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Rozprawy doktorskie na temat „Lentiviral vector”

Autor: Grafiati

Data publikacji: 4 czerwca 2021

Data aktualizacji: 10 marca 2023

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1

Trimby, Christopher Matthew. "STRATEGIES FOR TARGETING LENTIVIRAL VECTORS." UKnowledge, 2011. http://uknowledge.uky.edu/gradschool_diss/157.

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Lentiviral gene therapy has held great promise for treating a wide range of neurological disorders due to its ability to stably integrate into the genome of nondividing cells like neurons, in addition to dividing cells. The nervous system is a complex and highly heterogeneous system, and while a therapeutic intervention may have beneficial effects in one population of cells it may have severe side effects in another. For this reason, specific targeting of lentiviral vectors is crucial for their ultimate utility for research and clinical research use. Two different approaches for focusing the t

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Ingrao, Dina. "Etude de l'étape d'entrée des vecteurs lentiviraux dérivés du VIH-1 dans les cellules hématopoïétiques humaines." Thesis, Evry-Val d'Essonne, 2013. http://www.theses.fr/2013EVRY0021/document.

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Les vecteurs lentiviraux (LV) sont des outils efficaces de transfert de gène, largement utilisés en thérapie génique, en particulier pour la transduction ex vivo de cellules souches et progénitrices hématopoïétiques (CSPH). Afin d’étudier simultanément la fusion et la transduction dans les CSPH avec les LV, nous avons adapté une méthode basée sur latechnologie du transfert d’énergie entre deux molécules fluorescentes (FRET). Pour mettre en place cette technique, des LV capables d’incorporer spécifiquement une enzyme, la bétalactamase (BLAM-LV) et de coder une forme tronquée du récepteur au fac

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Thomas, Joan Helen. "Studies in gene transfer using pseudotyped lentiviral vector systems." Thesis, University of Cambridge, 2000. http://ethos.bl.uk/OrderDetails.do?uin=uk.bl.ethos.621818.

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Gelinas, Jean-Francois. "Enhancement of lentiviral vector production through alteration of virus-cell interactions." Thesis, University of Oxford, 2016. https://ora.ox.ac.uk/objects/uuid:9921b8b4-e2b5-4eec-9efc-6036765c8d55.

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Gene therapy is the introduction or alteration of genetic material with the intention to treat disease. To support this aim, viruses have been modified, with elements linked to viral pathogenicity removed from their genome and replaced by the genetic material to be delivered. Gene therapy vectors based on lentiviruses have many advantages, such as the ability to transduce non-dividing cells and to target specific cell types via pseudotyping. They have been successfully used in ex vivo clinical trials for several haematopoietic stem cell disorders. Lentiviral vectors, however, suffer from subst

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Zhang, Bing. "Lentiviral vector-mediated gene transfer in vitro and in vivo /." St. Lucia, Qld, 2004. http://www.library.uq.edu.au/pdfserve.php?image=thesisabs/absthe18024.pdf.

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Booth, C. A. "Lentiviral vector mediated gene therapy for X-linked lymphoproliferative disease." Thesis, University College London (University of London), 2012. http://discovery.ucl.ac.uk/1356299/.

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X-linked lymphoproliferative disease (XLP) is a rare primary immunodeficiency characterised by severe immune dysregulation and is caused by mutations in the SH2D1A gene. Clinical manifestations vary and include haemophagocytic lymphohistiocytosis (HLH), lymphoma and dysgammaglobulinaemia, often triggered by Epstein-Barr virus (EBV) infection. SLAM-associated protein (SAP) is a key regulator of immune function in T, NK, and NKT cells and defects in this protein lead to the cellular and humoral immune defects described in patients. Treatment options for XLP are limited and currently haematopoiet

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Macdonald, D. "Lentiviral vector vaccines for T-cell-mediated protection against influenza." Thesis, University College London (University of London), 2014. http://discovery.ucl.ac.uk/1417882/.

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Vaccines that induce T cells which recognize conserved viral proteins could confer cross-strain protection against pathogens with fast-mutating B cell epitopes. Influenza is an example of such a pathogen for which there is a pressing need for a universal vaccine. Lentiviral vectors are a counterintuitive choice as vaccines since they have low inherent immunogenicity. However, their efficient transduction of non-dividing cells and high capacity permits transduction of antigen presenting cells with not only antigen but also molecular adjuvants that directly or indirectly enhance the T cell respo

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Oakland, Mayumi. "Improving lentiviral vector-mediated gene transfer by understanding cellular barriers." Diss., University of Iowa, 2013. https://ir.uiowa.edu/etd/4709.

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Cystic fibrosis (CF) is an autosomal recessive genetic disorder of which lung disease is the leading cause of morbidity and mortality. One attractive strategy for the treatment of CF lung disease is to directly deliver CF transmembrane conductance regulator gene to airway epithelia. Although promising results have been reported, barriers present in the lung make successful gene transfer to the respiratory tract difficult. In order to improve gene transfer strategies in the intrapulmonary airways, we need to identify the bottlenecks of transduction for the vector system. A previous study report

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Mekkaoui, Leila. "Lentiviral vector purification using genetically encoded biotin mimic in packaging cell." Thesis, University College London (University of London), 2018. http://discovery.ucl.ac.uk/10053191/.

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Lentiviral vectors (LVs) are powerful tools in gene therapy that have recently witnessed an increasing demand in both research and clinical applications. Current LVs purification represents the main bottle neck in their application as several methods are employed which are time consuming, cumbersome and yield low recoveries. The aim of this project was to develop a one-step method to specifically and efficiently purify LVs, with high vector yields and reduced levels of impurities, using the biotin-streptavidin system. Herein, packaging 293T cells were genetically engineered with biotin mimicki

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FIRRITO, CLAUDIA. "Targeted Gene Correction and Reprogramming of SCID-X1 Fibroblasts to Rescue IL2RG Expression in iPSC-derived Hematopoietic Cells." Doctoral thesis, Università degli Studi di Milano-Bicocca, 2015. http://hdl.handle.net/10281/94656.

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La terapia genica basata sull’utilizzo di vettori integranti è stata già applicata con successo per la cura di varie malattie genetiche come le malattie da accumulo lisosomiale (LSD), la beta-talassemia (β-Thal) e le immunodeficienze primarie (PID). L’immunodeficienza combinata grave legata al cromosoma X (SCID-X1) è una malattia monogenica letale causata da mutazioni del gene codificante la catena comune gamma del recettore per l’interleuchina 2 (IL2RG). I primi studi clinici per la SCID-X1 hanno mostrato il potenziale terapeutico della terapia genica basata su vettori integranti, risultando

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Giorgi, Marie. "Optimisation de la stratégie de thérapie génique par vecteur lentiviral pour la β-thalassémie". Thesis, Sorbonne Paris Cité, 2019. http://www.theses.fr/2019USPCC047.

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La β-thalassémie est l’une des maladies génétiques les plus répandues au monde. Elle résulte d’un déséquilibre fort entre les chaînes alpha et beta de l’hémoglobine. La thérapie génique des cellules souches hématopoïétiques est une stratégie innovante et prometteuse pour le traitement des patients -thalassémiques. En l’occurrence, l’utilisation d’un vecteur lentiviral délivrant la chaîne β-globine, testé chez dix-huit individus, a fait ses preuves chez la majorité d’entre eux. Nous avons entrepris d’améliorer la balance bénéfice risque de cette stratégie de différentes manières. Grâce à un sy

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Zhang, Xinyu. "Expression of polysialic acid mediated by lentiviral vector to promote axonal regeneration." Thesis, Queen Mary, University of London, 2007. http://ethos.bl.uk/OrderDetails.do?uin=uk.bl.ethos.443583.

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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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SPINOZZI, GIULIO. "Anti-Cancer Drug Resistance Causal Modeling from Lentiviral-Vector Integration Site Studies." Doctoral thesis, Università degli Studi di Milano-Bicocca, 2017. http://hdl.handle.net/10281/151647.

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L'evoluzione nel cancro svolge un ruolo determinante attraverso l'accumulo di alterazioni genetiche, fornendo vantaggi selettivi ad una cellula tumorale, permettendo resistenza ai farmaci anti-cancro. Purtroppo, l'identificazione delle mutazioni driver e i meccanismi sottostanti la resistenza anti-tumorale ai farmaci (ACDR) rimangono ancora irrisolti. Abbiamo precedentemente dimostrato che vettori lentivirali, se adeguatamente modificati, possono integrare nei pressi di geni specifici, modificando la loro espressione e provocate il cancro o ACDR in vivo e in vitro. L'analisi delle integrazioni

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15

Galvan, Laurie. "Étude de l'implication potentielle des marqueurs du striatum dans la maladie de Huntington." Thesis, Paris 11, 2011. http://www.theses.fr/2011PA11T024.

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La maladie de Huntington (MH) est une maladie neurodégénérative héréditaire, incurable.Elle est due à une mutation dans le gène HD codant l'huntingtine (htt). Cette mutation setraduit dans la protéine par une augmentation de l'expansion polyglutamine (polyGln) qui larend toxique. Bien que la htt soit ubiquitaire dans le système nerveux central, ladégénérescence touche préférentiellement le striatum. Un patron d'expression de gènesspécifiques du striatum pourrait expliquer cette vulnérabilité préférentielle. Nous avons étudiéles effets "modificateurs" de 5 gènes préférentiellement exprimés dans

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Coleman, Jason Edward. "Efficient transduction and targeted expression of lentiviral vector transgenes in the developing retina." [Gainesville, Fla.]: University of Florida, 2003. http://purl.fcla.edu/fcla/etd/UFE0000665.

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Limberis, Maria. "A lentiviral gene transfer vector for the treatment of cystic fibrosis airway disease." Title page, synopsis and list of contents only, 2002. http://web4.library.adelaide.edu.au/theses/09PH/09phl735.pdf.

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"16th September 2002." Accompanying CD contains 2 MPEG clips with accompanying text, and a copy in PDF format of: Recovery of airway cystic fibrosis transmembrane conductance regulator function in mice with cystic fibrosis after single-dose lentivirus-mediated gene transfer / M. Limberis ... [et al.], published in Human gene therapy vol. 13 (2002). Bibliography: leaves xxix-li. This thesis focuses on modulating the physical barriers of the airway epithelium with mild detergents, so as to enhance gene transfer by a HIV-1 based lentivirus vector in vivo. The efficiency of the gene transfer was

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Nilsson, S. M. "Process development of lentiviral vector expression, purification and formulation for gene therapy applications." Thesis, University College London (University of London), 2016. http://discovery.ucl.ac.uk/1485725/.

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There is growing interest in the use of lentiviral vectors, particularly for cancer immunotherapy and the treatment of monogenic diseases. Manufacturing of these vectors is challenging primarily due to cytotoxic effects of vector components resulting in low cell culture titres and vector instability leading to low purification yields. In addition, currently used processes are typically not scalable as they rely on adherently cultured cells and unit operations such as batch centrifugation and gel filtration. To improve process scalability, suspension adaptation of a lentiviral vector packaging

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Hino, Shinjiro. "Sea Urchin Insulator Protects Lentiviral Vector from Silencing by Maintaining Active Chromatin Structure." Kyoto University, 2004. http://hdl.handle.net/2433/147507.

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Parker, Douglas George Anthony, and park0290@flinders edu au. "Lentivirus-mediated gene expression in corneal endothelium." Flinders University. Medicine, 2008. http://catalogue.flinders.edu.au./local/adt/public/adt-SFU20081204.094431.

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Modulation of corneal transplant rejection using gene therapy shows promise in experimental models but the most appropriate vector for gene transfer is yet to be determined. The overarching aim of the thesis was to evaluate the potential of a lentiviral vector for use in human corneal transplantation. Specific aims were: (i) to assess the ability of an HIV-1-based lentiviral vector to mediate expression of the enhanced yellow fluorescent protein (eYFP), and a model secreted protein interleukin-10 (IL10), in ovine and human corneal endothelium; and (ii) to examine the influence of lentivirus-me

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McLean, Rebecca Kathryn. "Development of a novel lentiviral vaccine vector and characterisation of in vitro immune responses." Thesis, University of Edinburgh, 2018. http://hdl.handle.net/1842/31135.

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Vaccines are a highly effective means of preventing infectious disease. However, for many diseases of livestock the available vaccines are ineffective or sub-optimal. This is partly due to challenges surrounding the specific targeting of antigen presenting cells (APCs). In order to improve the delivery of protective antigens to host APCs, a novel lentiviral vector derived from visna / maedi virus (VMV) has been developed. Initial characterisation using an enhanced green fluorescent protein (eGFP) reporter transgene found that the novel VMV vector efficiently transduced a wide range of cell lin

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Shinoda, Yasuhiko. "Efficient transduction of cytotoxic and anti-HIV-1 genes by a gene-regulatable lentiviral vector." Kyoto University, 2010. http://hdl.handle.net/2433/120584.

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Dresch, Christiane. "Antigen-specific tolerance induction by transcriptional targeting of dendritic cells with a novel lentiviral vector." Diss., kostenfrei, 2008. http://edoc.ub.uni-muenchen.de/9310/.

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Haynes, Soraya. "Construction of a novel bat coronavirus glycoprotein pseudotyped lentiviral vector and analysis of cell tropism." Thesis, Haynes, Soraya (2019) Construction of a novel bat coronavirus glycoprotein pseudotyped lentiviral vector and analysis of cell tropism. Honours thesis, Murdoch University, 2019. https://researchrepository.murdoch.edu.au/id/eprint/54215/.

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Since the SARS epidemic in 2003, and the subsequent identification of bats as the reservoir host, there has been a surge of interest in research into bat-borne viruses, with over 30 new bat coronaviruses alone being discovered. Investigating the cell tropism of these newly discovered coronaviruses deepens the understanding of their pathology and possible host ranges, as well as allowing assessments to be made on their zoonotic potential. Aside from biosafety concerns however, this is also reliant on isolation of the virus from its host, which can be difficult using conventional methods. Th

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Santoni, de Sio Francesca Romana. "Critical parameters and molecular analysis of lentiviral vector-mediated gene transfer into human haematopoietic stem cells." Thesis, Open University, 2006. http://oro.open.ac.uk/54821/.

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Camacho, Emely. "Optimization of Lentivirus Production for Cancer Therapy." Thesis, Uppsala universitet, Institutionen för medicinsk biokemi och mikrobiologi, 2011. http://urn.kb.se/resolve?urn=urn:nbn:se:uu:diva-164715.

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Vectors based on lentivirus backbones have revolutionized our ability to transfer genesinto many cell types. Lentiviral vectors integrate into the chromatin of target cells and do not transfer any viral genes causing vector replication. Both of these features arecommonly used in gene therapy and have been used clinically in individuals sufferingfrom cancer, infections and genetic diseases. It has been discovered that T-cells can be genetically modified to be used as effective weapons against cancer: therefore virus mustbe produced to deliver the gene of interest into the T-cells. In this proje

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Frank, Sander B., Veronique V. Schulz, and Cindy K. Miranti. "A streamlined method for the design and cloning of shRNAs into an optimized Dox-inducible lentiviral vector." BIOMED CENTRAL LTD, 2017. http://hdl.handle.net/10150/623280.

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Background: Short hairpin RNA (shRNA) is an established and effective tool for stable knock down of gene expression. Lentiviral vectors can be used to deliver shRNAs, thereby providing the ability to infect most mammalian cell types with high efficiency, regardless of proliferation state. Furthermore, the use of inducible promoters to drive shRNA expression allows for more thorough investigations into the specific timing of gene function in a variety of cellular processes. Moreover, inducible knockdown allows the investigation of genes that would be lethal or otherwise poorly tolerated if cons

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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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Ravache, Thaís Terpins, Renata Simões, and Marcelo Demarchi Goissis. "Geração de animais transgênicos por inoculação de vetor viral em meio de cultura de óvulos." reponame:Repositório Institucional da UFABC, 2014.

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Orientador: Prof. Dr. Marcelo Augusto Christoffolete<br>Dissertação (mestrado) - Universidade Federal do ABC, Programa de Pós-Graduação em Biotecnociência, 2014.<br>Desde o século XV, animais fazem parte da rotina na área da pesquisa, principalmente para estudos de doenças, e hoje em dia o modelo animal mais utilizado para estes estudos é o camundongo, tendo uma participação em mais de 90% das pesquisas em todo o mundo, sendo considerado como uma primeira via para definir funções de genes em mamíferos. Os camundongos são considerados os principais modelos nas técnicas de transgenia animal, por

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Molina, Gil Alberto. "Lentiviral vector packaging cell line development using genome editing to target optimal loci discovered by high throughput DNA barcoding." Thesis, University College London (University of London), 2017. http://discovery.ucl.ac.uk/1573558/.

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Lentiviral vectors are increasingly used as delivery methods in gene therapy clinical trials due to their high efficiency transducing cells and stability of transgene expression. The development of packaging and producer cell lines for the production of lentiviral vectors has always been a labour-intensive and lengthy process. Sequential introduction of vector components, adaptability to suspension cultures, autotransduction and genetic, transcriptional or cell line growth instability are some of the limitations that cause significant drops in productivity. Improved transcription of self-inact

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Chang, Chia-Wei. "Polycistronic lentiviral vector for hit and run reprogramming of mouse and human somatic cells to induced pluripotent stem cell." Thesis, Birmingham, Ala. : University of Alabama at Birmingham, 2009. https://www.mhsl.uab.edu/dt/2009p/changc.pdf.

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Darbey, Annalucia Leigh. "Targeting and repair of adult testicular somatic cells through viral gene therapy." Thesis, University of Edinburgh, 2018. http://hdl.handle.net/1842/33252.

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Androgens are essential for the maintenance of male health and wellbeing. A disturbance in androgen signalling has been associated with a number of clinically relevant disorders such as cardiovascular disease, diabetes and metabolic disorders as well as infertility. Primarily produced in the testis in males, the actions of androgens are mediated through binding to androgen receptor (AR), a member of the nuclear receptor superfamily of ligand-activated transcription factors. The somatic cells of the testis are known to have a number of key roles in both testis function and development and the S

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STARINIERI, FRANCESCO. "Investigating liver tissue dynamics to improve in vivo gene therapy." Doctoral thesis, Università Vita-Salute San Raffaele, 2022. http://hdl.handle.net/20.500.11768/122896.

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The liver is a relevant target organ for in vivo gene therapy, being involved in several coagulation disorders and metabolic diseases. Adeno-associated viral (AAV) vectors have been extensively used for liver gene therapy, obtaining successful results in clinical trials. Despite AAV vector genomes remain mostly episomal, the transgene has been shown to be maintained for several years in the adult liver. However, active hepatocyte proliferation during liver growth currently challenges application of AAV-vector gene therapy to young individuals. We have previously developed lentiviral vectors (L

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Hiraragi, Hajime. "Study of lentiviral vector for in utero gene transfer and functional analysis of human T-lymphotropic virus type p13(II)." Connect to resource, 2005. http://rave.ohiolink.edu/etdc/view?acc%5Fnum=osu1116532636.

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Thesis (Ph. D.)--Ohio State University, 2005.<br>Title from first page of PDF file. Document formatted into pages; contains xvii, 230 p.; also includes graphics. Includes bibliographical references (p. 200-230). Available online via OhioLINK's ETD Center

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Holder, Maxine Virginia. "Development of a lentiviral vector system based on equine infectious anaemia virus for use in liver directed fetal gene therapy." Thesis, Imperial College London, 2006. http://ethos.bl.uk/OrderDetails.do?uin=uk.bl.ethos.428014.

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Moussa, Maha. "Immunité et protection induites par un lentivecteur ADN innovant chez les modèles animaux de vaccination VIH-1." Thesis, Université Grenoble Alpes (ComUE), 2015. http://www.theses.fr/2015GREAV029/document.

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Nous avons récemment développé un prototype lentivecteur ADN non intégratif vaccinal contre VIH-1/SIDA que nous avons testé chez des modèles animaux. L'immunisation avec une dose unique de ce vaccin (CAL-SHIV-IN-) a permis la mise en place rapide de réponses immunes spécifiques contre tous les antigènes exprimés par le vaccin chez tous les animaux vaccinés. Les analyses longitudinales ont démontré la mise en place de réponses cellulaires et humorales spécifiques et persistantes sur une durée de plus de 74 semaines en absence de réintroduction d'antigènes chez tous les macaques vaccinés. La car

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Kitowski, Katherine Anne. "A LENTIVIRAL VECTOR CONFERRING COREGULATED, ERYTHROID-SPECIFIC EXPRESSION OF γ-GLOBIN AND shRNA SEQUENCES TO BCL11A FOR THE TREATMENT OF SICKLE CELL DISEASE". OpenSIUC, 2016. https://opensiuc.lib.siu.edu/theses/1995.

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Sickle cell disease (SCD) is a severe hemoglobin disorder caused by co-inheritance of a single mutation in the β-globin gene of adult hemoglobin (HbA; α2β2). This alteration leads to the formation of sickle hemoglobin (HbS; α2βS2) and deformed, sickle-shaped red blood cells (RBCs). Sickle RBCs obstruct small blood vessels resulting in anemia, excruciating pain crises, organ damage, and stroke. For the millions of people affected by this disease, life expectancy is only 40-60 years of age. The only cure for SCD is hematopoietic stem cell (HSC, CD34+) transplantation, which requires a human leuk

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Estève, Julie. "Transfert de gènes dans les cellules souches pluripotentes induites : application à la thérapie génique de l'hyperoxalurie primitive de type 1." Thesis, Bordeaux, 2018. http://www.theses.fr/2018BORD0280/document.

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L’hyperoxalurie primitive de type 1 (ou HP1) est une maladie héréditaire du métabolisme liée à un déficit en enzyme hépatocytaire AGT (alanine:glyoxylate aminotransférase), codée par le gène AGXT. Ce déficit entraîne, chez les patients atteints d’HP1, une excrétion hépatique accrue d’oxalate ; celui-ci est ensuite éliminé dans les urines où il se complexe avec le calcium pour former des néphrolithiases oxalo-calciques massives, pouvant conduire à une insuffisance rénale chronique. Le seul traitement curatif disponible pour cette pathologie est la greffe allogénique combinée hépatorénale, actue

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Lin, Yuan. "In Vivo Imaging of Engraftment and Enrichment of Lentiviral Transduced Hematopoietic Bone Marrow Cells Under MGMT-P140K Mediated Selection." Case Western Reserve University School of Graduate Studies / OhioLINK, 2011. http://rave.ohiolink.edu/etdc/view?acc_num=case1295039430.

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CABRIOLU, ANNALISA. "Sviluppo di vettori virali per la terapia genica della β−Talassemia". Doctoral thesis, Università degli Studi di Cagliari, 2012. http://hdl.handle.net/11584/266135.

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Beta−thalassemia major is a severe congenital anemi for which there is presently no curative therapy other than allogeneic hematopoietic stem cell transplantation. This therapeutic option, however, applies only to the minority of thalassemia patients who have an HLA−matched bone marrow donor. Gene therapy by the delivery of a regulated globin gene to autologous hematopoietic stem cells is an attractive alternative approach as it is in principle applicable to all thalassemic subjects. Current vectors, althougheffective in correcting thalassemia in murine models still suffer some drawbacks in

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Facchinello, Nicola. "Conditional inactivation of Emilin1 and Col6a1 genes." Doctoral thesis, Università degli studi di Padova, 2010. http://hdl.handle.net/11577/3422723.

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Conditional gene expression methods are important approaches for examining the function of particular genes in development and disease. In particular, a lentiviral vector system for RNAi- mediated in vivo silencing of Col6a1 and a Cre-loxP based procedure for conditional-inducible knockout (KO) of Emilin1 were used in this work. The use of lentiviral vectors can accelerate the generation of animals with substantial suppression of gene expression in an inducible way. Some authors have observed limitations of the technique due to low efficiency of transgenesis and mosaicism in transgenic mice.

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Ngom, Mor. "Small molecule stimulators for enhanced yield of human hematopoietic stem cells." Thesis, Sorbonne Paris Cité, 2017. http://www.theses.fr/2017USPCC320.

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Une transduction efficace des cellules souches hematopoïetiques est un préalable pour la thérapie génique des maladies génétiques comme la β‐thalassemie, l’Adrenoleucodystrophie et le Déficit Immunitaire Combiné Sévère. La petite molécule UM171 à été décrite comme étant une molécule capable de stimuler l’expansion in vitro des cellules souches hématopoïétiques humaines, permettant ainsi une plus large application des thérapies basées sur les cellules souches. Nous avons aussi conduit des études supplémetaires pour confirmer la capacité de UM171 à expandre les souches hématopoïétiques. Durant c

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Gagnepain, Anaïs. "Évaluation de nouveaux pseudotypes de vecteurs lentiviraux pour le transfert de gènes dans les cellules hématopoiétiques." Thesis, Lyon, École normale supérieure, 2014. http://www.theses.fr/2014ENSL0939/document.

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Le transfert de gènes dans les cellules souches hématopoïétiques par des vecteurs lentiviraux s’inscrit dans les protocoles actuels de traitement par thérapie génique de plusieurs maladies monogéniques (B-thalassémie, Adrénoleucodystrophie, SCID…). De même, le transfert de gènes dans les lymphocytes T et B ouvre des perspectives tant au niveau de la thérapie génique que pour l’immunothérapie. Nous avons mis au point des vecteurs lentiviraux pseudotypés par des glycoprotéines chimérique (BaEV/TR) et mutante (BaEVRLess) du rétrovirus endogène de babouin. Nous avons montré que ces nouveaux vecteu

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Vogiatzis, Stefania. "Generation of lentiviral vectors expressing chimeric NEDD4 ubiquitin ligases specifically targeting alpha-synuclein: a tool for studying Parkinson's disease pathogenesis and for the development of innovative therapeutic approaches." Doctoral thesis, Università degli studi di Padova, 2019. http://hdl.handle.net/11577/3424860.

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Background. The main pathological features of Parkinson's disease (PD) are the death of dopaminergic neurons and the diffuse accumulation of alpha-synuclein (aS) aggregates in neurons. The NEDD4 E3 ubiquitin ligase has been shown to promote aS degradation by the endosomal/lysosomal route. Interestingly, NEDD4 is protective against human aS toxicity in evolutionary distant models. Furthermore, a small molecule able to activate NEDD4 functions, was neuroprotective in evolutionary distant models of aS toxicity. While activation of E3s cannot be easily obtained pharmacologically, their flexibility

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ANNONI, ANDREA. "Strategies for tolerance induction to gene therapy derived products." Doctoral thesis, Università degli Studi di Roma "Tor Vergata", 2009. http://hdl.handle.net/2108/763.

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I vettori lentivirali (LV) rappresentano una concreta realtà per lo sviluppo di protocolli di terapia genica. I LV, infatti, hanno la capacità di trasdurre ed integrare stabilmente il proprio genoma anche in cellule che non sono in attivo stato di replicazione. Tuttavia, uno dei maggiori ostacoli al successo degli approcci di terapia genica che prevedono l’utilizzo di vettori virali è rappresentato dalla risposta immune innata ed adattativa da essi indotta. Le risposte immuni cellulo-mediate ed anticorpali dirette verso antigeni associati alle particelle virali e alla proteina derivante dal tr

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PASINI, SILVIA. "Role of activated transcription factor 4 (ATF4) in learning and memory." Doctoral thesis, Università degli Studi di Milano-Bicocca, 2011. http://hdl.handle.net/10281/27132.

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The aim of this study is to understand the role of Activated Transcription Factor 4 (ATF4) in the processes of learning and memory. The topic of learning and memory has always aroused great interest from time immemorial and although a lot of researches have been focused on this subject for a long time, many mechanisms have not yet been fully understood. Identifying the players and the mechanisms involved in learning and memory is of utmost importance because deficits in these cognitive functions are symptoms of common neurological diseases like stoke, depression, dementia and Alzheimer’s dis

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Zanatta, Daniela Bertolini. "Sequenciamento de um código de barras como ferramenta para quantificação de alterações na dinâmica de populações celulares transduzidas com vetores lentivirais." Universidade de São Paulo, 2012. http://www.teses.usp.br/teses/disponiveis/87/87131/tde-21092012-102415/.

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Os vetores retrovirais representam uma das melhores opções para transferência e terapia gênica, pois fornecem expressão do transgene em longo prazo. Entretanto, a inserção do provírus pode causar mutagênese insercional, induzindo proto-oncogenes. Eventos deste tipo têm sido descritos em protocolos clínicos para o tratamento de SCID-X1, doença granulomatosa crônica e talessemia beta, quando vetores retrovirais (oncorretrovirus) foram utilizados. Atualmente, existem poucos métodos simples e rápidos para revelar e quantificar a expansão clonal. Assim, descrevemos a construção uma biblioteca de ve

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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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Ward, N. J. "Lentiviral vectors for treatment of haemophilia." Thesis, University College London (University of London), 2010. http://discovery.ucl.ac.uk/19905/.

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Haemophilia A and B are X‐linked recessive disorders caused by defects in coagulation factors (F) VIII and IX, respectively. Severe cases of haemophilia are characterised by episodes of spontaneous bleeding, predominantly into the joints and muscles, and can result in permanent disability and even mortality if left untreated. The haemophilias are compelling candidates for treatment with gene therapy as therapeutic benefit only requires a modest increase in the endogenous coagulation factor level, response to treatment can be easily monitored, and factor expression can be mediated by many cell

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Reis, Luiza Cunha Junqueira. "Geração de células de pluripotência induzida (iPS) humanas utilizando vetores lentivirais e determinação do perfil de integração lentiviral." Universidade de São Paulo, 2012. http://www.teses.usp.br/teses/disponiveis/60/60135/tde-26022013-092913/.

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As células iPS surgiram com a promessa de contornar as limitações das células-tronco embrionárias, como questões éticas, segurança, compatibilidade e disponibilidade. Essas células podem ser obtidas a partir de células somáticas de indivíduos normais ou de pacientes com doenças genéticas, fazendo destas uma importante ferramenta para o screening de drogas, modelos de doenças e testes toxicológicos. Grandes avanços ocorreram na reprogramação de células diferenciadas pela expressão forçada de fatores de transcrição (FT), principalmente, através de vetores lentivirais (VL), que proporcionam uma r

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