Progresses towards safe and efficient gene therapy vectors

Oncotarget

Volumn 6, Issue 31, 2015, Pages 30675-30703

  Chira, Sergiu ^a   Jackson, Carlo S ^b   Oprea, Iulian ^c   Ozturk, Ferhat ^d   Pepper, Michael S ^b   Diaconu, Iulia ^f   Braicu, Cornelia ^a   Raduly, Lajos Zsolt ^a,h   Calin, George A ^g   Berindan Neagoe, Ioana ^a,e,g  

^a IULIU HATIEGANU UNIVERSITY OF MEDICINE AND PHARMACY   (Romania)

^b UNIVERSITY OF PRETORIA   (South Africa)

^c KAROLINSKA UNIVERSITY HOSPITAL   (Sweden)

^d CANIK BASARI UNIVERSITY   (Turkey)

^e THE ONCOLOGY INSTITUTE PROF DR ION CHIRICUTA   (Romania)

^f BLUEBIRD BIO   (United States)

^g UNIVERSITY OF TEXAS   (United States)

^h UNIVERSITY OF AGRICULTURAL SCIENCES AND VETERINARY MEDICINE   (Romania)

Author keywords

AAVP; Gene therapy; Hybrid vectors; Non viral vectors; Viral vectors

Indexed keywords

ADENOVIRUS VECTOR; CHITOSAN; HERPESVIRUS VECTOR; LENTIVIRUS VECTOR; LIPOPLEX; LIPOSOME; NANOPARTICLE; ONCORETROVIRUS VECTOR; PARVOVIRUS VECTOR; PLASMID DNA; POLYETHYLENEIMINE; POXVIRUS VECTOR; RETROVIRUS VECTOR; UNCLASSIFIED DRUG; SMALL INTERFERING RNA;

ARTICLE; DRUG EFFICACY; DRUG SAFETY; GENE THERAPY; GENETIC ENGINEERING; GENETIC MANIPULATION; HUMAN; NONHUMAN; NONVIRAL GENE DELIVERY SYSTEM; VIRAL GENE DELIVERY SYSTEM; ADENOVIRIDAE; DEPENDOPARVOVIRUS; GENE TRANSFER; GENE VECTOR; GENETICS; LENTIVIRINAE; PLASMID; PROCEDURES; RETROVIRIDAE; RNA INTERFERENCE; VIRUS REPLICATION;

ADENOVIRIDAE; DEPENDOVIRUS; GENE TRANSFER TECHNIQUES; GENETIC ENGINEERING; GENETIC THERAPY; GENETIC VECTORS; HUMANS; LENTIVIRUS; PLASMIDS; RETROVIRIDAE; RNA INTERFERENCE; RNA, SMALL INTERFERING; VIRUS REPLICATION;

EID: 84945574356     PISSN: None     EISSN: 19492553     Source Type: Journal    
DOI: 10.18632/oncotarget.5169     Document Type: Article

References (250)

1. Blaese RM, Culver KW, Miller AD, Carter CS, Fleisher T, Clerici M, Shearer G, Chang L, Chiang Y, Tolstoshev P, Greenblatt JJ, Rosenberg SA, Klein H, Berger M, Mullen CA, Ramsey WJ, et al. T lymphocyte-directed gene therapy for ADA-SCID initial trial results after 4 years. Science. 1995; 270:475-480.
2. Raper SE, Chirmule N, Lee FS, Wivel NA, Bagg A, Gao G-p, Wilson JM, Batshaw ML. Fatal systemic inflammatory response syndrome in a ornithine transcarbamylase deficient patient following adenoviral gene transfer. Mol Genet Metab. 2003; 80:148-158.
3. Hacein-Bey-Abina S, Von Kalle C, Schmidt M, McCormack MP, Wulffraat N, Leboulch P, Lim A, Osborne CS, Pawliuk R, Morillon E, Sorensen R, Forster A, Fraser P, Cohen JI, de Saint Basile G, Alexander I, et al. LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1. Science. 2003; 302:415-419.
4. Ginn SL, Alexander IE, Edelstein ML, Abedi MR, Wixon J. Gene therapy clinical trials worldwide to 2012-an update. J Gene Med. 2013; 15:65-77.
5. Ylä-Herttuala S. Endgame: glybera finally recommended for approval as the first gene therapy drug in the European union. Mol Ther. 2012; 20:1831-1832.
6. Kaminski JM, Huber MR, Summers JB, Ward MB. Design of a nonviral vector for site-selective, efficient integration into the human genome. FASEB J. 2002; 16:1242-1247.
7. Molas M, Gómez-Valadés AG, Vidal-Alabró A, MiguelTuru M, Bermudez J, Bartrons R, Perales JC. Receptormediated gene transfer vectors: progress towards genetic pharmaceuticals. Curr Gene Ther. 2003; 3:468-485.
8. Kawabata K, Takakura Y, Hashida M. The fate of plasmid DNA after intravenous injection in mice: involvement of scavenger receptors in its hepatic uptake. Pharm Res. 1995; 12:825-830.
9. Chia D, Dorsch CA, Levy L, Barnett EV. The metabolism of nucleic acids in mice. Immunology. 1979; 36:323-329.
10. Kobayashi N, Kuramoto T, Yamaoka K, Hashida M, Takakura Y. Hepatic uptake and gene expression mechanisms following intravenous administration of plasmid DNA by conventional and hydrodynamics-based procedures. J Pharmacol Exp Ther. 2001; 297:853-860.
11. Zhang G, Gao X, Song YK, Vollmer R, Stolz DB, Gasiorowski JZ, Dean DA, Liu D. Hydroporation as the mechanism of hydrodynamic delivery. Gene Ther. 2004; 11:675-682.
12. Budker VG, Subbotin VM, Budker T, Sebestyén MG, Zhang G, Wolff JA. Mechanism of plasmid delivery by hydrodynamic tail vein injection.II. Morphological studies. J Gene Med. 2006; 8:874-888.
13. Zhang Y, Li H, Sun J, Gao J, Liu W, Li B, Guo Y, Chen J. DC-Chol/DOPE cationic liposomes: a comparative study of the influence factors on plasmid pDNA and siRNA gene delivery. Int J Pharm. 2010; 390:198-207.
14. Alton EWFW, Boyd AC, Cheng SH, Davies JC, Davies LA, Dayan A, Gill DR, Griesenbach U, Higgins T, Hyde SC, Innes JA, McLachlan G, Porteous D, Pringle I, Scheule RK, Sumner-Jones S. Toxicology study assessing efficacy and safety of repeated administration of lipid/DNA complexes to mouse lung. Gene Ther. 2014; 21:89-95.
15. Kim H-K, Davaa E, Myung C-S, Park J-S. Enhanced siRNA delivery using cationic liposomes with new polyarginineconjugated PEG-lipid. Int J Pharm. 2010; 392:141-147.
16. Chan C-L, Majzoub RN, Shirazi RS, Ewert KK, Chen Y-J, Liang KS, Safinya CR. Endosomal escape and transfection efficiency of PEGylated cationic liposome-DNA complexes prepared with an acid-labile PEG-lipid. Biomaterials. 2012; 33:4928-4935.
17. Carrillo C, Sánchez-Hernández N, García-Montoya E, Pérez-Lozano P, Suñé-Negre JM, Ticó JR, Suñé C, Miñarro M. DNA delivery via cationic solid lipid nanoparticles (SLNs). Eur J Pharm Sci. 2013; 49:157-165.
18. Vankayala R, Chiang C-S, Chao J-I, Yuan C-J, Lin S-Y, Hwang KC. A general strategy to achieve ultra-high gene transfection efficiency using lipid-nanoparticle composites. Biomaterials. 2014; 35:8261-8272.
19. Schäfer J, Höbel S, Bakowsky U, Aigner A. Liposomepolyethylenimine complexes for enhanced DNA and siRNA delivery. Biomaterials. 2010; 31:6892-6900.
20. Wang Y-Q, Wang F, Deng X-Q, Sheng J, Chen S-Y, Su J. Delivery of therapeutic AGT shRNA by PEG-Bu for hypertension therapy. PLoS ONE. 2013; 8.
21. Wang M, Wu B, Lu P, Tucker JD, Milazi S, Shah SN, Lu QL. Pluronic-PEI copolymers enhance exon-skipping of 2'-O-methyl phosphorothioate oligonucleotide in cell culture and dystrophic mdx mice. Gene Ther. 2014; 21:52-59.
22. Gaspar VM, Correia IJ, Sousa A, Silva F, Paquete CM, Queiroz JA, Sousa F. Nanoparticle mediated delivery of pure P53 supercoiled plasmid DNA for gene therapy. J Control Release. 2011; 156:212-222.
23. Köping-Höggård M, Tubulekas I, Guan H, Edwards K, Nilsson M, Vårum KM, Artursson P. Chitosan as a nonviral gene delivery system.Structure-property relationships and characteristics compared with polyethylenimine in vitro and after lung administration in vivo. Gene Ther. 2001; 8:1108-1121.
24. Peng Y-S, Lai P-L, Peng S, Wu H-C, Yu S, Tseng T-Y, Wang L-F, Chu IM. Glial cell line-derived neurotrophic factor gene delivery via a polyethylene imine grafted chitosan carrier. Int J Nanomedicine. 2014; 9:3163-3174.
25. Lu H, Dai Y, Lv L, Zhao H. Chitosan-graftpolyethylenimine/DNA nanoparticles as novel non-viral gene delivery vectors targeting osteoarthritis. PLoS ONE. 2014; 9.
26. Yin Q, Gao Y, Zhang Z, Zhang P, Li Y. Bioreducible poly (β-amino esters)/shRNA complex nanoparticles for efficient RNA delivery. J Control Release. 2011; 151:35-44.
27. Khan M, Ang CY, Wiradharma N, Yong L-K, Liu S, Liu L, Gao S, Yang Y-Y. Diaminododecane-based cationic bolaamphiphile as a non-viral gene delivery carrier. Biomaterials. 2012; 33:4673-4680.
28. Luo K, Li C, Li L, She W, Wang G, Gu Z. Arginine functionalized peptide dendrimers as potential gene delivery vehicles. Biomaterials. 2012; 33:4917-4927.
29. Shen J, Zhao DJ, Li W, Hu QL, Wang QW, Xu FJ, Tang GP. A polyethylenimine-mimetic biodegradable polycation gene vector and the effect of amine composition in transfection efficiency. Biomaterials. 2013; 34:4520-4531.
30. Thierry AR, Lunardi-Iskandar Y, Bryant JL, Rabinovich P, Gallo RC, Mahan LC. Systemic gene therapy: biodistribution and long-term expression of a transgene in mice. Proc Natl Acad Sci USA. 1995; 92:9742-9746.
31. Yamada Y, Hashida M, Hayashi Y, Tabata M, Hyodo M, Ara MN, Ohga N, Hida K, Harashima H. An approach to transgene expression in liver endothelial cells using a liposome-based gene vector coated with hyaluronic acid. J Pharm Sci. 2013; 102:3119-3127.
32. Hu Y, Xu B, Ji Q, Shou D, Sun X, Xu J, Gao J, Liang W. A mannosylated cell-penetrating peptide-graftpolyethylenimine as a gene delivery vector. Biomaterials. 2014; 35:4236-4246.
33. Mohammadi Z, Dorkoosh FA, Hosseinkhani S, Gilani K, Amini T, Najafabadi AR, Tehrani MR. In vivo transfection study of chitosan-DNA-FAP-B nanoparticles as a new non viral vector for gene delivery to the lung. Int J Pharm. 2011; 421:183-188.
34. Andaloussi SEL, Lehto T, Mäger I, Rosenthal-Aizman K, Oprea II, Simonson OE, Sork H, Ezzat K, Copolovici DM, Kurrikoff K, Viola JR, Zaghloul EM, Sillard R, Johansson HJ, Said Hassane F, Guterstam P, et al. Design of a peptide-based vector, PepFect6, for efficient delivery of siRNA in cell culture and systemically in vivo. Nucleic Acids Res. 2011; 39:3972-3987.
35. Qi W, Ding D, Zhu H, Lu D, Wang Y, Ding J, Yan W, Jia M, Guo Y. Efficient siRNA transfection to the inner ear through the intact round window by a novel proteidic delivery technology in the chinchilla. Gene Ther. 2014; 21:10-18.
36. Fuminori K. Kaneda Y. Hemagglutinating Virus of Japan Envelope Vectors as High-Performance Vehicles for Delivery of Small RNAs. Journal of Genetic Syndromes & Gene Therapy. 2013; 4:1-9.
37. Kreiss P, Cameron B, Rangara R, Mailhe P, AguerreCharriol O, Airiau M, Scherman D, Crouzet J, Pitard B. Plasmid DNA size does not affect the physicochemical properties of lipoplexes but modulates gene transfer efficiency. Nucleic Acids Res. 1999; 27:3792-3798.
38. Brandén LJ, Mohamed AJ, Smith CI. A peptide nucleic acid-nuclear localization signal fusion that mediates nuclear transport of DNA. Nat Biotechnol. 1999; 17:784-787.
39. Lundin KE, Simonson OE, Moreno PMD, Zaghloul EM, Oprea II, Svahn MG, Smith CIE. Nanotechnology approaches for gene transfer. Genetica. 2009; 137:47-56.
40. Oprea II, Simonson OE, Moreno PMD, Viola JR, Lundin KE, Smith CIE. Temperature-assisted cyclic hybridization (TACH): an improved method for supercoiled DNA hybridization. Mol Biotechnol. 2010; 45:171-179.
41. Papadakis ED, Nicklin SA, Baker AH, White SJ. Promoters and control elements: designing expression cassettes for gene therapy. Curr Gene Ther. 2004; 4:89-113.
42. Wooddell CI, Reppen T, Wolff JA, Herweijer H. Sustained liver-specific transgene expression from the albumin promoter in mice following hydrodynamic plasmid DNA delivery. J Gene Med. 2008; 10:551-563.
43. Nishikawa M, Hashida M. Nonviral approaches satisfying various requirements for effective in vivo gene therapy. Biol Pharm Bull. 2002; 25:275-283.
44. Haase R, Magnusson T, Su B, Kopp F, Wagner E, Lipps H, Baiker A, Ogris M. Generation of a tumor-and tissue-specific episomal non-viral vector system. BMC Biotechnol. 2013; 13.
45. Chevalier-Mariette C, Henry I, Montfort L, Capgras S, Forlani S, Muschler J. CpG content affects gene silencing in mice: evidence from novel transgenes. Genome Biol. 2003; 4.
46. Pringle IA, Gill DR, Connolly MM, Lawton AE, Hewitt A-M, Nunez-Alonso G, Cheng SH, Scheule RK, Davies LA, Hyde SC. Rapid identification of novel functional promoters for gene therapy. J Mol Med. 2012; 90:1487-1496.
47. Goverdhana S, Puntel M, Xiong W, Zirger JM, Barcia C, Curtin JF, Soffer EB, Mondkar S, King GD, Hu J, Sciascia SA, Candolfi M, Greengold DS, Lowenstein PR, Castro MG. Regulatable gene expression systems for gene therapy applications: progress and future challenges. Mol Ther. 2005; 12:189-211.
48. Oprea II, Viola JR, Moreno PMD, Simonson OE, Rodin S, Teller N, Tryggvason K, Lundin KE, Girnita L, Smith CIE. Repeatable, Inducible Micro-RNA-Based Technology Tightly Controls Liver Transgene Expression. Mol Ther Nucleic Acids. 2014; 3.
49. Jackson DA, Juranek S, Lipps HJ. Designing nonviral vectors for efficient gene transfer and long-term gene expression. Mol Ther. 2006; 14:613-626.
50. Wong SP, Argyros O, Coutelle C, Harbottle RP. Non-viral S/MAR vectors replicate episomally in vivo when provided with a selective advantage. Gene Ther. 2011; 18:82-87.
51. Argyros O, Wong SP, Gowers K, Harbottle RP. Genetic modification of cancer cells using non-viral, episomal S/MAR vectors for in vivo tumour modelling. PLoS ONE. 2012; 7.
52. Glover DJ, Lipps HJ, Jans DA. Towards safe, non-viral therapeutic gene expression in humans. Nat Rev Genet. 2005; 6:299-310.
53. Sivalingam J, Phan TT, Kon OL. Intragenic integration in DLC1 sustains factor VIII expression in primary human cells without insertional oncogenicity. Gene Ther. 2014; 21:402-412.
54. Pace JK, Feschotte C. The evolutionary history of human DNA transposons: evidence for intense activity in the primate lineage. Genome Res. 2007; 17:422-432.
55. Cordaux R, Batzer MA. The impact of retrotransposons on human genome evolution. Nat Rev Genet. 2009; 10:691-703.
56. Smit AF, Riggs AD. Tiggers and DNA transposon fossils in the human genome. Proc Natl Acad Sci USA. 1996; 93:1443-1448.
57. Hackett PB, Largaespada DA, Cooper LJN. A transposon and transposase system for human application. Mol Ther. 2010; 18:674-683.
58. Hong I-S, Lee H-Y, Kim H-P. Novel therapeutic approaches for various cancer types using a modified sleeping beautybased gene delivery system. PLoS ONE. 2014; 9.
59. Di Matteo M, Samara-Kuko E, Ward NJ, Waddingon SN, McVey JH, Chuah MK, VenDriessche T. Hyperactive PiggyBac Transposons for Sustained and Robust Livertargeted Gene Therapy. Mol Ther. 2014; 22:1614-1624.
60. Meier O, Greber UF. Adenovirus endocytosis. J Gene Med. 2004; 1:S152-163.
61. Alba R, Bosch A, Chillon M. Gutless adenovirus: lastgeneration adenovirus for gene therapy. Gene Ther. 2005; 1:S18-27.
62. Gonzalez-Aparicio M, Mauleon I, Alzuguren P, Bunuales M, Gonzalez-Aseguinolaza G, San Martín C, Prieto J, Hernez-Alcoceba R. Self-inactivating helper virus for the production of high-capacity adenoviral vectors. Gene Ther. 2011; 18:1025-1033.
63. Ahn M, Gamble A, Witting SR, Magrisso J, Surendran S, Obici S, Morral N. Vector and helper genome rearrangements occur during production of helper-dependent adenoviral vectors. Hum Gene Ther Methods. 2013; 24:1-10.
64. Kushwah R, Oliver JR, Duan R, Zhang L, Keshavjee S, Hu J. Induction of immunological tolerance to adenoviral vectors by using a novel dendritic cell-based strategy. J Virol. 2012; 86:3422-3435.
65. Lau AA, Rozaklis T, Ibanes S, Luck AJ, Beard H, Hassiotis S, Mazouni K, Hopwood JJ, Kremer EJ, Hemsley KM. Helper-dependent canine adenovirus vectormediated transgene expression in a neurodegenerative lysosomal storage disorder. Gene. 2012; 491:53-57.
66. Fernandes P, Peixoto C, Santiago VM, Kremer EJ, Coroadinha AS, Alves PM. Bioprocess development for canine adenovirus type 2 vectors. Gene Ther. 2013; 20:353-360.
67. Szelechowski M, Bergeron C, Gonzalez-Dunia D, Klonjkowski B. Production and purification of non replicative canine adenovirus type 2 derived vectors. J Vis Exp. 2013.
68. Huard J, Lochmüller H, Acsadi G, Jani A, Massie B, Karpati G. The route of administration is a major determinant of the transduction efficiency of rat tissues by adenoviral recombinants. Gene Ther. 1995; 2:107-115.
69. Leggiero E, Astone D, Cerullo V, Lombardo B, Mazzaccara C, Labruna G, Sacchetti L, Salvatore F, Croyle M, Pastore L. PEGylated helper-dependent adenoviral vector expressing human Apo A-I for gene therapy in LDLR-deficient mice. Gene Ther. 2013; 20:1124-1130.
70. Yao H, Chen S-C, Shen Z, Huang Y-C, Zhu X, Wang Xm, Jiang W, Wang Z-F, Bian X-W, Ling E-A, Kung H-f, Lin MC. Functional characterization of a PEI-CyD-FAcoated adenovirus as delivery vector for gene therapy. Curr Med Chem. 2013; 20:2601-2608.
71. Singarapu K, Pal I, Ramsey JD. Polyethylene glycol-grafted polyethylenimine used to enhance adenovirus gene delivery. J Biomed Mater Res A. 2013; 101:1857-1864.
72. Kim S-Y, Lee S-J, Lim S-J. Formulation and in vitro and in vivo evaluation of a cationic emulsion as a vehicle for improving adenoviral gene transfer. Int J Pharm. 2014; 475:49-59.
73. Ballard EN, Trinh VT, Hogg RT, Gerard RD. Peptide targeting of adenoviral vectors to augment tumor gene transfer. Cancer Gene Ther. 2012; 19:476-488.
74. Reetz J, Genz B, Meier C, Kowtharapu BS, Timm F, Vollmar B, Herchenröder O, Abshagen K, Pützer BM. Development of Adenoviral Delivery Systems to Target Hepatic Stellate Cells In Vivo. PLoS ONE. 2013; 8.
75. Guse K, Suzuki M, Sule G, Bertin TK, Tyynismaa H, Ahola-Erkkilä S, Palmer D, Suomalainen A, Ng P, Cerullo V, Hemminki A, Lee B. Capsid-modified adenoviral vectors for improved muscle-directed gene therapy. Hum Gene Ther. 2012; 23:1065-1070.
76. Parker AL, White KM, Lavery CA, Custers J, Waddington SN, Baker AH. Pseudotyping the adenovirus serotype 5 capsid with both the fibre and penton of serotype 35 enhances vascular smooth muscle cell transduction. Gene Ther. 2013; 20:1158-1164.
77. Kim EK, Seo HS, Chae MJ, Jeon IS, Song BY, Park YJ, Ahn HM, Yun CO, Kang CY. Enhanced antitumor immunotherapeutic effect of B-cell-based vaccine transduced with modified adenoviral vector containing type 35 fiber structures. Gene Ther. 2014; 21:106-114.
78. Frauli M, Ribault S, Neuville P, Augé F, Calenda V. Adenoviral-mediated skeletal muscle transcriptional targeting using chimeric tissue-specific promoters. Med Sci Monit. 2003; 9:BR78-84.
79. Guo L, Li X, Yang S, Xu Y, Tao R, Wulan H, Su C, Han Y. A highly infectious chimeric adenovirus expressing basic fibroblast growth factor exerts potent targeted therapy for rabbit ear chronic ischemic wounds. Plast Reconstr Surg. 2014; 134:248e-256e.
80. Zhan Y, Yu B, Wang Z, Zhang Y, Zhang H-H, Wu H, Feng X, Geng R-S, Kong W, Yu X-H. A fiber-modified adenovirus co-expressing HSV-TK and Coli.NTR enhances antitumor activities in breast cancer cells. Int J Clin Exp Pathol. 2014; 7:2850-2860.
81. Appleby CE, Kingston PA, David A, Gerdes CA, Umaña P, Castro MG, Lowenstein PR, Heagerty AM. A novel combination of promoter and enhancers increases transgene expression in vascular smooth muscle cells in vitro and coronary arteries in vivo after adenovirus-mediated gene transfer. Gene Ther. 2003; 10:1616-1622.
82. Zheng C, Baum BJ. Integration of the hybrid adenoretroviral vector AdLTR-luc involves both MoMLV elements flanking the transgene. Int J Med Sci. 2014; 11:803-809.
83. Zheng C, Cotrim AP, Nikolov N, Mineshiba F, Swaim W, Baum BJ. A novel hybrid adenoretroviral vector with more extensive E3 deletion extends transgene expression in submandibular glands. Hum Gene Ther Methods. 2012; 23:169-181.
84. Nemunaitis J, Ganly I, Khuri F, Arseneau J, Kuhn J, McCarty T, Landers S, Maples P, Romel L, Randlev B, Reid T, Kaye S, Kirn D. Selective replication and oncolysis in p53 mutant tumors with ONYX-015, an E1B-55kD genedeleted adenovirus, in patients with advanced head and neck cancer: a phase II trial. Cancer Res. 2000; 60:6359-6366.
85. Kim J, Cho JY, Kim J-H, Jung KC, Yun C-O. Evaluation of E1B gene-attenuated replicating adenoviruses for cancer gene therapy. Cancer Gene Ther. 2002; 9:725-736.
86. Cherubini G, Kallin C, Mozetic A, Hammaren-Busch K, Müller H, Lemoine NR, Halldén G. The oncolytic adenovirus Adββ enhances selective cancer cell killing in combination with DNA-damaging drugs in pancreatic cancer models. Gene Ther. 2011; 18:1157-1165.
87. Fukuda K, Abei M, Ugai H, Kawashima R, Seo E, Wakayama M, Murata T, Endo S, Hamada H, Hyodo I, Yokoyama KK. E1A, E1B double-restricted replicative adenovirus at low dose greatly augments tumor-specific suicide gene therapy for gallbladder cancer. Cancer Gene Ther. 2009; 16:126-136.
88. Ma S, Qu W, Mao L, Zhu Z, Jia L, Zhao L, Zheng X. Antitumor effects of oncolytic adenovirus armed with Drosophila melanogaster deoxyribonucleoside kinase in colorectal cancer. Oncol Rep. 2012; 27:1443-1450.
89. Wang F, Wang Z, Tian H, Qi M, Zhai Z, Li S, Li R, Zhang H, Wang W, Fu S, Lu J, Rodriguez R, Guo Y, Zhou L. Biodistribution and safety assessment of bladder cancer specific recombinant oncolytic adenovirus in subcutaneous xenografts tumor model in nude mice. Curr Gene Ther. 2012; 12:67-76.
90. Wang L, Zhang Y, Zhao J, Xiao E, Lu J, Fu S, Wang Z. Combination of bladder cancer-specific oncolytic adenovirus gene therapy with cisplatin on bladder cancer in vitro. Tumour Biol. 2014.
91. Ding M, Cao X, Xu H-n, Fan J-k, Huang H-l, Yang D-q, Li Y-h, Wang J, Li R, Liu X-y. Prostate cancer-specific and potent antitumor effect of a DD3-controlled oncolytic virus harboring the PTEN gene. PLoS ONE. 2012; 7.
92. Zhu W, Wei L, Zhang H, Chen J, Qin X. Oncolytic adenovirus armed with IL-24 inhibits the growth of breast cancer in vitro and in vivo. J Exp Clin Cancer Res. 2012; 31.
93. Wang W, Ji W, Hu H, Ma J, Li X, Mei W, Xu Y, Hu H, Yan Y, Song Q, Li Z, Su C. Survivin promoter-regulated oncolytic adenovirus with Hsp70 gene exerts effective antitumor efficacy in gastric cancer immunotherapy. Oncotarget. 2014; 5:150-160.
94. Oneal MJ, Trujillo MA, Davydova J, McDonough S, Yamamoto M, Morris JC. Characterization of infectivity-enhanced conditionally replicating adenovectors for prostate cancer radiovirotherapy. Hum Gene Ther. 2012; 23:951-959.
95. Takagi-Kimura M, Yamano T, Tamamoto A, Okamura N, Okamura H, Hashimoto-Tamaoki T, Tagawa M, Kasahara N, Kubo S. Enhanced antitumor efficacy of fibermodified, midkine promoter-regulated oncolytic adenovirus in human malignant mesothelioma. Cancer Sci. 2013; 104:1433-1439.
96. Wakayama M, Abei M, Kawashima R, Seo E, Fukuda K, Ugai H, Murata T, Tanaka N, Hyodo I, Hamada H, Yokoyama KK. E1A, E1B double-restricted adenovirus with RGD-fiber modification exhibits enhanced oncolysis for CAR-deficient biliary cancers. Clin Cancer Res. 2007; 13:3043-3050.
97. Behr M, Kaufmann JK, Ketzer P, Engelhardt S, Mück Häusl M, Okun PM, Petersen G, Neipel F, Hassel JC, Ehrhardt A, Enk AH, Nettelbeck DM. Adenoviruses using the cancer marker EphA2 as a receptor in vitro and in vivo by genetic ligand insertion into different capsid scaffolds. PLoS ONE. 2014; 9.
98. Choi JW, Kang E, Kwon OJ, Yun TJ, Park HK, Kim PH, Kim SW, Kim JH, Yun CO. Local sustained delivery of oncolytic adenovirus with injectable alginate gel for cancer virotherapy. Gene Ther. 2013; 20:880-892.
99. Kim J, Kim P-H, Nam HY, Lee J-S, Yun C-O, Kim SW. Linearized oncolytic adenoviral plasmid DNA delivered by bioreducible polymers. J Control Release. 2012; 158:451-460.
100. Le Bec C, Douar AM. Gene therapy progress and prospects-vectorology: design and production of expression cassettes in AAV vectors. Gene Ther. 2006; 13:805-813.
101. Ghosh A, Yue Y, Lai Y, Duan D. A hybrid vector system expands adeno-associated viral vector packaging capacity in a transgene-independent manner. Mol Ther. 2008; 16:124-130.
102. Choi J-H, Yu N-K, Baek G-C, Bakes J, Seo D, Nam HJ, Baek SH, Lim C-S, Lee Y-S, Kaang B-K. Optimization of AAV expression cassettes to improve packaging capacity and transgene expression in neurons. Mol Brain. 2014; 7.
103. Shiau A-L, Liu P-S, Wu C-L. Novel strategy for generation and titration of recombinant adeno-associated virus vectors. J Virol. 2005; 79:193-201.
104. Lu H, Qu G, Yang X, Xu R, Xiao W. Systemic elimination of de novo capsid protein synthesis from replication-competent AAV contamination in the liver. Hum Gene Ther. 2011; 22:625-632.
105. Martin J, Frederick A, Luo Y, Jackson R, Joubert M, Sol B, Poulin F, Pastor E, Armentano D, Wadsworth S, Vincent K. Generation and characterization of adeno-associated virus producer cell lines for research and preclinical vector production. Hum Gene Ther Methods. 2013; 24:253-269.
106. Halbert CL, Metzger MJ, Lam SL, Miller AD. Capsidexpressing DNA in AAV vectors and its elimination by use of an oversize capsid gene for vector production. Gene Ther. 2011; 18:411-417.
107. Rabinowitz JE, Rolling F, Li C, Conrath H, Xiao W, Xiao X, Samulski RJ. Cross-packaging of a single adeno-associated virus (AAV) type 2 vector genome into multiple AAV serotypes enables transduction with broad specificity. J Virol. 2002; 76:791-801.
108. Keswani SG, Balaji S, Le L, Leung A, Katz AB, Lim FY, Habli M, Jones HN, Wilson JM, Crombleholme TM. Pseudotyped AAV vector-mediated gene transfer in a human fetal trachea xenograft model: implications for in utero gene therapy for cystic fibrosis. PLoS ONE. 2012; 7.
109. Keswani SG, Balaji S, Le L, Leung A, Lim F-Y, Habli M, Jones HN, Wilson JM, Crombleholme TM. Pseudotyped adeno-associated viral vector tropism and transduction efficiencies in murine wound healing. Wound Repair Regen. 2012; 20:592-600.
110. Balaji S, King A, Dhamija Y, Le LD, Shaaban AF, Crombleholme TM, Keswani SG. Pseudotyped adenoassociated viral vectors for gene transfer in dermal fibroblasts: implications for wound-healing applications. J Surg Res. 2013; 184:691-698.
111. Wu Z, Asokan A, Grieger JC, Govindasamy L, AgbandjeMcKenna M, Samulski RJ. Single amino acid changes can influence titer, heparin binding, and tissue tropism in different adeno-associated virus serotypes. J Virol. 2006; 80:11393-11397.
112. Sen D, Gadkari RA, Sudha G, Gabriel N, Kumar YS, Selot R, Samuel R, Rajalingam S, Ramya V, Nair SC, Srinivasan N, Srivastava A, Jayharan GR. Targeted modifications in adeno-associated virus serotype 8 capsid improves its hepatic gene transfer efficiency in vivo. Hum Gene Ther Methods. 2013; 24:104-116.
113. Mowat FM, Gornik KR, Dinculescu A, Boye SL, Hauswirth WW, Petersen-Jones SM, Bartoe JT. Tyrosine capsid-mutant AAV vectors for gene delivery to the canine retina from a subretinal or intravitreal approach. Gene Ther. 2014; 21:96-105.
114. Martino AT, Basner-Tschakarjan E, Markusic DM, Finn JD, Hinderer C, Zhou S, Ostrov DA, Srivastava A, Ertl HCJ, Terhorst C, High KA, Mingozzi F, Herzog RW. Engineered AAV vector minimizes in vivo targeting of transduced hepatocytes by capsid-specific CD8+ T cells. Blood. 2013; 121:2224-2233.
115. Aslanidi GV, Rivers AE, Ortiz L, Song L, Ling C, Govindasamy L, Van Vliet K, Tan M, AgbandjeMcKenna M, Srivastava A. Optimization of the capsid of recombinant adeno-associated virus 2 (AAV2) vectors: the final threshold? PLoS ONE. 2013; 8.
116. Kay CN, Ryals RC, Aslanidi GV, Min SH, Ruan Q, Sun J, Dyka FM, Kasuga D, Ayala AE, Van Vliet K, AgbandjeMcKenna M, Hauswirth WW, Boye SL, Boye SE. Targeting photoreceptors via intravitreal delivery using novel, capsid-mutated AAV vectors. PLoS ONE. 2013; 8.
117. Pulicherla N, Shen S, Yadav S, Debbink K, Govindasamy L, Agbandje-McKenna M, Asokan A. Engineering liverdetargeted AAV9 vectors for cardiac and musculoskeletal gene transfer. Mol Ther. 2011; 19:1070-1078.
118. Michelfelder S, Varadi K, Raupp C, Hunger A, Körbelin J, Pahrmann C, Schrepfer S, Müller OJ, Kleinschmidt JA, Trepel M. Peptide ligands incorporated into the threefold spike capsid domain to re-direct gene transduction of AAV8 and AAV9 in vivo. PLoS ONE. 2011; 6.
119. Hagen S, Baumann T, Wagner HJ, Morath V, Kaufmann B, Fischer A, Bergmann S, Schindler P, Arndt KM, Müller KM. Modular adeno-associated virus (rAAV) vectors used for cellular virus-directed enzyme prodrug therapy. Sci Rep. 2014; 4.
120. Zhong L, Li W, Yang Z, Chen L, Li Y, Qing K, WeigelKelley KA, Yoder MC, Shou W, Srivastava A. Improved transduction of primary murine hepatocytes by recombinant adeno-associated virus 2 vectors in vivo. Gene Ther. 2004; 11:1165-1169.
121. Jayandharan GR, Zhong L, Li B, Kachniarz B, Srivastava A. Strategies for improving the transduction efficiency of single-stranded adeno-associated virus vectors in vitro and in vivo. Gene Ther. 2008; 15:1287-1293.
122. Ma W, Li B, Ling C, Jayandharan GR, Srivastava A, Byrne BJ. A simple method to increase the transduction efficiency of single-stranded adeno-associated virus vectors in vitro and in vivo. Hum Gene Ther. 2011; 22:633-640.
123. Alaee F, Sugiyama O, Virk MS, Tang Y, Wang B, Lieberman JR. In vitro evaluation of a double-stranded self-complementary adeno-associated virus type2 vector in bone marrow stromal cells for bone healing. Genet Vaccines Ther. 2011; 9.
124. Ku CA, Chiodo VA, Boye SL, Goldberg AFX, Li T, Hauswirth WW, Ramamurthy V. Gene therapy using selfcomplementary Y733F capsid mutant AAV2/8 restores vision in a model of early onset Leber congenital amaurosis. Hum Mol Genet. 2011; 20:4569-4581.
125. Ryals RC, Boye SL, Dinculescu A, Hauswirth WW, Boye SE. Quantifying transduction efficiencies of unmodified and tyrosine capsid mutant AAV vectors in vitro using two ocular cell lines. Mol Vis. 2011; 17:1090-1102.
126. Wang L, Wang H, Morizono H, Bell P, Jones D, Lin J, McMenamin D, Yu H, Batshaw ML, Wilson JM. Sustained correction of OTC deficiency in spf( ash) mice using optimized self-complementary AAV2/8 vectors. Gene Ther. 2012; 19:404-410.
127. von Jonquieres G, Mersmann N, Klugmann CB, Harasta AE, Lutz B, Teahan O, Housley GD, Fröhlich D, Krämer-Albers E-M M. Glial promoter selectivity following AAV-delivery to the immature brain. PLoS ONE. 2013; 8.
128. Zhu H, Cao M, Mirandola L, Figueroa JA, Cobos E, Chiriva-Internati M, Hermonat PL. Comparison of efficacy of the disease-specific LOX1-and constitutive cytomegalovirus-promoters in expressing interleukin 10 through adeno-associated virus 2/8 delivery in atherosclerotic mice. PLoS ONE. 2014; 9.
129. Wang CY, Guo HY, Lim TM, Ng YK, Neo HP, Hwang PYK, Yee WC, Wang S. Improved neuronal transgene expression from an AAV-2 vector with a hybrid CMV enhancer/PDGF-beta promoter. J Gene Med. 2005; 7:945-955.
130. Bosch MK, Nerbonne JM, Ornitz DM. Dual transgene expression in murine cerebellar purkinje neurons by viral transduction in vivo. PLoS ONE. 2014; 9.
131. Vanrell L, Di Scala M, Blanco L, Otano I, Gil-Farina I, Baldim V, Paneda A, Berraondo P, Beattie SG, Chtarto A, Tenenbaum L, Prieto J, Gonzalez-Aseguinolaza G. Development of a liver-specific Tet-on inducible system for AAV vectors and its application in the treatment of liver cancer. Mol Ther. 2011; 19:1245-1253.
132. Goudy KS, Johnson MC, Garland A, Li C, Samulski RJ, Wang B, Tisch R. Inducible adeno-associated virus-mediated IL-2 gene therapy prevents autoimmune diabetes. J Immunol. 2011; 186:3779-3786.
133. Rosas LE, Grieves JL, Zaraspe K, La Perle KM, Fu H, McCarty DM. Patterns of scAAV vector insertion associated with oncogenic events in a mouse model for genotoxicity. Mol Ther. 2012; 20:2098-2110.
134. Debyser Z. A short course on virology/vectorology/gene therapy. Curr Gene Ther. 2003; 3:495-499.
135. Hacein-Bey-Abina S, Garrigue A, Wang GP, Soulier J, Lim A, Morillon E, Clappier E, Caccavelli L, Delabesse E, Beldjord K, Asnafi V, MacIntyre E, Dal Cortivo L, Radford I, Brousse N, Sigaux F, et al. Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1. Journal of Clinical Investigation. 2008; 118:3132-3142.
136. Xu W, Russ JL, Eiden MV. Evaluation of residual promoter activity in γ-retroviral self-inactivating (SIN) vectors. Mol Ther. 2012; 20:84-90.
137. Schambach A, Galla M, Maetzig T, Loew R, Baum C. Improving transcriptional termination of self-inactivating gamma-retroviral and lentiviral vectors. Mol Ther. 2007; 15:1167-1173.
138. Hwang BY, Schaffer DV. Engineering a serum-resistant and thermostable vesicular stomatitis virus G glycoprotein for pseudotyping retroviral and lentiviral vectors. Gene Ther. 2013; 20:807-815.
139. Stitz J, Buchholz CJ, Engelstädter M, Uckert W, Bloemer U, Schmitt I, Cichutek K. Lentiviral vectors pseudotyped with envelope glycoproteins derived from gibbon ape leukemia virus and murine leukemia virus 10A1. Virology. 2000; 273:16-20.
140. Sandrin V, Boson B, Salmon P, Gay W, Nègre D, Le Grand R, Trono D. Lentiviral vectors pseudotyped with a modified RD114 envelope glycoprotein show increased stability in sera and augmented transduction of primary lymphocytes and CD34+ cells derived from human and nonhuman primates. Blood. 2002; 100:823-832.
141. Bell AJ, Fegen D, Ward M, Bank A. RD114 envelope proteins provide an effective and versatile approach to pseudotype lentiviral vectors. Exp Biol Med (Maywood). 2010; 235:1269-1276.
142. Trobridge GD, Wu RA, Hansen M, Ironside C, Watts KL, Olsen P, Beard BC. Cocal-pseudotyped lentiviral vectors resist inactivation by human serum and efficiently transduce primate hematopoietic repopulating cells. Mol Ther. 2010; 18:725-733.
143. Lévy C, Amirache F, Costa C, Frecha C, Muller CP, Kweder H, Buckland R, Cosset F-L, Verhoeyen E. Lentiviral vectors displaying modified measles virus gp overcome pre-existing immunity in in vivo-like transduction of human T and B cells. Mol Ther. 2012; 20:1699-1712.
144. Enkirch T, Kneissl S, Hoyler B, Ungerechts G, Stremmel W, Buchholz CJ, Springfeld C. Targeted lentiviral vectors pseudotyped with the Tupaia paramyxovirus glycoproteins. Gene Ther. 2013; 20:16-23.
145. Mitomo K, Griesenbach U, Inoue M, Somerton L, Meng C, Akiba E, Tabata T, Ueda Y, Frankel GM, Farley R, Singh C, Chan M, Munkonge F, Brum A, Xenariou S, Escudero-Garcia S, et al. Toward gene therapy for cystic fibrosis using a lentivirus pseudotyped with Sendai virus envelopes. Mol Ther. 2010; 18:1173-1182.
146. Griesenbach U, Inoue M, Meng C, Farley R, Chan M, Newman NK, Brum A, You J, Kerton A, Shoemark A, Boyd AC, Davies JC, Higgins TE, Gill DR, Hyde SC, Innes JA, et al. Assessment of F/HN-pseudotyped lentivirus as a clinically relevant vector for lung gene therapy. Am J Respir Crit Care Med. 2012; 186:846-856.
147. Witting SR, Vallanda P, Gamble AL. Characterization of a third generation lentiviral vector pseudotyped with Nipah virus envelope proteins for endothelial cell transduction. Gene Ther. 2013; 20:997-1005.
148. Sakuma T, De Ravin SS, Tonne JM, Thatava T, Ohmine S, Takeuchi Y, Malech HL, Ikeda Y. Characterization of retroviral and lentiviral vectors pseudotyped with xenotropic murine leukemia virus-related virus envelope glycoprotein. Hum Gene Ther. 2010; 21:1665-1673.
149. Ou W, Marino MP, Suzuki A, Joshi B, Husain SR, Maisner A, Galanis E, Puri RK, Reiser J. Specific targeting of human interleukin (IL)-13 receptor a2-positive cells with lentiviral vectors displaying IL-13. Hum Gene Ther Methods. 2012; 23:137-147.
150. Zhang L, Feldman SA, Zheng Z, Chinnasamy N, Xu H, Nahvi AV, Dudley ME, Rosenberg SA, Morgan RA. Evaluation of γ-retroviral vectors that mediate the inducible expression of IL-12 for clinical application. J Immunother. 2012; 35:430-439.
151. Fontes AM, Melo FUF, Greene LJ, Faça VM, Lin Y, Gerson SL, Covas DT. Production of human factor VIII-FL in 293T cells using the bicistronic MGMT(P140K)-retroviral vector. Genet Mol Res. 2012; 11:775-789.
152. Johnston JM, Denning G, Doering CB, Spencer HT. Generation of an optimized lentiviral vector encoding a high-expression factor VIII transgene for gene therapy of hemophilia A. Gene Ther. 2013; 20:607-615.
153. Zhang N, Zhao L, Ma S, Gu M, Zheng X. Lentivirusmediated expression of Drosophila melanogaster deoxyribonucleoside kinase driven by the hTERT promoter combined with gemcitabine: a potential strategy for cancer therapy. Int J Mol Med. 2012; 30:659-665.
154. Lee C-J Fan, X Guo, X and Medin. Promoter-specific lentivectors for long-term, cardiac-directed therapy of Fabry disease. J Cardiol. 2011; 57:115-122.
155. Knight S, Zhang F, Mueller-Kuller U, Bokhoven M, Gupta A, Broughton T, Sha S, Antoniou MN, Brendel C, Grez M, Thrasher AJ, Collins M, Takeuchi Y. Safer, silencingresistant lentiviral vectors: optimization of the ubiquitous chromatin-opening element through elimination of aberrant splicing. J Virol. 2012; 86:9088-9095.
156. Yilmaz A, Fernandez S, Lairmore MD, Boris-Lawrie K. Coordinate enhancement of transgene transcription and translation in a lentiviral vector. Retrovirology. 2006; 3.
157. Benabdellah K, Cobo M, Muñoz P, Toscano MG, Martin F. Development of an all-in-one lentiviral vector system based on the original TetR for the easy generation of Tet-ON cell lines. PLoS ONE. 2011; 6.
158. Loew R, Vigna E, Lindemann D, Naldini L, Bujard H. Retroviral vectors containing Tet-controlled bidirectional transcription units for simultaneous regulation of two gene activities. J Mol Genet Med. 2006; 2:107-118.
159. Amendola M, Giustacchini A, Gentner B, Naldini L. A double-switch vector system positively regulates transgene expression by endogenous microRNA expression (miR-ON vector). Mol Ther. 2013; 21:934-946.
160. Hlavaty J, Schittmayer M, Stracke A, Jandl G, Knapp E, Felber BK, Salmons B, Günzburg WH, Renner M. Effect of posttranscriptional regulatory elements on transgene expression and virus production in the context of retrovirus vectors. Virology. 2005; 341:1-11.
161. Schambach A, Bohne J, Baum C, Hermann FG, Egerer L, von Laer D, Giroglou T. Woodchuck hepatitis virus posttranscriptional regulatory element deleted from X protein and promoter sequences enhances retroviral vector titer and expression. Gene Ther. 2006; 13:641-645.
162. Van Maele B, De Rijck J, De Clercq E, Debyser Z. Impact of the central polypurine tract on the kinetics of human immunodeficiency virus type 1 vector transduction. J Virol. 2003; 77:4685-4694.
163. Park F, Kay MA. Modified HIV-1 based lentiviral vectors have an effect on viral transduction efficiency and gene expression in vitro and in vivo. Mol Ther. 2001; 4:164-173.
164. Barry SC, Harder B, Brzezinski M, Flint LY, Seppen J, Osborne WR. Lentivirus vectors encoding both central polypurine tract and posttranscriptional regulatory element provide enhanced transduction and transgene expression. Hum Gene Ther. 2001; 12:1103-1108.
165. Hanawa H, Yamamoto M, Zhao H, Shimada T, Persons DA. Optimized lentiviral vector design improves titer and transgene expression of vectors containing the chicken beta-globin locus HS4 insulator element. Mol Ther. 2009; 17:667-674.
166. Anson DS. The use of retroviral vectors for gene therapywhat are the risks?.A review of retroviral pathogenesis and its relevance to retroviral vector-mediated gene delivery. Genet Vaccines Ther. 2004; 2.
167. Beard BC, Dickerson D, Beebe K, Gooch C, Fletcher J, Okbinoglu T, Miller DG, Jacobs MA, Kaul R, Kiem H-P, Trobridge GD. Comparison of HIV-derived lentiviral and MLV-based gammaretroviral vector integration sites in primate repopulating cells. Mol Ther. 2007; 15:1356-1365.
168. Bester AC, Kafri M, Maoz K, Kerem B. Infection with retroviral vectors leads to perturbed DNA replication increasing vector integrations into fragile sites. Sci Rep. 2013; 3.
169. Wu X, Li Y, Crise B, Burgess SM. Transcription start regions in the human genome are favored targets for MLV integration. Science. 2003; 300:1749-1751.
170. Mitchell RS, Beitzel BF, Schroder ARW, Shinn P, Chen H, Berry CC, Ecker JR, Bushman FD. Retroviral DNA integration: ASLV, HIV, and MLV show distinct target site preferences. PLoS Biol. 2004; 2.
171. Felice B, Cattoglio C, Cittaro D, Testa A, Miccio A, Ferrari G, Luzi L, Recchia A, Mavilio F. Transcription factor binding sites are genetic determinants of retroviral integration in the human genome. PLoS ONE. 2009; 4.
172. Cattoglio C, Pellin D, Rizzi E, Maruggi G, Corti G, Miselli F, Sartori D, Guffanti A, Di Serio C, Ambrosi A, De Bellis G, Mavilio F. High-definition mapping of retroviral integration sites identifies active regulatory elements in human multipotent hematopoietic progenitors. Blood. 2010; 116:5507-5517.
173. LaFave MC, Varshney GK, Gildea DE, Wolfsberg TG, Baxevanis AD, Burgess SM. MLV integration site selection is driven by strong enhancers and active promoters. Nucleic Acids Res. 2014; 42:4257-4269.
174. Ronen K, Negre O, Roth S, Colomb C, Malani N, Denaro M, Brady T, Fusil F, Gillet-Legrand B, Hehir K, Beuzard Y, Leboulch P, Down JD, Payen E, Bushman FD. Distribution of lentiviral vector integration sites in mice following therapeutic gene transfer to treat β-thalassemia. Mol Ther. 2011; 19:1273-1286.
175. Ambrosi A, Glad IK, Pellin D, Cattoglio C, Mavilio F, Di Serio C, Frigessi A. Estimated comparative integration hotspots identify different behaviors of retroviral gene transfer vectors. PLoS Comput Biol. 2011; 7.
176. Ranzani M, Cesana D, Bartholomae CC, Sanvito F, Pala M, Benedicenti F, Gallina P, Sergi LS, Merella S, Bulfone A, Doglioni C, von Kalle C, Kim YJ, Schmidt M, Tonon G, Naldini L, et al. Lentiviral vector-based insertional mutagenesis identifies genes associated with liver cancer. Nat Methods. 2013; 10:155-161.
177. Wang Z, Tang Z, Zheng Y, Yu D, Spear M, Iyer SR, Bishop B, Wu Y. Development of a nonintegrating Revdependent lentiviral vector carrying diphtheria toxin A chain and human TRAF6 to target HIV reservoirs. Gene Ther. 2010; 17:1063-1076.
178. Torres R, Garcia A, Jimenez M, Rodriguez S, Ramirez JC. An integration-defective lentivirus-based resource for sitespecific targeting of an edited safe-harbour locus in the human genome. Gene Ther. 2014; 21:343-352.
179. Hu WS, Pathak VK. Design of retroviral vectors and helper cells for gene therapy. Pharmacol Rev. 2000; 52:493-511.
180. Vigna E, Naldini L. Lentiviral vectors: excellent tools for experimental gene transfer and promising candidates for gene therapy. J Gene Med. 2000; 2:308-316.
181. Delenda C. Lentiviral vectors: optimization of packaging, transduction and gene expression. J Gene Med. 2004; 1:S125-138.
182. Farson D, Witt R, McGuinness R, Dull T, Kelly M, Song J, Radeke R, Bukovsky A, Consiglio A, Naldini L. A newgeneration stable inducible packaging cell line for lentiviral vectors. Hum Gene Ther. 2001; 12:981-997.
183. Stornaiuolo A, Piovani BM, Bossi S, Zucchelli E, Corna S, Salvatori F, Mavilio F, Bordignon C, Rizzardi GP, Bovolenta C. RD2-MolPack-Chim3, a packaging cell line for stable production of lentiviral vectors for antiHIV gene therapy. Hum Gene Ther Methods. 2013; 24:228-240.
184. Feldman SA, Xu H, Black MA, Park TS, Robbins PF, Kochenderfer JN, Morgan RA, Rosenberg SA. Use of the piggyBac Transposon to Create Stable Packaging Cell Lines for the Production of Clinical-Grade SelfInactivating γ-Retroviral Vectors. Hum Gene Ther Methods. 2014; 25:253-260.
185. Emery DW. The use of chromatin insulators to improve the expression and safety of integrating gene transfer vectors. Hum Gene Ther. 2011; 22:761-774.
186. Zhou S, Mody D, DeRavin SS, Hauer J, Lu T, Ma Z, Hacein-Bey Abina S, Gray JT, Greene MR, CavazzanaCalvo M, Malech HL, Sorrentino BP. A self-inactivating lentiviral vector for SCID-X1 gene therapy that does not activate LMO2 expression in human T cells. Blood. 2010; 116:900-908.
187. Papanikolaou E, Georgomanoli M, Stamateris E, Panetsos F, Karagiorga M, Tsaftaridis P, Graphakos S, Anagnou NP. The new self-inactivating lentiviral vector for thalassemia gene therapy combining two HPFH activating elements corrects human thalassemic hematopoietic stem cells. Hum Gene Ther. 2012; 23:15-31.
188. Uchida N, Hanawa H, Yamamoto M, Shimada T. The chicken hypersensitivity site 4 core insulator blocks promoter interference in lentiviral vectors. Hum Gene Ther Methods. 2013; 24:117-124.
189. Koldej RM, Carney G, Wielgosz MM, Zhou S, Zhan J, Sorrentino BP, Nienhuis AW. Comparison of insulators and promoters for expression of the Wiskott-Aldrich syndrome protein using lentiviral vectors. Hum Gene Ther Clin Dev. 2013; 24:77-85.
190. Benabdellah K, Gutierrez-Guerrero A, Cobo M, Muñoz P, Martín F. A chimeric HS4-SAR insulator (IS2) that prevents silencing and enhances expression of lentiviral vectors in pluripotent stem cells. PLoS ONE. 2014; 9.
191. Groth AC, Liu M, Wang H, Lovelett E, Emery DW. Identification and characterization of enhancer-blocking insulators to reduce retroviral vector genotoxicity. PLoS ONE. 2013; 8.
192. Hirata RK, Miller AD, Andrews RG, Russell DW. Transduction of hematopoietic cells by foamy virus vectors. Blood. 1996; 88:3654-3661.
193. Mergia A, Chari S, Kolson DL, Goodenow MM, Ciccarone T. The efficiency of simian foamy virus vector type-1 (SFV-1) in nondividing cells and in human PBLs. Virology. 2001; 280:243-252.
194. Vassilopoulos G, Trobridge G, Josephson NC, Russell DW. Gene transfer into murine hematopoietic stem cells with helper-free foamy virus vectors. Blood. 2001; 98:604-609.
195. Liu W, Liu Z, Cao X, Cao Z, Xue L, Zhu F, He X, Li W. Recombinant human foamy virus, a novel vector for neurological disorders gene therapy, drives production of GAD in cultured astrocytes. Mol Ther. 2007; 15:1834-1841.
196. Caprariello AV, Miller RH, Selkirk SM. Foamy virus as a gene transfer vector to the central nervous system. Gene Ther. 2009; 16:448-452.
197. Hill CL, Bieniasz PD, McClure MO. Properties of human foamy virus relevant to its development as a vector for gene therapy. J Gen Virol. 1999; 80:2003-2009.
198. Nasimuzzaman M, Persons DA. Cell Membraneassociated heparan sulfate is a receptor for prototype foamy virus in human, monkey, and rodent cells. Mol Ther. 2012; 20:1158-1166.
199. Trobridge GD, Miller DG, Jacobs MA, Allen JM, Kiem H-P, Kaul R, Russell DW. Foamy virus vector integration sites in normal human cells. Proc Natl Acad Sci USA. 2006; 103:1498-1503.
200. Deyle DR, Khan IF, Ren G, Russell DW. Lack of genotoxicity due to foamy virus vector integration in human iPSCs. Gene Ther. 2013; 20:868-873.
201. Nestler U, Heinkelein M, Lücke M, Meixensberger J, Scheurlen W, Kretschmer A, Rethwilm A. Foamy virus vectors for suicide gene therapy. Gene Ther. 1997; 4:1270-1277.
202. Trobridge GD, Russell DW. Helper-free foamy virus vectors. Hum Gene Ther. 1998; 9:2517-2525.
203. Wu M, Mergia A. Packaging cell lines for simian foamy virus type 1 vectors. J Virol. 1999; 73:4498-4501.
204. Bastone P, Romen F, Liu W, Wirtz R, Koch U, Josephson N, Langbein S, Löchelt M. Construction and characterization of efficient, stable and safe replication-deficient foamy virus vectors. Gene Ther. 2007; 14:613-620.
205. Grant KG, Krisky DM, Ataai MM, Glorioso JC. Engineering cell lines for production of replication defective HSV-1 gene therapy vectors. Biotechnol Bioeng. 2009; 102:1087-1097.
206. Rasmussen M, Kong L, Zhang G-r, Liu M, Wang X, Szabo G, Curthoys NP, Geller AI. Glutamatergic or GABAergic neuron-specific, long-term expression in neocortical neurons from helper virus-free HSV-1 vectors containing the phosphate-activated glutaminase, vesicular glutamate transporter-1, or glutamic acid decarboxylase promoter. Brain Res. 2007; 1144:19-32.
207. Zhang G-r, Geller AI. A helper virus-free HSV-1 vector containing the vesicular glutamate transporter-1 promoter supports expression preferentially in VGLUT1-containing glutamatergic neurons. Brain Res. 2010; 1331:12-19.
208. Schmeisser F, Donohue M, Weir JP. Tetracyclineregulated gene expression in replication-incompetent herpes simplex virus vectors. Hum Gene Ther. 2002; 13:2113-2124.
209. Jiang Y, Wei N, Zhu J, Zhai D, Wu L, Chen M, Xu G, Liu X. A new approach with less damage: intranasal delivery of tetracycline-inducible replication-defective herpes simplex virus type-1 vector to brain. Neuroscience. 2012; 201:96-104.
210. Ho IAW, Hui KM, Lam PYP. Glioma-specific and cell cycle-regulated herpes simplex virus type 1 amplicon viral vector. Hum Gene Ther. 2004; 15:495-508.
211. Wolfe D, Niranjan A, Trichel A, Wiley C, Ozuer A, Kanal E, Kondziolka D, Krisky D, Goss J, Deluca N, MurpheyCorb M, Glorioso JC. Safety and biodistribution studies of an HSV multigene vector following intracranial delivery to non-human primates. Gene Ther. 2004; 11:1675-1684.
212. Liu M, Wang X, Geller AI. Improved long-term expression from helper virus-free HSV-1 vectors packaged using combinations of mutated HSV-1 proteins that include the UL13 protein kinase and specific components of the VP16 transcriptional complex. BMC Mol Biol. 2009; 10.
213. Mineta T, Rabkin SD, Yazaki T, Hunter WD, Martuza RL. Attenuated multi-mutated herpes simplex virus-for the treatment of malignant gliomas. Nat Med. 1995; 1:938-943.
214. Fukuhara H, Ino Y, Kuroda T, Martuza RL, Todo T. Triple gene-deleted oncolytic herpes simplex virus vector doublearmed with interleukin 18 and soluble B7-1 constructed by bacterial artificial chromosome-mediated system. Cancer Res. 2005; 65:10663-10668.
215. Liu R, Varghese S, Rabkin SD. Oncolytic herpes simplex virus vector therapy of breast cancer in C3(1)/SV40 T-antigen transgenic mice. Cancer Res. 2005; 65:1532-1540.
216. Kanai R, Zaupa C, Sgubin D, Antoszczyk SJ, Martuza RL, Wakimoto H, Rabkin SD. Effect of γ34.5 deletions on oncolytic herpes simplex virus activity in brain tumors. J Virol. 2012; 86:4420-4431.
217. Nakamori M, Fu X, Meng F, Jin A, Tao L, Bast RC, Zhang X. Effective therapy of metastatic ovarian cancer with an oncolytic herpes simplex virus incorporating two membrane fusion mechanisms. Clin Cancer Res. 2003; 9:2727-2733.
218. Nakamori M, Fu X, Pettaway CA, Zhang X. Potent antitumor activity after systemic delivery of a doubly fusogenic oncolytic herpes simplex virus against metastatic prostate cancer. Prostate. 2004; 60:53-60.
219. Takaoka H, Takahashi G, Ogawa F, Imai T, Iwai S, Yura Y. A novel fusogenic herpes simplex virus for oncolytic virotherapy of squamous cell carcinoma. Virol J. 2011; 8.
220. Meshii N, Takahashi G, Okunaga S, Hamada M, Iwai S, Takasu A, Ogawa Y, Yura Y. Enhancement of systemic tumor immunity for squamous cell carcinoma cells by an oncolytic herpes simplex virus. Cancer Gene Ther. 2013; 20:493-498.
221. Gambini E, Reisoli E, Appolloni I, Gatta V, CampadelliFiume G, Menotti L, Malatesta P. Replication-competent herpes simplex virus retargeted to HER2 as therapy for high-grade glioma. Mol Ther. 2012; 20:994-1001.
222. Nanni P, Gatta V, Menotti L, De Giovanni C, Ianzano M, Palladini A, Grosso V, Dall'ora M, Croci S, Nicoletti G, Landuzzi L, Iezzi M. Preclinical therapy of disseminated HER-2+ ovarian and breast carcinomas with a HER-2-retargeted oncolytic herpesvirus. PLoS Pathog. 2013; 9.
223. Fu X, Rivera A, Tao L, De Geest B, Zhang X. Construction of an oncolytic herpes simplex virus that precisely targets hepatocellular carcinoma cells. Mol Ther. 2012; 20:339-346.
224. Hogervorst EJ, Schouls L, Wagenaar JP, Boog CJ, Spaan WJ, van Embden JD, van Eden W. Modulation of experimental autoimmunity: treatment of adjuvant arthritis by immunization with a recombinant vaccinia virus. Infect Immun. 1991; 59:2029-2035.
225. Lee SS, Eisenlohr LC, McCue PA, Mastrangelo MJ, Lattime EC. Intravesical gene therapy: in vivo gene transfer using recombinant vaccinia virus vectors. Cancer Res. 1994; 54:3325-3328.
226. Xie YC, Hwang C, Overwijk W, Zeng Z, Eng MH, Mulé JJ, Imperiale MJ, Restifo NP, Sa MG. Induction of tumor antigen-specific immunity in vivo by a novel vaccinia vector encoding safety-modified simian virus 40 T antigen. J Natl Cancer Inst. 1999; 91:169-175.
227. Nagorsen D, Panelli M, Dudley ME, Finkelstein SE, Rosenberg SA, Marincola FM. Biased epitope selection by recombinant vaccinia-virus (rVV)-infected mature or immature dendritic cells. Gene Ther. 2003; 10:1754-1765.
228. Fodor I, Timiryasova T, Denes B, Yoshida J, Ruckle H, Lilly M. Vaccinia virus mediated p53 gene therapy for bladder cancer in an orthotopic murine model. J Urol. 2005; 173:604-609.
229. Ramírez JC, Gherardi MM, Esteban M. Biology of attenuated modified vaccinia virus Ankara recombinant vector in mice: virus fate and activation of B-and T-cell immune responses in comparison with the Western Reserve strain and advantages as a vaccine. J Virol. 2000; 74:923-933.
230. Puhlmann M, Brown CK, Gnant M, Huang J, Libutti SK, Alexander HR, Bartlett DL. Vaccinia as a vector for tumor-directed gene therapy: biodistribution of a thymidine kinase-deleted mutant. Cancer Gene Ther. 2000; 7:66-73.
231. Erbs P, Findeli A, Kintz J, Cordier P, Hoffmann C, Geist M, Balloul JM. Modified vaccinia virus Ankara as a vector for suicide gene therapy. Cancer Gene Ther. 2008; 15:18-28.
232. Hiley CT, Yuan M, Lemoine NR, Wang Y. Lister strain vaccinia virus, a potential therapeutic vector targeting hypoxic tumours. Gene Ther. 2010; 17:281-287.
233. Thirunavukarasu P, Sathaiah M, Gorry MC, O'Malley ME, Ravindranathan R, Austin F, Thorne SH, Guo ZS, Bartlett DL. A rationally designed A34R mutant oncolytic poxvirus: improved efficacy in peritoneal carcinomatosis. Mol Ther. 2013; 21:1024-1033.
234. Lee JH, Roh MS, Lee YK, Kim MK, Han JY, Park BH, Trown P, Kirn DH, Hwang TH. Oncolytic and immunostimulatory efficacy of a targeted oncolytic poxvirus expressing human GM-CSF following intravenous administration in a rabbit tumor model. Cancer Gene Ther. 2010; 17:73-79.
235. Yu F, Wang X, Guo ZS, Bartlett DL, Gottschalk SM. T-cell engager-armed oncolytic vaccinia virus significantly enhances antitumor therapy. Mol Ther. 2014; 22:102-111.
236. Kaufman HL, Bines SD. OPTIM trial: a Phase III trial of an oncolytic herpes virus encoding GM-CSF for unresectable stage III or IV melanoma. Future Oncol. 2010; 6:941-949.
237. Heo J, Reid T, Ruo L, Breitbach CJ, Rose S, Bloomston M, Cho M, Lim HY, Chung HC, Kim CW, Burke J, Lencioni R, Hickman T, Moon A, Lee YS, Kim MK, et al. Randomized dose-finding clinical trial of oncolytic immunotherapeutic vaccinia JX-594 in liver cancer. Nat Med. 2013; 19:329-336.
238. Chorny M, Fishbein I, Alferiev IS, Nyanguile O, Gaster R, Levy RJ. Adenoviral gene vector tethering to nanoparticle surfaces results in receptor-independent cell entry and increased transgene expression. Mol Ther. 2006; 14:382-391.
239. Pushko P, Pumpens P, Grens E. Development of virus-like particle technology from small highly symmetric to large complex virus-like particle structures. Intervirology. 2013; 56:141-165.
240. Zeltins A. Construction and characterization of virus-like particles: a review. Mol Biotechnol. 2013; 53:92-107.
241. Erbacher P, Remy JS, Behr JP. Gene transfer with synthetic virus-like particles via the integrin-mediated endocytosis pathway. Gene Ther. 1999; 6:138-145.
242. Krauzewicz N, Stokrová J, Jenkins C, Elliott M, Higgins CF, Griffin BE. Virus-like gene transfer into cells mediated by polyoma virus pseudocapsids. Gene Ther. 2000; 7:2122-2131.
243. Müller K, Nahde T, Fahr A, Müller R, Brüsselbach S. Highly efficient transduction of endothelial cells by targeted artificial virus-like particles. Cancer Gene Ther. 2001; 8:107-117.
244. Keswani RK, Pozdol IM, Pack DW. Design of hybrid lipid/retroviral-like particle gene delivery vectors. Mol Pharm. 2013; 10:1725-1735.
245. Keswani R, Su K, Pack DW. Efficient in vitro gene delivery by hybrid biopolymer/virus nanobiovectors. J Control Release. 2014; 192:40-46.
246. Hajitou A, Rangel R, Trepel M, Soghomonyan S, Gelovani JG, Alauddin MM, Pasqualini R, Arap W. Design and construction of targeted AAVP vectors for mammalian cell transduction. Nat Protoc. 2007; 2:523-531.
247. Asavarut P, O'Neill K, Syed N, Hajitou A. Chimeric adeno-associated virus and bacteriophage: a potential targeted gene therapy vector for malignant glioma. Ther Deliv. 2014; 5:975-990.
248. Pranjol MZI, Hajitou A. Bacteriophage-derived vectors for targeted cancer gene therapy. Viruses. 2015; 7:268-284.
249. Stoneham CA, Hollinshead M, Hajitou A. Clathrinmediated endocytosis and subsequent endo-lysosomal trafficking of adeno-associated virus/phage. J Biol Chem. 2012; 287:35849-35859.
250. Przystal JM, Umukoro E, Stoneham CA, Yata T, O'Neill K, Syed N, Hajitou A. Proteasome inhibition in cancer is associated with enhanced tumor targeting by the adeno-associated virus/phage. Mol Oncol. 2013; 7:55-66.