AAV6 Vexosomes Mediate Robust Suicide Gene Delivery in a Murine Model of Hepatocellular Carcinoma
Tài liệu tham khảo
Rolling, 1995, AAV as a viral vector for human gene therapy. Generation of recombinant virus, Mol. Biotechnol., 3, 9, 10.1007/BF02821330
Srivastava, 2016, In vivo tissue-tropism of adeno-associated viral vectors, Curr. Opin. Virol., 21, 75, 10.1016/j.coviro.2016.08.003
Nathwani, 2011, Adenovirus-associated virus vector-mediated gene transfer in hemophilia B, N. Engl. J. Med., 365, 2357, 10.1056/NEJMoa1108046
Bainbridge, 2008, Effect of gene therapy on visual function in Leber’s congenital amaurosis, N. Engl. J. Med., 358, 2231, 10.1056/NEJMoa0802268
Hauswirth, 2008, Treatment of leber congenital amaurosis due to RPE65 mutations by ocular subretinal injection of adeno-associated virus gene vector: short-term results of a phase I trial, Hum. Gene Ther., 19, 979, 10.1089/hum.2008.107
Nathwani, 2014, Long-term safety and efficacy of factor IX gene therapy in hemophilia B, N. Engl. J. Med., 371, 1994, 10.1056/NEJMoa1407309
Mingozzi, 2011, Therapeutic in vivo gene transfer for genetic disease using AAV: progress and challenges, Nat. Rev. Genet., 12, 341, 10.1038/nrg2988
Yuan, 2011, A versatile adeno-associated virus vector producer cell line method for scalable vector production of different serotypes, Hum. Gene Ther., 22, 613, 10.1089/hum.2010.241
Clark, 2002, Recent advances in recombinant adeno-associated virus vector production, Kidney Int., 61, S9, 10.1046/j.1523-1755.2002.0610s1009.x
Mary, 2019, Post-translational modifications in capsid proteins of recombinant adeno-associated virus (AAV) 1-rh10 serotypes, FEBS J., 286, 4964, 10.1111/febs.15013
Potter, 2014, A simplified purification protocol for recombinant adeno-associated virus vectors, Mol. Ther. Methods Clin. Dev., 1, 14034, 10.1038/mtm.2014.34
Gao, 2000, Purification of recombinant adeno-associated virus vectors by column chromatography and its performance in vivo, Hum. Gene Ther., 11, 2079, 10.1089/104303400750001390
Vandenberghe, 2010, Efficient serotype-dependent release of functional vector into the culture medium during adeno-associated virus manufacturing, Hum. Gene Ther., 21, 1251, 10.1089/hum.2010.107
Maguire, 2012, Microvesicle-associated AAV vector as a novel gene delivery system, Mol. Ther., 20, 960, 10.1038/mt.2011.303
Hudry, 2016, Exosome-associated AAV vector as a robust and convenient neuroscience tool, Gene Ther., 23, 380, 10.1038/gt.2016.11
György, 2014, Naturally enveloped AAV vectors for shielding neutralizing antibodies and robust gene delivery in vivo, Biomaterials, 35, 7598, 10.1016/j.biomaterials.2014.05.032
Meliani, 2017, Enhanced liver gene transfer and evasion of preexisting humoral immunity with exosome-enveloped AAV vectors, Blood Adv., 1, 2019, 10.1182/bloodadvances.2017010181
György, 2017, Rescue of Hearing by Gene Delivery to Inner-Ear Hair Cells Using Exosome-Associated AAV, Mol. Ther., 25, 379, 10.1016/j.ymthe.2016.12.010
Wassmer, 2017, Exosome-associated AAV2 vector mediates robust gene delivery into the murine retina upon intravitreal injection, Sci. Rep., 7, 45329, 10.1038/srep45329
Khan, 2019, Combination Suicide Gene Delivery with an Adeno-Associated Virus Vector Encoding Inducible Caspase-9 and a Chemical Inducer of Dimerization Is Effective in a Xenotransplantation Model of Hepatocellular Carcinoma, Bioconjug. Chem., 30, 1754, 10.1021/acs.bioconjchem.9b00291
Patel, 2019, Comparative analysis of exosome isolation methods using culture supernatant for optimum yield, purity and downstream applications, Sci. Rep., 9, 5335, 10.1038/s41598-019-41800-2
Andreu, 2014, Tetraspanins in extracellular vesicle formation and function, Front. Immunol., 5, 442, 10.3389/fimmu.2014.00442
Schiller, 2018, Enhanced Production of Exosome-Associated AAV by Overexpression of the Tetraspanin CD9, Mol. Ther. Methods Clin. Dev., 9, 278, 10.1016/j.omtm.2018.03.008
Okada, 2009, Scalable purification of adeno-associated virus serotype 1 (AAV1) and AAV8 vectors, using dual ion-exchange adsorptive membranes, Hum. Gene Ther., 20, 1013, 10.1089/hum.2009.006
Lock, 2010, Rapid, simple, and versatile manufacturing of recombinant adeno-associated viral vectors at scale, Hum. Gene Ther., 21, 1259, 10.1089/hum.2010.055
Piras, 2016, Distribution of AAV8 particles in cell lysates and culture media changes with time and is dependent on the recombinant vector, Mol. Ther. Methods Clin. Dev., 3, 16015, 10.1038/mtm.2016.15
Yagyu, 2015, An Inducible Caspase-9 Suicide Gene to Improve the Safety of Therapy Using Human Induced Pluripotent Stem Cells, Mol. Ther., 23, 1475, 10.1038/mt.2015.100
Sápi, 2015, Tumor Volume Estimation and Quasi-Continuous Administration for Most Effective Bevacizumab Therapy, PLoS ONE, 10, e0142190, 10.1371/journal.pone.0142190
Singh, 2009, Comparison of ACINUS, caspase-3, and TUNEL as apoptotic markers in determination of tumor growth rates of clinically localized prostate cancer using image analysis, Prostate, 69, 1603, 10.1002/pros.21019
Duan, 2003, Comparison of immunohistochemistry for activated caspase-3 and cleaved cytokeratin 18 with the TUNEL method for quantification of apoptosis in histological sections of PC-3 subcutaneous xenografts, J. Pathol., 199, 221, 10.1002/path.1289
Wieder, 2005, TUNEL assay as a measure of chemotherapy-induced apoptosis, Methods Mol. Med., 111, 43
Mathivanan, 2010, Exosomes: extracellular organelles important in intercellular communication, J. Proteomics, 73, 1907, 10.1016/j.jprot.2010.06.006
Record, 2014, Exosomes as new vesicular lipid transporters involved in cell-cell communication and various pathophysiologies, Biochim. Biophys. Acta, 1841, 108, 10.1016/j.bbalip.2013.10.004
Raposo, 2013, Extracellular vesicles: exosomes, microvesicles, and friends, J. Cell Biol., 200, 373, 10.1083/jcb.201211138
Baixauli, 2014, Exosomes and autophagy: coordinated mechanisms for the maintenance of cellular fitness, Front. Immunol., 5, 403, 10.3389/fimmu.2014.00403
Hessvik, 2016, PIKfyve inhibition increases exosome release and induces secretory autophagy, Cell. Mol. Life Sci., 73, 4717, 10.1007/s00018-016-2309-8
Harding, 1983, Receptor-mediated endocytosis of transferrin and recycling of the transferrin receptor in rat reticulocytes, J. Cell Biol., 97, 329, 10.1083/jcb.97.2.329
Mittelbrunn, 2011, Unidirectional transfer of microRNA-loaded exosomes from T cells to antigen-presenting cells, Nat. Commun., 2, 282, 10.1038/ncomms1285
Nolte-’t Hoen, 2012, Deep sequencing of RNA from immune cell-derived vesicles uncovers the selective incorporation of small non-coding RNA biotypes with potential regulatory functions, Nucleic Acids Res., 40, 9272, 10.1093/nar/gks658
Pigati, 2010, Selective release of microRNA species from normal and malignant mammary epithelial cells, PLoS ONE, 5, e13515, 10.1371/journal.pone.0013515
Kouwaki, 2017, Extracellular Vesicles Deliver Host and Virus RNA and Regulate Innate Immune Response, Int. J. Mol. Sci., 18, 666, 10.3390/ijms18030666
Feng, 2013, A pathogenic picornavirus acquires an envelope by hijacking cellular membranes, Nature, 496, 367, 10.1038/nature12029
Yang, 2017, Exosomes mediate hepatitis B virus (HBV) transmission and NK-cell dysfunction, Cell. Mol. Immunol., 14, 465, 10.1038/cmi.2016.24
Wiley, 2006, Immature dendritic cell-derived exosomes can mediate HIV-1 trans infection, Proc. Natl. Acad. Sci. USA, 103, 738, 10.1073/pnas.0507995103
Masciopinto, 2004, Association of hepatitis C virus envelope proteins with exosomes, Eur. J. Immunol., 34, 2834, 10.1002/eji.200424887
Jiang, 2017, Exosomes as novel bio-carriers for gene and drug delivery, Int. J. Pharm., 521, 167, 10.1016/j.ijpharm.2017.02.038
Orefice, 2019, Real-Time Monitoring of Exosome Enveloped-AAV Spreading by Endomicroscopy Approach: A New Tool for Gene Delivery in the Brain, Mol. Ther. Methods Clin. Dev., 14, 237, 10.1016/j.omtm.2019.06.005
Forner, 2012, Hepatocellular carcinoma, Lancet, 379, 1245, 10.1016/S0140-6736(11)61347-0
Cheng, 2009, Efficacy and safety of sorafenib in patients in the Asia-Pacific region with advanced hepatocellular carcinoma: a phase III randomised, double-blind, placebo-controlled trial, Lancet Oncol., 10, 25, 10.1016/S1470-2045(08)70285-7
Calcedo, 2009, Worldwide epidemiology of neutralizing antibodies to adeno-associated viruses, J. Infect. Dis., 199, 381, 10.1086/595830
Sayroo, 2016, Development of novel AAV serotype 6 based vectors with selective tropism for human cancer cells, Gene Ther., 23, 18, 10.1038/gt.2015.89
Rezvani, 2016, In Vivo Hepatic Reprogramming of Myofibroblasts with AAV Vectors as a Therapeutic Strategy for Liver Fibrosis, Cell Stem Cell, 18, 809, 10.1016/j.stem.2016.05.005
Urabe, 2002, Insect cells as a factory to produce adeno-associated virus type 2 vectors, Hum. Gene Ther., 13, 1935, 10.1089/10430340260355347
Aurnhammer, 2012, Universal real-time PCR for the detection and quantification of adeno-associated virus serotype 2-derived inverted terminal repeat sequences, Hum. Gene Ther. Methods, 23, 18, 10.1089/hgtb.2011.034
Tomayko, 1989, Determination of subcutaneous tumor size in athymic (nude) mice, Cancer Chemother. Pharmacol., 24, 148, 10.1007/BF00300234