Skip to main content
SpringerLink
Log in
Book cover

Gene Therapy and Gene Delivery Systems pp 147–167Cite as

Advanced Targeting Strategies for Murine Retroviral and Adeno-associated Viral Vectors

  • Chapter
  • First Online:

Part of the book series: Advances in Biochemical Engineering/Biotechnology ((ABE,volume 99))

Abstract

Targeted gene delivery involves broadening viral tropism to infect previously nonpermissive cells, replacing viral tropism to infect a target cell exclusively, or stealthing the vector against nonspecific interactions with host cells and proteins. These approaches offer the potential advantages of enhanced therapeutic effects, reduced side effects, lowered dosages, and enhanced therapeutic economics. This review will discuss a variety of targeting strategies, both genetic and nongenetic, for re-engineering the tropism of two representative enveloped and nonenveloped viruses, murine retrovirus and adeno-associated virus. Basic advances in understanding the structural biology and virology of the parent viruses have aided rational design efforts to engineer novel properties into the viral attachment proteins. Furthermore, even in the absence of basic, mechanistic knowledge of viral function, high-throughput library and directed evolution approaches can yield significant improvements in vector function. These two complementary strategies offer the potential to gain enhanced molecular control over vector properties and overcome challenges in generating high titer, stealthy, retargeted vectors.

This is a preview of subscription content, log in via an institution.

Preview

Unable to display preview. Download preview PDF.

Unable to display preview. Download preview PDF.

References

  1. Robson T, Hirst DG (2003) J Biomed Biotechnol 2003:110–137

    Article  Google Scholar 

  2. Romano G (2004) Drug News Perspect 17:85–90

    CAS  Google Scholar 

  3. Burton EA, Bai Q, Goins WF, Glorioso JC (2001) Adv Drug Deliv Rev 53:155–170

    Article  CAS  Google Scholar 

  4. Wickham TJ (2003) Nat Med 9:135–139

    Article  CAS  Google Scholar 

  5. Cavazzana-Calvo M, Hacein-Bey S, de Saint Basile G, Gross F, Yvon E et al. (2000) Science 288:669–672

    Article  CAS  Google Scholar 

  6. Hacein-Bey-Abina S, Von Kalle C, Schmidt M, McCormack MP, Wulffraat N et al. (2003) Science 302:415–419

    Article  CAS  Google Scholar 

  7. Edelstein M (2004) The Journal of Gene Medicine website for gene therapy clinical trials worldwide. Wiley, Chichester, UK (see http://www.wiley.co.uk/genmed/clinical/, last accessed 20th September 2005)

    Google Scholar 

  8. Fields BN, Knipe DM, Howley PM, Griffin DE (2001) Fields Virology. Lippincott Williams & Wilkins, Philadelphia, PA

    Google Scholar 

  9. Kayman SC, Park H, Saxon M, Pinter A (1999) J Virol 73:1802–1808

    CAS  Google Scholar 

  10. Wu BW, Lu J, Gallaher TK, Anderson WF, Cannon PM (2000) Virology 269:7–17

    Article  CAS  Google Scholar 

  11. Rothenberg SM, Olsen MN, Laurent LC, Crowley RA, Brown PO (2001) J Virol 75:11851–11862

    Article  CAS  Google Scholar 

  12. Naldini L, Blomer U, Gallay P, Ory D, Mulligan R et al. (1996) Science 272:263–267

    CAS  Google Scholar 

  13. Russell DW, Miller AD (1996) J Virol 70:217–222

    CAS  Google Scholar 

  14. Wilson C, Reitz MS, Okayama H, Eiden MV (1989) J Virol 63:2374–2378

    CAS  Google Scholar 

  15. Burns JC, Friedmann T, Driever W, Burrascano M, Yee JK (1993) P Natl Acad Sci USA 90:8033–8037

    CAS  Google Scholar 

  16. Kasahara N, Dozy AM, Kan YW (1994) Science 266:1373–1376

    CAS  Google Scholar 

  17. Cosset FL, Morling FJ, Takeuchi Y, Weiss RA, Collins MK et al. (1995) J Virol 69:6314–6322

    CAS  Google Scholar 

  18. Nguyen TH, Pages JC, Farge D, Briand P, Weber A (1998) Hum Gene Ther 9:2469–2479

    Article  CAS  Google Scholar 

  19. Valsesia-Wittmann S, Drynda A, Deleage G, Aumailley M, Heard JM et al. (1994) J Virol 68:4609–4619

    CAS  Google Scholar 

  20. Gollan TJ, Green MR (2002) J Virol 76:3558–3563

    CAS  Google Scholar 

  21. Somia NV, Zoppe M, Verma IM (1995) P Natl Acad Sci USA 92:7570–7574

    CAS  Google Scholar 

  22. Chu TH, Dornburg R (1995) J Virol 69:2659–2663

    CAS  Google Scholar 

  23. Chu TH, Dornburg R (1997) J Virol 71:720–725

    CAS  Google Scholar 

  24. Martin F, Kupsch J, Takeuchi Y, Russell SJ, Cosset FL et al. (1998) Hum Gene Ther 9:737–746

    CAS  Google Scholar 

  25. Roux P, Jeanteur P, Piechaczyk M (1989) P Natl Acad Sci USA 86:9079–9083

    CAS  Google Scholar 

  26. Etienne-Julan M, Roux P, Carillo S, Jeanteur P, Piechaczyk M (1992) J Gen Virol 73 (Pt 12):3251–3255

    Google Scholar 

  27. Ohno K, Meruelo D (1997) Biochem Mol Med 62:123–127

    Article  CAS  Google Scholar 

  28. Ohno K, Sawai K, Iijima Y, Levin B, Meruelo D (1997) Nat Biotechnol 15:763–767

    Article  CAS  Google Scholar 

  29. Morizono K, Bristol G, Xie YM, Kung SK, Chen IS (2001) J Virol 75:8016–8020

    Article  CAS  Google Scholar 

  30. Hall FL, Gordon EM, Wu L, Zhu NL, Skotzko MJ et al. (1997) Hum Gene Ther 8:2183–2192

    CAS  Google Scholar 

  31. Hall FL, Liu L, Zhu NL, Stapfer M, Anderson WF et al. (2000) Hum Gene Ther 11:983-993

    CAS  Google Scholar 

  32. Gordon EM, Chen ZH, Liu L, Whitley M, Wei D et al. (2001) Hum Gene Ther 12:193–204

    CAS  Google Scholar 

  33. Russell SJ, Cosset FL (1999) J Gene Med 1:300–311

    Article  CAS  Google Scholar 

  34. Lavillette D, Russell SJ, Cosset FL (2001) Curr Opin Biotechnol 12:461–466

    Article  CAS  Google Scholar 

  35. Zhao Y, Zhu L, Lee S, Li L, Chang E et al. (1999) Natl Acad Sci USA 96:4005–4010

    CAS  Google Scholar 

  36. Barnett AL, Davey RA, Cunningham JM (2001) P Natl Acad Sci USA 98:4113–4118

    CAS  Google Scholar 

  37. Valsesia-Wittmann S, Morling FJ, Hatziioannou T, Russell SJ, Cosset FL (1997) Embo J 16:1214–1223

    Article  CAS  Google Scholar 

  38. Martin F, Chowdhury S, Neil SJ, Chester KA, Cosset FL et al. (2003) J Virol 77:2753–2756

    Article  CAS  Google Scholar 

  39. Martin F, Neil S, Kupsch J, Maurice M, Cosset FL et al. (1999) J Virol 73:6923–6929

    CAS  Google Scholar 

  40. Konishi H, Ochiya T, Chester KA, Begent RH, Muto T et al. (1998) Hum Gene Ther 9:235–248

    CAS  Google Scholar 

  41. Khare PD, Shao-Xi L, Kuroki M, Hirose Y, Arakawa F et al. (2001) Cancer Res 61:3705

    Google Scholar 

  42. Martin F, Chowdhury S, Neil S, Phillipps N, Collins MK (2002) Mol Ther 5:269–274

    Article  CAS  Google Scholar 

  43. Boerger AL, Snitkovsky S, Young JA (1999) P Natl Acad Sci USA 96:9867–9872

    Article  CAS  Google Scholar 

  44. Snitkovsky S, Young JA (1998) P Natl Acad Sci USA 95:7063–7068

    Article  CAS  Google Scholar 

  45. Snitkovsky S, Niederman TM, Mulligan RC, Young JA (2001) J Virol 75:1571–1575

    Article  CAS  Google Scholar 

  46. Snitkovsky S, Young JA (2002) Virology 292:150–155

    Article  CAS  Google Scholar 

  47. Mothes W, Boerger AL, Narayan S, Cunningham JM, Young JA (2000) Cell 103:679–689

    Article  CAS  Google Scholar 

  48. Melikyan GB, Barnard RJ, Markosyan RM, Young JA, Cohen FS (2004) J Virol 78:3753–3762

    Article  CAS  Google Scholar 

  49. Tai CK, Logg CR, Park JM, Anderson WF, Press MF et al. (2003) Hum Gene Ther 14:789–802

    Article  CAS  Google Scholar 

  50. Endres MJ, Jaffer S, Haggarty B, Turner JD, Doranz BJ et al. (1997) Science 278:1462–1464

    Article  CAS  Google Scholar 

  51. Balliet JW, Bates P (1998) J Virol 72:671–676

    CAS  Google Scholar 

  52. Somia NV, Miyoshi H, Schmitt MJ, Verma IM (2000) J Virol 74:4420–4424

    Article  CAS  Google Scholar 

  53. Bittner A, Mitnacht-Kraus R, Schnierle BS (2002) J Virol Methods 104:83–92

    Article  CAS  Google Scholar 

  54. Pizzato M, Marlow SA, Blair ED, Takeuchi Y (1999) J Virol 73:8599–8611

    CAS  Google Scholar 

  55. Pizzato M, Blair ED, Fling M, Kopf J, Tomassetti A et al. (2001) Gene Ther 8:1088–1096

    CAS  Google Scholar 

  56. Sharma S, Miyanohara A, Friedmann T (2000) J Virol 74:10790–10795

    Article  CAS  Google Scholar 

  57. Hatziioannou T, Delahaye E, Martin F, Russell SJ, Cosset FL (1999) Hum Gene Ther 10:1533–1544

    Article  CAS  Google Scholar 

  58. Lin AH, Kasahara N, Wu W, Stripecke R, Empig CL et al. (2001) Hum Gene Ther 12:323–332

    Article  CAS  Google Scholar 

  59. DePolo NJ, Reed JD, Sheridan PL, Townsend K, Sauter SL et al. (2000) Mol Ther 2:218–222

    Article  CAS  Google Scholar 

  60. Croyle MA, Callahan SM, Auricchio A, Schumer G, Linse KD et al. (2004) J Virol 78:912–921

    Article  CAS  Google Scholar 

  61. Guibinga GH, Hall FL, Gordon EM, Ruoslahti E, Friedmann T (2004) Mol Ther 9:76–84

    Article  CAS  Google Scholar 

  62. Fielding AK, Maurice M, Morling FJ, Cosset FL, Russell SJ (1998) Blood 91:1802–1809

    CAS  Google Scholar 

  63. Nilson BH, Morling FJ, Cosset FL, Russell SJ (1996) Gene Ther 3:280–286

    CAS  Google Scholar 

  64. Merten CA, Engelstaedter M, Buchholz CJ, Cichutek K (2003) Virology 305:106–114

    Article  CAS  Google Scholar 

  65. Chadwick MP, Morling FJ, Cosset FL, Russell SJ (1999) J Mol Biol 285:485–494

    Article  CAS  Google Scholar 

  66. Peng KW, Pham L, Ye H, Zufferey R, Trono D et al. (2001) Gene Ther 8:1456–1463

    CAS  Google Scholar 

  67. Peng KW, Vile R, Cosset FL, Russell SJ (1999) Gene Ther 6:1552–1557

    CAS  Google Scholar 

  68. Chowdhury S, Chester KA, Bridgewater J, Collins MK, Martin F (2004) Mol Ther 9:8592

    Article  CAS  Google Scholar 

  69. Morling FJ, Peng KW, Cosset FL, Russell SJ (1997) Virology 234:51–61

    Article  CAS  Google Scholar 

  70. Stemmer WP (1994) Nature 370:389–391

    Article  CAS  Google Scholar 

  71. Boder ET, Wittrup KD (1997) Nat Biotechnol 15:553–557

    Article  CAS  Google Scholar 

  72. Arnold FH, Volkov AA (1999) Curr Opin Chem Biol 3:54–59

    Article  CAS  Google Scholar 

  73. Soong NW, Nomura L, Pekrun K, Reed M, Sheppard L et al. (2000) Nat Genet 25:436–439

    Article  CAS  Google Scholar 

  74. Barry MA, Dower WJ, Johnston SA (1996) Nat Med 2:299–305

    Article  CAS  Google Scholar 

  75. Engelstadter M, Bobkova M, Baier M, Stitz J, Holtkamp N et al. (2000) Hum Gene Ther 11:293–303

    Article  CAS  Google Scholar 

  76. Buchholz CJ, Peng KW, Morling FJ, Zhang J, Cosset FL et al. (1998) Nat Biotechnol 16:951–954

    CAS  Google Scholar 

  77. Bupp K, Roth MJ (2002) Mol Ther 5:329–335

    Article  CAS  Google Scholar 

  78. Bupp K, Roth MJ (2003) Hum Gene Ther 14:1557–1564

    Article  CAS  Google Scholar 

  79. Schneider RM, Medvedovska Y, Hartl I, Voelker B, Chadwick MP et al. (2003) Gene Ther 10:1370–1380

    CAS  Google Scholar 

  80. Xiao X, Li J, McCown TJ, Samulski RJ (1997) Exp Neurol 144:113–124

    Article  CAS  Google Scholar 

  81. Bueler H (1999) Biol Chem 380:613–622

    Article  CAS  Google Scholar 

  82. Gao GP, Alvira MR, Wang L, Calcedo R, Johnston J et al. (2002) P Natl Acad Sci USA 99:11854–11859

    CAS  Google Scholar 

  83. Gao G, Alvira MR, Somanathan S, Lu Y, Vandenberghe LH et al. (2003) P Natl Acad Sci USA 100:6081–6086

    CAS  Google Scholar 

  84. Rutledge EA, Halbert CL, Russell DW (1998) J Virol 72:309–319

    CAS  Google Scholar 

  85. Gao GP, Alvira MR, Wang L, Calcedo R, Johnston J et al. (2002) P Natl Acad Sci USA 99:11854–11859

    CAS  Google Scholar 

  86. Samulski RJ, Chang LS, Shenk T (1989) J Virol 63:3822–3828

    CAS  Google Scholar 

  87. Bartlett JS, Kleinschmidt J, Boucher RC, Samulski RJ (1999) Nat Biotechnol 17:181–186

    Article  CAS  Google Scholar 

  88. Ponnazhagan S, Mahendra G, Kumar S, Thompson JA, Castillas M Jr (2002) J Virol 76:12900–12907

    Article  CAS  Google Scholar 

  89. Lee G, Maheshri N, Kaspar B, Schaffer DV (2005) Biotechnol Bioeng 92:24–34

    Article  CAS  Google Scholar 

  90. Chiorini JA, Yang L, Liu Y, Safer B, Kotin RM (1997) J Virol 71:6823–6833

    CAS  Google Scholar 

  91. Chiorini JA, Kim F, Yang L, Kotin RM (1999) J Virol 73:1309–1319

    CAS  Google Scholar 

  92. Chirmule N, Propert K, Magosin S, Qian Y, Qian R et al. (1999) Gene Ther 6:1574–1583

    CAS  Google Scholar 

  93. Davidson BL, Stein CS, Heth JA, Martins I, Kotin RM et al. (2000) P Natl Acad Sci USA 97:3428–3432

    Article  CAS  Google Scholar 

  94. Summerford C, Samulski RJ (1998) J Virol 72:1438–1445

    CAS  Google Scholar 

  95. Summerford C, Bartlett JS, Samulski RJ (1999) Nat Med 5:78–82

    CAS  Google Scholar 

  96. Qing K, Mah C, Hansen J, Zhou S, Dwarki V et al. (1999) Nat Med 5:71–77

    Article  CAS  Google Scholar 

  97. Walters RW, Yi SM, Keshavjee S, Brown KE, Welsh MJ et al. (2001) J Biol Chem 276:20610–20616

    Article  CAS  Google Scholar 

  98. Di Pasquale G, Davidson BL, Stein CS, Martins I, Scudiero D et al. (2003) Nat Med 9:1306–1312

    Article  CAS  Google Scholar 

  99. Rabinowitz JE, Rolling F, Li C, Conrath H, Xiao W et al. (2002) J Virol 76:791–801

    Article  CAS  Google Scholar 

  100. Auricchio A, Kobinger G, Anand V, Hildinger M, O'Connor E et al. (2001) Hum Mol Genet 10:3075–3081

    Article  CAS  Google Scholar 

  101. Yang Q, Mamounas M, Yu G, Kennedy S, Leaker B et al. (1998) Hum Gene Ther 9:1929–1937

    CAS  Google Scholar 

  102. Girod A, Ried M, Wobus C, Lahm H, Leike K et al. (1999) Nat Med 5:1052–1056

    Article  CAS  Google Scholar 

  103. Hermonat PL, Labow MA, Wright R, Berns KI, Muzyczka N (1984) J Virol 51:329–339

    CAS  Google Scholar 

  104. Rabinowitz JE, Xiao W, Samulski RJ (1999) Virology 265:274–285

    Article  CAS  Google Scholar 

  105. Wu P, Xiao W, Conlon T, Hughes J, Agbandje-McKenna M et al. (2000) J Virol 74:8635–8647

    CAS  Google Scholar 

  106. Shi W, Arnold GS, Bartlett JS (2001) Hum Gene Ther 12:1697–1711

    Article  CAS  Google Scholar 

  107. Shi W, Bartlett JS (2003) Mol Ther 7:515–525

    Article  CAS  Google Scholar 

  108. Grifman M, Trepel M, Speece P, Gilbert LB, Arap W et al. (2001) Mol Ther 3:964–975

    Article  CAS  Google Scholar 

  109. White SJ, Nicklin SA, Buning H, Brosnan MJ, Leike K et al. (2004) Circulation 109:513–519

    CAS  Google Scholar 

  110. Ried MU, Girod A, Leike K, Buning H, Hallek M (2002) J Virol 76:4559–4566

    Article  CAS  Google Scholar 

  111. Xie Q, Bu W, Bhatia S, Hare J, Somasundaram T et al. (2002) P Natl Acad Sci USA 99:10405–10410

    Article  CAS  Google Scholar 

  112. Walters RW, Agbandje-McKenna M, Bowman VD, Moninger TO, Olson NH et al. (2004) J Virol 78:3361–3371

    CAS  Google Scholar 

  113. Muller OJ, Kaul F, Weitzman MD, Pasqualini R, Arap W et al. (2003) Nat Biotechnol 21:1040–1046

    Article  CAS  Google Scholar 

  114. Bartlett JS, Wilcher R, Samulski RJ (2000) J Virol 74:2777–2785

    Article  CAS  Google Scholar 

  115. Duan D, Li Q, Kao AW, Yue Y, Pessin JE et al. (1999) J Virol 73:10371–10376

    CAS  Google Scholar 

  116. Hansen J, Qing K, Srivastava A (2001) J Virol 75:4080–4090

    CAS  Google Scholar 

  117. Qing K, Hansen J, Weigel-Kelley KA, Tan M, Zhou S et al. (2001) J Virol 75:8968–8976

    Article  CAS  Google Scholar 

  118. Hansen J, Qing K, Srivastava A (2001) Mol Ther 4:289–296

    Article  CAS  Google Scholar 

Download references

Acknowledgments

The authors wish to thank James Koerber for a critical reading of this manuscript. J.Y. is supported by a Graduate Research Fellowship from the Whitaker Foundation. D.S. is supported by EB003007 and generous funding from the ALS Association.

Author information

Authors and Affiliations

  1. Department of Chemical Engineering and Helen Wills Neuroscience Institute, University of California, 201 Gilman Hall, CA 94720, Berkeley, USA

    Julie H. Yu & David V. Schaffer

Authors
  1. Julie H. Yu
    View author publications

    You can also search for this author in PubMed Google Scholar

  2. David V. Schaffer
    View author publications

    You can also search for this author in PubMed Google Scholar

Corresponding author

Correspondence to David V. Schaffer .

Editor information

David V. Schaffer Weichang Zhou

Rights and permissions

Reprints and permissions

About this chapter

Cite this chapter

Yu, J.H., Schaffer, D.V. Advanced Targeting Strategies for Murine Retroviral and Adeno-associated Viral Vectors. In: Schaffer, D.V., Zhou, W. (eds) Gene Therapy and Gene Delivery Systems. Advances in Biochemical Engineering/Biotechnology, vol 99. Springer, Berlin, Heidelberg. https://doi.org/10.1007/10_006

Download citation

Publish with us

Policies and ethics

Search

Navigation