Abstract
This chapter gives a comparative review of the different vector systems applied to date in prenatal gene therapy experiments highlighting the need for versatility and choice for application in accordance with the actual aim of the study. It reviews the key characteristics of the four main gene therapy vector systems and gives examples for their successful application in prenatal gene therapy experiments.
Access this chapter
Tax calculation will be finalised at checkout
Purchases are for personal use only
References
Rahman SH, Maeder ML, Joung JK, Cathomen T (2011) Zinc-finger nucleases for somatic gene therapy: the next frontier. Hum Gene Ther 22:925–933
Galetto R, Duchateau P, Paques F (2009) Targeted approaches for gene therapy and the emergence of engineered meganucleases. Expert Opin Biol Ther 9:1289–1303
Li T, Huang S, Jiang WZ, et al (2010) TAL nucleases (TALNs): hybrid proteins composed of TAL effectors and FokI DNA-cleavage domain. Nucleic Acids Res 39:359–372
Urnov FD, Miller JC, Lee YL et al (2005) Highly efficient endogenous human gene correction using designed zinc-finger nucleases. Nature 435:646–651
Li H, Haurigot V, Doyon Y, et al (2011) In vivo genome editing restores haemostasis in a mouse model of haemophilia. Nature 475:217–221
Toscano MG, Romero Z, Munoz P et al (2011) Physiological and tissue-specific vectors for treatment of inherited diseases. Gene Ther 18:117–127
Gonzaga S, Henriques-Coelho T, Davey M et al (2008) Cystic adenomatoid malformations are induced by localized FGF10 overexpression in fetal rat lung. Am J Respir Cell Mol Biol 39:346–355
Krieg AM (1999) Direct immunologic activities of CpG DNA and implications for gene therapy. J Gene Med 1:56–63
Yew NS, Zhao H, Przybylska M et al (2002) CpG-depleted plasmid DNA vectors with enhanced safety and long-term gene expression in vivo. Mol Ther 5:731–738
Mason CA, Bigras JL, O’Blenes SB et al (1999) Gene transfer in utero biologically engineers a patent ductus arteriosus in lambs by arresting fibronectin-dependent neointimal formation. Nat Med 5:176–182
Saada J, Oudrhiri N, Bonnard A et al (2010) Combining keratinocyte growth factor transfection into the airways and tracheal occlusion in a fetal sheep model of congenital diaphragmatic hernia. J Gene Med 12(5):413–422
Darquet A, Cameron B, Wils P et al (1997) A new DNA vehicle for nonviral gene delivery: supercoiled minicircle. Gene Ther 4:1341–1349
Bigger BW, Tolmachov O, Collombet JM et al (2001) An araC-controlled bacterial cre expression system to produce DNA minicircle vectors for nuclear and mitochondrial gene therapy. J Biol Chem 276:23018–23027
Chen ZY, He CY, Ehrhardt A, Kay MA (2003) Minicircle DNA vectors devoid of bacterial DNA result in persistent and high-level transgene expression in vivo. Mol Ther 8:495–500
Argyros O, Wong SP, Niceta M et al (2008) Persistent episomal transgene expression in liver following delivery of a scaffold/matrix attachment region containing non-viral vector. Gene Ther 15:1593–1605
Argyros O, Wong SP, Fedonidis C et al (2011) Development of S/MAR minicircles for enhanced and persistent transgene expression in the mouse liver. J Mol Med 89:515–529
Hagedorn C, Wong SP, Harbottle R, Lipps HJ (2011) Scaffold/Matrix attached region-based nonviral episomal vectors. Hum Gene Ther 22:915–923
Kootstra NA, Verma IM (2003) Gene therapy with viral vectors. Annu Rev Pharmacol Toxicol 43:413–439
Strauss SE (1984) Adenovirus infections in humans. In: Ginsberg HS (ed) Adenoviruses. Plenum Press, New York, London, pp 451–496
Horowitz MS (1990) Adenoviruses NO. In: Fields BN, Knipe DM (eds) Virology. Raven Press, Ltd, New York, pp 1723–1740
Yang Y, Li Q, Ertl HCJ, Wilson JM (1995) Cellular and humoral immune responses to viral antigens create barriers to lung-directed gene therapy with recombinant adenoviruses. J Virol 69:2004–2015
Danthinne X, Imperiale MJ (2000) Production of first generation adenovirus vectors: a review. Gene Ther 7:1707–1714
Kochanek S (1999) High-capacity adenoviral vectors for gene transfer and somatic gene therapy. Hum Gene Ther 10:2451–2459
Kochanek S, Clemens PR, Mitani K et al (1996) A new adenoviral vector: Replacement of all viral coding sequences with 28 kb of DNA independently expressing both full-length dystrophin and ß-galactosidase. Proc Natl Acad Sci U S A 93:5731–5752
Schiedner G, Morral N, Parks RJ et al (1998) Genomic DNA transfer with a high-capacity adenovirus vector results in improved in vivo gene expression and decreased toxicity. Nat Genet 18:180–183
Kreppel F, Biermann V, Kochanek S, Schiedner G (2002) A DNA-based method to assay total and infectious particle contents and helper virus contamination in high-capacity adenoviral vector preparations. Hum Gene Ther 13:1151–1156
Waddington S, Buckley SMK, David AL et al (2007) Fetal gene transfer. Curr Opin Mol Ther 9:432–438
Katz AB, Keswani SG, Habli M et al (2009) Placental gene transfer: transgene screening in mice for trophic effects on the placenta. Am J Obstet Gynecol 201(499):e491–e498
Laurema A, Vanamo K, Heikkila A et al (2004) Fetal membranes act as a barrier for adenoviruses: gene transfer into exocoelomic cavity of rat fetuses does not affect cells in the fetus. Am J Obstet Gynecol 190:264–267
Larson J, Morrow SL, Happel L et al (1997) Reversal of cystic fibrosis phenotype in mice by gene therapy in utero. Lancet 349:619–620
Buckley SM, Waddington SN, Jezzard S et al (2008) Intra-amniotic delivery of CFTR-expressing adenovirus does not reverse cystic fibrosis phenotype in inbred CFTR-knockout mice. Mol Ther 16:819–824
Davies LA, Varathalingam A, Painter H et al (2008) Adenovirus-mediated in utero expression of CFTR does not improve survival of CFTR knockout mice. Mol Ther 16:812–818
Bilbao R, Reay DP, Wu E et al (2005) (2005) Comparison of high-capacity and first-generation adenoviral vector gene delivery to murine muscle in utero. Gene Ther 12:39–47
Roberts DM, Nanda A, Havenga MJ et al (2006) Hexon-chimaeric adenovirus serotype 5 vectors circumvent pre-existing anti-vector immunity. Nature 441:239–243
Fang B, Eisensmith RC, Wang H et al (1995) Gene therapy for Hemophilia B: host immunosuppression prolongs the therapeutic effect of adenovirus-mediated Factor IX expression. Hum Gene Ther 6:1039–1044
Zennou V, Petit C, Guetard D et al (2000) HIV-1 genome nuclear import is mediated by a central DNA flap. Cell 101:173–185
Mitchell RS, Beitzel BF, Schroder AR et al (2004) Retroviral DNA integration: ASLV, HIV, and MLV show distinct target site preferences. PLoS Biol 2:E234
Bartosch B, Cosset FL (2004) Strategies for retargeted gene delivery using vectors derived from lentiviruses NO. Curr Gene Ther 4:427–443
Baum C, Kustikova O, Modlich U et al (2006) Mutagenesis and oncogenesis by chromosomal insertion of gene transfer vectors. Hum Gene Ther 17:253–263
Pitt BR, Schwarz MA, Pilewski JM et al (1995) Retrovirus-mediated gene transfer in lungs of living fetal sheep. Gene Ther 2:344–350
Douar AM, Adebakin S, Themis M et al (1997) Foetal gene delivery in mice by intra-amniotic administration of retroviral producer cells and adenovirus. Gene Ther 4:883–890
Seppen J, van der Rijt R, Looije N et al (2003) Long-term correction of bilirubin UDP glucuronyltransferase deficiency in rats by in utero lentiviral gene transfer. Mol Ther 8:593–599
Niiya M, Endo M, Shang D et al (2008) Correction of ADAMTS13 Deficiency by In Utero Gene Transfer of Lentiviral Vector encoding ADAMTS13 Genes, Mol Ther 17:34–41
Waddington S, Nivsarkar M, Mistry A et al (2004) Permanent phenotypic correction of Haemophilia B in immunocompetent mice by prenatal gene therapy. Blood 104:2714–2721
Yu ZY, McKay K, van Asperen P et al (2007) Lentivirus vector-mediated gene transfer to the developing bronchiolar airway epithelium in the fetal lamb. J Gene Med 9:429–439
Sinn PL, Penisten AK, Burnight ER et al (2005) Gene transfer to respiratory epithelia with lentivirus pseudotyped with Jaagsiekte sheep retrovirus envelope glycoprotein. Hum Gene Ther 16:479–488
Themis M, Waddington SN, Schmidt M et al (2005) Oncogenesis following delivery of a non-primate lentiviral gene therapy vector to fetal mice. Mol Ther Mol Ther 12:763–771
Buning H, Perabo L, Coutelle O et al (2008) Recent developments in adeno-associated virus vector technology. J Gene Med 10:717–733
Douar A-M, Themis M, Coutelle C (1996) Fetal somatic gene therapy. Hum Mol Reprod 2:633–641
Manno CS, Pierce GF, Arruda VR et al (2006) Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response. Nature 12:342–347
Mingozzi F, Maus MV, Hui DJ et al (2007) CD8(+) T-cell responses to adeno-associated virus capsid in humans. Nat Med 13:419–422
Nathwani AC, Gray JT, Ng CY et al (2006) Self-complementary adeno-associated virus vectors containing a novel liver-specific human factor IX expression cassette enable highly efficient transduction of murine and nonhuman primate liver. Blood 107:2653–2661
Davidoff AM, Gray JT, Ng CY et al (2005) Comparison of the ability of adeno-associated viral vectors pseudotyped with serotype 2, 5, and 8 capsid proteins to mediate efficient transduction of the liver in murine and nonhuman primate models. Mol Ther 11:875–888
Cecchini S, Virag T, Kotin RM (2011) Reproducible high yields of recombinant adeno-associated virus produced using invertebrate cells in 0.02- to 200-liter cultures. Hum Gene Ther 22:1021–1030
Mueller C, Flotte TR (2008) Clinical gene therapy using recombinant adeno-associated virus vectors. Gene Ther 15:858–863
Mah C, Sarkar R, Zolotukhin I et al (2003) Dual vectors expressing murine factor VIII result in sustained correction of hemophilia A mice. Hum Gene Ther 14:143–152
Lu H, Chen L, Wang J, Huack B et al (2008) Complete correction of hemophilia A with adeno-associated viral vectors containing a full-size expression cassette. Hum Gene Ther 19:648–654
Allocca M, Doria M, Petrillo M et al (2008) Serotype-dependent packaging of large genes in adeno-associated viral vectors results in effective gene delivery in mice. J Clin Invest 118:1955–1964
Hirsch ML, Agbandje-McKenna M, Samulski RJ (2010) Little vector, big gene transduction: fragmented genome reassembly of adeno-associated virus. Mol Ther 18(1):6–8
Rucker M, Fraites TJ Jr, Porvasnik SL et al (2004) Rescue of enzyme deficiency in embryonic diaphragm in a mouse model of metabolic myopathy: Pompe disease. Development 131:3007–3019
Dejneka NS, Surace EM, Aleman TS et al (2004) In utero gene therapy rescues vision in a murine model of congenital blindness. Mol Ther 9:182–188
Karolewski BA, Wolfe JH (2006) Genetic correction of the fetal brain increases the lifespan of mice with the severe multisystemic disease mucopolysaccharidosis type VII. Mol Ther 14:14–24
Koppanati BM, Li J, Reay DP et al (2010) Improvement of the mdx mouse dystrophic Âphenotype by systemic in utero AAV8 delivery of a minidystrophin gene. Gene Ther 17:1355–1362
Sabatino DE, Mackenzie TC, Peranteau W et al (2007) Persistent expression of hF.IX after tolerance induction by in utero or neonatal administration of AAV-1-F.IX in hemophilia B mice. Mol Ther 15:1677–1685
Koppanati BM, Li J, Xiao X, Clemens PR (2009) Systemic delivery of AAV8 in utero results in gene expression in diaphragm and limb muscle: treatment implications for muscle disorders. Gene Ther 16:1130–1137
Tarantal AF, Lee CC (2010) Long-term luciferase expression monitored by bioluminescence imaging after adeno-associated virus-mediated fetal gene delivery in rhesus monkeys (Macaca mulatta). Hum Gene Ther 21:143–148
David AL, Peebles DM, Gregory L et al (2006) Clinically applicable procedure for gene delivery to fetal gut by ultrasound-guided gastric injection: toward prenatal prevention of early-onset intestinal diseases. Hum Gene Ther 17:767–779
Mattar CN, Nathwani AC, Waddington SN et al (2011) Stable human FIX expression after 0.9G intrauterine gene transfer of self-complementary adeno-associated viral vector 5 and 8 in macaques, Mol Ther 19:1950–1960
Rahim AA, Wong AM, Hoefer K, et al (2011) Intravenous administration of AAV2/9 to the fetal and neonatal mouse leads to differential targeting of CNS cell types and extensive transduction of the nervous system. FASEB J 25:3505–3518
Mattar CN, Waddington SN, Biswas A et al (2012) Systemic delivery of scAAV9 in fetal macaques facilitates neuronal transduction of the scAAV9 in fetal macaques facilitates neuronal transduction of the central and peripheral nervous systems, Gene Ther. In Press
Donsante A, Miller DG, Li Y et al (2007) AAV vector integration sites in mouse hepatocellular carcinoma. Science 317:477
Donsante A, Vogler C, Muzyczka N et al (2001) Observed incidence of tumorigenesis in long-term rodent studies of rAAV vectors. Gene Ther 8:1343–1346
Russell DW (2007) AAV vectors, insertional mutagenesis, and cancer. Mol Ther 15:1740–1743
Kay MA (2007) AAV vectors and tumorigenicity. Nat Biotechnol 25:1111–1113
Author information
Authors and Affiliations
Corresponding author
Editor information
Editors and Affiliations
Rights and permissions
Copyright information
© 2012 Springer Science+Business Media, LLC.
About this protocol
Cite this protocol
Coutelle, C., Waddington, S.N. (2012). Vector Systems for Prenatal Gene Therapy: Choosing Vectors for Different Applications. In: Coutelle, C., Waddington, S. (eds) Prenatal Gene Therapy. Methods in Molecular Biology, vol 891. Humana Press, Totowa, NJ. https://doi.org/10.1007/978-1-61779-873-3_3
Download citation
DOI: https://doi.org/10.1007/978-1-61779-873-3_3
Published:
Publisher Name: Humana Press, Totowa, NJ
Print ISBN: 978-1-61779-872-6
Online ISBN: 978-1-61779-873-3
eBook Packages: Springer Protocols