-mediated delivery of erythropoietin leads to sustained elevation of hematocrit in nonhuman primates. Gene Therapy 1998; 5: 665-670.
    • (1998) Gene Therapy , vol.5 , pp. 665-670
    • Zhou, S.1
  • 42
    • 0343399184 scopus 로고    scopus 로고
    • Direct intracranial injection of recombinant adeno-associated virus at birth corrects lysosomal storage and improves cognitive function in the mucopolysaccharidosis VII mouse
    • Washington, DC, 12a (Abstr. 43)
    • Frisella Jr W et al. Direct intracranial injection of recombinant adeno-associated virus at birth corrects lysosomal storage and improves cognitive function in the mucopolysaccharidosis VII mouse. American Society of Gene Therapy, 2nd Annual Meeting, Washington, DC, 1999, 12a (Abstr. 43).
    • (1999) American Society of Gene Therapy, 2nd Annual Meeting
    • Frisella W., Jr.1
  • 43
    • 0031880563 scopus 로고    scopus 로고
    • Ribozyme rescue of photoreceptor cells in a transgenic rat model of autosomal dominant retinitis pigmentosa
    • Lewin A et al. Ribozyme rescue of photoreceptor cells in a transgenic rat model of autosomal dominant retinitis pigmentosa. Nature Med 1998; 4: 967-971.
    • (1998) Nature Med , vol.4 , pp. 967-971
    • Lewin, A.1
  • 44
    • 0029741735 scopus 로고    scopus 로고
    • Safety of single-dose administration of an adeno-associated virus (AAV)-CFTR in the primate lung
    • Conrad C et al. Safety of single-dose administration of an adeno-associated virus (AAV)-CFTR in the primate lung. Gene Therapy 1996; 3: 658-668.
    • (1996) Gene Therapy , vol.3 , pp. 658-668
    • Conrad, C.1
  • 45
    • 0032853032 scopus 로고    scopus 로고
    • Repeated delivery of adeno-associated virus vectors to the rabbit airway
    • Beck S et al. Repeated delivery of adeno-associated virus vectors to the rabbit airway. J Virol 1999; 73: 9446-9455.
    • (1999) J Virol , vol.73 , pp. 9446-9455
    • Beck, S.1
  • 46
    • 0032490381 scopus 로고    scopus 로고
    • Efficient and persistent gene transfer of AAV-CFTR in maxillary sinus
    • Wagner J et al. Efficient and persistent gene transfer of AAV-CFTR in maxillary sinus. Lancet 1998; 351: 1702-1703.
    • (1998) Lancet , vol.351 , pp. 1702-1703
    • Wagner, J.1
  • 47
    • 0032862984 scopus 로고    scopus 로고
    • Persistent expression of canine factor IX in hemophilia B canines
    • Chao HJ et al. Persistent expression of canine factor IX in hemophilia B canines. Gene Therapy 1999; 6: 1695-1704.
    • (1999) Gene Therapy , vol.6 , pp. 1695-1704
    • Chao, H.J.1
  • 48
    • 17444387438 scopus 로고    scopus 로고
    • Long-term correction of canine hemophilia B by gene therapy of blood coagulation factor IX mediated by adeno-associated viral vector
    • Herzog RW et al. Long-term correction of canine hemophilia B by gene therapy of blood coagulation factor IX mediated by adeno-associated viral vector. Nature Med 1999; 5: 56-63.
    • (1999) Nature Med , vol.5 , pp. 56-63
    • Herzog, R.W.1


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