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.
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Robson T, Hirst DG (2003) J Biomed Biotechnol 2003:110–137
Romano G (2004) Drug News Perspect 17:85–90
Burton EA, Bai Q, Goins WF, Glorioso JC (2001) Adv Drug Deliv Rev 53:155–170
Wickham TJ (2003) Nat Med 9:135–139
Cavazzana-Calvo M, Hacein-Bey S, de Saint Basile G, Gross F, Yvon E et al. (2000) Science 288:669–672
Hacein-Bey-Abina S, Von Kalle C, Schmidt M, McCormack MP, Wulffraat N et al. (2003) Science 302:415–419
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)
Fields BN, Knipe DM, Howley PM, Griffin DE (2001) Fields Virology. Lippincott Williams & Wilkins, Philadelphia, PA
Kayman SC, Park H, Saxon M, Pinter A (1999) J Virol 73:1802–1808
Wu BW, Lu J, Gallaher TK, Anderson WF, Cannon PM (2000) Virology 269:7–17
Rothenberg SM, Olsen MN, Laurent LC, Crowley RA, Brown PO (2001) J Virol 75:11851–11862
Naldini L, Blomer U, Gallay P, Ory D, Mulligan R et al. (1996) Science 272:263–267
Russell DW, Miller AD (1996) J Virol 70:217–222
Wilson C, Reitz MS, Okayama H, Eiden MV (1989) J Virol 63:2374–2378
Burns JC, Friedmann T, Driever W, Burrascano M, Yee JK (1993) P Natl Acad Sci USA 90:8033–8037
Kasahara N, Dozy AM, Kan YW (1994) Science 266:1373–1376
Cosset FL, Morling FJ, Takeuchi Y, Weiss RA, Collins MK et al. (1995) J Virol 69:6314–6322
Nguyen TH, Pages JC, Farge D, Briand P, Weber A (1998) Hum Gene Ther 9:2469–2479
Valsesia-Wittmann S, Drynda A, Deleage G, Aumailley M, Heard JM et al. (1994) J Virol 68:4609–4619
Gollan TJ, Green MR (2002) J Virol 76:3558–3563
Somia NV, Zoppe M, Verma IM (1995) P Natl Acad Sci USA 92:7570–7574
Chu TH, Dornburg R (1995) J Virol 69:2659–2663
Chu TH, Dornburg R (1997) J Virol 71:720–725
Martin F, Kupsch J, Takeuchi Y, Russell SJ, Cosset FL et al. (1998) Hum Gene Ther 9:737–746
Roux P, Jeanteur P, Piechaczyk M (1989) P Natl Acad Sci USA 86:9079–9083
Etienne-Julan M, Roux P, Carillo S, Jeanteur P, Piechaczyk M (1992) J Gen Virol 73 (Pt 12):3251–3255
Ohno K, Meruelo D (1997) Biochem Mol Med 62:123–127
Ohno K, Sawai K, Iijima Y, Levin B, Meruelo D (1997) Nat Biotechnol 15:763–767
Morizono K, Bristol G, Xie YM, Kung SK, Chen IS (2001) J Virol 75:8016–8020
Hall FL, Gordon EM, Wu L, Zhu NL, Skotzko MJ et al. (1997) Hum Gene Ther 8:2183–2192
Hall FL, Liu L, Zhu NL, Stapfer M, Anderson WF et al. (2000) Hum Gene Ther 11:983-993
Gordon EM, Chen ZH, Liu L, Whitley M, Wei D et al. (2001) Hum Gene Ther 12:193–204
Russell SJ, Cosset FL (1999) J Gene Med 1:300–311
Lavillette D, Russell SJ, Cosset FL (2001) Curr Opin Biotechnol 12:461–466
Zhao Y, Zhu L, Lee S, Li L, Chang E et al. (1999) Natl Acad Sci USA 96:4005–4010
Barnett AL, Davey RA, Cunningham JM (2001) P Natl Acad Sci USA 98:4113–4118
Valsesia-Wittmann S, Morling FJ, Hatziioannou T, Russell SJ, Cosset FL (1997) Embo J 16:1214–1223
Martin F, Chowdhury S, Neil SJ, Chester KA, Cosset FL et al. (2003) J Virol 77:2753–2756
Martin F, Neil S, Kupsch J, Maurice M, Cosset FL et al. (1999) J Virol 73:6923–6929
Konishi H, Ochiya T, Chester KA, Begent RH, Muto T et al. (1998) Hum Gene Ther 9:235–248
Khare PD, Shao-Xi L, Kuroki M, Hirose Y, Arakawa F et al. (2001) Cancer Res 61:3705
Martin F, Chowdhury S, Neil S, Phillipps N, Collins MK (2002) Mol Ther 5:269–274
Boerger AL, Snitkovsky S, Young JA (1999) P Natl Acad Sci USA 96:9867–9872
Snitkovsky S, Young JA (1998) P Natl Acad Sci USA 95:7063–7068
Snitkovsky S, Niederman TM, Mulligan RC, Young JA (2001) J Virol 75:1571–1575
Snitkovsky S, Young JA (2002) Virology 292:150–155
Mothes W, Boerger AL, Narayan S, Cunningham JM, Young JA (2000) Cell 103:679–689
Melikyan GB, Barnard RJ, Markosyan RM, Young JA, Cohen FS (2004) J Virol 78:3753–3762
Tai CK, Logg CR, Park JM, Anderson WF, Press MF et al. (2003) Hum Gene Ther 14:789–802
Endres MJ, Jaffer S, Haggarty B, Turner JD, Doranz BJ et al. (1997) Science 278:1462–1464
Balliet JW, Bates P (1998) J Virol 72:671–676
Somia NV, Miyoshi H, Schmitt MJ, Verma IM (2000) J Virol 74:4420–4424
Bittner A, Mitnacht-Kraus R, Schnierle BS (2002) J Virol Methods 104:83–92
Pizzato M, Marlow SA, Blair ED, Takeuchi Y (1999) J Virol 73:8599–8611
Pizzato M, Blair ED, Fling M, Kopf J, Tomassetti A et al. (2001) Gene Ther 8:1088–1096
Sharma S, Miyanohara A, Friedmann T (2000) J Virol 74:10790–10795
Hatziioannou T, Delahaye E, Martin F, Russell SJ, Cosset FL (1999) Hum Gene Ther 10:1533–1544
Lin AH, Kasahara N, Wu W, Stripecke R, Empig CL et al. (2001) Hum Gene Ther 12:323–332
DePolo NJ, Reed JD, Sheridan PL, Townsend K, Sauter SL et al. (2000) Mol Ther 2:218–222
Croyle MA, Callahan SM, Auricchio A, Schumer G, Linse KD et al. (2004) J Virol 78:912–921
Guibinga GH, Hall FL, Gordon EM, Ruoslahti E, Friedmann T (2004) Mol Ther 9:76–84
Fielding AK, Maurice M, Morling FJ, Cosset FL, Russell SJ (1998) Blood 91:1802–1809
Nilson BH, Morling FJ, Cosset FL, Russell SJ (1996) Gene Ther 3:280–286
Merten CA, Engelstaedter M, Buchholz CJ, Cichutek K (2003) Virology 305:106–114
Chadwick MP, Morling FJ, Cosset FL, Russell SJ (1999) J Mol Biol 285:485–494
Peng KW, Pham L, Ye H, Zufferey R, Trono D et al. (2001) Gene Ther 8:1456–1463
Peng KW, Vile R, Cosset FL, Russell SJ (1999) Gene Ther 6:1552–1557
Chowdhury S, Chester KA, Bridgewater J, Collins MK, Martin F (2004) Mol Ther 9:8592
Morling FJ, Peng KW, Cosset FL, Russell SJ (1997) Virology 234:51–61
Stemmer WP (1994) Nature 370:389–391
Boder ET, Wittrup KD (1997) Nat Biotechnol 15:553–557
Arnold FH, Volkov AA (1999) Curr Opin Chem Biol 3:54–59
Soong NW, Nomura L, Pekrun K, Reed M, Sheppard L et al. (2000) Nat Genet 25:436–439
Barry MA, Dower WJ, Johnston SA (1996) Nat Med 2:299–305
Engelstadter M, Bobkova M, Baier M, Stitz J, Holtkamp N et al. (2000) Hum Gene Ther 11:293–303
Buchholz CJ, Peng KW, Morling FJ, Zhang J, Cosset FL et al. (1998) Nat Biotechnol 16:951–954
Bupp K, Roth MJ (2002) Mol Ther 5:329–335
Bupp K, Roth MJ (2003) Hum Gene Ther 14:1557–1564
Schneider RM, Medvedovska Y, Hartl I, Voelker B, Chadwick MP et al. (2003) Gene Ther 10:1370–1380
Xiao X, Li J, McCown TJ, Samulski RJ (1997) Exp Neurol 144:113–124
Bueler H (1999) Biol Chem 380:613–622
Gao GP, Alvira MR, Wang L, Calcedo R, Johnston J et al. (2002) P Natl Acad Sci USA 99:11854–11859
Gao G, Alvira MR, Somanathan S, Lu Y, Vandenberghe LH et al. (2003) P Natl Acad Sci USA 100:6081–6086
Rutledge EA, Halbert CL, Russell DW (1998) J Virol 72:309–319
Gao GP, Alvira MR, Wang L, Calcedo R, Johnston J et al. (2002) P Natl Acad Sci USA 99:11854–11859
Samulski RJ, Chang LS, Shenk T (1989) J Virol 63:3822–3828
Bartlett JS, Kleinschmidt J, Boucher RC, Samulski RJ (1999) Nat Biotechnol 17:181–186
Ponnazhagan S, Mahendra G, Kumar S, Thompson JA, Castillas M Jr (2002) J Virol 76:12900–12907
Lee G, Maheshri N, Kaspar B, Schaffer DV (2005) Biotechnol Bioeng 92:24–34
Chiorini JA, Yang L, Liu Y, Safer B, Kotin RM (1997) J Virol 71:6823–6833
Chiorini JA, Kim F, Yang L, Kotin RM (1999) J Virol 73:1309–1319
Chirmule N, Propert K, Magosin S, Qian Y, Qian R et al. (1999) Gene Ther 6:1574–1583
Davidson BL, Stein CS, Heth JA, Martins I, Kotin RM et al. (2000) P Natl Acad Sci USA 97:3428–3432
Summerford C, Samulski RJ (1998) J Virol 72:1438–1445
Summerford C, Bartlett JS, Samulski RJ (1999) Nat Med 5:78–82
Qing K, Mah C, Hansen J, Zhou S, Dwarki V et al. (1999) Nat Med 5:71–77
Walters RW, Yi SM, Keshavjee S, Brown KE, Welsh MJ et al. (2001) J Biol Chem 276:20610–20616
Di Pasquale G, Davidson BL, Stein CS, Martins I, Scudiero D et al. (2003) Nat Med 9:1306–1312
Rabinowitz JE, Rolling F, Li C, Conrath H, Xiao W et al. (2002) J Virol 76:791–801
Auricchio A, Kobinger G, Anand V, Hildinger M, O'Connor E et al. (2001) Hum Mol Genet 10:3075–3081
Yang Q, Mamounas M, Yu G, Kennedy S, Leaker B et al. (1998) Hum Gene Ther 9:1929–1937
Girod A, Ried M, Wobus C, Lahm H, Leike K et al. (1999) Nat Med 5:1052–1056
Hermonat PL, Labow MA, Wright R, Berns KI, Muzyczka N (1984) J Virol 51:329–339
Rabinowitz JE, Xiao W, Samulski RJ (1999) Virology 265:274–285
Wu P, Xiao W, Conlon T, Hughes J, Agbandje-McKenna M et al. (2000) J Virol 74:8635–8647
Shi W, Arnold GS, Bartlett JS (2001) Hum Gene Ther 12:1697–1711
Shi W, Bartlett JS (2003) Mol Ther 7:515–525
Grifman M, Trepel M, Speece P, Gilbert LB, Arap W et al. (2001) Mol Ther 3:964–975
White SJ, Nicklin SA, Buning H, Brosnan MJ, Leike K et al. (2004) Circulation 109:513–519
Ried MU, Girod A, Leike K, Buning H, Hallek M (2002) J Virol 76:4559–4566
Xie Q, Bu W, Bhatia S, Hare J, Somasundaram T et al. (2002) P Natl Acad Sci USA 99:10405–10410
Walters RW, Agbandje-McKenna M, Bowman VD, Moninger TO, Olson NH et al. (2004) J Virol 78:3361–3371
Muller OJ, Kaul F, Weitzman MD, Pasqualini R, Arap W et al. (2003) Nat Biotechnol 21:1040–1046
Bartlett JS, Wilcher R, Samulski RJ (2000) J Virol 74:2777–2785
Duan D, Li Q, Kao AW, Yue Y, Pessin JE et al. (1999) J Virol 73:10371–10376
Hansen J, Qing K, Srivastava A (2001) J Virol 75:4080–4090
Qing K, Hansen J, Weigel-Kelley KA, Tan M, Zhou S et al. (2001) J Virol 75:8968–8976
Hansen J, Qing K, Srivastava A (2001) Mol Ther 4:289–296
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.
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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
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DOI: https://doi.org/10.1007/10_006
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