Improved adeno-associated virus (AAV) serotype 1 and 5 vectors for gene therapy

Scientific Reports

Volumn 6, Issue , 2013, Pages

  Sen, Dwaipayan ^a   Balakrishnan, Balaji ^a   Gabriel, Nishanth ^a   Agrawal, Prachi ^b   Roshini, Vaani ^a   Samuel, Rekha ^b   Srivastava, Alok ^a,b   Jayandharan, Giridhara R ^a,b  

^a Department of Hematology ^*  (India)

^b CHRISTIAN MEDICAL COLLEGE   (India)

Author keywords

[No Author keywords available]

Indexed keywords

CAPSID PROTEIN; ENHANCED GREEN FLUORESCENT PROTEIN; GREEN FLUORESCENT PROTEIN; NEUTRALIZING ANTIBODY; SERINE; THREONINE;

ADENO ASSOCIATED VIRUS; ANIMAL; ARTICLE; C57BL MOUSE; CHEMISTRY; CHO CELL; GENE THERAPY; GENE VECTOR; GENETIC TRANSDUCTION; GENETICS; HAMSTER; IMMUNOLOGY; LIVER; METABOLISM; MOUSE; MUTATION; PHOSPHORYLATION; SITE DIRECTED MUTAGENESIS; UBIQUITINATION; VIROLOGY;

ANIMALS; ANTIBODIES, NEUTRALIZING; CAPSID PROTEINS; CHO CELLS; CRICETINAE; DEPENDOVIRUS; GENETIC THERAPY; GENETIC VECTORS; GREEN FLUORESCENT PROTEINS; LIVER; MICE; MICE, INBRED C57BL; MUTAGENESIS, SITE-DIRECTED; MUTATION; PHOSPHORYLATION; SERINE; THREONINE; TRANSDUCTION, GENETIC; UBIQUITINATION;

EID: 84878737483     PISSN: None     EISSN: 20452322     Source Type: Journal    
DOI: 10.1038/srep01832     Document Type: Article

References (41)

1. Bennett, J., Ashtari, M., Wellman, J., Marshall, K. A., Cyckowski, L. L., Chung, D. C. et al. AAV2 gene therapy readministration in three adults with congenital blindness. Sci Transl Med 4, 120ra115 (2012).
2. Bartel, M. A., Weinstein, J. R. & Schaffer, D. V. Directed evolution of novel adeno-associated viruses for therapeutic gene delivery. Gene Ther 19, 694-700 (2012).
3. Grieger, J. C. & Samulski, R. J. Adeno-associated virus as a gene therapy vector: vector development, production and clinical applications. Adv Biochem Eng Biotechnol 99, 119-145 (2005). (Pubitemid 44617179)
4. Daya, S. & Berns, K. I. Gene therapy using adeno-associated virus vectors. Clin Microbiol Rev 21, 583-593 (2008).
5. High, K. A. The gene therapy journey for hemophilia: are we there yet? Blood 120, 4482-4487 (2012).
6. Manno, C. S., Pierce, G. F., Arruda, V. R., Glader, B., Ragni, M., Rasko, J. J. et al. Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response. Nat Med 12, 342-347 (2006). (Pubitemid 43355084)
7. Buchlis, G., Podsakoff, G. M., Radu, A., Hawk, S. M., Flake, A. W., Mingozzi, F. et al. Factor IX expression in skeletal muscle of a severe hemophilia B patient 10 years after AAV-mediated gene transfer. Blood 119, 3038-3041 (2012).
8. Mingozzi, F., Maus, M. V., Hui, D. J., Sabatino, D. E., Murphy, S. L., Rasko, J. E. et al. CD8(1) T-cell responses to adeno-associated virus capsid in humans. Nat Med 13, 419-422 (2007). (Pubitemid 46559826)
9. Madsen, D., Cantwell, E. R., O'Brien, T., Johnson, P. A. & Mahon, B. P. Adeno-associated virus serotype 2 induces cell-mediated immune responses directed against multiple epitopes of the capsid protein VP1. J Gen Virol 90, 2622-2633 (2009).
10. Zincarelli, C., Soltys, S., Rengo, G. & Rabinowitz, J. E. Analysis of AAV serotypes 1-9 mediated gene expression and tropism in mice after systemic injection. Mol Ther 16, 1073-1080 (2008). (Pubitemid 351737072)
11. Hu, C. & Lipshutz, G. S. AAV-based neonatal gene therapy for hemophilia A: long-term correction and avoidance of immune responses in mice. Gene Ther 19, 1166-1176 (2012).
12. Maheshri, N., Koerber, J. T., Kaspar, B. K. & Schaffer, D. V. Directed evolution of adeno-associated virus yields enhanced gene delivery vectors. Nat Biotechnol 24, 198-204 (2006). (Pubitemid 43221704)
13. Finn, J. D., Hui, D., Downey, H. D., Dunn, D., Pien, G. C., Mingozzi, F. et al. Proteasome inhibitors decrease AAV2 capsid derived peptide epitope presentation on MHC class I following transduction. Mol Ther 18, 135-142 (2010).
14. Mingozzi, F., Hasbrouck, N. C., Basner-Tschakarjan, E., Edmonson, S. A., Hui, D. J., Sabatino, D. E. et al. Modulation of tolerance to the transgene product in a nonhuman primate model of AAV-mediated gene transfer to liver. Blood 110, 2334-2341 (2007). (Pubitemid 47523153)
15. Nathwani, A. C., Tuddenham, E. G., Rangarajan, S., Rosales, C., McIntosh, J., Linch, D. C. et al. Adenovirus-associated virus vector-mediated gene transfer in hemophilia B. N Engl J Med 365, 2357-2365 (2011).
16. Gabriel, N., Hareendran, S., Sen, D., Gadkari, R. A., Sudha, G., Selot, R. et al. Bio-engineering of AAV-2 capsid at specific serine, threonine or lysine residues improves its transduction efficiencyin vitro and in vivo. Hum Gene Ther Methods (2013). EPUB doi: 10.1089/hgtb.2012.194.
17. Zhong, L., Li, B., Mah, C. S., Govindasamy, L., Agbandje-McKenna, M., Cooper, M. et al. Next generation of adeno-associated virus 2 vectors: point mutations in tyrosines lead to high-efficiency transduction at lower doses. Proc Natl Acad Sci U S A 105, 7827-7832 (2008). (Pubitemid 351872723)
18. Sen, D., Gadkari, R. A., Sudha, G., Gabriel, N., Sathish Kumar, Y., Selot, R. et al. Targeted modifications in adeno-associated virus (AAV) serotype-8 capsid improves its hepatic gene transfer efficiency in vivo. Hum Gene Ther Methods (2013). EPUB doi: 10.1089/hgtb.2012.195.
19. Boutin, S., Monteilhet, V., Veron, P., Leborgne, C., Benveniste, O., Montus, M. F. et al. Prevalence of serum IgG and neutralizing factors against adeno-associated virus (AAV) types 1, 2, 5, 6, 8, and 9 in the healthy population: implications for gene therapy using AAV vectors. Hum Gene Ther 21, 704-712 (2010).
20. Calcedo, R., Vandenberghe, L. H., Gao, G., Lin, J. & Wilson, J. M. Worldwide epidemiology of neutralizing antibodies to adeno-associated viruses. J Infect Dis 199, 381-390 (2009).
21. Li, C., Narkbunnam, N., Samulski, R. J., Asokan, A., Hu, G., Jacobson, L. J. et al. Neutralizing antibodies against adeno-associated virus examined prospectively in pediatric patients with hemophilia. Gene Ther 19, 288-294 (2012).
22. Murphy, S. L., Li, H., Zhou, S., Schlachterman, A. & High, K. A. Prolonged susceptibility to antibody-mediated neutralization for adeno-associated vectors targeted to the liver. Mol Ther 16, 138-145 (2008).
23. Paneda, A., Vanrell, L., Mauleon, I., Crettaz, J. S., Berraondo, P., Timmermans, E. J. et al. Effect of adeno-associated virus serotype and genomic structure on liver transduction and biodistribution in mice of both genders. Hum Gene Ther 20, 908-917 (2009).
24. Xiao, W., Chirmule, N., Berta, S. C., McCullough, B., Gao, G. & Wilson, J.M. Gene therapy vectors based on adeno-associated virus type 1. J Virol 73, 3994-4003 (1999). (Pubitemid 29189844)
25. Lorain, S., Gross, D. A., Goyenvalle, A., Danos, O., Davoust, J. & Garcia, L. Transient immunomodulation allows repeated injections of AAV1 and correction of muscular dystrophy in multiple muscles. Mol Ther 16, 541-547 (2008). (Pubitemid 351314994)
26. Zabner, J., Seiler, M., Walters, R., Kotin, R. M., Fulgeras, W., Davidson, B. L. et al. Adeno-associated virus type 5 (AAV5) but not AAV2 binds to the apical surfaces of airway epithelia and facilitates gene transfer. J Virol 74, 3852-3858 (2000). (Pubitemid 30180327)
27. Hildinger, M., Auricchio, A., Gao, G., Wang, L., Chirmule, N. & Wilson, J. M. Hybrid vectors based on adeno-associated virus serotypes 2 and 5 for muscle-directed gene transfer. J Virol 75, 6199-6203 (2001). (Pubitemid 32553151)
28. Nathwani, A. C., Gray, J. T., McIntosh, J., Ng, C. Y., Zhou, J., Spence, Y. et al. Safe and efficient transduction of the liver after peripheral vein infusion of self-complementary AAV vector resultsinstable therapeutic expressionofhuman FIX in nonhuman primates. Blood 109, 1414-1421 (2007). (Pubitemid 46239572)
29. Halbert, C. L., Miller, A. D., McNamara, S., Emerson, J., Gibson, R. L., Ramsey, B. et al. Prevalence of neutralizing antibodies against adeno-associated virus (AAV) types 2, 5, and 6 in cystic fibrosis and normal populations: Implications for gene therapy using AAV vectors. Hum Gene Ther 17, 440-447 (2006).
30. Gaudet, D., Methot, J., Dery, S., Brisson, D., Essiembre, C., Tremblay, G. et al. Efficacy and long-term safety of alipogene tiparvovec (AAV1-LPL(S447X)) gene therapy for lipoprotein lipase deficiency: an open-label trial. Gene Ther (2012).
31. Zincarelli, C., Soltys, S., Rengo, G., Koch, W. J. & Rabinowitz, J. E. Comparative cardiac gene delivery of adeno-associated virus serotypes 1-9 reveals that AAV6 mediates the most efficient transduction in mouse heart. Clin Transl Sci 3, 81-89 (2010).
32. Gao, G., Vandenberghe, L. H. & Wilson, J. M. New recombinant serotypes of AAV vectors. Curr Gene Ther 5, 285-297 (2005). (Pubitemid 40895013)
33. Wu, Z., Asokan, A. & Samulski, R. J. Adeno-associated virus serotypes: vector toolkit for human gene therapy. Mol Ther 14, 316-327 (2006). (Pubitemid 44184972)
34. Rebuffat, A., Harding, C. O., Ding, Z. & Thony, B. Comparison of adeno-associated virus pseudotype 1, 2, and 8 vectors administered by intramuscular injection in the treatment of murine phenylketonuria. Hum Gene Ther 21, 463-477 (2010).
35. Markakis, E. A., Vives, K. P., Bober, J., Leichtle, S., Leranth, C., Beecham, J. et al. Comparative transduction efficiency of AAV vector serotypes 1-6 in the substantia nigra and striatum of the primate brain. Mol Ther 18, 588-593 (2010).
36. Wu, Z., Asokan, A., Grieger, J. C., Govindasamy, L., Agbandje-McKenna, M. & Samulski, R. J. Single amino acid changes can influence titer, heparin binding, and tissue tropism in different adeno-associated virus serotypes. J Virol 80, 11393-11397 (2006). (Pubitemid 44706580)
37. Hida, K., Won, S. Y., Di Pasquale, G., Hanes, J., Chiorini, J. A. & Ostermeier, M. Sites in the AAV5 capsid tolerant to deletions and tandem duplications. Arch Biochem Biophys 496, 1-8 (2010).
38. Aneja, M. K. & Rudolph, C. Gene therapy of surfactant protein B deficiency. Curr Opin Mol Ther 8, 432-438 (2006). (Pubitemid 44566784)
39. Flotte, T. R., Ng, P., Dylla, D. E., McCray, P. B., Jr., Wang, G., Kolls, J. K. et al. Viral vector-mediated and cell-based therapies for treatment of cystic fibrosis. Mol Ther 15, 229-241 (2007). (Pubitemid 46111871)
40. Kube, D. M. & Srivastava, A. Quantitative DNA slot blot analysis: inhibition of DNA binding tomembranesbymagnesium ions. Nucleic Acids Res 25, 3375-3376 (1997). (Pubitemid 27338702)
41. Aurnhammer, C., Haase, M., Muether, N., Hausl, M., Rauschhuber, C., Huber, I. et al. Universal Real-Time PCR for the Detection and Quantification of Adeno-Associated Virus Serotype 2-Derived Inverted Terminal Repeat Sequences. Hum Gene Ther 23, 18-28 (2012).