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1

Nouveau, Thibaut. "Nébulisation de nouveaux polyplexes pour le transfert de gènes." Electronic Thesis or Diss., Sorbonne université, 2023. https://accesdistant.sorbonne-universite.fr/login?url=https://theses-intra.sorbonne-universite.fr/2023SORUS734.pdf.

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La thérapie génique est une forme de thérapie pour traiter les maladies génétiques héréditaires ou acquises tels que les cancers ou la mucoviscidose. L’introduction d’un polynucléotide, par voie systémique ou locale (orale ou nasale par exemple), au sein des cellules malades permet de corriger les défauts à l’origine des mutations génétiques. Néanmoins, le franchissement des différentes barrières biologiques nécessaire à l’internalisation de l’ADN ne peut se faire que par l’intermédiaire d’un vecteur qui va le protéger et lui permettre d’atteindre le noyau de la cellule où il sera transcrit. D
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2

Terp, Megan Cavanaugh. "Improved Nanoparticle Preparation and Delivery Technology for DOTAP and Oligonucleotide Based Lipoplexes." The Ohio State University, 2012. http://rave.ohiolink.edu/etdc/view?acc_num=osu1338301430.

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3

Ho, J. K. W. "Synthesis and biophysical studies of cationic lipids as gene delivery vectors." Thesis, University College London (University of London), 2012. http://discovery.ucl.ac.uk/1389331/.

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Non–viral gene vectors exhibit several desirable properties, over their viral counterparts. This has generated vast interest in the development of efficient non–viral vectors for DNA in gene therapy. The first generation lipid/peptide/DNA (LID) vector was comprised of Lipofectin™, a 1:1 mixture of DOTMA and DOPE, together with a targeting peptide. The resulting LID vector exhibited significant advantages over cationic liposome vectors (lipoplex systems) for gene delivery, transfecting a range of cells with high transfection efficiencies. The LID vector is introduced, and its advantages are out
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4

Mohammadi, A. "Design and development of tuneable cationic lipopolyplexes as vectors in gene therapy." Thesis, University College London (University of London), 2013. http://discovery.ucl.ac.uk/1389064/.

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Gene therapy is a promising technique that offers great potential for the treatment of inherited and acquired disorders. However, one of the major obstacles is identifying suitable vectors with high targeting and transfection efficiency. A literature review on gene therapy and studies on the developments of vectors in gene therapy is presented in Chapter 1. The results and discussion for the development of novel targeting peptides is outlined in Chapter 2. Biophysical analyses of the peptides were performed in PD (peptide-DNA) and LPD (lipid-peptide-DNA) formulations, and found to be stable ov
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Belmadi, Nawal. "Développement, formulation et biodistribution de vecteurs synthétiques pour le transfert de gènes dans le cadre de la thérapie génique de la mucoviscidose." Thesis, Brest, 2015. http://www.theses.fr/2015BRES0093/document.

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La mucoviscidose est une maladie monogénique, caractérisée par des mutations survenant au niveau du gène CFTR (Cystic Fibrosis Transmembrane Conductance Regulator). Le clonage en 1989 du gène CFTR a permis d’envisager de traiter cette maladie par thérapie génique. Cela consiste à transférer à l’aide d’un vecteur, une version normale du gène CFTR dans les cellules atteintes des patients. En raison de la gravité des complications pulmonaires, c’est l’épithélium respiratoire qui constitue aujourd’hui le tissu cible pour le transfert de gènes. Le principe de la thérapie génique est évidemment très
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Cruz, Claudemir Mota da. "Estudo dos campos vetoriais polinomiais quadráticos que possuem integral primeira racional de grau 3." Universidade Federal da Paraí­ba, 2011. http://tede.biblioteca.ufpb.br:8080/handle/tede/7354.

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Made available in DSpace on 2015-05-15T11:46:00Z (GMT). No. of bitstreams: 1 arquivototal.pdf: 3489954 bytes, checksum: 857373bbfa0d1a2ba6e371f0fab25e09 (MD5) Previous issue date: 2011-04-18<br>Coordenação de Aperfeiçoamento de Pessoal de Nível Superior - CAPES<br>This work was dedicated to classify all the global phase portraits of the quadratic polynomial vector fields having a rational first integral of degree three. For this, techniques were used as blow-up, classification of singular points, invariant curves for a system of ordinary diferential equations and vector fields induced o
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Ver?ssimo, Lourena Mafra. "Desenvolvimento de nanosistemas farmac?uticos para terapia g?nica." Universidade Federal do Rio Grande do Norte, 2011. http://repositorio.ufrn.br:8080/jspui/handle/123456789/12638.

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Made available in DSpace on 2014-12-17T14:05:18Z (GMT). No. of bitstreams: 1 LourenaMF_TESE_Capa_ate_pag79.pdf: 4572586 bytes, checksum: 2630047160ff526cc964f166fb86ab33 (MD5) Previous issue date: 2011-03-14<br>Coordena??o de Aperfei?oamento de Pessoal de N?vel Superior<br>Gene therapy is one of the major challenges of the post-genomic research and it is based on the transfer of genetic material into a cell, tissue or organ in order to cure or improve the patient s clinical status. In general, gene therapy consists in the insertion of functional genes aiming substitute, complement or inhib
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8

Le, Bihan Olivier. "Etude par microscopie électronique des mécanismes d'action de vecteurs synthétiques pour le transfert de gènes." Thesis, Bordeaux 1, 2009. http://www.theses.fr/2009BOR13972/document.

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La grande majorité des essais cliniques de transfert de gènes in vivo utilise des vecteurs viraux. Si ces derniers sont efficaces, ils présentent des risques immunogènes, toxiques, voire mutagènes avérés. Les vecteurs synthétiques (non viraux), par leur grande modularité et leur faible toxicité représentent une alternative très prometteuse. Le principal frein à leur utilisation est leur manque d’efficacité. L’objectif majeur de ce travail de thèse a été de comprendre le mécanisme de transfert de gènes associé à différents complexes vecteurs synthétiques/ADN plasmidique, ce qui est indispensabl
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9

Feng, Tzu-Hua, and 馮子驊. "Synthesis and Characterization of Star-Shaped Cationic Copolymer Hybrid Micelles as a Gene Vector." Thesis, 2012. http://ndltd.ncl.edu.tw/handle/52853289244065649927.

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碩士<br>高雄醫學大學<br>醫藥暨應用化學研究所<br>100<br>In this study, amphiphilic star-shaped cationic copolymers were synthesized of hydrophobic monomer and hydrophilic cationic monomer. The synthesized polymer was determined by 1H-NMR spectroscopy and gel permeation chromatography (GPC). The synthesized star-shaped cationic copolymer micelles and hybrid micelles contained PEG are formed by oil-in-water solvent evaporation method. The polyplex binding ability, transfection efficiency and cytotoxicity of micelles are all compared by gel electrophoresis performance, luciferase assay and MTT assay.
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10

Lee, Chien Hsing, and 李建興. "THE STUDY OF POLYCATIONS AND CATIONIC LIPOSOMES AS A NON-VIRAL VECTOR FOR GENE DELIVERY." Thesis, 2003. http://ndltd.ncl.edu.tw/handle/67776008321623946734.

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博士<br>國立臺灣大學<br>生物化學暨分子生物學研究所<br>91<br>During the past years, non-viral gene delivery vector was emerging as a new modality as well as a technology in laboratory and in medical practice. A promising area of research is the design of targeted lipid-DNA complexes. It is clear that the physicochemical properties of lipid-DNA complexes are very important in the design of the ideal lipidic vector. The requirements for such an ideal vector can be summarized as follows: (1) the particles should be small enough to be taken up by organs and surface protected by polyethylene glycol (PEG), to avoid pr
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11

CHO, Shun-Hsing, and 卓順興. "Study on the shelf-life of asialofetuin cationic liposome as a vector and the preliminary test in the rats' hepatoma." Thesis, 1998. http://ndltd.ncl.edu.tw/handle/48557436660373568000.

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碩士<br>國立臺灣大學<br>藥學系<br>86<br>Abstract Gene therapy could represent an important advance in the treatment of both inherited and acquired disease. Successful of human gene therapy depends upon the development of delivery vehicles or vectors which can selectively deliver therapeutic genes to the target cells with efficiency and safety. Various methods of nonviral vectors have been proposed. Cationic liposomes are particularly attractive due to their favorable characteristics suc
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12

LIU, KAI-WEI, and 劉鎧維. "Evaluation of Cholesterol Derivatives as Lipid Content of Cationic Liposomes and Study on Improvement of the stability of Liposomes as Gene Delivery Vector." Thesis, 2001. http://ndltd.ncl.edu.tw/handle/91362161057078766271.

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碩士<br>國立臺灣大學<br>藥學研究所<br>89<br>Abstract The main purpose of the present study was to evaluate the influence on the transfection efficiency of Gal-lipoosme/DNA complexes after freeze-thawing or freeze-drying in the presense of sucrose as cryoprotectant. Meanwhile, the possibility of two cholesterol derivatives to prepare cationic liposomes was also evaluated. The mouse embryonic normal liver cell line (BNL CL.2) was used to be the transfection target. DC-chol (3β[N-(N’,N’-dimethylaminoethane)-carbamoyl]cholesterol) and DOPE (dioleoyl phosphatidyl ethanolamine) (1/1 molar r
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13

Liang, Hsiang-Liang, and 梁翔亮. "Modification of cationic polyester as gene vectors by blending." Thesis, 2008. http://ndltd.ncl.edu.tw/handle/35972645543468687650.

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碩士<br>嘉南藥理科技大學<br>生物科技系暨研究所<br>96<br>Efficient transfect ion and nontoxicity were two of the most important requirements of an idea gene delivery vector. The main objective of this study was the development of new carrier system by blending. These polyesters were obtained in high through a Michael-type conjugated addition of diacrylate monomers with amine monomers. We identified the structure and molecular weights of two cationic polymers: PEDP and PE-3D by NMR、FT-IR and GPC. PEDP and PE-3D had less cytotoxicity in the different cells. PEDP and PE-3D were able to combine with DNA yield comple
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14

Tseng, S.-ja, and 曾士傑. "Synthesis and Characterizations of Novel Biodegradable Cationic Polyurethanes as Gene Delivery Vectors." Thesis, 2005. http://ndltd.ncl.edu.tw/handle/48327514361439926123.

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碩士<br>嘉南藥理科技大學<br>生物科技系暨研究所<br>93<br>The goal of this project is to design and synthesize the biodegradable cationic polymer with low cytotoxicity that can be used as vector for gene delivery. To optimize the relation between transfection efficiency, the characterizations of molecular weight, buffering capacity, the structure of the polyurethane, and complexes formed by the plasmid DNA with polycation. In this assay show the protection effect of polycations to plasmid DNA against endonucleases and bovine albumin by agarose gel band assay. The acid-base titration profile was obtained for polyme
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15

Incani, Ramirez Vanessa. "Improving gene delivery efficiency by lipid modification of cationic polymers." Phd thesis, 2010. http://hdl.handle.net/10048/1616.

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This thesis explores the capabilities of cationic polymers modified with lipids of different carbon chain length to deliver DNA molecules to primary cells and transformed cell lines. Our studies focus on two different polymers: polyethylenimine (PEI) and poly(L-lysine) (PLL). Firstly, PEI and PLL were conjugated to palmitic acid (C16). The delivery of plasmid DNA to rat bone marrow stromal cells (rat-BMSC) was evaluated by using a Green Fluorescent Protein gene expressing plasmid (pEGFP-N2) as a reporter system. The rationale for lipid substitution is to give the polymer an amphiphilic charact
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16

Silva, João Paulo Neves da. "Physicochemical characterization of DNA/DODAB:MO Cationic Liposome complexes and study of its potential as nonviral vectors." Doctoral thesis, 2013. http://hdl.handle.net/1822/29565.

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Tese de doutoramento em Ciências (área de especialização em Física)<br>Throughout the last decades, several liposomal formulations have been developed with the goal of condensing, transporting and releasing complementary DNA (cDNA) into cells, thus allowing the treatment of several genetic diseases via DNA replacement therapy. Although this technology has been partially substituted with the more contemporary use of small interference RNA (siRNA) therapy, the intracellular delivery of cDNA still remains the only viable gene therapy approach for stable host genome modification instead of t
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17

HOU, TING-YI, and 侯婷譯. "Synthesis and characterization of novel biodegradable cationic polymers:poly-L-lysine grafted polyethylenimine as effective gene vectors." Thesis, 2013. http://ndltd.ncl.edu.tw/handle/66140037950494538945.

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碩士<br>國立中正大學<br>化學暨生物化學研究所<br>101<br>Over the past few years, a number of studies have been conducted to focus on the hydrolysis property of imine groups. Research on cationic polymers contain imine groups in related to their capability of gene delivery has not yet been investigated. In this research, a new series of poly(L-lysine) – grafted – polyethylenimine(PLI)were successfully synthesized. The grafted polymer contains poly(L-lysine) as a backbone with and low-molecular-weight polyethylenimine(LMW-PEI)in the side chains via imine linkages, was examined for their potentials as non-viral gen
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18

Chen, Shanshan. "Synthesis and characterization of cationic polymers derived from cholic acid." Thèse, 2006. http://hdl.handle.net/1866/17970.

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19

Matyášová, Veronika. "Biokompatibilita a imunokompatibilita polymerů určených pro genovou terapii." Master's thesis, 2010. http://www.nusl.cz/ntk/nusl-296266.

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Gene therapy is a potential strategy for treatment of diseases caused by a gene defect. Recent studies are involved particulary in the cure of diseases caused by single gene defect (cystic fibrosis, haemophilia, muscular dystrophy etc.). Our work is part of a project aiming at developing ex vivo non-viral gene delivery systems that could be used for the treatment of ocular and cardiovascular diseases. The gene vectors are biodegradable polymeric carriers based on poly-α-amino acids. These polyplexes should transfect target cells which are supposed to be seeded on polyimide membranes. The biode
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Costa, Catarina Dias da. "Development of lipofection vectors based in novel serine-based cationic gemini surfactants finely tuned by monoolein for therapeutic siRNA delivery." Master's thesis, 2015. http://hdl.handle.net/1822/35709.

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Dissertação de mestrado em Biofísica e Bionanossistemas<br>Gene therapy based on gene silencing with siRNA has evolved and gained great importance over the last few years as a method with great potential in the treatment of genetic diseases. However, a major challenge to the use of RNAi-based therapies is their efficient delivery through an appropriate vector. Cationic liposomes are an example of the most versatile nonviral vectors for the delivery of nucleic acids and have been researched in the past few years as a strategy to promote effective transfection of genetic material. This pro
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Teixeira, Marina da Silva. "Development of new hybrid gene delivery carriers." Master's thesis, 2017. http://hdl.handle.net/10362/36889.

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Gene therapy has gained increased attention over the last decades due to the possibility to treat a disease at its routes. Several vehicles intended to carry and deliver a functional copy of the deficient gene have been developed. Amongst these, viral vectors are highly effective systems, capable to deliver the genetic cargo to the nucleus. However, these carriers have raised safety concerns regarding to immunogenicity and insertional mutagenesis, creating the need to develop equally efficient vehicles with higher safety profiles. Therefore, non-viral vectors have been suggested as an alternat
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