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2013 | 73 | 1 |

Tytuł artykułu

Genetic engineering of stem cells for enhanced therapy

Warianty tytułu

Języki publikacji

EN

Abstrakty

EN
Stem cell therapy is a promising strategy for overcoming the limitations of current treatment methods. The modification of stem cell properties may be necessary to fully exploit their potential. Genetic engineering, with an abundance of methodology to induce gene expression in a precise and well-controllable manner, is particularly attractive for this purpose. There are virus-based and non-viral methods of genetic manipulation. Genome-integrating viral vectors are usually characterized by highly efficient and long-term transgene expression, at a cost of safety. Non-integrating viruses are also highly efficient in transduction, and, while safer, offer only a limited duration of transgene expression. There is a great diversity of transfectable forms of nucleic acids; however, for efficient shuttling across cell membranes, additional manipulation is required. Both physical and chemical methods have been employed for this purpose. Stem cell engineering for clinical applications is still in its infancy and requires further research. There are two main strategies for inducing transgene expression in therapeutic cells: transient and permanent expression. In many cases, including stem cell trafficking and using cell therapy for the treatment of rapid-onset disease with a short healing process, transient transgene expression may be a sufficient and optimal approach. For that purpose, mRNA-based methods seem ideally suited, as they are characterized by a rapid, highly efficient transfection, with outstanding safety. Permanent transgene expression is primarily based on the application of viral vectors, and, due to safety concerns, these methods are more challenging. There is active, ongoing research toward the development of non-viral methods that would induce permanent expression, such as transposons and mammalian artificial chromosomes.

Słowa kluczowe

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-

Rocznik

Tom

73

Numer

1

Opis fizyczny

p.1-18,fig.,ref.

Twórcy

  • NeuroRepair Department, Mossakowski Medical Research Centre, Polish Academy of Sciences, Warsaw, Poland
  • NeuroRepair Department, Mossakowski Medical Research Centre, Polish Academy of Sciences, Warsaw, Poland
autor
  • NeuroRepair Department, Mossakowski Medical Research Centre, Polish Academy of Sciences, Warsaw, Poland
  • Russell H. Morgan Department of Radiology and Radiological Science, Division of MR Research, the Johns Hopkins University School of Medicine, Baltimore, USA
  • Cellular Imaging Section and Vascular Biology Program, Institute for Cell Engineering, The Johns Hopkins University School of Medicine, Baltimore, USA
  • Department of Neurosurgery, Mossakowski Medical Research Centre, Polish Academy of Sciences, Warsaw, Poland
autor
  • Russell H. Morgan Department of Radiology and Radiological Science, Division of MR Research, the Johns Hopkins University School of Medicine, Baltimore, USA
  • Cellular Imaging Section and Vascular Biology Program, Institute for Cell Engineering, The Johns Hopkins University School of Medicine, Baltimore, USA
autor
  • NeuroRepair Department, Mossakowski Medical Research Centre, Polish Academy of Sciences, Warsaw, Poland

Bibliografia

  • Alba R, Bosch A, Chillon M (2005) Gutless adenovirus: last-generation adenovirus for gene therapy. Gene Ther 1: S18-27.
  • Aluigi M, Fogli M, Curtil A, Isidori A, Gruppioni E, Chiodoni C, Colombo M, Versura P, D'Errico-Grigioni A, Ferri E, Baccarani M, Lemoli RM (2006) Nucleofection is an efficient nonviral transfection technique for human bone marrow-derived mesenchymal stem cells. Stem Cells 24: 454-461.
  • Apolonia L, Waddington SN, Fernandes C, Ward NJ, Bouma G, Blundell MP, Thrasher AJ, Collins MK, Philpott NJ (2007) Stable gene transfer to muscle using non-integrat¬ing lentiviral vectors. Mol Ther 15: 1947-1954.
  • Aronovich EL, McIvor RS, Hackett PB (2011) The Sleeping Beauty transposon system: a non-viral vector for gene therapy. Hum Mol Genet 20: R14-R20.
  • Asano T, Hanazono Y, Ueda Y, Muramatsu S, Kume A, Suemori H, Suzuki Y, Kondo Y, Harii K, Hasegawa M, Nakatsuji N, Ozawa K (2002) Highly efficient gene trans¬fer into primate embryonic stem cells with a simian lenti- virus vector. Mol Ther 6: 162-168.
  • Bachrach U (2005) Naturally occurring polyamines: interaction with macromolecules. Curr Protein Pept Sci 6: 559-566.
  • Bell AJ Jr, Fegen D, Ward M, Bank A (2010) RD114 enve¬lope proteins provide an effective and versatile approach to pseudotype lentiviral vectors. Exp Biol Med (Maywood) 235: 1269-1276.
  • Bersenev A, Levine BL (2012) Convergence of gene and cell therapy. Regen Med 7: 50-56.
  • Burt RK, Loh Y, Cohen B, Stefoski D, Balabanov R, Katsamakis G, Oyama Y, Russell EJ, Stern J, Muraro P, Rose J, Testori A, Bucha J, Jovanovic B, Milanetti F, Storek J, Voltarelli JC, Burns WH (2009) Autologous non-myeloablative haemopoi- etic stem cell transplantation in relapsing-remitting multiple sclerosis: a phase I/II study. Lancet Neurol 8: 244-253.
  • Cai B, Li J, Wang J, Luo X, Ai J, Liu Y, Wang N, Liang H, Zhang M, Chen N, Wang G, Xing S, Zhou X, Yang B, Wang X, Lu Y (2012) microRNA-124 regulates cardio- myocyte differentiation of bone marrow-derived mesen- hymal stem cells via targeting STAT3 signaling. Stem Cells 30: 1746-1755.
  • Candotti F, Shaw KL, Muul L, Carbonaro D, Sokolic R, Choi C, Schurman SH, Garabedian E, Kesserwan C, Jagadeesh GJ, Fu PY, Gschweng E, Cooper A, Tisdale JF, Weinberg KI, Crooks GM, Kapoor N, Shah A, Abdel- Azim H, Yu XJ, Smogorzewska M, Wayne AS, Rosenblatt HM, Davis CM, Hanson C, Rishi RG, Wang X, Gjertson D, Yang OO, Balamurugan A, Bauer G, Ireland JA, Engel BC, Podsakoff GM, Hershfield MS, Blaese RM, Parkman R, Kohn DB (2012) Gene therapy for adenosine deami- nase-deficient severe combined immune deficiency: clin¬ical comparison of retroviral vectors and treatment plans. Blood 120: 3635-3646.
  • Cao F, Xie X, Gollan T, Zhao L, Narsinh K, Lee RJ, Wu JC (2010) Comparision of Gene - Transfer Efficiency in Human Embryonic Stem Cells. Mol Imaging Biol 12: 15-24.
  • Cao X, Deng W, Wei Y, Su W, Yang Y, Wei Y, Yu J, Xu X (2010) Encapsulation of plasmid DNA in calcium phos¬phate nanoparticles: stem cell uptake and gene transfer efficiency. Int J Nanomedicine 6: 3335-3349.
  • Carson SD, Hobbs JT, Tracy SM, Chapman NM (1999) Expression of the coxsackievirus and adenovirus receptor in cultured human umbilical vein endothelial cells: regu¬lation in response to cell density. J Virol 73: 7077-7079.
  • Challita PM, Kohn DB (1994) Lack of expression from a retroviral vector after transduction of murine hematopoietic stem cells is associated with methylation in vivo. Proc Natl Acad Sci USA 91: 2567-2571.
  • Cho JW, Lee CY, Ko Y (2012) Therapeutic potential of mes- enchymal stem cells overexpressing human forkhead box A2 gene in the regeneration of damaged liver tissues. J Gastroenterol Hepatol 27: 1362-1370.
  • Ciani L, Casini A, Gabbiani C, Ristori S, Messori L, Martini G (2007) DOTAP/DOPE and DC-Chol/DOPE lipoplexes for gene delivery studied by circular dichroism and other biophysical tehniques. Biophys Chem 127: 213-220.
  • D'Costa J, Mansfield SG, Humeau LM (2009) Lentiviral vectors in clinical trials: Current status. Curr Opin Mol Ther 11: 554-564.
  • Dalgleish AG, Beverley PCL, Clapham PR, Crawford DH, Greaves MF, Weiss RA (1984) The CD4 (T4) antigen is an essential component of the receptor for the AIDS ret- rovirus. Nature 312: 763-767.
  • Dewey RA, Morrissey G, Cowsill CM, Stone D, Bolognani F, Dodd NJ, Southgate TD, Klatzmann D, Lassmann H, Castro MG, Lowenstein PR (1999) Chronic brain inflam¬mation and persistent herpes simplex virus 1 thymidine kinase expression in survivors of syngeneic glioma treated by adenovirus-mediated gene therapy: implica¬tions for clinical trials. Nat Med 5: 1256-1263.
  • Di Matteo M, Belay E, Chuah MK, Vandendriessche T (2012) Recent developments in transposon-mediated gene therapy. Expert Opin Biol Ther 12: 841-858.
  • Dunbar CE, Takatoku M, Donahue RE (2001) The impact of ex vivo cytokine stimulation on engraftment of primitive hematopoietic cells in a non-human primate model. Ann N Y Acad Sci 938: 236-245.
  • Ferreira E, Potier E, Vaudin P, Oudina K, Bensidhoum M, Logeart-Avramoglou D, Mir LM, Petite H (2012) Sustained and promoter dependent bone morphogenetic protein expression by rat mesenchymal stem cells after BMP-2 transgene electrotransfer. Eur Cell Mater 24: 18-28.
  • Flanagan M, Gimble JM, Yu G, Wu X, Li S (2011) Competitive electroporation formulation for cell therapy. Cancer Gene Ther 18: 579-586.
  • Fusaki N, Ban H, Nishiyama A, Saeki K, Hasegawa M (2009) Efficient induction of transgene-free human pluri- potent stem cells using a vector based on Sendai virus, an RNA virus that does not integrate into the host genome. Proc Jpn Acad Ser B Phys Biol Sci 85: 348-362.
  • Gage FH (2012) Transplantation in the future. Prog Brain Res 200: 7-13.
  • Gall J, Kass-Eisler A, Leinwand L, Falck-Pedersen E (1996) Adenovirus type 5 and 7 capsid chimera: fiber replace¬ment alters receptor tropism without affecting primary immune neutralization epitopes. J Virol 70: 2116-2123.
  • Gehl J (2003) Electroporation: theory and methods, perspec¬tives for drug delivery, gene therapy and research. Acta Physiol Scand 177: 437-447.
  • Gorelik M, Orukari I, Wang J, Galpoththawela S, Kim H, Levy M, Gilad AA, Bar-Shir A, Kerr DA, Levchenko A, Bulte JW, Walczak P (2012) Use of MR cell tracking to evaluate targeting of glial precursor cells to inflammatory tissue by exploiting the very late antigen-4 docking receptor. Radiology 265: 175-185.
  • Gracey Maniar LE, Maniar JM, Chen ZY, Lu J, Fire AZ, Kay MA (2013) Minicircle DNA vectors achieve sus¬tained expression reflected by active chromatin and tran- scriptional level. Mol Ther 21: 131-138.
  • Gresch O, Engel FB, Nesic D, Tran TT, England HM, Hickman ES, Korner I, Gan L, Chen S, Castro-Obregon S, Hammermann R, Wolf J, Muller-Hartmann H, Nix M, Siebenkotten G, Kraus G, Lun K (2004) New non-viral method for gene transfer into primary cells. Methods 33: 151-163.
  • Gul-Uludag H, Xu P, Marquez-Curtis LA, Xing J, Janowska- Wieczorek A, Chen J (2012) Cationic liposome-mediated CXCR4 gene delivery into hematopoietic stem/progeni¬tor cells: implications for clinical transplantation and gene therapy. Stem Cells Dev 21: 1587-1596.
  • Guzman-Villanueva D, El-Sherbiny IM, Herrera-Ruiz D, Vlassov AV, Smyth HDC (2012) Formulation Approaches to Short Interfering RNA and MicroRNA: Challenges and Implications. J Pharm Sci 101: 4046-4066.
  • Hacein-Bey-Abina S, Hauer J, Lim A, Picard C, Wang GP, Berry CC, Martinache C, Rieux-Laucat F, Latour S, Belohradsky BH, Leiva L, Sorensen R, Debre M, Casanova JL, Blanche S, Durandy A, Bushman FD, Fischer A, Cavazzana-Calvo M (2010) Efficacy of gene therapy for X-linked severe combined immunodeficiency. N Engl J Med 363: 355-364.
  • Han SW, Nakamura C, Kotobuki N, Obataya I, Ohgushi H, Nagamune T, Miyake J (2008) High-efficiency DNA injection into a single human mesenchymal stem cell using a nanoneedle and atomic force microscopy. Nanomedicine 4: 215-225.
  • Hanazono Y, Asano T, Ueda Y, Ozawa K (2003) Genetic manipulation of primate embryonic and hematopoietic stem cells with simian lentivirus vectors. Trends Cardiovasc Med 13: 106-110.
  • Hayashi T, Lamba DA, Slowik A, Reh TA, Bermingham- McDonogh O (2010) A method for stabilizing RNA for transfection that allows control of expression duration. Dev Dyn 239: 2034-2040.
  • Hollis RP, Nightingale SJ, Wang X, Pepper KA, Yu XJ, Barsky L, Crooks GM, Kohn DB (2006) Stable gene trans¬fer to human CD34 hematopoietic cells using the Sleeping Beauty transposon. Exp Hematol 34: 1333-1343.
  • Hosoya N, Miura T, Kawana-Tachikawa A, Koibuchi T, Shioda T, Odawara T, Nakamura T, Kitamura Y, Kano M, Kato A, Hasegawa M, Nagai Y, Iwamoto A (2008) Comparison between Sendai virus and adenovirus vectors to transduce HIV-1 genes into human dendritic cells. J Med Virol 80: 373-382.
  • Huang S, Terstappen LW (1994) Lymphoid and myeloid dif¬ferentiation of single human CD34+, HLA-DR+, CD38- hematopoietic stem cells. Blood 83: 1515-1526.
  • Ito H, Goater JJ, Tiyapatanaputi P, Rubery PT, O'Keefe RJ, Schwarz EM (2004) Light-activated gene transduction of recombinant adeno-associated virus in human mesenchy¬mal stem cells. Gene Ther 11: 34-41.
  • Ivics Z, Hackett PB, Plasterk RH, Izsvak Z (1997) Molecular reconstruction of sleeping beauty, a Tc1-like transposon from fish, and its transposition in human cells. Cell 91: 501-510.
  • Izsvak Z, Chuah MKL, VandenDriessche T, Ivics Z (2009) Efficient stable gene transfer into human cells by the Sleeping Beauty transposon vectors. Methods 49: 287¬297.
  • Janowski M, Date I (2009) Systemic neurotransplantation--a problem-oriented systematic review. Rev Neurosci 20: 39-60.
  • Jeon SY, Park JS, Yang HN, Woo DG, Park KH (2012) Co-delivery of SOX9 genes and anti-Cbfa siRNA coated onto PLGA nanoparticles for chondrogenesis of human MSCs. Biomaterials 33: 4413-4423.
  • Jo J, Nagaya N, Miyahara Y, Kataoka M, Harada-Shiba M, Kangawa K, Tabata Y (2007) Transplantation of geneti¬cally engineered mesenchymal stem cells improves car¬diac function in rats with myocardial infarction: Benefit of a novel nonviral vector, cationized dextran. Tissue Eng 13: 313-322.
  • Jordan M, Wurm F (2004) Transfection of adherent and suspended cells by calcium phosphate. Methods 33: 136-143.
  • Kang W, Hebert M (2012) A Sox2 BAC yransgenic approach for targeting adult neural stem cells. PLoS One 7: 1-7.
  • Katona RL, Vanderbyl SL, Perez CF (2011) Mammalian artificial chromosomes and clinical applications for genetic modification of stem cells: an overview. Methods Mol Biol 738: 199-216.
  • Kazuki Y, Hiratsuka M, Takiguchi M, Osaki M, Kajitani N, Hoshiya H, Hiramatsu K, Yoshino T, Kazuki K, Ishihara C, Takehara S, Higaki K, Nakagawa M, Takahashi K, Yamanaka S, Oshimura M (2010) Complete genetic cor¬rection of ips cells from Duchenne muscular dystrophy. Mol Ther 18: 386-393.
  • Kazuki Y, Oshimura M (2011) Human artificial chromo¬somes for gene delivery and the development of animal Models. Mol Ther 19: 1591-1601.
  • Keravala A, Ormerod BK, Palmer TD, Calos MP (2008) Long-term transgene expression in mouse neural pro¬genitor cells modified with PhiC31 integrase. J Neurosci Methods 173: 299-305.
  • Kim HH, Choi HS, Yang JM, Shin S (2007) Characterization of gene delivery in vitro and in vivo by the arginine pep- tide system. Int J Pharm 335: 70-78.
  • Kim HJ, Im GI (2011) Electroporation-Mediated Transfer of SOX trio genes (SOX-5, SOX-6, and SOX-9) to enhance the chondrogenesis of mesenchymal stem cells. Stem Cells Dev 20: 2103-2114.
  • Kim NY, Choi YB, Kang CI, Kim HH, Yang JM, Shin S (2010) An hydrophobically modified arginine peptide vector system effectively delivers DNA into human mes- enchymal stem cells and maintains transgene expression with differentiation. J Gene Med 12: 779-789.
  • Kim SJ, Lee WI, Heo H, Shin O, Kwon YK, Lee H (2007) Stable gene expression by self-complementary adeno-as- sociated viruses in human MSCs. Biochem Biophys Res Commun 360: 573-579.
  • Kim TH, Kim M, Eltohamy M, Yun YR, Jang JH, Kim HW (2012) Efficacy of mesoporous silica nanoparticles in delivering BMP-2 plasmid DNA for in vitro osteogenic stimulation of mesenchymal stem cells. J Biomed Mater Res A (in press).
  • Knaän-Shanzer S, van de Watering MJ, van der Velde I, Goncalves MA, Valerio D, de Vries AA (2005) Endowing human adenovirus serotype 5 vectors with fiber domains of species B greatly enhances gene transfer into human mesenchymal stem cells. Stem Cells 23: 1598-1607.
  • Krause CD, Izotova LS, Ren G, Yuan ZR, Shi Y, Chen CC, Ron Y, Pestka S (2011) Efficient co-expression of bicis- tronic proteins in mesenchymal stem cells by develop¬ment and optimization of a multifunctional plasmid. Stem Cell Res Ther 2: 15.
  • Kuhn AN, Beißert T, Simon P, Vallazza B, Buck J, Davies BP, Tureci O, Sahin U (2012) mRNA as a versatile tool for exogenous protein expression. Curr Gene Ther 12: 347-361.
  • Laker C, Meyer J, Schopen A, Friel J, Heberlein C, Ostertag W, Stocking C (1998) Host cis-mediated extinction of a retrovirus permissive for expression in embryonal stem cells during differentiation. J Virol 72: 339-348.
  • Lamb BT, Gearhart JD (1995) YAC transgenics and the study of genetics and human disease. Curr Opin Genet Dev 5: 342-348.
  • Lamb RA, Kolakofsky D (2001) Paramyxoviridae: The viruses and their replication. In: fields Virology (4th edi¬tion) (Knipe DM, Howley PM, Eds). Lippincott Williams and Wilkins, Philadeplphia, PA. p. 1305-1340.
  • Lan F, Liu J, Narsinh KH, Hu S, Han L, Lee AS, Karow M, Nguyen PK, Nag D, Calos MP, Robbins RC, Wu JC (2012) Safe Genetic Modification of Cardiac Stem Cells Using a Site-Specific Integration Technique. Circulation 126: S20 -S28.
  • Li W, Ma N, Ong LL, Nesselmann C, Klopsch C, Ladilov Y, Furlani D, Piechaczek C, Moebius JM, Tzow KL, Lendlein A, Stamm C, Li RK, Steinhoff G (2007) Bcl-2 engineered MSCs inhibited apoptosis and improved heart function. Stem Cells 25: 2118-2127.
  • Lim JY, Park SH, Jeong CH, Oh JH, Kim SM, Ryu CH, Park SA, Ahn JG, Oh W, Jeun SS, Chang JW (2010) Microporation is a valuable transfection method for effi¬cient gene delivery into human umbilical cord blood-de¬rived mesenchymal stem cells. BMC Biotechnol 10: 38.
  • Lindenbaum M, Perkins E, Csonka E, Fleming E, Garcia L, Greene A, Gung L, Hadlaczky G, Lee E, Leung J,
  • MacDonald N, Maxwell A, Mills K, Monteith D, Perez CF, Shellard J, Stewart S, Stodola T, Vandenborre D, Vanderbyl S, Ledebur HC Jr (2004) A mammalian artifi¬cial chromosome engineering system (ACE System) applicable to biopharmaceutical protein production, trans¬genesis and gene-based cell therapy. Nucleic Acids Res 32: 1-15.
  • Liu L, Wang Y, Fan H, Zhao X, Liu D, Hu Y, Kidd AR III, Bao J Hou Y (2012) MicroRNA-181a regulates local immune balance by inhibiting proliferation and immuno- suppressive properties of mesenchyml stem cells. Stem Cells 30: 1756-1770.
  • Lodish H, Berk A, Zipursky SL, Matsudaira P, Baltimore D, Darnell J (2000) Molecular Cell Biology (4th edition). W.H. Freeman, New York, NY [Bookshelf ID: NBK21498]
  • Ma Y, Ramezani A, Lewis R, Hawley RG, Thomson JA (2003) High-level sustained transgene expression in human embryonic stem cells using lentiviral vectors. Stem Cells 21: 111-117.
  • Masotti A, Mossa G, Cametti C, Ortaggi G, Bianco A, del Grosso N, Malizia D, Esposito C (2009) Comparision of different commercially available cationic liposome-DNA lipoplexes: Parameters influencing toxicity and transfection efficiency. Colloids Surf B Biointerfaces 68: 136-144.
  • McCarter SD, Scott JR, Lee PJ, Zhang X, Choi AM, McLean CA, Badhwar A, Dungey AA, Bihari A, Harris KA, Potter RF (2003) Cotransfection of heme oxygenase-1 prevents the acute inflammation elicited by a second adenovirus. Gene Ther 10: 1629-1635.
  • McGinley L, McMahon J, Strappe P, Barry F, Murphy M, O'Toole D, O'Brien T (2011) Lentiviral vector mediated modification of mesenchymal stem cells & enhanced survival in an in vitro model of ischaemia. Stem Cell Res Ther 2: 12.
  • McLenachan S, Sarsero JP, Ioannou PA (2007) Flow- cytometric analysis of mouse embryonic stem cell lipo- fection using small and large DNA constructs. Genomics 89: 708-720.
  • Miyoshi H, Blömer U, Takahashi M, Gage FH, Verma IM (1998) Development of a self-inactivating lentivirus vec¬tor. J Virol 72: 8150-8157.
  • Muenthaisong S, Ujhelly O, Polgar Z, Varga E, Ivics Z, Pirity MK, Dinnyes A (2012) Generation of mouse induced pluripotent stem cells from different genetic backgrounds using Sleeping beauty transposon mediated gene transfer. Exp Cell Res 318: 2482-2489.
  • Nagane K, Jo J, Tabata Y (2010) Promoted adipogenesis of rat mesenchymal stem cells by transfection of small inter¬fering RNA complexed with a cationized dextran. Tissue Eng Part A 16: 21-31.
  • Nakashima M, Tachibana K, Iohara K, Ito M, Ishikawa M, Akamine A (2003) Induction of reparative dentin forma¬tion by ultrasound-mediated gene delivery of growth/dif¬ferentiation factor 11. Hum Gene Ther 14: 591-597.
  • Nakashima S, Matsuyama Y, Nitta A,Sakai Y, Ishiguro N (2005) Highly efficient transfection of human marrow stromal cells by nucleofection. Transplant Proc 37: 2290-2292.
  • Naldini L, Blomer U, Gage FH, Trono D, Verma IM (1996) Efficient transfer, integration, and sustained long-term expression of the transgene in adult rat brains injected with a lentiviral vector. Proc Natl Acad Sci USA 93: 11382-11388.
  • Otani K, Yamahara K, Ohnishi S, Obata H, Kitamura S, Nagaya N (2009) Nonviral delivery of siRNA into mes- enchymal stem cells by a combination of ultrasound and microbubbles. J Control Release 133: 146-153.
  • Park SA, Ryu CH, Kim SM, Lim JY, Park SI, Jeong CH, Jun JA, Oh JH, Park SH, Oh W, Jeun SS (2011) CXCR4- transfected human umbilical cord blood-derived mesen- chymal stem cells exhibit enhanced migratory capacity toward gliomas. Int J Oncol 38: 97-103.
  • Park SJ, Na K (2012) The transfection efficiency of photo- sensitizer-induced gene delivery to human MSCs and internalization rates of eGFP and Runx2 genes. Biomaterials 33: 6485-6494.
  • Pollock K, Stroemer P, Patel S, Stevanato L, Hope A, Miljan E, Dong Z, Hodges H, Price J, Sinden JD (2006) A con¬ditionally immortal clonal stem cell line from human cortical neuroepithelium for the treatment of ischemic stroke. Exp Neurol 199: 143-155.
  • Pozzi D, Marhini C, Cardarelli F, Amenitsch H, Garulli C, Bifone A, Caracciolo G (2012) Transfection efficiency boost of cholesterol-containing lipoplexes. Biochim Biophys Acta 1818: 2335-2343.
  • Raimondi E (2011) Naturally occurring minichromosome platforms in chromosome engineering: an overview. Methods Mol Biol 738: 41-56.
  • Ramalingam S, London V, Kandavelou K, Cebotaru L, Guggino W, Civin C, Chandrasegaran S (2013) Generation and genetic engineering of human induced pluripotent stem cells using designed zinc finger nucleases. Stem Cells Dev 22: 595-610.
  • Rejman J, Conese M, Hoekstra D (2006) Gene transfer by means of lipo- and polyplexes: role of clatrin and caveo- lae-mediated endocytosis. J Liposome Res 16: 237-247.
  • Rejman J, Tavernier G, Bavarsad N, Demeester J, de Smedt SC (2010) mRNA transfection of cervical carcinoma and mesenchymal stem cells mediated by cationic carriers. J Control Release 147: 385-391.
  • Rome C, Deckers R, Moonen CTW (2008) The use of ultra¬sound in transfection and transgene expression. Handb Exp Pharmacol 185: 225-243.
  • Ruponen M, Ronkko S,Honkakoski P, Pelkonen J, Tammi M, Urtti A (2001) Extracellular glycosaminoglycans modify cellular trafficking of lipoplexes and polyplexes. J Biol Chem 276: 33875-33880.
  • Ryser MF, Ugarte F, Thieme S, Bornhauser M, Roesen- Wolff A, Brenner S (2008) mRNA Transfection of CXCR4-GFP fusion-simply generated by PCR-results in efficient migration of primary human mesenchymal stem cells. Tissue Eng Part C Methods 14: 179-184.
  • Sakuma T, Hosoi S, Woltjen K, Suzuki KI, Kashiwagi K, Wada H, Ochiai H, Miyamoto T, Kawai N, Sasakura Y, Matsuura S, Okada Y, Kawahara A, Hayashi S, Yamamoto T (2013) Efficient TALEN construction and evaluation methods for human cell and animal applications. Genes Cells 18: 315-326.
  • Sanchez-Antequera Y, Mykhaylyk O, van Til NP, Cengizeroglu A, Henk de Jong J, Huston MW, Anton M, Johnston ICD, Pojda Z, Wagemaker G, Plank C (2011) Magselectofection: an integrated method of nanomag- netic separation and genetic modification of target cells. Blood 117: 171-181.
  • Santos JL, Oliveira H, Pandita D, Rodrigues J, Pego AP, Granja PL, Tomas H (2010) Functionalization of poly(amidoamine) dendrimers with hydrophobic chains for improved gene delivery in mesenchymal stem cells. J Control Release 144: 55-64.
  • Scaife MD, Neschadim A, Fowler DH, Medin JA (2009) Novel application of lentiviral vectors towards treatment of graft- versus-host disease. Expert Opin Biol Ther 9: 749-761.
  • Schambach A, Zychlinski D, Ehrnstrom B, Baum C (2013) Biosafety features of lentiviral vectors. Hum Gene Ther 24: 132-142.
  • Simoes S, Slepushkin V, Pires P, Gaspar R, Pedroso de Lima MC, Duzgunes N (1999) Mechanisms of gene transfer mediated by lipolexes associated with targeting ligands or pH-sensitive peptides. Gene Ther 6: 1798¬1807.
  • Sinden JD, Vishnubhatla I, Muir KW (2012) Prospects for stem cell-derived therapy in stroke. Prog Brain Res 201: 119-167.
  • Slowing II, Vivero-Escoto JL, Wu CW, Lin VS (2008) Mesoporous silica nanoparticles as controlled release drug delivery and gene transfection carriers. Adv Drug Deliv Rev 60: 1278-1288.
  • Snead NM, Rossi JJ (2012) RNA Interference Trigger Variants: Getting the Most Out of RNA for RNA Interference-Based Therapeutics. Nucleic Acid Ther 22: 139-146.
  • Stadtfeld M, Nagaya M, Utikal J, Weir G, Hochedlinger K (2008) Induced pluripotent stem cells generated without viral integration. Science 322: 945-949.
  • Stroemer P, Hope A, Patel S, Pollock K, Sinden J (2008) Development of a human neural stem cell line for use in recovery from disability after stroke. Front Biosci 13: 2290-2292.
  • Suzuki K, Mitsui K, Aizawa E, Hasegawa K, Kawase E, Yamagishi T, Shimizu Y, Suemori H, Nakatsuji N, Mitani K (2008) Highly efficient transient gene expression and gene targeting in primate embryonic stem cells with helper-dependent adenoviral vectors. Proc Natl Acad Sci U S A 105: 13781-13786.
  • Thakor DK, Teng YD, Obata H, Nagane K, Saito S, Tabata Y (2011) Nontoxic genetic engineering of esenchymal stem cells using serum-compatible pullulan-spermine/ DNA anioplexes. Tissue Eng Part C Methods 17: 131¬144.
  • Tinsley RB, Vesey MJ, Barati S, Rush RA, Ferguson IA (2004) Improved non-viral transfection of glial and adult neural stem cell lines and of primary astrocytes by com¬bining agents with complementary modes of action. J Gene Med 6: 1023-1032.
  • Treacy O, Ryan AE, Heinzl T, O'Flynn L, Cregg M, Wilk M, Odoardi F, Lohan P, O'Brien T, Nosov M, Ritter T (2012) Adenoviral transduction of mesenchymal stem cells: in vitro responses and in vivo immune responses after cell transplantation. PLoS One 7: e42662.
  • Wen Y, Guo Z, Du Z, Fang R, Wu H, Zeng X, Wang C, Feng M, Pan S (2012) Serum tolerance and endosomal escape capacity of histidine-modified pDNA-loaded complexes based on polyamidoamine dendrimer derivatives. Biomaterials 33: 8111-8121.
  • Wiehe JM, Ponsaerts P, Rojewski MT, Homann JM, Greiner J, Kronawitter D, Schrezenmeier H, Hombach V, Wiesneth M, Zimmermann O, Torzewski J (2007) mRNA-mediated gene delivery into human progenitor cells promotes highly efficient protein expression. J Cell Mol Med 11: 521-530.
  • Wiehe JM, Kaya Z, Homann JM, Wohrle J, Vogt K, Nguyen T, Rottbauer W, Torzewski J, Fekete N, Rojewski M, Schrezenmeier H, Moepps B, Zimmermann O (2012) GMP-adapted overexpression of CXCR4 in human mes- enchymal stem cells for cardiac repair. Int J Cardiol (in press).
  • Woltjen K, Hamalainen R, Kibschull M, Mileikovsky M, Nagy A (2011) Transgene-free production of pluripotent stem cells using piggyBac transposons. Methods Mol Biol 767: 87-103.
  • Wright JF (2009) Transient transfection methods for clinical adeno-associated viral vector production. Hum Gene Ther 20: 698-706.
  • Wu C, Dunbar CE (2011) Stem cell gene therapy: the risks of insertional mutagenesis and approaches to minimize genotoxicity. Front Med 5: 356-371.
  • Yang Y, Nunes FA, Berencsi K, Furth EE, Gonczol E, Wilson JM (1994) Cellular immunity to viral antigens limits E1-deleted adenoviruses for gene therapy. Proc Natl Acad Sci U S A 91: 4407-4411.
  • Yoshida A, Yamaguchi Y, Nonomura K, Kawakami K, Takahashi Y, Miura M (2010) Simultaneous expression of different transgenes in neurons and glia by combin¬ing in utero electroporation with the Tol2 transposon- mediated gene transfer system. Genes Cells 15: 501¬512.
  • Yu X, Cohen DM, Chen CS (2012) miR-125b is an adhe¬sion-regulated microRNA that protects mesenchymal stem cells from anoikis. Stem Cells 30: 956-964.
  • Zaiss AK, Liu Q, Bowen GP, Wong NC, Bartlett JS, Muruve DA (2002) Differential activation of innate immune responses by adenovirus and adeno-associated virus vec¬tors. J Virol 76: 4580-4590.
  • Zaldumbide A, Carlotti F, Gongalves MA, Knaän-Shanzer S, Cramer SJ, Roep BO, Wiertz EJ, Hoeben RC (2012) Adenoviral vectors stimulate glucagon transcription in human mesenchymal stem cells expressing pancreatic transcription factors. PLoS One 7: e48093.
  • Zhang XY, La Russa VF, Bao L, Kolls J, Schwarzenberger P, Reiser J (2002) Lentiviral vectors for sustained trans¬gene expression in human bone marrowderived stromal cells. Mol Ther 5: 555-565.
  • Ziv R, Steinhardt Y, Pelled G, Gazit D, Rubinsky B (2009) Micro-electroporation of mesenchymal stem cells with alternating electrical current pulses. Biomed Microdevices 11: 95-101.

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