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Rozprawy doktorskie na temat „Gene therapy”

Autor: Grafiati

Data publikacji: 4 czerwca 2021

Data aktualizacji: 25 lipca 2025

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1

Vasanwala, Farha Huseini. "Gene manipulations for cancer gene therapy." Diss., The University of Arizona, 2002. http://hdl.handle.net/10150/289776.

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Tumor cells can be modified with cytokine genes such as the Interleukin-2 (IL-2) gene. The levels of IL-2 expressed are critical for successful treatment. We have tried to achieve higher levels of IL-2 than those currently available by conventional plasmids. Use of a transcriptional activator, e.g; the tat gene along with the HIV promoter driving the IL-2 gene, greatly increased IL-2 levels compared to widely used cytomegalovirus (CMV) driven plasmids. Control of the tat gene with an inducible promoter, i.e; the human HSP70B promoter, permitted control of gene expression. The inducibility of t

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Santos, João Miguel Almeida. "Gene therapy: development of a new nanocarrier system for mitochondrial gene therapy." Master's thesis, Universidade da Beira Interior, 2013. http://hdl.handle.net/10400.6/1627.

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Mitochondria are unique organelles that have their own genome, the mitochondrial DNA (mtDNA). Although quite small compared to nuclear DNA (nDNA), mutations in mtDNA are quite frequent due to the lack of protection and repair mechanisms. Per consequence, cytopathies and diseases are quite common and mostly associated with high energy demanding tissues such as muscles and the brain. Therefore, the development of a new and efficient mitochondrial gene therapy protocol is seen as a promising approach. During this MSc thesis we try to bring together a new nanocarrier system with the ability

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3

Nanda, Dharminderkoemar. "Gene therapy for gliomas." [S.l.] : Rotterdam : [The Author] ; Erasmus University [Host], 2008. http://hdl.handle.net/1765/13140.

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Hayes, E. A. L. "Anti-angiogenic gene therapy." Thesis, University of Cambridge, 2006. http://ethos.bl.uk/OrderDetails.do?uin=uk.bl.ethos.603877.

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The aim of this project was to assess a novel anti-angiogenic gene therapy in which a therapeutic gene is activated exclusively in proliferating endothelial cells using tissue-specific promoters and the Cre/IoxP recombination system. Adenoviruses and transgenic mice were generated in parallel to test individual components of the targeting system. One of the therapeutic effector strategies investigated was the herpes simplex virus-1 thymidine kinase (HSV-1TK)/ganciclovir (GCV)-mediated suicide system, which is reported to kill proliferating cells selectively. Administration of GCV to pTie2-TK t

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5

Bilsland, Alan. "Telomerase directed gene therapy." Thesis, University of Glasgow, 2002. http://ethos.bl.uk/OrderDetails.do?uin=uk.bl.ethos.272871.

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6

Katabi, Maha M. "Transcriptional targeting of suicide genes in cancer gene therapy." Thesis, National Library of Canada = Bibliothèque nationale du Canada, 1999. http://www.collectionscanada.ca/obj/s4/f2/dsk1/tape7/PQDD_0021/NQ55345.pdf.

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7

Morin, Kevin Wayne. "Scintigraphic imaging during gene therapy." Thesis, National Library of Canada = Bibliothèque nationale du Canada, 1997. http://www.collectionscanada.ca/obj/s4/f2/dsk3/ftp04/nq21605.pdf.

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8

Singwi, Sanjeev. "HIV gene therapy using nucleases." Thesis, National Library of Canada = Bibliothèque nationale du Canada, 1999. http://www.collectionscanada.ca/obj/s4/f2/dsk1/tape7/PQDD_0001/MQ46100.pdf.

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9

Horst, Maarten ter. "Gene therapy of malignant gliomas." [S.l.] : Rotterdam : [The Author] ; Erasmus University [Host], 2008. http://hdl.handle.net/1765/10864.

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10

Lau, Cara Jean. "Gene therapy for malignant gliomas." Thesis, McGill University, 2007. http://digitool.Library.McGill.CA:80/R/?func=dbin-jump-full&object_id=18478.

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Gliomas are the most common primary brain tumours found in adults. The median survival of patients diagnosed with the most malignant form, glioblastoma multiforme (GBM), is 9-12 months and has changed little over the years despite advances in medical technology. Gene therapy may offer new solutions to treat this resistant disease. Hence, we tested three different gene therapy strategies. In our first study, we tested the efficacy of targeted therapy to correct common aberrations found in gliomas including amplification/mutation of receptor tyrosine kinases (RTK) and loss of PTEN, which result

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11

Harris, Jonathan David. "Targeted gene therapy for cancer." Thesis, Imperial College London, 1995. http://ethos.bl.uk/OrderDetails.do?uin=uk.bl.ethos.309239.

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12

Rigg, Anne Sagar. "Gene therapy for human cancer." Thesis, Imperial College London, 2000. http://ethos.bl.uk/OrderDetails.do?uin=uk.bl.ethos.341902.

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13

Pandha, Hardev Singh. "Gene transfer therapy for cancer." Thesis, Imperial College London, 1998. http://ethos.bl.uk/OrderDetails.do?uin=uk.bl.ethos.299872.

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14

Page, Sean Michael. "Gene therapy for haemophilia B." Thesis, University of Oxford, 1996. http://ethos.bl.uk/OrderDetails.do?uin=uk.bl.ethos.318970.

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15

White, Stephen John. "Ex vivo keratinocyte gene therapy." Thesis, University of Oxford, 1998. http://ethos.bl.uk/OrderDetails.do?uin=uk.bl.ethos.268103.

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16

Bainbridge, James William Braithwaite. "Gene therapy for ocular angiogenesis." Thesis, University College London (University of London), 2003. http://ethos.bl.uk/OrderDetails.do?uin=uk.bl.ethos.409631.

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17

Miller, Gaynor. "Modelling gene therapy for haemophilia." Thesis, University College London (University of London), 1999. http://ethos.bl.uk/OrderDetails.do?uin=uk.bl.ethos.369099.

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18

Roeder, Geraldine Elizabeth. "Gene therapy for cervical cancer." Thesis, University of Bristol, 2002. http://ethos.bl.uk/OrderDetails.do?uin=uk.bl.ethos.268704.

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19

Knight, S. B. "Lentiviral vectors for gene therapy." Thesis, University College London (University of London), 2012. http://discovery.ucl.ac.uk/1346459/.

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Lentiviral vectors, derived from HIV-1, are promising tools for gene therapy. Recent clinical trials have demonstrated the translation of their effectiveness in laboratory studies to clinical trials. However there are still limitations, relating to vector safety and efficiency of production, that could confine their future use. I investigated the ability of lentiviral vectors to perturb cellular gene expression by insertional mutagenesis (IM), using an in vitro model that detects aberrant splicing from lentiviral vectors to the growth hormone receptor gene (Ghr). The lentiviral vector pHV with

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20

Savina, Yulia. "Gene therapy of Parkinson's disease." Thesis, Київський національний університет технологій та дизайну, 2019. https://er.knutd.edu.ua/handle/123456789/13159.

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21

Haidet, Amanda M. "Gene Therapy for Neuromuscular Disorders." The Ohio State University, 2010. http://rave.ohiolink.edu/etdc/view?acc_num=osu1270479273.

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22

CATTANEO, STEFANO. "Combinatorial gene therapy for epilepsy." Doctoral thesis, Università Vita-Salute San Raffaele, 2022. http://hdl.handle.net/20.500.11768/128275.

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Epilepsy is a neurological disease characterized by a persistent predisposition to generate seizures, that affects about 1% of the world population. About 30% of epileptic patients are drug-resistant, thus refractory to currently available anti-epileptic drugs (AEDs). Less than 10% of these drug-resistant patients are eligible for resective brain surgery, often due to generalized or multiple epileptic foci, or due to proximity of the epileptic focus to eloquent brain areas. Therefore, gene therapy may represent a doable approach for the unmet medical need of these patients. Neuropeptide Y (NP

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23

Rück, Andreas. "Myocardial gene therapy and gene expression in angina pectoris /." Stockholm, 2006. http://diss.kib.ki.se/2006/91-7140-648-4/.

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24

Abunasra, Haitham Juma. "Gene therapy in myocardial ischemia-reperfusion." Thesis, Imperial College London, 2004. http://ethos.bl.uk/OrderDetails.do?uin=uk.bl.ethos.404964.

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25

Piccirillo, Ciriaco A. "Cytokine gene therapy of autoimmune disease." Thesis, National Library of Canada = Bibliothèque nationale du Canada, 1998. http://www.collectionscanada.ca/obj/s4/f2/dsk1/tape11/PQDD_0023/NQ50237.pdf.

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26

Aints, Alar. "Vector development for suicide gene therapy /." Stockholm, 2002. http://diss.kib.ki.se/2002/91-7349-199-3.

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27

Samani, Amir Abbas. "IGF-IR targeted cancer gene therapy." Thesis, McGill University, 2004. http://digitool.Library.McGill.CA:80/R/?func=dbin-jump-full&object_id=18206.

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Since the declaration of “War on cancer” in 1971, and with the insight provided by recent advances in human genetics and in molecular technology , the field of cancer biology has been expanded rapidly providing hope that a cure for this lethal disease will be found. One of the major contributions of the field of cell biology to the understanding of malignant diseases has been the identification of growth factors and their receptors as major promoters of transformation and malignant progression. In particular, the appreciation of the central role that receptor tyrosine kinases (RTK) play in d

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28

Abdelgany, Amr. "Gene therapy for congenital myasthenic syndromes." Thesis, University of Oxford, 2005. http://ethos.bl.uk/OrderDetails.do?uin=uk.bl.ethos.441062.

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29

Salooja, Nina. "Towards gene therapy for haemophilia B." Thesis, University of Oxford, 2001. http://ethos.bl.uk/OrderDetails.do?uin=uk.bl.ethos.393512.

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30

Wakefield, Philip M. "Gene therapy for duchenne muscular dystrophy." Thesis, University of Oxford, 1999. http://ethos.bl.uk/OrderDetails.do?uin=uk.bl.ethos.365743.

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31

Chen, Ming-Jen. "Combination gene therapy for colorectal cancer." Thesis, University of Birmingham, 2003. http://ethos.bl.uk/OrderDetails.do?uin=uk.bl.ethos.273724.

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Oncolytic virotherapy with the adenovirus mutant dl 1520 in combination with chemotherapy has shown clinical response. Approaches to cancer gene therapy involving delivery of enzymes to activate the prodrugs CB 1954 and 5-FC are currently being tested in clinical trials. We hypothesised that the combination of an adenoviral vector equivalent to dl1520 with activation ofCB 1954or 5-FC and the combination of CB 1954 activation with 5-FU may further improve the antitumour effects for colorectal cancer therapy. The initial in vitro data showed that the combination of dl 1520 with CB 1954 activatio

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32

Liljenfeldt, Lina. "CD40L Gene Therapy for Solid Tumors." Doctoral thesis, Uppsala universitet, Institutionen för immunologi, genetik och patologi, 2014. http://urn.kb.se/resolve?urn=urn:nbn:se:uu:diva-222705.

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Adenoviral CD40L gene therapy (AdCD40L) is a strong inducer of anti-tumor immune responses via its activation of dendritic cells (DCs). Activated DCs can in turn activate T cells, which are key players in an efficient anti-tumor response. This thesis includes three papers that focus on different aspects of AdCD40L gene therapy. In the first paper, the infiltration of suppressive CD11b+Gr-1+ cells in orthotopic MB49 bladder tumors was investigated and found to be significantly reduced while activated T cells were increased when the tumors had been treated with local AdCD40L gene therapy. Furthe

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33

Chung-Faye, Guy Allen. "Gene therapy strategies for colorectal cancer." Thesis, University of Birmingham, 2002. http://ethos.bl.uk/OrderDetails.do?uin=uk.bl.ethos.246708.

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34

Riley, Steven James. "Improving adenovirus vectors for gene therapy." Thesis, University of Warwick, 1998. http://ethos.bl.uk/OrderDetails.do?uin=uk.bl.ethos.323303.

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35

Zweiri, Jehad Ahmed. "Suicide immune gene therapy of cancer." Thesis, King's College London (University of London), 2002. http://ethos.bl.uk/OrderDetails.do?uin=uk.bl.ethos.270793.

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36

Azevedo, Ferreira Sónia. "Novel approaches to thalassaemia gene therapy." Thesis, King's College London (University of London), 2013. https://kclpure.kcl.ac.uk/portal/en/theses/novel-approaches-to-thalassaemia-gene-therapy(117f19f2-b791-44ea-9a79-232ce3e600a6).html.

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The haemoglobinopathies are the most prevalent genetically inherited disorders worldwide. Gene therapy for p-thalassaemia is particularly challenging given the requirement for high p-globin chain production in a lineage-specific manner. Advances in lentiviral vector (LV) technology, together with efforts to incorporate erythroid-specific regulatory elements into gene therapy vectors have yielded some promising results in both in vitro and in vivo studies with a number of clinical trials either underway or planned all using an ex vivo approach for the genetic correction of patient haematopoieti

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37

Shan, Haidong. "Neuroprotection gene therapy in retinal degeneration." Thesis, University of Oxford, 2013. http://ora.ox.ac.uk/objects/uuid:30f9b5c3-b9a6-4ad0-a952-6840bd73bbc3.

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Retinal degenerative disease, which includes age-related macular degeneration and retinitis pigmentosa, is the main cause of blindness in developed countries. Degeneration of the retinal pigment epithelium (RPE) and photoreceptor cells through apoptosis is believed to be the main mechanism of cell death. X-linked inhibitor of apoptosis (XIAP) is an endogenous anti-apoptotic protein that mediates its effects through the inhibition of caspases - the proteins regulating the final stages of apoptosis. The neuroprotection of XIAP has been demonstrated in various neurodegenerative models. Retinal ge

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38

Allen, Edwin Henry Alexander. "Gene therapy for the ocular surface." Thesis, Ulster University, 2014. http://ethos.bl.uk/OrderDetails.do?uin=uk.bl.ethos.669690.

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Meesmann's epithelial corneal dystrophy (MEeD), which clinically presents with microcysts that can cause irritation, blurred vision or photophobia, is a genetic disorder caused by dominant-negative mutations in the KRT3 and KRT12 genes. Eradicating the mutant protein or tipping the balance strongly in favour of the wild type allele are viable options for therapeutic intervention. Here we studied two therapeutic approaches for suppression of the mutant KRTl2 allele and have developed, characterised and initiated in vivo testing using two novel KRTl2 mouse models. For a transient therapeutic app

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39

Sundaram, V. "Gene therapy for inherited retinal diseases." Thesis, University College London (University of London), 2014. http://discovery.ucl.ac.uk/1418145/.

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Inherited retinal diseases include a number of disorders which typically affect photoreceptor/retinal pigment epithelial function, and can lead to severe visual impairment. Advances in molecular genetics have allowed the identification of many of the genes responsible for particular conditions, and progress in viral gene transfer technology has enabled the replacement of specific genes into the retina. The first human clinical trial of gene therapy for inherited retinal disease was carried out at Moorfields Eye Hospital and UCL Institute of Ophthalmology, involving 12 patients with RPE65 defic

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40

Eriksson, Jonas. "Gene therapy tools: oligonucleotides and peptides." Doctoral thesis, Stockholms universitet, Institutionen för neurokemi, 2016. http://urn.kb.se/resolve?urn=urn:nbn:se:su:diva-132271.

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Genetic mutations can cause a wide range of diseases, e.g. cancer. Gene therapy has the potential to alleviate or even cure these diseases. One of the many gene therapies developed so far is RNA-cleaving deoxyribozymes, short DNA oligonucleotides that specifically bind to and cleave RNA. Since the development of these synthetic catalytic oligonucleotides, the main way of determining their cleavage kinetics has been through the use of a laborious and error prone gel assay to quantify substrate and product at different time-points. We have developed two new methods for this purpose. The first on

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41

Wallace, Lindsay M. "Gene Therapy for Facioscapulohumeral Muscular Dystrophy." The Ohio State University, 2012. http://rave.ohiolink.edu/etdc/view?acc_num=osu1338315498.

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42

Tiwari, Swati. "Gene Therapy Approaches for Hemophagocytic Lymphohistiocytosis." University of Cincinnati / OhioLINK, 2015. http://rave.ohiolink.edu/etdc/view?acc_num=ucin1447690858.

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Brown, Iain. "Gene therapy for sporadic ovarian cancer." Thesis, University of Aberdeen, 2000. http://digitool.abdn.ac.uk/R?func=search-advanced-go&find_code1=WSN&request1=AAIU602008.

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Ovarian cancer accounts for more deaths than all other gynaecological cancers taken together. The 5 year survival rate can be as high as 80% for cases diagnosed early, but the asymptomatic nature of the disease means that it is most frequently detected in the later stages. By this time, disease has invariably spread beyond the ovaries and the survival rate drops to around 30%. Treatment of ovarian cancer often fails due to a high rate of chemoresistance and novel methods of treatment and detection are required to increase the survival chances of patients. This study sought to determine whether

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44

Demetriades, Anna-Maria. "Periocular gene therapy for ocular angiogenesis." Thesis, University of Oxford, 2005. http://ethos.bl.uk/OrderDetails.do?uin=uk.bl.ethos.559857.

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Protein delivery to the eye remains an important area in the clinical arena as new therapeutic proteins are being discovered. The focus of this thesis was to establish periocular gene therapy as a novel way of providing localised and sustained release of endogenous anti-angiogenic proteins to the eye. Three endogenous anti-angiogenic proteins of different sizes with established anti-angiogenic activity were chosen for the purpose of our study. The soluble extracellular domain of the vascular endothelial growth factor (VEGF) receptor-1 (sFlt-l; 78kDa), pigment epithelium-derived factor (PEDF; 5

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45

Chen, Ian Ying-Li. "Molecular imaging of cardiac gene therapy /." May be available electronically:, 2008. http://proquest.umi.com/login?COPT=REJTPTU1MTUmSU5UPTAmVkVSPTI=&clientId=12498.

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46

Carty, Nikisha Christine. "Recombinant AAV Gene Therapy and Delivery." Scholar Commons, 2009. https://scholarcommons.usf.edu/etd/1890.

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Alzheimer's disease (AD), first characterized in the early 20th century, is a common form of dementia which can occur as a result of genetic mutations in the genes encoding presenilin 1, presenilin 2, or amyloid precursor protein (APP). These genetic alterations can accelerate the pathological characteristics of AD, including the formation of extracellular neuritic plaques composed of amyloid beta peptides and the formation of intracellular neurofibrillary tangles consisting of hyperphosphorylated tau protein. Ultimately, AD results in gross neuron loss in the brain which is evidenced clinical

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Shaw, Paul Andrew. "Improving gene delivery for gene therapy and DNA vaccination applications." Thesis, University of Cambridge, 2006. http://ethos.bl.uk/OrderDetails.do?uin=uk.bl.ethos.614094.

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水野, 正明, 純. 吉田, Masaaki Mizuno та Jun Yoshida. "脳腫瘍の遺伝子治療". 日本脳神経外科コングレス, 2003. http://hdl.handle.net/2237/10863.

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Thanaketpaisarn, Oranuch. "Optimization of nonviral gene delivery system for in vivo gene therapy." 京都大学 (Kyoto University), 2005. http://hdl.handle.net/2433/144625.

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Wang, Wen-Hua 1965. "Cytokine gene expression and gene therapy in experimental corneal graft rejection." Thesis, McGill University, 2001. http://digitool.Library.McGill.CA:80/R/?func=dbin-jump-full&object_id=38529.

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It has been proposed that CD4+ T cells and cell-mediated immunity play a central role in corneal allograft rejection. Two subsets of CD4+ T cells, Th1 and Th2 cells, are known to cross-regulate each other through their cytokine pattern and the immune response might be directed predominantly in one or the other direction. As such, it has been hypothesized here that predominant Th1 type immune response could lead to corneal allograft rejection, and that previous inflamed corneal beds (high-risk eyes) might augment the Th1 response and thus accelerate the graft rejection.<br>Reverse transcription

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