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Journal articles on the topic 'Cell-mediated gene therapy'

Author: Grafiati

Published: 3 June 2025

Last updated: 31 July 2025

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1

Rosenblum, Warren D., Won H. Lee, and Michael T. Lotze. "Dendritic cell mediated gene therapy in cardiac transplantation." Journal of Cardiac Failure 5, no. 3 (1999): 26. http://dx.doi.org/10.1016/s1071-9164(99)91438-0.

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2

Yong, Seok-Beom, Jee Young Chung, Yoonsung Song, and Yong-Hee Kim. "Recent challenges and advances in genetically-engineered cell therapy." Journal of Pharmaceutical Investigation 48, no. 2 (2017): 199–208. http://dx.doi.org/10.1007/s40005-017-0381-1.

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Abstract Cells naturally sense and actively response to their environment. Cell-therapy has long been studied and shown therapeutic effects in various diseases. However, several hurdles should be overcome to improve cell-based therapy. Gene delivery-mediated cellular modification has shown improvement of cell function by obstacle gene silencing and therapeutic gene expression. Especially, CRISPR/Cas9-mediated genome editing is a very promising method for gene modification. In this review, we describe the recent advances in genetic modification for cell therapy. Stem cells are still promising s

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3

Saeed, A., I. H. Tarner, C. F. Michael, et al. "T-cell mediated gene therapy for CIED in mice." Journal of Allergy and Clinical Immunology 111, no. 2 (2003): S263. http://dx.doi.org/10.1016/s0091-6749(03)80940-5.

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4

Aboody, K. S., J. Najbauer, and M. K. Danks. "Stem and progenitor cell-mediated tumor selective gene therapy." Gene Therapy 15, no. 10 (2008): 739–52. http://dx.doi.org/10.1038/gt.2008.41.

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5

Ishihara, Akikazu, and Alicia L. Bertone. "Cell-mediated and direct gene therapy for bone regeneration." Expert Opinion on Biological Therapy 12, no. 4 (2012): 411–23. http://dx.doi.org/10.1517/14712598.2012.661709.

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6

Wang, Xiaojie, Mark Meloche, C. Bruce Verchere, Dawei Ou, Alice Mui, and Garth L. Warnock. "Improving Islet Engraftment by Gene Therapy." Journal of Transplantation 2011 (2011): 1–7. http://dx.doi.org/10.1155/2011/594851.

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Islet cell transplantation is currently the only feasible long-term treatment option for patients with type 1 diabetes. However, the majority of transplanted islets experience damage and apoptosis during the isolation process, a blood-mediated inflammatory microenvironment in the portal vein upon islet infusion, hypoxia induced by the low oxygenated milieu, and poor-revascularization-mediated lack of nutrients, and impaired hormone modulation in the local transplanted site. Strategies using genetic modification methods through overexpression or silencing of those proteins involved in promoting

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7

Dwyer, Roisin M., Sonja Khan, Frank P. Barry, Timothy O'Brien, and Michael J. Kerin. "Advances in mesenchymal stem cell-mediated gene therapy for cancer." Stem Cell Research & Therapy 1, no. 3 (2010): 25. http://dx.doi.org/10.1186/scrt25.

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8

Aboody, K. S., J. Najbauer, and M. K. Danks. "Erratum: Stem and progenitor cell-mediated tumor selective gene therapy." Gene Therapy 15, no. 14 (2008): 1072. http://dx.doi.org/10.1038/gt.2008.106.

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9

Moutsatsos, Ioannis K., Gadi Turgeman, Shuanhu Zhou, et al. "Exogenously Regulated Stem Cell-Mediated Gene Therapy for Bone Regeneration." Molecular Therapy 3, no. 4 (2001): 449–61. http://dx.doi.org/10.1006/mthe.2001.0291.

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10

Liberatore, Concetta, Marusca Capanni, Nicola Albi, et al. "Natural Killer Cell–mediated Lysis of Autologous Cells Modified by Gene Therapy." Journal of Experimental Medicine 189, no. 12 (1999): 1855–62. http://dx.doi.org/10.1084/jem.189.12.1855.

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This study investigated the role of natural killer (NK) cells as effectors of an immune response against autologous cells modified by gene therapy. T lymphocytes were transduced with LXSN, a retroviral vector adopted for human gene therapy that carries the selectable marker gene neo, and the autologous NK response was evaluated. We found that (i) infection with LXSN makes cells susceptible to autologous NK cell–mediated lysis; (ii) expression of the neo gene is responsible for conferring susceptibility to lysis; (iii) lysis of neo-expressing cells is clonally distributed and mediated only by N

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11

Altanerova, Ursula, Jana Jakubechova, Katarina Benejova, et al. "Intracellular prodrug gene therapy for cancer mediated by tumor cell suicide gene exosomes." International Journal of Cancer 148, no. 1 (2020): 128–39. http://dx.doi.org/10.1002/ijc.33188.

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12

Alexander, Ian E., and David W. Russell. "The Potential of AAV-Mediated Gene Targeting for Gene and Cell Therapy Applications." Current Stem Cell Reports 1, no. 1 (2015): 16–22. http://dx.doi.org/10.1007/s40778-014-0001-1.

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13

Davidson, Beverly L., Scott M. Graham, Eric A. Goebel, Joseph Zabner, and Jeffrey A. Kern. "Adenovirus-Mediated Gene Therapy for Head and Neck Squamous Cell Carcinomas." Annals of Otology, Rhinology & Laryngology 105, no. 7 (1996): 562–67. http://dx.doi.org/10.1177/000348949610500713.

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Advanced head and neck squamous cell carcinomas (HNSCCs) have a poor prognosis despite aggressive multimodal therapy. The goal of our study was to test the feasibility of gene transduction as a novel therapy for head and neck cancer. Three human HNSCC cell lines were transduced in vitro with a replication-deficient recombinant adenovirus containing the lacZ marker gene (Ad2/CMVßgal). Gene transduction efficiency was dependent on multiplicity of infection, duration of exposure to the virus, and viral concentration. Next, the HEp-2 cell line was transduced with an adenoviral vector (Ad.RSV tk) c

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14

Nagaya, Noritoshi, Hidezo Mori, Shinsuke Murakami, Kenji Kangawa, and Soichiro Kitamura. "Adrenomedullin: angiogenesis and gene therapy." American Journal of Physiology-Regulatory, Integrative and Comparative Physiology 288, no. 6 (2005): R1432—R1437. http://dx.doi.org/10.1152/ajpregu.00662.2004.

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Adrenomedullin (AM) is a potent, long-lasting vasodilator peptide that was originally isolated from human pheochromocytoma. AM signaling is of particular significance in endothelial cell biology since the peptide protects cells from apoptosis, promotes angiogenesis, and affects vascular tone and permeability. The angiogenic effect of AM is mediated by activation of Akt, mitogen-activated protein kinase/extracellular signal-regulated kinase 1/2, and focal adhesion kinase in endothelial cells. Both AM and its receptor, calcitonin receptor-like receptor, are upregulated through a hypoxia-inducibl

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15

Nemerow, Glen R. "Integrin-Targeting Strategies for Adenovirus Gene Therapy." Viruses 16, no. 5 (2024): 770. http://dx.doi.org/10.3390/v16050770.

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Numerous human adenovirus (AdV) types are endowed with arginine–glycine–aspartic acid (RGD) sequences that enable them to recognize vitronectin-binding (αv) integrins. These RGD-binding cell receptors mediate AdV entry into host cells, a crucial early step in virus infection. Integrin interactions with adenoviruses not only initiate receptor-mediated endocytosis but also facilitate AdV capsid disassembly, a prerequisite for membrane penetration by AdV protein VI. This review discusses fundamental aspects of AdV–host interactions mediated by integrins. Recent efforts to re-engineer AdV vectors

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16

Huard, J., T. Yokoyama, R. Pruchnic, et al. "Muscle-derived cell-mediated ex vivo gene therapy for urological dysfunction." Gene Therapy 9, no. 23 (2002): 1617–26. http://dx.doi.org/10.1038/sj.gt.3301816.

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17

Ley, Déborah, Ruthger Van Zwieten, Stefania Puttini, Pavithra Iyer, Alessia Cochard, and Nicolas Mermod. "A PiggyBac-mediated approach for muscle gene transfer or cell therapy." Stem Cell Research 13, no. 3 (2014): 390–403. http://dx.doi.org/10.1016/j.scr.2014.08.007.

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18

Kawabata, Ken, Makoto Migita, Hideki Mochizuki, et al. "Ex vivo cell-mediated gene therapy for metachromatic leukodystrophy using neurospheres." Brain Research 1094, no. 1 (2006): 13–23. http://dx.doi.org/10.1016/j.brainres.2006.03.116.

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19

Noh, Moon Jong. "Cell mediated gene therapy: A guide for doctors in the clinic." World Journal of Medical Genetics 5, no. 1 (2015): 1. http://dx.doi.org/10.5496/wjmg.v5.i1.1.

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20

Nakajima, Atsuo, Christine M. Seroogy, Matthew R. Sandora, et al. "Antigen-specific T cell–mediated gene therapy in collagen-induced arthritis." Journal of Clinical Investigation 107, no. 10 (2001): 1293–301. http://dx.doi.org/10.1172/jci12037.

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21

Chen, Chiann-Chyi, Amariliz Rivera, Naomi Ron, Joseph P. Dougherty, and Yacov Ron. "A gene therapy approach for treating T-cell–mediated autoimmune diseases." Blood 97, no. 4 (2001): 886–94. http://dx.doi.org/10.1182/blood.v97.4.886.

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Abstract Experimental autoimmune encephalomyelitis (EAE) is a demyelinating disease of the central nervous system (CNS) that serves as a model for multiple sclerosis (MS) in humans. In mice, EAE is mediated by Th1 type CD4+ T cells specific for various myelin proteins which migrate from the periphery to the CNS. Removal or blocking of CD4+ cells before or shortly after disease induction was shown to prevent disease onset and/or disease progression but also results in general immune suppression. Most treatment regimens for autoimmune diseases currently rely on general suppression of the T-cell

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22

Schug, Christina, Sarah Urnauer, Carsten Jaeckel, et al. "TGFB1-driven mesenchymal stem cell-mediated NIS gene transfer." Endocrine-Related Cancer 26, no. 1 (2019): 89–101. http://dx.doi.org/10.1530/erc-18-0173.

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Based on their excellent tumor-homing capacity, genetically engineered mesenchymal stem cells (MSCs) are under investigation as tumor-selective gene delivery vehicles. Transgenic expression of the sodium iodide symporter (NIS) in genetically engineered MSCs allows noninvasive tracking of MSC homing by imaging of functional NIS expression as well as therapeutic application of 131I. The use of tumor stroma-activated promoters can improve tumor-specific MSC-mediated transgene delivery. The essential role of transforming growth factor B1 (TGFB1) and the SMAD downstream target in the signaling betw

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23

Lee, Jung-Hoon, and Min-Jae Lee. "Liposome-Mediated Cancer Gene Therapy: Clinical Trials and their Lessons to Stem Cell Therapy." Bulletin of the Korean Chemical Society 33, no. 2 (2012): 433–42. http://dx.doi.org/10.5012/bkcs.2012.33.2.433.

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24

Hosseinkhani, Hossein, Abraham J. Domb, Ghorbanali Sharifzadeh, and Victoria Nahum. "Gene Therapy for Regenerative Medicine." Pharmaceutics 15, no. 3 (2023): 856. http://dx.doi.org/10.3390/pharmaceutics15030856.

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The development of biological methods over the past decade has stimulated great interest in the possibility to regenerate human tissues. Advances in stem cell research, gene therapy, and tissue engineering have accelerated the technology in tissue and organ regeneration. However, despite significant progress in this area, there are still several technical issues that must be addressed, especially in the clinical use of gene therapy. The aims of gene therapy include utilising cells to produce a suitable protein, silencing over-producing proteins, and genetically modifying and repairing cell fun

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25

Younan, Patrick M., Patricia Polacino, John P. Kowalski, et al. "Positive selection of mC46-expressing CD4+ T cells and maintenance of virus specific immunity in a primate AIDS model." Blood 122, no. 2 (2013): 179–87. http://dx.doi.org/10.1182/blood-2013-01-482224.

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Key Points Stem cell gene therapy results in enhanced virus-specific immunity and recovery of CD4+ T cells in a nonhuman primate model of AIDS. Gene therapy–mediated protection of stem cells results in a disease state similar to that observed in long-term nonprogressors.

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26

Nienhuis, Arthur W. "Development of gene therapy for blood disorders: an update." Blood 122, no. 9 (2013): 1556–64. http://dx.doi.org/10.1182/blood-2013-04-453209.

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Abstract This review addresses the current status of gene therapy for immunodeficiencies, chronic granulomatous disease, suicide gene therapy for graft-versus-host disease, viral infections, malignant hematologic disorders, hemophilia, and the hemoglobin disorders. New developments in vector design have fostered improved expression as well as enhanced safety, particularly of integrating retroviral vectors. Several immunodeficiencies have been treated successfully by stem cell–targeted, retroviral-mediated gene transfer with reconstitution of the immune system following infusion of the transduc

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27

Rodrigues, OA, and BE Strauss. "DEVELOPMENT OF ADENOVIRAL VECTOR-MEDIATED GENE THERAPY AGAINST CANINE MELANOMA CELL LINES." Cytotherapy 23, no. 4 (2021): 9. http://dx.doi.org/10.1016/j.jcyt.2021.02.032.

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28

Laje, Pablo, Dezhi Shang, Masayuki Endo, et al. "Correction of Murine Adamts13 Deficiency by Hematopoietic Progenitor Cell-Mediated Gene Therapy." Blood 112, no. 11 (2008): 2356. http://dx.doi.org/10.1182/blood.v112.11.2356.2356.

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Abstract ADAMTS13, a metalloprotease that cleaves von Willebrand factor (vWF), is primarily synthesized in liver, endothelial cells and megakaryocytes/platelets. It circulates in plasma as an active protease with concentrations between 0.5 and 1.0 μg per milliliter of plasma. Proteolytic cleavage of endothelial cell bound and plasma vWF by ADAMTS13 is critical for maintaining normal hemostasis. Inability to cleave newly released unusually large vWF as a result of hereditary or acquired deficiency of ADAMTS13 activity leads to a potentially fatal syndrome, thrombotic thrombocytopenic purpura (T

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29

Shi, Shuo, Min Zhang, Rui Guo, Ying Miao, and Biao Li. "Bone Marrow–Derived Mesenchymal Stem Cell–Mediated Dual-Gene Therapy for Glioblastoma." Human Gene Therapy 30, no. 1 (2019): 106–17. http://dx.doi.org/10.1089/hum.2018.092.

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30

Paul, Stephane, Etienne Regulier, Ronald Rooke, et al. "Tumor gene therapy by MVA-mediated expression of T-cell–stimulating antibodies." Cancer Gene Therapy 9, no. 5 (2002): 470–77. http://dx.doi.org/10.1038/sj.cgt.7700461.

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31

Miyake, K., K. Inokuchi, N. Miyake, K. Dan, and T. Shimada. "HIV vector-mediated targeted suicide gene therapy for adult T-cell leukemia." Gene Therapy 14, no. 23 (2007): 1662–67. http://dx.doi.org/10.1038/sj.gt.3303024.

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32

Rando, T. A., and H. M. Blau. "Primary mouse myoblast purification, characterization, and transplantation for cell-mediated gene therapy." Journal of Cell Biology 125, no. 6 (1994): 1275–87. http://dx.doi.org/10.1083/jcb.125.6.1275.

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The transplantation of cultured myoblasts into mature skeletal muscle is the basis for a new therapeutic approach to muscle and non-muscle diseases: myoblast-mediated gene therapy. The success of myoblast transplantation for correction of intrinsic muscle defects depends on the fusion of implanted cells with host myofibers. Previous studies in mice have been problematic because they have involved transplantation of established myogenic cell lines or primary muscle cultures. Both of these cell populations have disadvantages: myogenic cell lines are tumorigenic, and primary cultures contain a su

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33

Migita, Makoto, Makoto Migita, Jun Hayakawa, et al. "Cell-mediated Gene Therapy Using Bone Marrow Derived Cells Towards Regenerative Medicine." Journal of Nippon Medical School 70, no. 5 (2003): 432–35. http://dx.doi.org/10.1272/jnms.70.432.

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34

Laje, Pablo, Dezhi Shang, Wenjing Cao, et al. "Correction of murine ADAMTS13 deficiency by hematopoietic progenitor cell–mediated gene therapy." Blood 113, no. 10 (2009): 2172–80. http://dx.doi.org/10.1182/blood-2008-08-173021.

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Abstract ADAMTS13, a metalloprotease primarily synthesized in liver and endothelial cells, cleaves von Willebrand factor (VWF) at the central A2 domain, thereby reducing the sizes of circulating VWF multimers. Genetic or acquired deficiency of plasma ADAMTS13 activity leads to a potentially fatal syndrome, thrombotic thrombocytopenic purpura (TTP). To date, plasma infusion or exchange is the only proven effective therapy for TTP. In search for a better therapy, an autologous transplantation of hematopoietic progenitor cells transduced ex vivo with a self-inactivating lentiviral vector encoding

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35

Yan, Ziqing. "Historical Evolution and CRISPR-Mediated Gene Editing Applications in NK Cell Therapy." Theoretical and Natural Science 90, no. 1 (2025): 107–15. https://doi.org/10.54254/2753-8818/2025.gu20967.

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Natural Killer (NK) cell therapy has emerged as a transformative approach in cancer immunotherapy, driven by decades of research since their discovery in the 1970s. NK cells possess a unique ability to target tumor cells through antigen-independent recognition, which has been expanded with the discovery of antigen-specific memory and licensing mechanisms. This review explores the evolution of NK cell biology, highlighting key findings such as the enhanced response of NK cells upon re-exposure to cytomegalovirus (CMV), defying conventional immune paradigms. Recent advancements in gene editing,

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36

Mitrovic, Tatjana, and Sinisa Radulovic. "Cancer gene therapy." Archive of Oncology 13, no. 1 (2005): 23–26. http://dx.doi.org/10.2298/aoo0501023m.

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Cancer gene therapy can be defined as transfer of nucleic acids into tumor or normal cells with aim to eradicate or reduce tumor mass by direct killing of cells, immunomodulation or correction of genetic errors, and reversion of malignant status. Initially started with lots of optimism and enthusiasm, cancer gene therapy has shown limited success in treatment of patients. This review highlights current limitations and almost endless possibilities of cancer gene therapy. The major difficulty in advancing gene therapy technology from the bench to the clinical practice is problem with gene delive

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37

Deng, Weiwen, Trinity J. Bivalacqua, Natasha N. Chattergoon, Albert L. Hyman, James R. Jeter, and Philip J. Kadowitz. "Adenoviral gene transfer of eNOS: high-level expression in ex vivo expanded marrow stromal cells." American Journal of Physiology-Cell Physiology 285, no. 5 (2003): C1322—C1329. http://dx.doi.org/10.1152/ajpcell.00141.2003.

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Endothelial nitric oxide synthase (eNOS) is an attractive target for cardiovascular gene therapy. Marrow stromal cells (MSCs), also known as mesenchymal stem cells, hold great promise for use in adult stem cell-based cell and gene therapy. To determine the feasibility of adenoviral-mediated eNOS gene transfer into ex vivo expanded MSCs, rat MSCs (rMSCs) were isolated, expanded ex vivo, and transduced with Ad5RSVeNOS, an adenoviral vector containing the eNOS gene under the control of the Rous sarcoma virus promoter. The presence of eNOS protein in Ad5RSVeNOS-transduced rMSCs was confirmed by im

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38

Yang, Chengbin, Rui Hu, Tommy Anderson, et al. "Biodegradable nanoparticle-mediated K-ras down regulation for pancreatic cancer gene therapy." Journal of Materials Chemistry B 3, no. 10 (2015): 2163–72. http://dx.doi.org/10.1039/c4tb01623h.

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39

Islamov, Rustem, Farid Bashirov, Filip Fadeev, et al. "Epidural Stimulation Combined with Triple Gene Therapy for Spinal Cord Injury Treatment." International Journal of Molecular Sciences 21, no. 23 (2020): 8896. http://dx.doi.org/10.3390/ijms21238896.

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The translation of new therapies for spinal cord injury to clinical trials can be facilitated with large animal models close in morpho-physiological scale to humans. Here, we report functional restoration and morphological reorganization after spinal contusion in pigs, following a combined treatment of locomotor training facilitated with epidural electrical stimulation (EES) and cell-mediated triple gene therapy with umbilical cord blood mononuclear cells overexpressing recombinant vascular endothelial growth factor, glial-derived neurotrophic factor, and neural cell adhesion molecule. Prelimi

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40

Bennett, Michael J., Michael H. Nantz, Rajiv P. Balasubramaniam, Dieter C. Gruenert, and Robert W. Malone. "Cholesterol enhances cationic liposome-mediated DNA transfection of human respiratory epithelial cells." Bioscience Reports 15, no. 1 (1995): 47–53. http://dx.doi.org/10.1007/bf01200214.

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Cationic liposome transfection reagents are useful for transferring polynucleotides into cells, and have been proposed for human pulmonary gene therapy. The effect of adding cholesterol to cationic lipid preparations has been tested by first formulating the cationic lipid N-[1-(2,3-dioleoyloxy)propyl-N-[1-(2-hydroxy)ethyl]-N,N-dimethyl ammonium iodide (DORI) with varying amounts of dioleoylphos-phatidylethanolamine (DOPE) and cholesterol. Cholesterol was found to enhance lipid-mediated transfection in both the respiratory epithelial cells and mouse fibroblasts. These findings will facilitate n

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41

Decary, Stéphanie, Vincent Mouly, and Gillian S. Butler-Browne. "Telomere Length as a Tool to Monitor Satellite Cell Amplification for Cell-Mediated Gene Therapy." Human Gene Therapy 7, no. 11 (1996): 1347–50. http://dx.doi.org/10.1089/hum.1996.7.11-1347.

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42

DiNardo, Andrew R., Kimal Rajapakshe, Tomoki Nishiguchi, et al. "Epigenetic mediated immune exhaustion persists after successful Tuberculosis therapy." Journal of Immunology 202, no. 1_Supplement (2019): 194.17. http://dx.doi.org/10.4049/jimmunol.202.supp.194.17.

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Abstract BACKGROUND Chronic antigen stimulation from HIV, HPV, LCMV and schistosomiasis induce epigenetic-mediated immune exhaustion. Individuals with successful anti-TB therapy (ATT) have a thirteen-fold increased risk of developing recurrent TB. METHODS From a cohort of HIV infected and uninfected individuals with TB and their asymptomatic household contacts (n=32), genome-wide DNA methylation (DNA MethylEPIC) and epigenetic deconvolution (EDEC) identified cell-specific DNA methylation. Cell-specific results were validated with MSRE-PCR, gene expression and flow cytometry-based Mtb-specific

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Jonuschies, Jacqueline, Michael Antoniou, Simon Waddington, et al. "The Human Desmin Promoter Drives Robust Gene Expression for Skeletal Muscle Stem Cell-Mediated Gene Therapy." Current Gene Therapy 14, no. 4 (2014): 276–88. http://dx.doi.org/10.2174/1566523214666140612154521.

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Kiisholts, Kristina, Kaido Kurrikoff, Piret Arukuusk, et al. "Cell-Penetrating Peptide and siRNA-Mediated Therapeutic Effects on Endometriosis and Cancer In Vitro Models." Pharmaceutics 13, no. 10 (2021): 1618. http://dx.doi.org/10.3390/pharmaceutics13101618.

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Gene therapy is a powerful tool for the development of new treatment strategies for various conditions, by aiming to transport biologically active nucleic acids into diseased cells. To achieve that goal, we used highly potential delivery vectors, cell-penetrating peptides (CPPs), as oligonucleotide carriers for the development of a therapeutic approach for endometriosis and cancer. Despite marked differences, both of these conditions still exhibit similarities, like excessive, uncoordinated, and autonomous cellular proliferation and invasion, accompanied by overlapping gene expression patterns

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45

Grimm, Dirk, and Mark A. Kay. "RNAi and Gene Therapy: A Mutual Attraction." Hematology 2007, no. 1 (2007): 473–81. http://dx.doi.org/10.1182/asheducation-2007.1.473.

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AbstractThe phylogenetically conserved cellular phenomenon of RNA interference (RNAi)—the sequence-specific post-transcriptional silencing of gene expression mediated by small double-stranded RNAs—holds substantial promise for basic research and for drug development. Particularly attractive from a medical standpoint is the juxtaposition of new RNAi methodology with established gene transfer strategies, especially viral vectors for efficient and tissue-specific RNAi delivery to patients. Here, we summarize the latest experimental and clinical advances in RNAi-based gene therapy approaches. We b

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46

Luo, Yumei, Detu Zhu, Dang Hoang Lam, et al. "A Double-Switch Cell Fusion-Inducible Transgene Expression System for Neural Stem Cell-Based Antiglioma Gene Therapy." Stem Cells International 2015 (2015): 1–8. http://dx.doi.org/10.1155/2015/649080.

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Recent progress in neural stem cell- (NSC-) based tumor-targeted gene therapy showed that NSC vectors expressing an artificially engineered viral fusogenic protein, VSV-G H162R, could cause tumor cell death specifically under acidic tumor microenvironment by syncytia formation; however, the killing efficiency still had much room to improve. In the view that coexpression of another antitumoral gene with VSV-G can augment the bystander effect, a synthetic regulatory system that triggers transgene expression in a cell fusion-inducible manner has been proposed. Here we have developed a double-swit

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47

Bodine, DM, T. Moritz, RE Donahue, et al. "Long-term in vivo expression of a murine adenosine deaminase gene in rhesus monkey hematopoietic cells of multiple lineages after retroviral mediated gene transfer into CD34+ bone marrow cells." Blood 82, no. 7 (1993): 1975–80. http://dx.doi.org/10.1182/blood.v82.7.1975.1975.

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Abstract Retroviral mediated gene transfer into stem cells has been proposed as therapy for many inherited hematopoietic diseases. Deficiency of the enzyme adenosine deaminase (ADA) results in depletion of T lymphocytes, causing severe combined immunodeficiency syndrome (SCIDS). In this report, we describe retroviral mediated gene transfer of a murine ADA cDNA into Rhesus monkey hematopoietic stem cells. Immunoselected CD34+ bone marrow cells were exposed to medium containing the ADA retrovirus during culture on a stromal cell line engineered to express the transmembrane form of stem cell fact

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48

Bodine, DM, T. Moritz, RE Donahue, et al. "Long-term in vivo expression of a murine adenosine deaminase gene in rhesus monkey hematopoietic cells of multiple lineages after retroviral mediated gene transfer into CD34+ bone marrow cells." Blood 82, no. 7 (1993): 1975–80. http://dx.doi.org/10.1182/blood.v82.7.1975.bloodjournal8271975.

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Retroviral mediated gene transfer into stem cells has been proposed as therapy for many inherited hematopoietic diseases. Deficiency of the enzyme adenosine deaminase (ADA) results in depletion of T lymphocytes, causing severe combined immunodeficiency syndrome (SCIDS). In this report, we describe retroviral mediated gene transfer of a murine ADA cDNA into Rhesus monkey hematopoietic stem cells. Immunoselected CD34+ bone marrow cells were exposed to medium containing the ADA retrovirus during culture on a stromal cell line engineered to express the transmembrane form of stem cell factor. After

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49

Mouly, V., A. Aamiri, A. Bigot, et al. "The mitotic clock in skeletal muscle regeneration, disease and cell mediated gene therapy." Acta Physiologica Scandinavica 184, no. 1 (2005): 3–15. http://dx.doi.org/10.1111/j.1365-201x.2005.01417.x.

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Pala, Francesca, Henner Morbach, Maria Carmina Castiello, et al. "Lentiviral-mediated gene therapy restores B cell tolerance in Wiskott-Aldrich syndrome patients." Journal of Clinical Investigation 125, no. 10 (2015): 3941–51. http://dx.doi.org/10.1172/jci82249.

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