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Relevant bibliographies by topics / Cell-mediated gene therapy / Dissertations / Theses

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Dissertations / Theses on the topic 'Cell-mediated gene therapy'

Author: Grafiati

Published: 3 June 2025

Last updated: 31 July 2025

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1

Langford-Smith, Alexander William Walker. "Lentiviral vector mediated haematopoietic stem cell gene therapy for mucopolysaccharidosis type IIIA." Thesis, University of Manchester, 2012. https://www.research.manchester.ac.uk/portal/en/theses/lentiviral-vector-mediated-haematopoietic-stem-cell-gene-therapy-for-mucopolysaccharidosis-type-iiia(89f8e108-58f3-42bb-8b80-0e0a1fe45fd7).html.

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Mucopolysaccharidosis type III (Sanfilippo) is comprised of four phenotypically similar lysosomal storage disorders (MPS IIIA-D) caused by the deficiency of enzymes that catabolise heparan sulphate (HS). Progressive accumulation of HS results in abnormal behaviour, progressive cognitive and motor impairment and death in mid-teens. There are currently no treatments for MPS III. To assess the effect of novel therapeutics in the mouse models of MPS III it is necessary to examine the effect on primary storage of HS, secondary storage and behaviour. The reported behaviour of MPS IIIA and B mice is

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2

Queen, Jillian May. "Viral mediated gene transfer for modification of cell signalling and remodelling in the ischaemic myocardium." Thesis, University of Glasgow, 1998. http://ethos.bl.uk/OrderDetails.do?uin=uk.bl.ethos.301367.

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3

Zeng, Yan. "T-cell mediated suppression of neuroblastoma following fractalkine gene therapy is amplified by targeted IL-2." Doctoral thesis, Humboldt-Universität zu Berlin, Medizinische Fakultät - Universitätsklinikum Charité, 2006. http://dx.doi.org/10.18452/15401.

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Das Induzieren und Aufrechterhalten einer tumor-protektiven Immunität sind wesentliche Ziele in der Immuntherapie des Neuroblastoms. Eine Erhöhung der Anzahl von tumor-infiltrierenden Leukozyten könnte ein Weg sein, um dieses Ziel zu erreichen. Fractalkine ist ein besonderes TH1 CX3C Chemokin, welches sowohl Adhäsion und Migration von Leukozyten vermittelt. Gerichtetes IL-2 (ch14.18-IL-2) wurde durch eine genetische Fusion von anti-GD2 Antikörper mit IL-2 hergestellt, damit IL-2 spezifisch in das Mikromilieu von Neuroblastomen gebracht werden kann. In dieser Arbeit habe ich die Hypothese gete

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4

Wootton, Virginia. "Evaluation of lentivirus-mediated reconstitution of the B-cell compartment in XLA patient-derived CD34+ cells in humanised mice." Thesis, The University of Sydney, 2016. http://hdl.handle.net/2123/15864.

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X-Linked Agammaglobulinaemia (XLA) is a primary immune deficiency resulting in an absence of B-cells and antibody production due to mutations in the Bruton’s tyrosine kinase (Btk) gene. Treatment requires lifelong immunoglobulin replacement and carries significant morbidity making gene therapy an attractive possibility. The aim of this thesis was to directly test reconstitution of B-cell ontogeny in patient-derived CD34+ cells in a humanised mouse model following lentivirus-mediated gene transfer. This thesis has established a minimum non-XLA CD34+ cell dose of 0.5 x10^6 is required to reconst

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5

Knoop, Kerstin. "Molecular imaging and radionuclide therapy in non-thyroidal tumors after mesenchymal stem cell- mediated sodium/iodide symporter (NIS) gene transfer." Diss., Ludwig-Maximilians-Universität München, 2014. http://nbn-resolving.de/urn:nbn:de:bvb:19-178550.

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6

Tutter, Mariella [Verfasser], and Ernst [Akademischer Betreuer] Wagner. "Challenges and chances of the combination of hyperthermia with mesenchymal stem cell-mediated sodium iodide symporter gene therapy / Mariella Tutter ; Betreuer: Ernst Wagner." München : Universitätsbibliothek der Ludwig-Maximilians-Universität, 2020. http://d-nb.info/1211957543/34.

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7

Knoop, Kerstin [Verfasser], and Ernst [Akademischer Betreuer] Wagner. "Molecular imaging and radionuclide therapy in non-thyroidal tumors after mesenchymal stem cell- mediated sodium/iodide symporter (NIS) gene transfer / Kerstin Knoop. Betreuer: Ernst Wagner." München : Universitätsbibliothek der Ludwig-Maximilians-Universität, 2014. http://d-nb.info/1065180403/34.

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8

Zeng, Yan [Verfasser], H. [Gutachter] Lode, R. [Gutachter] Erttmann, and R. [Gutachter] Xiang. "T-cell mediated suppression of neuroblastoma following fractalkine gene therapy is amplified by targeted IL-2 / Yan Zeng ; Gutachter: H. Lode, R. Erttmann, R. Xiang." Berlin : Humboldt-Universität zu Berlin, 2006. http://d-nb.info/1208077031/34.

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9

Nicklin, Stuart Anthony. "Targeted adenovirus-mediated gene transfer to human vascular endothelium." Thesis, University of Bristol, 2000. http://ethos.bl.uk/OrderDetails.do?uin=uk.bl.ethos.310591.

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10

Xu, Dan. "Cellular Immunity in Recombinant Adeno-Associated Virus Vector Mediated Gene Therapy." The Ohio State University, 2011. http://rave.ohiolink.edu/etdc/view?acc_num=osu1313504203.

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11

Kaminsky, Paul Michael. "Focal adhesion kinase signaling regulates highly productive transduction of adeno-associated virus through integrin-mediated endocytosis." Thesis, The University of Iowa, 2013. http://pqdtopen.proquest.com/#viewpdf?dispub=3566662.

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<p> Recombinant adeno-associated virus (rAAV) is a widely used gene therapy vector. Although a wide range of rAAV serotypes can effectively enter most cell types, their transduction efficiencies (i.e., transgene expression) can vary widely depending on the target cell type. Integrins play important roles as co-receptors for rAAV infection, however, it remains unclear how integrin-dependent and -independent mechanisms of rAAV endocytosis influence the efficiency of intracellular virus processing and ultimately transgene expression. In this thesis, I examined the contribution of integrin-medi

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12

Bunse, Mario. "RNAi-mediated knockdown of the endogenous TCR improves safety of immunotherapy with TCR gene-modified T cells." Doctoral thesis, Humboldt-Universität zu Berlin, Lebenswissenschaftliche Fakultät, 2015. http://dx.doi.org/10.18452/17155.

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Durch den Transfer der Gene des heterodimeren T-Zellrezeptors (TZR) mithilfe viraler Vektoren können T-Zellen programmiert werden, ein ausgewähltes Antigen spezifisch zu erkennen. In klinischen Studien wurden solche T-Zellen bereits mit Erfolg zur Immuntherapie von Krebs und viralen Infektionen eingesetzt. Genmodifizierte T-Zellen unterscheiden sich jedoch von normalen T-Zellen, weil sie neben den beiden zelleigenen auch die zwei übertragenen TZR-Gene exprimieren. Diese Situation erlaubt die Bildung vier verschiedener TZR-Heterodimere: der zelleigene TZR, der übertragene TZR und zwei gemischte

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13

Naughton, Bartholomew J. IV. "Brain Region and Cell Type Specific Approaches to Study Drug Abuse." The Ohio State University, 2011. http://rave.ohiolink.edu/etdc/view?acc_num=osu1314715486.

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14

Scaife, Matthew. "Truncated Cell Surface Markers Fused with Mutant Human Tmpk: Versatile Cell Fate Control Safety Cassettes for Lentiviral Vector Mediated Correction of Fabry Disease." Thesis, 2010. http://hdl.handle.net/1807/25794.

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Lentivirus-mediated gene therapy has curative potential for a variety of disorders, however, insertional oncogenesis still remains a concern. One approach to increase safety of such treatment modalities is to include a ‘cell fate control safety cassette’ in lentiviral vectors (LVs), enabling pharmacological control over the survival of gene-modified cells (GMCs). Two novel LVs with engineered expression of truncated cell surface molecules (CD19 or LNGFR) fused to a ‘cell fate control’ gene (TmpkF105YR200A) were constructed. Results demonstrated these safety cassettes could be used to contro

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15

Wei-Hua, Huang, and 黃緯華. "Establishment of a bone marrow stem cell- mediated BMP2 lentiviral ex vivo gene therapy system." Thesis, 2009. http://ndltd.ncl.edu.tw/handle/05313563924971834835.

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碩士<br>國立陽明大學<br>口腔生物研究所<br>97<br>Bone grafting is often used clinically for treating osseous defects. In addition to many inherent limitations for bone grafting, its regenerative effect in large osseous defects are still less than ideal. Tissue engineering, by using scaffold, cell and growth factors, is an emergent regenerative technique with great potential. Several special concerns exist when applying tissue engineering for treating large defects. The scaffold needs sufficient mechanical strength to maintain space for regeneration. Cells with high regenerative capacity such as stem cells sho

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16

Zeng, Yan [Verfasser]. "T-cell mediated suppression of neuroblastoma following fractalkine gene therapy is amplified by targeted IL-2 / Yan Zeng." 2005. http://d-nb.info/978557972/34.

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17

Ha, Sung Pil. "CD4+ T cell mediated tumor immunity following transplantation of TRP-1 TCR gene modified hematopoietic stem cells." Thesis, 2013. http://hdl.handle.net/1805/3739.

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Indiana University-Purdue University Indianapolis (IUPUI)<br>Immunotherapy for cancer has held much promise as a potent modality of cancer treatment. The ability to selectively destroy diseased cells and leave healthy cells unharmed has been the goal of cancer immunotherapy for the past thirty years. However, the full capabilities of cancer immunotherapies have been elusive. Cancer immunotherapies have been consistently hampered by limited immune reactivity, a diminishing immune response over time, and a failure to overcome self-tolerance. Many of these deficiencies have been borne-out by

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