Gene therapy: Design and prospects for craniofacial regeneration

Journal of Dental Research

Volumn 88, Issue 7, 2009, Pages 585-596

  Scheller, E L ^a   Krebsbach, P H ^a  

^a UNIVERSITY OF MICHIGAN   (United States)

Author keywords

Gene therapy; Gene transfer; Regeneration; Tissue engineering; Vector design; Virus

Indexed keywords

ANIMAL; BONE REGENERATION; FACIAL BONE; GENE THERAPY; GENE TRANSFER; GENE VECTOR; GENETICS; HEAD AND NECK TUMOR; HUMAN; METHODOLOGY; PHYSIOLOGY; REVIEW; SKIN TRANSPLANTATION; SQUAMOUS CELL CARCINOMA; TISSUE ENGINEERING; VIRUS;

ANIMALS; BONE REGENERATION; CARCINOMA, SQUAMOUS CELL; FACIAL BONES; GENE THERAPY; GENE TRANSFER TECHNIQUES; GENETIC VECTORS; HEAD AND NECK NEOPLASMS; HUMANS; SKIN TRANSPLANTATION; TISSUE ENGINEERING; VIRUSES;

EID: 68249161047     PISSN: 00220345     EISSN: None     Source Type: Journal    
DOI: 10.1177/0022034509337480     Document Type: Review

References (157)

1. Aframian DJ, Palmon A (2008). Current status of the development of an artificial salivary gland. Tissue Eng Part B Rev 14:187-198.
2. Agarwal M, Austin TW, Morel F, Chen J, Bohnlein E, Plavec I (1998). Scaffold attachment region-mediated enhancement of retroviral vector expression in primary T cells. J Virol 72:3720-3728.
3. Aiuti A, Bachoud-Levi AC, Blesch A, Brenner MK, Cattaneo F, Chiocca EA, et al. (2007). Progress and prospects: gene therapy clinical trials (part 2). Gene Ther 14:1555-1563.
4. Akinc A, Anderson DG, Lynn DM, Langer R (2003). Synthesis of poly(beta-amino ester)s optimized for highly effective gene delivery. Bioconjug Chem 14:979-988.
5. Alden TD, Beres EJ, Laurent JS, Engh JA, Das S, London SD, et al. (2000). The use of bone morphogenetic protein gene therapy in craniofacial bone repair. J Craniofac Surg 11:24-30.
6. Alexander BL, Ali RR, Alton EW, Bainbridge JW, Braun S, Cheng SH, et al. (2007). Progress and prospects: gene therapy clinical trials (part 1). Gene Ther 14:1439-1447.
7. Anderson WF (1990). September 14, 1990: the beginning. Hum Gene Ther 1:371-372.
8. Apperley JF, Luskey BD, Williams DA (1991). Retroviral gene transfer of human adenosine deaminase in murine hematopoietic cells: effect of selectable marker sequences on long-term expression. Blood 78:310-317.
9. Barnett BG, Crews CJ, Douglas JT (2002). Targeted adenoviral vectors. Biochim Biophys Acta 1575:1-14.
10. Baum BJ, Zheng C, Cotrim AP, Goldsmith CM, Atkinson JC, Brahim JS, et al. (2006). Transfer of the AQP1 cDNA for the correction of radiation-induced salivary hypofunction. Biochim Biophys Acta 1758:1071-1077.
11. Betz VM, Betz OB, Harris MB, Vrahas MS, Evans CH (2008). Bone tissue engineering and repair by gene therapy. Front Biosci 13:833-841.
12. Blaese RM, Culver KW, Chang L, Anderson WF, Mullen C, Nienhuis A, et al. (1993). Treatment of severe combined immunodeficiency disease (SCID) due to adenosine deaminase deficiency with CD34+ selected autologous peripheral blood cells transduced with a human ADA gene. Amendment to clinical research project, Project 90-C-195, January 10, 1992. Hum Gene Ther 4:521-527.
13. Blomer U, Naldini L, Kafri T, Trono D, Verma IM, Gage FH (1997). Highly efficient and sustained gene transfer in adult neurons with a lentivirus vector. J Virol 71:6641-6649.
14. Bonadio J, Smiley E, Patil P, Goldstein S (1999). Localized, direct plasmid gene delivery in vivo: prolonged therapy results in reproducible tissue regeneration. Nat Med 5:753-759.
15. Boussif O, Lezoualc'h F, Zanta MA, Mergny MD, Scherman D, Demeneix B, et al. (1995). A versatile vector for gene and oligonucleotide transfer into cells in culture and in vivo: polyethylenimine. Proc Natl Acad Sci USA 92:7297-7301.
16. Breitbart AS, Grande DA, Laser J, Barcia M, Porti D, Malhotra S, et al (2001). Treatment of ischemic wounds using cultured dermal fibroblasts transduced retrovirally with PDGF-B and VEGF121 genes. Ann Plast Surg 46:555-561.
17. Buchschacher GL Jr (2001). Introduction to retroviruses and retroviral vectors. Somat Cell Mol Genet 26(1-6): 1-11.
18. Carter BJ (2005). Adeno-associated virus vectors in clinical trials. Hum Gene Ther 16:541-550.
19. Chang AH, Sadelain M (2007). The genetic engineering of hematopoietic stem cells: the rise of lentiviral vectors, the conundrum of the LTR, and the promise of lineage-restricted vectors. Mol Ther 15:445-456.
20. Chang SC, Chuang HL, Chen YR, Chen JK, Chung HY, Lu YL, et al. (2003). Ex vivo gene therapy in autologous bone marrow stromal stem cells for tissue-engineered maxillofacial bone regeneration. Gene Ther 10:2013-2019.
21. Chen H, Frankenburg EP, Goldstein SA, McCauley LK (2003). Combination of local and systemic parathyroid hormone enhances bone regeneration. Clin Orthop Relat Res 416:291-302.
22. Cho YW, Kim JD, Park K (2003). Polycation gene delivery systems: escape from endosomes to cytosol. J Pharm Pharmacol 55:721-734.
23. Choi YH, Liu F, Kim JS, Choi YK, Park JS, Kim SW (1998). Polyethylene glycol-grafted poly-L-lysine as polymeric gene carrier. J Control Release 54:39-48.
24. Chu K, Cornetta KG, Econs MJ (2008). Efficient and stable gene expression into human osteoclasts using an HIV-1-based lentiviral vector. DNA Cell Biol 27:315-320.
25. Clayman GL, Frank DK, Bruso PA, Goepfert H (1999). Adenovirus-mediated wild-type p53 gene transfer as a surgical adjuvant in advanced head and neck cancers. Clin Cancer Res 5:1715-1722.
26. Connolly JB (2002). Lentiviruses in gene therapy clinical research. Gene Ther 9:1730-1734.
27. Cucchiarini M, Thurn T, Weimer A, Kohn D, Terwilliger EF, Madry H (2007). Restoration of the extracellular matrix in human osteoarthritic articular cartilage by overexpression of the transcription factor SOX9. Arthritis Rheum 56:158-167.
28. Dai Y, Schwarz EM, Gu D, Zhang WW, Sarvetnick N, Verma IM (1995). Cellular and humoral immune responses to adenoviral vectors containing factor IX gene: tolerization of factor IX and vector antigens allows for long-term expression. Proc Natl Acad Sci USA 92.T401-1405.
29. Dambach MJ, Trecki J, Martin N, Markovitz NS (2006). Oncolytic viruses derived from the gamma34.5-deleted herpes simplex virus recombinant R3616 encode a truncated UL3 protein. Mol Ther 13:891-898.
30. Dang JM, Leong KW (2006). Natural polymers for gene delivery and tissue engineering. Adv Drug Deliv Rev 58:487-499.
31. Davidson BL, Stein CS, Heth JA, Martins I, Kotin RM, Derksen TA, et al. (2000). Recombinant adeno-associated virus type 2, 4, and 5 vectors: transduction of variant cell types and regions in the mammalian central nervous system. Proc Natl Acad Sci USA 97:3428-3432.
32. Delviks KA, Pathak VK (1999). Development of murine leukemia virus-based self-activating vectors that efficiently delete the selectable drug resistance gene during reverse transcription. J Virol 73:8837-8842.
33. Dewannieux M, Harper F, Richaud A, Letzelter C, Ribet D, Pierron G, et al. (2006). Identification of an infectious progenitor for the multiple-copy HERV-K human endogenous retroelements. Genome Res 16:1548-1556.
34. Ding W, Zhang L, Yan Z, Engelhardt JF (2005). Intracellular trafficking of adeno-associated viral vectors. Gene Ther 12:873-880.
35. Eaglstein WH, Iriondo M, Laszlo K (1995). A composite skin substitute (graftskin) for surgical wounds. A clinical experience. Dermatol Surg 21:839-843.
36. Edwards PC, Ruggiero S, Fantasia J, Burakoff R, Moorji SM, Parie E, et al. (2005). Sonic hedgehog gene-enhanced tissue engineering for bone regeneration. Gene Ther 12:75-86.
37. Engelman A (1999). In vivo analysis of retroviral integrase structure and function. Adv Virus Res 52:411-426.
38. Eto Y, Yoshioka Y, Mukai Y, Okada N, Nakagawa S (2008). Development of PEGylated adenovirus vector with targeting ligand. Int J Pharm 354:3-8.
39. Feng G, Wan Y, Balian G, Laurencin CT, Li X (2008). Adenovirus-mediated expression of growth and differentiation factor-5 promotes chondrogen-esis of adipose stem cells. Growth Factors 26:132-142.
40. Fleury S, Driscoll R, Simeoni E, Dudler J, von Segesser LK, Kappenberger L, et al. (2004). Helper-dependent adenovirus vectors devoid of all viral genes cause less myocardial inflammation compared with first-generation adenovirus vectors. Basic Res Cardiol 99:247-256.
41. Flotte TR, Zeitlin PL, Reynolds TC, Heald AE, Pedersen P, Beck S, et al. (2003). Phase I trial of intranasal and endobronchial administration of a recombinant adeno-associated virus serotype 2 (rAAV2)-CFTR vector in adult cystic fibrosis patients: a two-part clinical study. Hum Gene Ther 14:1079-1088.
42. Franceschi RT (2005). Biological approaches to bone regeneration by gene therapy. J Dent Res 84:1093-1103.
43. Frankel AD, Young JA (1998). HIV-1: fifteen proteins and an RNA. Annu Rev Biochem 67:1-25.
44. Freytag SO, Strieker H, Peabody J, Pegg J, Paielli D, Movsas B, et al. (2007). Five-year follow-up of trial of replication-competent adenovirus-mediated suicide gene therapy for treatment of prostate cancer. Mol Ther 15:636-642.
45. Gafni Y, Pelled G, Zilberman Y, Turgeman G, Apparailly F, Yotvat H, et al. (2004). Gene therapy platform for bone regeneration using an exoge-nously regulated, AAV-2-based gene expression system. Mol Ther 9:587-595.
46. Gao GP, Alvira MR, Wang L, Calcedo R, Johnston J, Wilson JM (2002). Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy. Proc Natl Acad Sci USA 99:11854-11859.
47. Ginsberg HS (1999). The life and times of adenoviruses. Adv Virus Res 54:1-13.
48. Girod A, Ried M, Wobus C, Lahm H, Leike K, Kleinschmidt J, et al. (1999). Genetic capsid modifications allow efficient re-targeting of adeno-associated virus type 2. Nat Med 5:1438.
49. Goff SP (2004). Retrovirus restriction factors. Mol Cell 16:849-859.
50. Gonzalez H, Hwang SJ, Davis ME (1999). New class of polymers for the delivery of macromolecular therapeutics. Bioconjug Chem 10:1068-1074.
51. Gu DL, Nguyen T, Gonzalez AM, Printz MA, Pierce GF, Sosnowski BA, et al. (2004). Adenovirus encoding human platelet-derived growth fac-tor-B delivered in collagen exhibits safety, biodistribution, and immu-nogenicity profiles favorable for clinical use. Mol Ther 9:699-711.
52. Henry TD, Grines CL, Watkins MW, Dib N, Barbeau G, Moreadith R, et al. (2007). Effects of Ad5FGF-4 in patients with angina: an analysis of pooled data from the AGENT-3 and AGENT-4 trials. J Am Coll Cardiol 50:1038-1046.
53. Hidaka C, Quitoriano M, Warren RF, Crystal RG (2001). Enhanced matrix synthesis and in vitro formation of cartilage-like tissue by genetically modified chondrocytes expressing BMP-7. J Orthop Res 19:751-758.
54. Hu B, Nadiri A, Kuchler-Bopp S, Perrin-Schmitt F, Peters H, Lesot H (2006). Tissue engineering of tooth crown, root, and periodontium. Tissue Eng 12:2069-2075.
55. Hu WW, Wang Z, Hollister SJ, Krebsbach PH (2007). Localized viral vector delivery to enhance in situ regenerative gene therapy. Gene Ther 14:891-901.
56. Izal I, Acosta CA, Ripalda P, Zaratiegui M, Ruiz J, Forriol F (2008). IGF-1 gene therapy to protect articular cartilage in a rat model of joint damage. Arch Orthop Trauma Surg 128:239-247.
57. Jaalouk DE, Eliopoulos N, Couture C, Mader S, Galipeau J (2000). Glucocorticoid-inducible retrovector for regulated transgene expression in genetically engineered bone marrow stromal cells. Hum Gene Ther 11:1837-1849.
58. Jane JA Jr, Dunford BA, Kron A, Pittman DD, Sasaki T, Li JZ, et al. (2002). Ectopic osteogenesis using adenoviral bone morphogenetic protein (BMP)-4 and BMP-6 gene transfer. Mol Ther 6:464-470.
59. Jiang J, Fan CY, Zeng BF (2008). Osteogenic differentiation effects on rat bone marrow-derived mesenchymal stromal cells by lentivirus-mediated co-transfection of human BMP2 gene and VEGF165 gene. Biotechnol Lett 30:197-203.
60. Jin Q, Anusaksathien O, Webb SA, Printz MA, Giannobile WV (2004). Engineering of tooth-supporting structures by delivery of PDGF gene therapy vectors. Mol Ther 9:519-526.
61. Jin QM, Anusaksathien O, Webb SA, Rutherford RB, Giannobile WV (2003). Gene therapy of bone morphogenetic protein for periodontal tissue engineering. J Periodontal 74:202-213.
62. Kafri T, Blomer U, Peterson DA, Gage FH, Verma IM (1997). Sustained expression of genes delivered directly into liver and muscle by lentiviral vectors. Nat Genet 17:314-317.
63. Kaigler D, Cirelli JA, Giannobile WV (2006). Growth factor delivery for oral and periodontal tissue engineering. Expert Opin Drug Deliv 3:647-662.
64. Kaiser J (2003). Gene therapy. Seeking the cause of induced leukemias in X-SCID trial. Science 299:495.
65. Kang Y, Liao WM, Yuan ZH, Sheng PY, Zhang LJ, Yuan XW, et al. (2007). In vitro and in vivo induction of bone formation based on adeno-associated virus-mediated BMP-7 gene therapy using human adipose-derived mesenchymal stem cells. Acta Pharmacol Sin 28:839-849.
66. Kay MA, Manno CS, Ragni MV, Larson PJ, Couto LB, McClelland A, et al. (2000). Evidence for gene transfer and expression of factor IX in haemophilia B patients treated with an AAV vector. Nat Genet 24:257-261.
67. Kemeny N, Brown K, Covey A, Kim T, Bhargava A, Brody L, et al. (2006). Phase I, open-label, dose-escalating study of a genetically engineered herpes simplex virus, NV1020, in subjects with metastatic colorectal carcinoma to the liver. Hum Gene Ther 17:1214-1224.
68. Khan TA, Sellke FW, Laham RJ (2003). Gene therapy progress and prospects: therapeutic angiogenesis for limb and myocardial ischemia. Gene Ther 10:285-291.
69. Kim WJ, Yockman JW, Lee M, Jeong JH, Kim YH, Kim SW (2005). Soluble Flt-1 gene delivery using PEI-g-PEG-RGD conjugate for anti-angio-genesis. J Control Release 106:224-234.
70. Koefoed M, Ito H, Gromov K, Reynolds DG, Awad HA, Rubery PT, et al. (2005). Biological effects of rAAV-caAlk2 coating on structural allograft healing. Mol Ther 12:212-218.
71. Kohn DB, Sadelain M, Dunbar C, Bodine D, Kiem HP, Candotti F, et al. (2003). American Society of Gene Therapy (ASGT) ad hoc subcommittee on retroviral-mediated gene transfer to hematopoietic stem cells. Mol Ther 8:180-187.
72. Korin YD, Zack JA (1998). Progression to the G1b phase of the cell cycle is required for completion of human immunodeficiency virus type 1 reverse transcription in T cells. J Virol 72:3161-3168.
73. Kotin RM, Siniscalco M, Samulski RJ, Zhu XD, Hunter L, Laughlin CA, et al. (1990). Site-specific integration by adeno-associated virus. Proc Natl Acad Sci USA 87:2211-2215.
74. Krebsbach PH, Gu K, Franceschi RT, Rutherford RB (2000). Gene therapy-directed osteogenesis: BMP-7-transduced human fibroblasts form bone in vivo. Hum Gene Ther 11:1201-1210.
75. Krebsbach PH, Zhang K, Malik AK, Kurachi K (2003). Bone marrow stromal cells as a genetic platform for systemic delivery of therapeutic proteins in vivo: human factor IX model. J Gene Med 5:11-17.
76. Kurian KM, Watson CJ, Wyllie AH (2000). Retroviral vectors. Mol Pathol 53:173-176.
77. Laemmli UK (1975). Characterization of DNA condensates induced by poly(ethylene oxide) and polylysine. Proc Natl Acad Sci USA 72: 4288-4292.
78. Lawrence F, director (2007). "I Am Legend." Warners Brothers Pictures [movie].
79. Lee JY, Peng H, Usas A, Musgrave D, Cummins J, Pelinkovic D, et al. (2002). Enhancement of bone healing based on ex vivo gene therapy using human muscle-derived cells expressing bone morphogenetic protein 2. Hum Gene Ther 13:1201-1211.
80. Lee KH, Song SU, Hwang TS, Yi Y, Oh IS, Lee JY, et al. (2001). Regeneration of hyaline cartilage by cell-mediated gene therapy using transforming growth factor beta 1-producing fibroblasts. Hum Gene Ther 12:1805-1813.
81. Lee M, Kim SW (2005). Polyethylene glycol-conjugated copolymers for plasmid DNA delivery. Pharm Res 22.T-10.
82. Lewis PF, Emerman M (1994). Passage through mitosis is required for oncoretroviruses but not for the human immunodeficiency virus. J Virol 68:510-516.
83. Li W, Asokan A, Wu Z, Van Dyke T, DiPrimio N, Johnson JS, et al. (2008). Engineering and selection of shuffled AAV genomes: a new strategy for producing targeted biological nanoparticles. Mol Ther 16:1252-1260.
84. Li Y, Tew SR, Russell AM, Gonzalez KR, Hardingham TE, Hawkins RE (2004). Transduction of passaged human articular chondrocytes with adenoviral, retroviral, and lentiviral vectors and the effects of enhanced expression of SOX9. Tissue Eng 10:575-584.
85. Lin L, Fu X, Zhang X, Chen LX, Zhang JY, Yu CL, et al. (2006). Rat adipose-derived stromal cells expressing BMP4 induce ectopic bone formation in vitro and in vivo. Acta Pharmacol Sin 27:1608-1615.
86. Lin X, Huang H, Li S, Li H, Li Y, Cao Y, et al. (2007). A phase I clinical trial of an adenovirus-mediated endostatin gene (E10A) in patients with solid tumors. Cancer Biol Ther 6:648-653.
87. Liu G, Molas M, Grossmann GA, Pasumarthy M, Perales JC, Cooper MJ, et al. (2001). Biological properties of poly-L-lysine-DNA complexes generated by cooperative binding of the polycation. J Biol Chem 276:34379-34387.
88. Loeb JE, Weitzman MD, Hope TJ (2002). Enhancement of green fluorescent protein expression in adeno-associated virus with the woodchuck hepatitis virus post-transcriptional regulatory element. Methods Mol Biol 183:331-340.
89. Lusky M, Christ M, Rittner K, Dieterle A, Dreyer D, Mourot B, et al. (1998). In vitro and in vivo biology of recombinant adenovirus vectors with E1, E1/E2A, or E1/E4 deleted. J Virol 72:2022-2032.
90. Madden MR, Finkelstein JL, Staiano-Coico L, Goodwin CW, Shires GT, Nolan EE, et al. (1986). Grafting of cultured allogeneic epidermis on second- and third-degree burn wounds on 26 patients. J Trauma 26:955-962.
91. Mahato RI, Lee M, Han S, Maheshwari A, Kim SW (2001). Intratumoral delivery of p2CMVmIL-12 using water-soluble lipopolymers. Mol Ther 4:130-138.
92. Manno CS, Pierce GF, Arruda VR, Glader B, Ragni M, Rasko JJ, et al. (2006). Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response. Nat Med 12:342-347.
93. Martin GS (2004). The road to Src. Oncogene 23:7910-7917.
94. Matheson R (1954). I am legend. Sept 15, 1997 ed. New York: Tor Books.
95. McCarty DM, Monahan PE, Samulski RJ (2001). Self-complementary recombinant adeno-associated virus (scAAV) vectors promote efficient transduction independently of DNA synthesis. Gene Ther 8:1248-1254.
96. McKie R (2000). Gene researchers face crisis as man's saviour turns killer. London: The Observer, Jan. 2.
97. Melvin JE, Yule D, Shuttleworth T, Begenisich T (2005). Regulation of fluid and electrolyte secretion in salivary gland acinar cells. Annu Rev Physiol 67:445-469.
98. Mishra S, Webster P, Davis ME (2004). PEGylation significantly affects cellular uptake and intracellular trafficking of non-viral gene delivery particles. Eur J Cell Biol 83:97-111.
99. Mitchell RS, Beitzel BF, Schroder AR, Shinn P, Chen H, Berry CC, et al. (2004). Retroviral DNA integration: ASLV, HIV, and MLV show distinct target site preferences. PLoS Biol 2(8):E234.
100. Moyad MA (2007). What happened to the Provenge (Sipuleucel-T) vaccine for hormone refractory prostate cancer? Urol Nurs 27:256-257.
101. Muul LM, Tuschong LM, Soenen SL, Jagadeesh GJ, Ramsey WJ, Long Z, et al. (2003). Persistence and expression of the adenosine deaminase gene for 12 years and immune reaction to gene transfer components: long-term results of the first clinical gene therapy trial. Blood 101:2563-2569.
102. Muzyczka N, Warrington KH Jr (2005). Custom adeno-associated virus capsids: the next generation of recombinant vectors with novel tropism. Hum Gene Ther 16:408-416.
103. Nah JW, Yu L, Han SO, Ahn CH, Kim SW (2002). Artery wall binding peptide-poly(ethylene glycol)-grafted-poly(1-lysine)-based gene delivery to artery wall cells. J Control Release 78:273-284.
104. Nakai H, Storm TA, Kay MA (2000). Increasing the size of rAAV-mediated expression cassettes in vivo by intermolecular joining of two complementary vectors. Nat Biotechnol 18:527-532.
105. Nakashima M, Iohara K, Zheng L (2006). Gene therapy for dentin regeneration with bone morphogenetic proteins. Curr Gene Ther 6:551-560.
106. Niculescu-Duvaz I, Springer CJ (2005). Introduction to the background, principles, and state of the art in suicide gene therapy. Mol Biotechnol 30:71-88.
107. NIH (2008a). Genetic Modification Clinical Research Information System (GeMCRIS). Bethesda, MD: US National Institutes of Health.
108. NIH (2008b). ClinicalTrials.gov: US National Institutes of Health, http://www.clinicaltrials.gov/
109. O'Malley BW Jr, Li D, McQuone SJ, Ralston R (2005). Combination non-viral interleukin-2 gene immunotherapy for head and neck cancer: from bench top to bedside. Laryngoscope 115:391-404.
110. Pagnotto MR, Wang Z, Karpie JC, Ferretti M, Xiao X, Chu CR (2007). Adeno-associated viral gene transfer of transforming growth factor-betal to human mesenchymal stem cells improves cartilage repair. Gene Ther 14:804-813.
111. Park TG, Jeong JH, Kim SW (2006). Current status of polymeric gene delivery systems. Adv Drug Deliv Rev 58:467-486.
112. Peng H, Usas A, Gearhart B, Young B, Olshanski A, Huard J (2004). Development of a self-inactivating tet-on retroviral vector expressing bone morphogenetic protein 4 to achieve regulated bone formation. Mol Ther 9:885-894.
113. Perabo L, Endell J, King S, Lux K, Goldnau D, Hallek M, et al. (2006). Combinatorial engineering of a gene therapy vector: directed evolution of adeno-associated virus. J Gene Med 8:155-162.
114. Purdue GF, Hunt JL, Still JM Jr, Law EJ, Herndon DN, Goldfarb IW, et al. (1997). A multicenter clinical trial of a biosynthetic skin replacement, Dermagraft-TC, compared with cryopreserved human cadaver skin for temporary coverage of excised burn wounds. J Burn Care Rehabil 18(1 Pt 1):52-57.
115. Qing K, Mah C, Hansen J, Zhou S, Dwarki V, Srivastava A (1999). Human fibroblast growth factor receptor 1 is a co-receptor for infection by adeno-associated virus 2. Nat Med 5:71-77.
116. Quick DJ, Anseth KS (2004). DNA delivery from photocrosslinked PEG hydrogels: encapsulation efficiency, release profiles, and DNA quality. J Control Release 96:341-351.
117. Rabie AB, Dai J, Xu R (2007). Recombinant AAV-mediated VEGF gene therapy induces mandibular condylar growth. Gene Ther 14:972-980.
118. Roberts L (1989). Human gene transfer test approved. Science 243:473.
119. Rowe WP, Huebner RJ, Bell JA (1957). Definition and outline of contemporary information on the adenovirus group. Ann NY Acad Sci 67:255-261.
120. Roy K, Mao HQ, Huang SK, Leong KW (1999). Oral gene delivery with chitosan-DNA nanoparticles generates immunologic protection in a murine model of peanut allergy. Nat Med 5:387-391.
121. Russell WC (2000). Update on adenovirus and its vectors. J Gen Virol 81 (Pt 11):2573-2604.
122. Samulski RJ, Berns KI, Tan M, Muzyczka N (1982). Cloning of adenoassociated virus into pBR322: rescue of intact virus from the recombi-nantplasmid in human cells. Proc Natl Acad Sci USA 79:2077-2081.
123. Schek RM, Taboas JM, Segvich SJ, Hollister SJ, Krebsbach PH (2004). Engineered osteochondral grafts using biphasic composite solid free-form fabricated scaffolds. Tissue Eng 10:1376-1385.
124. Schek RM, Taboas JM, Hollister SJ, Krebsbach PH (2005). Tissue engineering osteochondral implants for temporomandibular joint repair. Orthod Craniofac Res 8:313-319.
125. Scheller EL, Chang J, Wang CY (2008). Wnt/beta-catenin inhibits dental pulp stem cell differentiation. J Dent Res 87:126-130.
126. Schnepp BC, Clark KR, Klemanski DL, Pacak CA, Johnson PR (2003). Genetic fate of recombinant adeno-associated virus vector genomes in muscle. J Virol 77:3495-3504.
127. Schultz BR, Chamberlain JS (2008). Recombinant adeno-associated virus transduction and integration. Mol Ther 16:1189-1199.
128. Shen HC, Peng H, Usas A, Gearhart B, Cummins J, Fu FH, et al. (2004). Ex vivo gene therapy-induced endochondral bone formation: comparison of muscle-derived stem cells and different subpopulations of primary muscle-derived cells. Bone 34:982-992.
129. Shi W, Bartlett JS (2003). RGD inclusion in VP3 provides adeno-associated virus type 2 (AAV2)-based vectors with a heparan sulfate-independent cell entry mechanism. Mol Ther 7:515-525.
130. Smith AE (1995). Viral vectors in gene therapy. Annu Rev Microbiol 49: 807-838.
131. Smith P, Shuler FD, Georgescu HI, Ghivizzani SC, Johnstone B, Niyibizi C, et al. (2000). Genetic enhancement of matrix synthesis by articular chondrocytes: comparison of different growth factor genes in the presence and absence of interleukin-1. Arthritis Rheum 43:1156-1164.
132. Somia N, Verma IM (2000). Gene therapy: trials and tribulations. Nat Rev Genet 1:91-99.
133. Springen K (2004). Using genes as medicine. Newsweek, Dec 6.
134. Sumimoto H, Kawakami Y (2007). Lentiviral vector-mediated RNAi and its use for cancer research. Future Oncol 3:655-664.
135. Summerford C, Samulski RJ (1998). Membrane-associated heparan sulfate proteoglycan is a receptor for adeno-associated virus type 2 virions. J Virol 72:1438-1445.
136. Summerford C, Bartlett JS, Samulski RJ (1999). AlphaVbeta5 integrin: a co-receptor for adeno-associated virus type 2 infection. Nat Med 5:78-82.
137. Tarkka T, Sipola A, Jamsa T, Soini Y, Yla-Herttuala S, Tuukkanen J, et al. (2003). Adenoviral VEGF-A gene transfer induces angiogenesis and promotes bone formation in healing osseous tissues. J Gene Med 5:560-566.
138. Tenenbaum L, Lehtonen E, Monahan PE (2003). Evaluation of risks related to the use of adeno-associated virus-based vectors. Curr Gene Ther 3:545-565.
139. Thomas CE, Storm TA, Huang Z, Kay MA (2004). Rapid uncoating of vector genomes is the key to efficient liver transduction with pseudotyped adeno-associated virus vectors. J Virol 78:3110-3122.
140. Tran SD, Wang J, Bandyopadhyay BC, Redman RS, Dutra A, Pak E, et al. (2005). Primary culture of polarized human salivary epithelial cells for use in developing an artificial salivary gland. Tissue Eng 11:172-181.
141. Tran SD, Sugito T, Dipasquale G, Cotrim AP, Bandyopadhyay BC, Riddle K, et al. (2006). Re-engineering primary epithelial cells from rhesus monkey parotid glands for use in developing an artificial salivary gland. Tissue Eng 12:2939-2948.
142. Ulrich-Vinther M (2007). Gene therapy methods in bone and joint disorders. Evaluation of the adeno-associated virus vector in experimental models of articular cartilage disorders, periprosthetic osteolysis and bone healing. Acta Orthop Suppl 78:1-64.
143. Urabe M, Ding C, Kotin RM (2002). Insect cells as a factory to produce adeno-associated virus type 2 vectors. Hum Gene Ther 13:1935-1943.
144. VanEpps HL (2005). Peyton Rous: father of the tumor virus. J Exp Med 201:320.
145. Verma IM, Somia N (1997). Gene therapy-promises, problems and prospects. Nature 389:239-242.
146. West AG, Gaszner M, Felsenfeld G (2002). Insulators: many functions, many mechanisms. Genes Dev 16:271-288.
147. Wright V, Peng H, Usas A, Young B, Gearhart B, Cummins J, et al. (2002). BMP4-expressing muscle-derived stem cells differentiate into osteogenic lineage and improve bone healing in immunocompetent mice. Mol Ther 6:169-178.
148. Wu Z, Asokan A, Samulski RJ (2006). Adeno-associated virus serotypes: vector toolkit for human gene therapy. Mol Ther 14:316-327.
149. Xie Q, Bu W, Bhatia S, Hare J, Somasundaram T, Azzi A, et al. (2002). The atomic structure of adeno-associated virus (AAV-2), a vector for human gene therapy. Proc Natl Acad Sci USA 99.T0405-10410.
150. Yoo GH, Piechocki MP, Oliver J, Lonardo F, Zumstein L, Lin HS, et al. (2004). Enhancement of Ad-p53 therapy with docetaxel in head and neck cancer. Laryngoscope 114:1871-1879.
151. Young CS, Abukawa H, Asrican R, Ravens M, Troulis MJ, Kaban LB, et al. (2005). Tissue-engineered hybrid tooth and bone. Tissue Eng 11: 1599-1610.
152. Yu SF, von Ruden T, Kantoff PW, Garber C, Sciberg M, Ruther U, et al. (1986). Self-inactivating retroviral vectors designed for transfer of whole genes into mammalian cells. Proc Natl Acad Sci USA 83:3194-3198.
153. Zennou V, Petit C, Guetard D, Nerhbass U, Montagnier L, Chameau P (2000). HIV-1 genome nuclear import is mediated by a central DNA flap. Cell 101:173-185.
154. Zhang D, Schwarz EM, Rosier RN, Zuscik MJ, Puzas JE, O'Keefe RJ (2003). ALK2 functions as a BMP type I receptor and induces Indian hedgehog in chondrocytes during skeletal development. J Bone Miner Res 18:1593-1604.
155. Zhang X, Godbey WT (2006). Viral vectors for gene delivery in tissue engineering. Adv Drug Deliv Rev 58:515-534.
156. Zhao J, Jia L, Chen D, Xiao J, Pan X, Jing X (2002). Adenovirus-mediated TGF-beta(1) gene transfer to human degenerative lumbar intervertebral disc cells. Chin Med J (Engl) 115:409-412.
157. Zufferey R, Dull T, Mandel RJ, Bukovsky A, Quiroz D, Naldini L, et al. (1998). Self-inactivating lentivirus vector for safe and efficient in vivo gene delivery. J Virol 72:9873-9880.