Enhancing transduction of the liver by adeno-associated viral vectors

Gene Therapy

Volumn 16, Issue 1, 2009, Pages 60-69

  Nathwani, A C ^a,b   Cochrane, M ^a   McIntosh, J ^a   Ng, C Y C ^c   Zhou, J ^c   Gray, J T ^c   Davidoff, A M ^c  

^a UNIVERSITY COLLEGE LONDON   (United Kingdom)

^b NHS BLOOD AND TRANSPLANT   (United Kingdom)

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

Author keywords

[No Author keywords available]

Indexed keywords

BETA GALACTOSIDASE; BLOOD CLOTTING FACTOR 9; BORTEZOMIB; CAPSID PROTEIN; MESSENGER RNA; PARVOVIRUS VECTOR;

ADENO ASSOCIATED VIRUS; ANIMAL CELL; ANIMAL EXPERIMENT; ARTICLE; CONTROLLED STUDY; DRUG POTENCY; FEMALE; GENE EXPRESSION; GENE THERAPY; GENE TRANSFER; GENETIC TRANSDUCTION; LIVER CELL; MALE; MOUSE; NONHUMAN; PRIORITY JOURNAL; PROBABILITY; PROCESS OPTIMIZATION; PSEUDOTYPING; SEROTYPE; SEX DIFFERENCE; TRANSGENE; VIRAL GENE DELIVERY SYSTEM;

ANIMALS; ANTIBODIES, VIRAL; BORONIC ACIDS; DEPENDOVIRUS; FACTOR IX; FEMALE; GENE EXPRESSION; GENE THERAPY; GENETIC VECTORS; HUMANS; INJECTIONS, INTRAVENOUS; LIVER; MALE; MICE; MICE, INBRED BALB C; MICE, INBRED C57BL; PROTEASE INHIBITORS; PYRAZINES; TRANSDUCTION, GENETIC; TRANSGENES;

ADENO-ASSOCIATED VIRUS; ADENOVIRIDAE; MIRIDAE; MURINAE; MUS;

EID: 58149459993     PISSN: 09697128     EISSN: 14765462     Source Type: Journal    
DOI: 10.1038/gt.2008.137     Document Type: Article

References (40)

1. Nathwani AC, Benjamin R, Nienhuis AW, Davidoff AM. Current status and prospects for gene therapy. Vox Sang 2004; 87: 73-81.
2. Manno CS, Chew AJ, Hutchison S, Larson PJ, Herzog RW, Arruda VR et al. AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B. Blood 2003; 101: 2963-2972.
3. Manno CS, Arruda VR, Pierce GF, Glader B, Ragni M, Rasko J et al. Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response. Nat Med 2006; 12: 342-347.
4. Flotte TR, Brantly ML, Spencer LT, Byrne BJ, Spencer CT, Baker DJ et al. Phase I trial of intramuscular injection of a recombinant adeno-associated virus alpha 1-antitrypsin (rAAV2-CB-hAAT) gene vector to AAT-deficient adults. Hum Gene Ther 2004; 15: 93-128.
5. Stedman H, Wilson JM, Finke R, Kleckner AL, Mendell J. Phase I clinical trial utilizing gene therapy for limb girdle muscular dystrophy: Alpha-, beta-, gamma-, or delta-sarcoglycan gene delivered with intramuscular instillations of adeno-associated vectors. Hum Gene Ther 2000; 11: 777-790.
6. Kaplitt MG, Feigin A, Tang C, Fitzsimons HL, Mattis P, Lawlor PA et al. Safety and tolerability of gene therapy with an adeno-associated virus (AAV) borne GAD gene for Parkinson's disease: An open label, phase I trial. Lancet 2007; 369: 2097-2105.
7. Davidoff AM, Gray JT, Ng CY, Zhang Y, Zhou J, Spence Y et al. 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. Mol Ther 2005; 11: 875-888.
8. Nathwani AC, Gray JT, Ng CY, Zhou J, Spence Y, Waddington SN et al. Self complementary adeno-associated virus vectors containing a novel liver-specific human factor IX expression cassette enable highly efficient transduction of murine and nonhuman primate liver. Blood 2006; 107: 2653-2661.
9. Nathwani AC, Gray JT, McIntosh J, Ng CY, Zhou J, Spence Y et al. Safe and efficient transduction of the liver after peripheral vein infusion of self complementary AAV vector results in stable therapeutic expression of human FIX in nonhuman primates. Blood 2007; 109 1414-1421.
10. Gao GP, Alvira MR, Wang L, Calcedo R, Johnston J, Wilson JM. Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy. Proc Natl Acad Sci USA 2002; 99: 11854-11859.
11. Gao G, Vandenberghe LH, Alvira MR, Lu Y, Calcedo R, Zhou X et al. Clades of adeno-associated viruses are widely disseminated in human tissues. J Virol 2004; 78: 6381-6388.
12. Mackus WJ, Frakking FN, Grummels A, Gamadia LE, De Bree GJ, Hamann D et al. Expansion of CMV-specific CD8+CD45RA+. Blood 2003; 102: 1057-1063.
13. Yan Z, Zak R, Luxton GW, Ritchie TC, Bantel-Schaal U, Engelhardt JF. Ubiquitination of both adeno-associated virus type 2 and 5 capsid proteins affects the transduction efficiency of recombinant vectors. J Virol 2002; 76: 2043-2053.
14. Fisher KJ, Gao GP, Weitzman MD, DeMatteo R, Burda JF, Wilson JM. Transduction with recombinant adeno-associated virus for gene therapy is limited by leading-strand synthesis. J Virol 1996; 70: 520-532.
15. Davidoff AM, Ng CY, Zhou J, Spence Y, Nathwani AC. Sex significantly influences transduction of murine liver by recombinant adeno-associated viral vectors through an androgen-dependent pathway. Blood 2003; 102: 480-488.
16. Duan D, Yue Y, Yan Z, Yang J, Engelhardt JF. Endosomal processing limits gene transfer to polarized airway epithelia by adeno-associated virus. J Clin Invest 2000; 105: 1573-1587.
17. Thomas CE, Storm TA, Huang Z, Kay MA. Rapid uncoating of vector genomes is the key to efficient liver transduction with pseudotyped adeno-associated virus vectors. J Virol 2004; 78: 3110-3122.
18. McCarty DM, Fu H, Monahan PE, Toulson CE, Naik P, Samulski RJ. Adeno-associated virus terminal repeat (TR) mutant generates self-complementary vectors to overcome the rate-limiting step to transduction in vivo. Gene Therapy 2003; 10: 2112-2118.
19. Wang Z, Ma HI, Li J, Sun L, Zhang J, Xiao X. Rapid and highly efficient transduction by double-stranded adeno-associated virus vectors in vitro and in vivo. Gene Therapy 2003; 10: 2105-2111.
20. Fisher KJ, Jooss K, Alston J, Yang Y, Haecker SE, High K et al. Recombinant adeno-associated virus for muscle directed gene therapy. Nat Med 1997; 3: 306-312.
21. Schwarzmeier JD, Hubmann R, Duchler M, Jager U, Shehata M. Regulation of CD23 expression by Notch2 in B-cell chronic lymphocytic leukemia. Leuk Lymphoma 2005; 46: 157-165.
22. De BP, Heguy A, Hackett NR, Ferris B, Leopold PL, Lee J et al. High levels of persistent expression of alpha1-antitrypsin mediated by the nonhuman primate serotype rh.10 adeno-associated virus despite preexisting immunity to common human adeno-associated viruses. Mol Ther 2006; 13: 67-76.
23. Nathwani AC, Davidoff A, Hanawa H, Zhuo F, Vanin EF, Nienhuis AW. Factors influencing in-vivo transduction by recombinant adeno-associated viral vectors expressing the human factor IX cDNA. Blood 2001; 97: 1258-1265.
24. Davidoff AM, Ng CY, Zhang Y, Streck CJ, Mabry SJ, Barton SH et al. Careful decoy receptor titering is required to inhibit tumor angiogenesis while avoiding adversely altering VEGF bioavailability. Mol Ther 2005; 11: 300-310.
25. Nathwani AC, Persons DA, Stevenson SC, Frare P, McClelland A, Nienhuis AW et al. Adenovirus-mediated expresssion of the murine ecotropic receptor facilitates transduction of human hematopoietic cells with an ecotropic retroviral vector. Gene Therapy 1999; 6: 1456-1468.
26. Wang L, Takabe K, Bidlingmaier SM, Ill CR, Verma IM. Sustained correction of bleeding disorder in hemophilia B mice by gene therapy. Proc Natl Acad Sci USA 1999; 96: 3906-3910.
27. Chiorini JA, Yang L, Liu Y, Safer B, Kotin RM. Cloning of adeno-associated virus type 4 (AAV4) and generation of recombinant AAV4 particles. J Virol 1997; 71: 6823-6833.
28. Chiorini JA, Kim F, Yang L, Kotin RM. Cloning and characterization of adeno-associated virus type 5. J Virol 1999; 73: 1309-1319.
29. Rutledge EA, Halbert CL, Russell DW. Infectious clones and vectors derived from adeno-associated virus (AAV) serotypes other than AAV type 2. J Virol 1998; 72: 309-319.
30. Akache B, Grimm D, Pandey K, Yant SR, Xu H, Kay MA. The 37/ 67-kilodalton laminin receptor is a receptor for adeno-associated virus serotypes 8, 2, 3, and 9. J Virol 2006; 80: 9831-9836.
31. Fournier S, Delespesse G, Rubio M, Biron G, Sarfati M. CD23 antigen regulation and signaling in chronic lymphocytic leukemia. J Clin Invest 1992; 89: 1312-1321.
32. Ogura T, Mizukami H, Mimuro J, Madoiwa S, Okada T, Matsushita T et al. Utility of intraperitoneal administration as a route of AAV serotype 5 vector-mediated neonatal gene transfer. J Gene Med 2006; 8: 990-997.
33. Sarkar R, Mucci M, Addya S, Tetreault R, Bellinger DA, Nichols TC et al. Long-term efficacy of adeno-associated virus serotypes 8 and 9 in hemophilia a dogs and mice. Hum Gene Ther 2006; 17: 427-439.
34. Wang L, Calcedo R, Nichols TC, Bellinger DA, Dillow A, Verma IM et al. Sustained correction of disease in naive and AAV2-pretreated hemophilia B dogs: AAV2/8-mediated, liver-directed gene therapy. Blood 2005; 105: 3079-3086.
35. Wu Z, Sun J, Zhang T, Yin C, Yin F, Van Dyke T et al. Optimization of self-complementary AAV vectors for liverdirected expression results in sustained correction of hemophilia B at low vector dose. Mol Ther 2008; 16: 280-289.
36. Montini E, Cesana D, Schmidt M, Sanvito F, Ponzoni M, Bartholomae C et al. Hematopoietic stem cell gene transfer in a tumor-prone mouse model uncovers low genotoxicity of lentiviral vector integration. Nat Biotechnol 2006; 24: 687-696.
37. Humeau LM, Binder GK, Lu X, Slepushkin V, Merling R, Echeagaray P et al. Efficient lentiviral vector-mediated control of HIV-1 replication in CD4 lymphocytes from diverse HIV+ infected patients grouped according to CD4 count and viral load. Mol Ther 2004; 9: 902-913.
38. Davidoff AM, Ng CY, Sleep S, McIntosh J, Azam S, Zhao Y et al. Purification of recombinant adeno-associated virus type 8 vectors by ion exchange chromatography generates clinical grade vector stock. J Virol Methods 2004; 121: 209-215.
39. Nathwani AC, Davidoff AM, Hanawa H, Hu Y, Hoffer FA, Nikanorov A 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 2002; 100: 1662-1669.
40. Nathwani AC, Hanawa H, Vandergriff J, Kelly P, Vanin EF, Nienhuis AW. Efficient gene transfer into human cord blood CD34+ cells and the CD34+CD38- subset using highly purified recombinant adeno-associated viral vector preparations that are free of helper virus and wild-type AAV. Gene Therapy 2000; 7: 183-195.