Efficient liver gene transfer with foamy virus vectors.

作者: Dimitris Zacharoulis , Chris Rountas , Michalis Katsimpoulas , John Morianos , Ilenia Chatziandreou

DOI: 10.12659/MSMBR.883996

关键词: VirologyVirusPolymerase chain reactionBiologyGene expressionGlyceraldehyde 3-phosphate dehydrogenaseMolecular biologyGene deliveryPlasmidVector (molecular biology)Real-time polymerase chain reaction

摘要: BACKGROUND Liver gene transfer offers hope for the correction of genetic and acquired disorders. Efficient in large animals can be obtained with hydrodynamic (HGT), a method that achieve sufficient levels delivery. MATERIAL AND METHODS To test relative efficiency between plasmid versus foamy virus (FV) vector-based liver efficiency, we applied HGT 4 juvenile pigs, using same backbone, either naked or coated as FV vector particle. Gene persistence expression was assayed by PCR real-time PCR, respectively, at 1 week month after infusions. RESULTS tolerated well no adverse reactions were observed. Plasmid injections resulted detectable DNA sequences week. At time point, 2/15 sections analyzed positive presence DNA. When vectors infused under identical conditions, 18/28 (64.3%) samples sequences, reached 29.7 15.6% endogenous GAPDH injected adjacent lobes. CONCLUSIONS Our results indicate medium-term therapeutic vectors, an effect attributed to potential procedure natural affinity hepatocytes.

参考文章(33)
Paul C Hendrie, Yunwen Huo, Raisa B Stolitenko, David W Russell, A rapid and quantitative assay for measuring neighboring gene activation by vector proviruses. Molecular Therapy. ,vol. 16, pp. 534- 540 ,(2008) , 10.1038/SJ.MT.6300398
Simon J. Eastman, Kevin M. Baskin, Bradley L. Hodges, Qiuming Chu, Amy Gates, Rebecca Dreusicke, Scott Anderson, Ronald K. Scheule, Development of Catheter-Based Procedures for Transducing the Isolated Rabbit Liver with Plasmid DNA Human Gene Therapy. ,vol. 13, pp. 2065- 2077 ,(2002) , 10.1089/10430340260395910
Catherine S Manno, Glenn F Pierce, Valder R Arruda, Bertil Glader, Margaret Ragni, John J E Rasko, Margareth C Ozelo, Keith Hoots, Philip Blatt, Barbara Konkle, Michael Dake, Robin Kaye, Mahmood Razavi, Albert Zajko, James Zehnder, Pradip Rustagi, Hiroyuki Nakai, Amy Chew, Debra Leonard, J Fraser Wright, Ruth R Lessard, Jürg M Sommer, Michael Tigges, Denise Sabatino, Alvin Luk, Haiyan Jiang, Federico Mingozzi, Linda Couto, Hildegund C Ertl, Katherine A High, Mark A Kay, Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response Nature Medicine. ,vol. 12, pp. 342- 347 ,(2006) , 10.1038/NM1358
S F Aliño, M J Herrero, I Noguera, F Dasí, M Sánchez, Pig liver gene therapy by noninvasive interventionist catheterism. Gene Therapy. ,vol. 14, pp. 334- 343 ,(2007) , 10.1038/SJ.GT.3302873
John W. Fabre, Michael Whitehorne, Aidan Grehan, Greta J. Sawyer, Xiaohong Zhang, Mark Davenport, Mohamed Rela, Critical Physiological and Surgical Considerations for Hydrodynamic Pressurization of Individual Segments of the Pig Liver Human Gene Therapy. ,vol. 22, pp. 879- 887 ,(2011) , 10.1089/HUM.2010.144
RICHARD A. MacDONALD, "Lifespan" of Liver Cells Archives of Internal Medicine. ,vol. 107, pp. 335- 343 ,(1961) , 10.1001/ARCHINTE.1961.03620030023003
H Yoshino, K Hashizume, E Kobayashi, Naked plasmid DNA transfer to the porcine liver using rapid injection with large volume. Gene Therapy. ,vol. 13, pp. 1696- 1702 ,(2006) , 10.1038/SJ.GT.3302833
Michalis Katsimpoulas, Dimitrios Zacharoulis, Chris Rountas, Constantinos Dimitriou, Georgios Mantziaras, Nikolaos Kostomitsopoulos, Nagy Habib, Alkiviadis Kostakis, Minimal invasive technique for gene delivery in porcine liver lobe segment. Journal of Investigative Surgery. ,vol. 24, pp. 13- 17 ,(2011) , 10.3109/08941939.2010.519815
Gijsbert A. Patijn, Andre Lieber, Leonard Meuse, Brian Winther, Mark A. Kay, High-Efficiency Retrovirus-Mediated Gene Transfer into the Livers of Mice Human Gene Therapy. ,vol. 9, pp. 1449- 1456 ,(1998) , 10.1089/HUM.1998.9.10-1449
Dirk Lindemann, Axel Rethwilm, Foamy Virus Biology and Its Application for Vector Development Viruses. ,vol. 3, pp. 561- 585 ,(2011) , 10.3390/V3050561