Comparison of the ability of adeno-associated viral vectors pseudotyped with serotype 2, 5, and 8 capsid proteins to mediate efficient transduction of the liver in murine and nonhuman primate models

Molecular Therapy

Volumn 11, Issue 6, 2005, Pages 875-888

  Davidoff, Andrew M ^a   Gray, John T ^a   Ng, Catherine Y C ^a   Zhang, Youbin ^a   Zhou, Junfang ^a   Spence, Yunyu ^a   Bakar, Yusura ^b   Nathwani, Amit C ^b,c  

^a ST JUDE CHILDREN S RESEARCH HOSPITAL   (United States)

^b UNIVERSITY COLLEGE LONDON   (United Kingdom)

^c NHS BLOOD AND TRANSPLANT   (United Kingdom)

Author keywords

AAV serotypes; Hemophilia B gene therapy; Liver directed gene transfer

Indexed keywords

ALPHA 1 ANTITRYPSIN; APOLIPOPROTEIN C1; APOLIPOPROTEIN E; BETA ACTIN; BLOOD CLOTTING FACTOR 9; CAPSID PROTEIN; COMPLEMENTARY DNA; DOUBLE STRANDED DNA; PARVOVIRUS VECTOR; VIRUS ANTIBODY;

ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ANTIBODY SPECIFICITY; ARTICLE; CONTROLLED STUDY; DOSE RESPONSE; DRUG BLOOD LEVEL; GENE EXPRESSION; GENETIC TRANSCRIPTION; GENETIC TRANSDUCTION; LIVER; LOW DRUG DOSE; MALE; MOLECULAR BIOLOGY; MOUSE; NONHUMAN; PRIMATE; SEROTYPE; STATISTICAL SIGNIFICANCE; STEADY STATE; TRANSGENE; VIRAL GENE DELIVERY SYSTEM; VIRUS IMMUNITY; VIRUS RECOMBINANT; VIRUS TYPING;

ANIMALS; ANTIBODY FORMATION; CAPSID PROTEINS; DEPENDOVIRUS; DNA, VIRAL; GENE THERAPY; GENETIC VECTORS; HEMOPHILIA B; HEPATOCYTES; LIVER; MACACA MULATTA; MALE; MICE; SEROTYPING; TRANSDUCTION, GENETIC; TRANSGENES;

MIRIDAE; MURINAE; PRIMATES;

EID: 19444365150     PISSN: 15250016     EISSN: None     Source Type: Journal    
DOI: 10.1016/j.ymthe.2004.12.022     Document Type: Article

References (35)

1. T.R. Flotte Immune responses to recombinant adeno-associated virus vectors: Putting preclinical findings into perspective Hum. Gene Ther. 15 2004 716-717
2. K. High et al. Immune response to AAV and to factor IX in a Phase I study of AAV-mediated liver directed gene transfer for hemophilia B Mol. Ther. 9 2004 S383-S384
3. A.C. Nathwani et al. Factors influencing in-vivo transduction by recombinant adeno-associated viral vectors expressing the human factor IX cDNA Blood 97 2001 1258-1265
4. V.R. Arruda et al. Lack of germ-line transmission of vector sequences following systemic administration of recombinant AAV-2 vector in males Mol. Ther. 4 2001 586-592
5. J.D. Mount et al. Sustained phenotypic correction of hemophilia B dogs with a factor IX null mutation by liver-directed gene therapy Blood 99 2002 2670-2676
6. R.O. Snyder et al. Correction of hemophilia B in canine and murine models using recombinant adeno-associated viral vectors Nat. Med. 5 1999 64-70
7. F. Mingozzi et al. Improved hepatic gene transfer by using an adeno-associated virus serotype 5 vector J. Virol. 76 2002 10497-10502
8. J.E. Rabinowitz et al. Cross-packaging of a single adeno-associated virus (AAV) type 2 vector genome into multiple AAV serotypes enables transduction with broad specificity J. Virol. 76 2002 791-801
9. M. Hildinger A. Auricchio E. O'Connor and J.M. Wilson Hybrid vectors of adeno-associated virus type 2 and 5 efficiently transduce muscle, lung and hepatocytes Mol. Ther. 3 2001 S187 [Abstract]
10. G.P. Gao et al. Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy Proc. Natl. Acad. Sci. USA 99 2002 11854-11859
11. J.A. Chiorini F. Kim L. Yang and R.M. Kotin Cloning and characterization of adeno-associated virus type 5 J. Virol. 73 1999 1309-1319
12. D. Grimm et al. Preclinical in vivo evaluation of pseudotyped adeno-associated virus vectors for liver gene therapy Blood 102 2003 2412-2419
13. P.G. Di et al. Identification of PDGFR as a receptor for AAV-5 transduction Nat. Med. 9 2003 1306-1312
14. R. Sarkar et al. AAV 8 serotype: Total correction of hemophilia A mice and partial correction in dogs with canine FVIII Mol. Ther. 7 2003 S26
15. C.E. Thomas T.A. Storm Z. Huang and M.A. Kay Rapid uncoating of vector genomes is the key to efficient liver transduction with pseudotyped adeno-associated virus vectors J. Virol. 78 2004 3110-3122
16. A.C. Nathwani et al. Sustained high-level expression of human factor IX (hFIX) after liver-targeted delivery of recombinant adeno-associated virus encoding the hFIX gene in rhesus macaques Blood 100 2002 1662-1669
17. J. Qiu R. Nayak G.E. Tullis and D.J. Pintel Characterization of the transcription profile of adeno-associated virus type 5 reveals a number of unique features compared to previously characterized adeno-associated viruses J. Virol. 76 2002 12435-12447
18. H. Nakai et al. A limited number of transducible hepatocytes restricts a wide-range linear vector dose response in recombinant adeno-associated virus-mediated liver transduction J. Virol. 76 2002 11343-11349
19. I.E. Alexander D.W. Russell and A.D. Miller Transfer of contaminants in adeno-associated virus vector stocks can mimic transduction and lead to artifactual results Hum. Gene Ther. 8 1997 1911-1920
20. R. Sarkar et al. Total correction of hemophilia A mice with canine FVIII using an AAV 8 serotype Blood 103 2004 1253-1260
21. R.W. Herzog et al. Long-term correction of canine hemophilia B by gene transfer of blood coagulation factor IX mediated by adeno-associated viral vector Nat. Med. 5 1999 56-63
22. S.C. Hung M.S. Kang and E. Kieff Maintenance of Epstein-Barr virus (EBV) oriP-based episomes requires EBV-encoded nuclear antigen-1 chromosome-binding domains, which can be replaced by high-mobility group-I or histone H1 Proc. Natl. Acad. Sci. USA 98 2001 1865-1870
23. R. Wade-Martins J. Frampton and M.R. James Long-term stability of large insert genomic DNA episomal shuttle vectors in human cells Nucleic Acids Res. 27 1999 1674-1682
24. H. Nakai et al. AAV serotype 2 vectors preferentially integrate into active genes in mice Nat. Genet. 34 2003 297-302
25. C.L. Halbert E.A. Rutledge J.M. Allen D.W. Russell and A.D. Miller Repeat transduction in the mouse lung by using adeno-associated virus vectors with different serotypes J. Virol. 74 2000 1524-1532
26. W.P. Parks D.W. Boucher J.L. Melnick L.H. Taber and M.D. Yow Seroepidemiological and ecological studies of the adenovirus-associated satellite viruses Infect. Immun. 2 1970 716-722
27. K. Erles P. Sebokova and J.R. Schlehofer Update on the prevalence of serum antibodies (IgG and IgM) to adeno-associated virus (AAV) J. Med. Virol. 59 1999 406-411
28. A.M. Davidoff C.Y. Ng J. Zhou Y. Spence and A.C. Nathwani Sex significantly influences transduction of murine liver by recombinant adeno-associated viral vectors through an androgen-dependent pathway Blood 102 2003 480-488
29. C.H. Miao et al. Inclusion of the hepatic locus control region, an intron, and untranslated region increases and stabilizes hepatic factor IX gene expression in vivo but not in vitro Mol. Ther. 1 2000 522-532
30. D.G. Hafenrichter et al. Quantitative evaluation of liver-specific promoters from retroviral vectors after in vivo transduction of hepatocytes Blood 84 1994 3394-3404
31. X. Xiao J. and Li R.J. Samulski Production of high-titer recombinant adeno-associated virus vectors in the absence of helper adenovirus J. Virol. 72 1998 2224-2232
32. K.R. Clark X. Liu J.P. McGrath and P.R. Johnson Highly purified recombinant adeno-associated virus vectors are biologically active and free of detectable helper and wild-type viruses Hum. Gene Ther. 10 1999 1031-1039
33. A. Auricchio E. O'Connor M. Hildinger and J.M. Wilson A single-step affinity column for purification of serotype-5 based adeno-associated viral vectors Mol. Ther. 4 2001 372-374
34. A.M. Davidoff et al. Purification of recombinant adeno-associated virus type 8 vectors by ion exchange chromatography generates clinical grade vector stock J. Virol. Methods 121 2004 209-215
35. - subset using highly purified recombinant adeno-associated viral vector preparations that are free of helper virus and wild-type AAV Gene Ther. 7 2000 183-195