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Relevant bibliographies by topics / CRISPR, Cas9, genome editing, gRNA / Dissertations / Theses

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Dissertations / Theses on the topic 'CRISPR, Cas9, genome editing, gRNA'

Author: Grafiati

Published: 11 March 2023

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1

Roidos, Paris. "Genome editing with the CRISPR Cas9 system." Thesis, KTH, Skolan för bioteknologi (BIO), 2014. http://urn.kb.se/resolve?urn=urn:nbn:se:kth:diva-163694.

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2

Ran, Fei Ann. "CRISPR-Cas: Development and applications for mammalian genome editing." Thesis, Harvard University, 2014. http://dissertations.umi.com/gsas.harvard:11610.

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The ability to introduce targeted modifications into genomes and engineer model organisms holds enormous promise for biomedical and technological applications, and has driven the development of tools such as zinc finger nucleases (ZFNs) and transcription activator-like effector nucleases (TALENs). To facilitate genome engineering in mammalian cells, we have engineered the CRISPR (clustered regularly interspaced short palindromic repeats)-Cas9 programmable nuclease systems from Streptococcus pyogenes SF370 (SpCas9) and S. thermophilus LMD-9 (St1Cas9) for mouse and human cell gene editing throug

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3

Hirosawa, Moe. "Cell-type-specific genome editing with a microRNA-responsive CRISPR-Cas9 switch." Kyoto University, 2019. http://hdl.handle.net/2433/242421.

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4

Castanon, velasco Oscar. "Targeting the transposable elements of the genome to enable large-scale genome editing and bio-containment technologies." Thesis, Université Paris-Saclay (ComUE), 2019. http://www.theses.fr/2019SACLX006.

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Les nucléases programmables et site-spécifiques comme CRISPR-Cas9 sont des signes avant-coureurs d’une nouvelle révolution en génie génétique et portent en germe un espoir de modification radicale de la santé humaine. Le « multiplexing » ou la capacité d’introduire plusieurs modifications simultanées dans le génome sera particulièrement utile en recherche tant fondamentale qu’appliquée. Ce nouvel outil sera susceptible de sonder les fonctions physiopathologiques de circuits génétiques complexes et de développer de meilleures thérapies cellulaires ou traitements antiviraux. En repoussant les li

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5

Valladares, Rodrigo, and Hanna Briheim. "Metoder och tillämpningar av CRISPR-Cas9 i cancerforskning. : Samt hur CRISPR-Cas9 kan implementeras i skolundervisningen." Thesis, Linköpings universitet, Institutionen för fysik, kemi och biologi, 2020. http://urn.kb.se/resolve?urn=urn:nbn:se:liu:diva-166140.

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CRISPR-Cas9 är ett effektivt genredigeringsverktyg som har upptäckts på senare år. Verktyget härstammar från ett adaptivt immunförsvar hos prokaryoter. Tekniken används för att modifiera DNA hos växter, djur och människor på ett enkelt och billigt sätt. CRISPR-Cas9 har visat sig ha stor potential vid bekämpning av olika sjukdomar däribland cancer som idag är ett globalt hälsoproblem. Inom cancerforskningen ses CRISPR-Cas9 som ett lovande verktyg vid cancerterapi och läkemedelsutveckling. I denna studie sammanställer vi aktuella metoder och användningsområden med CRISPR-Cas9 inom cancerforsknin

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6

Toffessi, Tcheuyap Vanina. "Development of von Willebrand Factor Zebrafish Mutant Using CRISPR/Cas9 Mediated Genome Editing." Thesis, University of North Texas, 2017. https://digital.library.unt.edu/ark:/67531/metadc984227/.

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von Willebrand factor (VWF) protein acts in the intrinsic coagulation pathway by stabilizing FVIII from proteolytic clearance and at the site of injury, by promoting the adhesion and aggregation of platelets to the exposed subendothelial wall. von Willebrand disease (VWD) results from quantitative and qualitative deficiencies in VWF protein. The variability expressivity in phenotype presentations is in partly caused by the action of modifier genes. Zebrafish has been used as hemostasis animal model. However, it has not been used to evaluate VWD. Here, we report the development of a heterozygo

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7

Canver, Matthew. "Elucidation of Mechanisms of Fetal Hemoglobin Regulation by CRISPR/Cas9 Mediated Genome Editing." Thesis, Harvard University, 2016. http://nrs.harvard.edu/urn-3:HUL.InstRepos:33493407.

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Despite nearly complete understanding of the genetics of the β-hemoglobinopathies for several decades, definitive treatment options have lagged behind. Fetal hemoglobin (HbF) reinduction represents a “silver bullet” for therapy of the β-globin disorders. Recent development of the clustered regularly interspaced short palindromic repeat (CRISPR)/Cas9 nuclease system has allowed for facile manipulation of the genome for the study of genes and genetic elements. Here we developed CRISPR/Cas9-based methodology to reliably engender targeted genomic deletions ranging from 1.3 kilobases to over 1 mega

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8

Antoniani, Chiara. "A genome editing approach to induce fetal hemoglobin expression for the treatment of β-hemoglobinopathies". Thesis, Sorbonne Paris Cité, 2017. http://www.theses.fr/2017USPCB077.

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Les β-hémoglobinopathies (β-thalassémies et drépanocytose) sont des anémies génétiques qui touchent des milliers de nouveaux nés chaque année dans le monde. Ces maladies sont causées par des mutations affectant l'expression de l'hémoglobine chez l'adulte. Le seul traitement disponible est la transfusion sanguine à vie, associée à une chélation du fer. Pour les patients les plus touchés, la greffe de cellule souche hématopoïétique (CSH) demeure le seul traitement curatif. Néanmoins, la transplantation autologue de cellules souches génétiquement corrigées représente une alternative thérapeutique

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9

Lin, ChieYu. "Characterization and Optimization of the CRISPR/Cas System for Applications in Genome Engineering." Thesis, Harvard University, 2014. http://etds.lib.harvard.edu/hms/admin/view/61.

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The ability to precisely manipulate the genome in a targeted manner is fundamental to driving both basic science research and development of medical therapeutics. Until recently, this has been primarily achieved through coupling of a nuclease domain with customizable protein modules that recognize DNA in a sequence-specific manner such as zinc finger or transcription activator-like effector domains. Though these approaches have allowed unprecedented precision in manipulating the genome, in practice they have been limited by the reproducibility, predictability, and specificity of targeted cleav

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10

Hsu, Patrick David. "Development of the CRISPR nuclease Cas9 for high precision mammalian genome engineering." Thesis, Harvard University, 2014. http://nrs.harvard.edu/urn-3:HUL.InstRepos:13068392.

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Recent advances in genome engineering technologies based on the CRISPR-associated RNA-guided endonuclease Cas9 are enabling the systematic interrogation of genome function. Analogous to the search function in modern word processors, Cas9 can be guided to specific locations within complex genomes by a short RNA search string. Using this system, DNA sequences within the endogenous genome and their functional outputs are now easily edited or modulated in virtually any organism of choice. Cas9-mediated genetic perturbation is simple and scalable, empowering researchers to elucidate the functional

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11

Bolukbasi, Mehmet F. "Development of Chimeric Cas9 Nucleases for Accurate and Flexible Genome Editing." eScholarship@UMMS, 2017. https://escholarship.umassmed.edu/gsbs_diss/941.

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There has been tremendous amount of effort focused on the development and improvement of genome editing applications over the decades. Particularly, the development of programmable nucleases has revolutionized genome editing with regards to their improvements in mutagenesis efficacy and targeting feasibility. Programmable nucleases are competent for a variety of genome editing applications. There is growing interest in employing the programmable nucleases in therapeutic genome editing applications, such as correcting mutations in genetic disorders. Type II CRISPR-Cas9 bacterial adaptive immuni

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12

Amai, Takamitsu. "Development of genome editing technology of mitochondrial DNA in Saccharomyces cerevisiae." Doctoral thesis, Kyoto University, 2021. http://hdl.handle.net/2433/263707.

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13

Jo, Norihide. "Platforms of in vivo genome editing with inducible Cas9 for advanced cancer modeling." Kyoto University, 2019. http://hdl.handle.net/2433/242397.

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14

Fang, Yufeng. "Nuclear Localization of Proteins and Genome Editing in the Oomycete Phytophthora sojae." Diss., Virginia Tech, 2016. http://hdl.handle.net/10919/74232.

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Oomycetes are fungi-like eukaryotic microorganisms, which are actually phylogenetic relatives of diatoms and brown algae, within the kingdom Stramenopila. Many oomycete species, mainly in the genera Phytophthora, Pythium and downy mildews, are devastating plant pathogens that cause multibillion-dollar losses to agriculture annually in the world. Some oomycetes are also animal pathogens, causing severe losses in aquaculture and fisheries, and occasionally causing dangerous infections of humans. Phytophthora species, represented by the Irish Potato Famine pathogen P. infestans and the soybean pa

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15

Feehan, Joanna Marie. "Development of methodology for genome editing in Xenopus laevis using CRISPR/Cas9, targeting the rhodopsin gene." Thesis, University of British Columbia, 2016. http://hdl.handle.net/2429/57863.

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Xenopus laevis is a commonly used research subject for retinal physiology and cell biology studies, but its utility is limited by the lack of a robust technology for generation of knock-out (KO) or knock-down (KD) phenotypes. However, new genome manipulation techniques involving CRISPR/Cas9 offer an opportunity for generating gene KOs in X. laevis. RNA-guided Cas9 endonuclease introduces double-stranded DNA breaks into the genome, which are either repaired by error-prone non-homologous-end joining (NHEJ), facilitating indel generation, or by less error-prone homology-directed repair (HDR), fac

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Lam, Phuong T. "Crispr/cas9-mediated genome editing of human pluripotent stem cells to advance human retina regeneration research." Miami University / OhioLINK, 2019. http://rave.ohiolink.edu/etdc/view?acc_num=miami1575372014701457.

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17

Kishimoto, Kenta. "Application of genome editing to marine aquaculture as a new breeding technology." Kyoto University, 2019. http://hdl.handle.net/2433/242704.

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Kyoto University (京都大学)<br>0048<br>新制・課程博士<br>博士(農学)<br>甲第21827号<br>農博第2340号<br>新制||農||1067(附属図書館)<br>学位論文||H31||N5199(農学部図書室)<br>京都大学大学院農学研究科応用生物科学専攻<br>(主査)教授佐藤健司, 准教授豊原治彦, 准教授田川正朋<br>学位規則第4条第1項該当

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18

Xu, Huaigeng. "Targeted Disruption of HLA genes via CRISPR-Cas9 generates iPSCs with Enhanced Immune Compatibility." Kyoto University, 2019. http://hdl.handle.net/2433/242420.

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19

Mosqueira, Diogo. "Disease modeling hypertrophic cardiomyopathy using CRISPR/Cas9 genome editing technology in human pluripotent stem cell-derived cardiomyocytes." Thesis, University of Nottingham, 2018. http://eprints.nottingham.ac.uk/51359/.

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Hypertrophic cardiomyopathy (HCM) is a prevalent genetic cardiovascular disease affecting 1:500 individuals whose cardiac function is deteriorated due to thickening of the left ventricle of the heart, mostly owing to mutations in sarcomeric genes. Modeling HCM in vitro using human pluripotent stem cell-derived cardiomyocytes (hPSC-CM) offers promise to further investigate the disease mechanisms, towards the development of effective drugs. Herein, nickase CRISPR/Cas9 genome editing technology was harnessed to introduce the R453C pathological mutation in the MYH7 sarcomeric gene, in three health

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Carstairs, Alice. "Development of in vitro skeletal disease models using CRISPR/Cas9 genome editing in immortalised mesenchymal stem cells." Thesis, University of York, 2017. http://etheses.whiterose.ac.uk/18513/.

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The emergence of engineered nucleases for genome editing has allowed for greater understanding of human biology in health and disease, particularly through combination with stem cells and differentiation protocols. Mesenchymal stem cells (MSCs) are a multipotent adult stem cell able to differentiate into osteoblasts, chondrocytes and adipocytes. Early mesoderm differentiation pathways are relatively well understood, yet the understanding of how mesoderm transcription factors drive post-natal differentiation is less well studied. Additionally, the impact of skeletal disease on MSCs is often neg

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Schneider, Sara Jane. "Delivery of CRISPR/Cas9 RNAs into Blood Cells of Zebrafish: Potential for Genome Editing in Somatic Cells." Thesis, University of North Texas, 2017. https://digital.library.unt.edu/ark:/67531/metadc1011754/.

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Factor VIII is a clotting factor found on the intrinsic side of the coagulation cascade. A mutation in the factor VIII gene causes the disease Hemophilia A, for which there is no cure. The most common treatment is administration of recombinant factor VIII. However, this can cause an immune response that renders the treatment ineffective in certain hemophilia patients. For this reason a new treatment, or cure, needs to be developed. Gene editing is one solution to correcting the factor VIII mutation. CRISPR/Cas9 mediated gene editing introduces a double stranded break in the genomic DNA. Where

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22

Vicencio, Jeremy 1990. "Optimizing CRISPR-Cas technologies in Caenorhabditis elegans : Nested CRISPR and expanded targeting with Cas variants." Doctoral thesis, TDX (Tesis Doctorals en Xarxa), 2021. http://hdl.handle.net/10803/672604.

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In this thesis, I present an alternative, cloning-free method for the generation of endogenous fluorescent reporters in the nematode Caenorhabditis elegans. I demonstrate that Nested CRISPR is an efficient method that can be customized for the insertion of a suite of fluorescent tags and epitopes at endogenous loci using a combination of single-stranded and double-stranded DNA repair templates. In this thesis, I also demonstrate the use of enzymes other than Cas9 to target non-NGG PAM sites. The results show that AsCas12a can perform efficient genome editing in TTTV PAMs. Furthermore, the stru

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23

Tennant, Peter Andrew. "Genome editing using site-specific nucleases : targeting highly expressed genomic regions for robust transgene expression and genetic analysis." Thesis, University of Edinburgh, 2016. http://hdl.handle.net/1842/22857.

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Integration and expression of exogenous genetic material – in particular, transgenes – into the genomes of model organisms, cell lines or patients is widely used for the creation of genetically modified experimental systems and gene therapy. However, loss of transgene expression due to silencing is still a major hurdle which remains to be overcome. Judicious selection of integration loci can help alleviate the risk of silencing; in recent years the ability to efficiently and specifically target transgene integration has been improved by the advent of site-specific nucleases (SSNs). SSNs can be

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24

Vitarelli, Marcela de Oliveira. "Humanização específica do sistema de glicosilação de Pichia pastoris pela técnica CRISPR/Cas9 visando a expressão de glicoproteínas humanas." Universidade de São Paulo, 2016. http://www.teses.usp.br/teses/disponiveis/46/46131/tde-11042017-084657/.

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A produção de proteínas terapêuticas recombinantes compreende moléculas complexas e de alto valor agregado, incluindo a enzima glucocerebrosidase (GCase). Sua deficiência resulta na Doença de Gaucher, passível de tratamento por meio da terapia de reposição enzimática. A forma ativa da GCase recombinante usada na terapia apresenta resíduos terminais de manose expostos no seu perfil de glicosilação. Perfil este que espera-se ser reproduzido por meio da construção de uma linhagem de Pichia pastoris com um padrão de glicosilação humanizado, por meio da deleção de dois genes envolvidos no sistema d

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Amaya, Colina Anais Karime. "Towards the Treatment of Human Genetic Liver Disease by AAV-Mediated Genome Editing and Selective Expansion of Repaired Hepatocytes." Thesis, The University of Sydney, 2019. https://hdl.handle.net/2123/21893.

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Gene repair involves the correction of the genetic mutation directly at the defective locus with retention of physiological expression. The biggest challenge of this approach, however, is that gene repair by homologous recombination occurs at levels that are unlikely to be sufficient to confer therapeutic benefit in the majority of cell-autonomous liver disease phenotypes, such as OTC deficiency, the most common urea cycle disorder. To overcome this challenge, gene correction can be complemented by selective expansion strategies designed to expand repaired hepatocytes to frequencies required f

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Edraki, Alireza. "Compact Cas9s and Their Natural Inhibitors for Genome Editing." eScholarship@UMMS, 2019. https://escholarship.umassmed.edu/gsbs_diss/1052.

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Recent advances with the bacterial CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) defense system as genome editing tools have opened a new avenue for targeting disease-causing mutations. The programmability of the Cas9 endonuclease by RNA makes it a potentially powerful therapeutic tool to correct such mutations. The CRISPR-Cas9 system consists of a Cas9 endonuclease that is guided by RNA (sgRNA) to create double-stranded breaks in a target DNA segment complementary to the guide. This process is dependent on a 2-8 nucleotide sequence (called PAM) that is adjacent to the tar

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27

Bressan, Raul Bardini. "Genome editing as a tool to explore transcriptional and epigenetic regulation in neural stem cells and brain cancer." Thesis, University of Edinburgh, 2018. http://hdl.handle.net/1842/31095.

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Mammalian neural stem cell (NSC) lines provide a useful experimental model for basic and applied research across stem cell and developmental biology, regenerative medicine and neuroscience. NSCs are clonally expandable, genetically stable, and easily transfectable - experimental attributes compatible with functional genetic analyses. However, targeted genetic manipulations have not been reported for mammalian NSC lines. Here, we deploy the CRISPR/Cas9 technology and demonstrate a variety of diverse targeted genetic modifications in both mouse and human NSC lines such as: targeted transgene ins

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Fine, Eli Jacob. "A toolkit for analysis of gene editing and off-target effects of engineered nucleases." Diss., Georgia Institute of Technology, 2015. http://hdl.handle.net/1853/54875.

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Several tools were developed to help researchers facilitate clinical translation of the use of engineered nucleases towards their disease gene of interest. Two major issues addressed were the inability to accurately predict nuclease off-target sites by user-friendly \textit{in silico} methods and the lack of a high-throughput, sensitive measurement of gene editing activity at endogenous loci. These objectives were accomplished by the completion of the following specific aims. An online search interface to allow exhaustive searching of a genome for potential nuclease off-target sites was implem

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29

Charpentier, Marine. "Développement de nouvelles approches d’édition du génome à l’aide de nucléases artificielles (TALENs et CRISPR/Cas9)." Thesis, Paris, EPHE, 2016. http://www.theses.fr/2016EPHE3106/document.

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L’édition du génome repose sur la création de cassures double brin à un endroit précis du génome à l’aide de nucléases artificielles (ZFN, TALEN, CRISPR/Cas9) et sur les différents systèmes de réparation que la cellule va mettre en place pour réparer ces dommages. Les deux systèmes de réparation principaux sont le NHEJ (Non Homologous End Joining) et la RH (Recombinaison Homologue). Le NHEJ consiste en une ligation directe des extrémités de la coupure pouvant induire de petites insertions ou délétions avant la ligation. Ces mutations, si elles sont introduites dans un exon, vont modifier le ca

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LLADO, SANTAEULARIA MANEL. "THERAPEUTIC GENOME EDITING IN RETINA AND LIVER." Doctoral thesis, Università degli Studi di Milano, 2020. http://hdl.handle.net/2434/696628.

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In vivo gene therapy with adeno-associated viral (AAV) vectors has been successful at treating several inherited diseases, specifically those caused by loss of function mutations which require transfer of a correct copy of a gene. This would not benefit dominant diseases due to gain of function mutations which produce toxic protein products. In addition, since AAV genomes persist as episomes in target cells, AAV mediated transgene expression might be short lived in tissues where cell proliferation occurs when newborn or after damage, like for example the liver. To overcome these challenges, I

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Ballmann, Cora [Verfasser]. "Gezielte Sequenzierung von USP8 bei PatientInnen mit Morbus Cushing und Genome Editing in HAC15 Zellen mittels CRISPR/Cas9 / Cora Ballmann." Düsseldorf : Universitäts- und Landesbibliothek der Heinrich-Heine-Universität Düsseldorf, 2021. http://d-nb.info/1231075147/34.

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Kapahnke, Marcel [Verfasser]. "Knock-out of Flotillins in Human Cells Using the CRISPR-Cas9 Genome Editing System: Effects on mRNA Splicing / Marcel Kapahnke." Gießen : Universitätsbibliothek, 2020. http://d-nb.info/1223462137/34.

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33

Stringa, Blerta. "The effect of germline variants on the genesis of early somatic events in cancer explored via Cas9 genome editing." Doctoral thesis, Università degli studi di Trento, 2019. http://hdl.handle.net/11572/242372.

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Although the understanding of genetic predisposition to prostate cancer (PCa) has been improved through genome-wide association studies (GWAS), little is known about the biological implication of germline variants residing in coding or non-coding regions in cancer development and progression. Our hypothesis is that inherited variants may predispose to specific early recurrent genomic events observed in PCa adenocarcinomas, possibly in the context of variable androgen receptor (AR) signaling that changes during a man’s lifetime. Recent in silico analysis by our group on potential association b

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Stringa, Blerta. "The effect of germline variants on the genesis of early somatic events in cancer explored via Cas9 genome editing." Doctoral thesis, Università degli studi di Trento, 2019. http://hdl.handle.net/11572/242372.

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Although the understanding of genetic predisposition to prostate cancer (PCa) has been improved through genome-wide association studies (GWAS), little is known about the biological implication of germline variants residing in coding or non-coding regions in cancer development and progression. Our hypothesis is that inherited variants may predispose to specific early recurrent genomic events observed in PCa adenocarcinomas, possibly in the context of variable androgen receptor (AR) signaling that changes during a man’s lifetime. Recent in silico analysis by our group on potential association b

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Murakami, Yu. "Establishment of a practical gene knock-in system and its application in medaka." Kyoto University, 2020. http://hdl.handle.net/2433/253339.

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Kyoto University (京都大学)<br>0048<br>新制・課程博士<br>博士(農学)<br>甲第22503号<br>農博第2407号<br>新制||農||1077(附属図書館)<br>学位論文||R2||N5283(農学部図書室)<br>京都大学大学院農学研究科応用生物科学専攻<br>(主査)教授佐藤健司, 教授澤山茂樹, 准教授豊原治彦<br>学位規則第4条第1項該当

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MINGOIA, MAURA. "Terapia genica della β Talassemia mediante editing del DNA". Doctoral thesis, Università degli Studi di Cagliari, 2016. http://hdl.handle.net/11584/266632.

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β (HBB) gene, resulting in absence (β0) or deficiency (β+) of β globin chain synthesis. This genetic disorder occurs most frequently in people from Mediterranean countries, such as Italy. In particular, the data indicates that about 12.6% of the Sardinian subjects are carriers of β thalassemia and these are among the highest frequencies of thalassemia genes found in a Caucasian population. In Sardinia, the disease is generally determined by a nonsense mutation at codon 39 (E39X) of exon 2 causing the interruption of β globin synthesis. Patients homozygous with E39X mutation have a severe anemi

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BORRELLI, VIRGINIA MARIA GRAZIA. "Caratterizzazione del gene LIPOSSIGENASI 4 e approccio CRISPR-Cas9 per aumentare la resistenza alla fusariosi di mais." Doctoral thesis, Università Cattolica del Sacro Cuore, 2018. http://hdl.handle.net/10280/53792.

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Il Fusarium verticillioides (Fv) causa il marciume rosa della spiga e contamina le cariossidi con fumonisine, una famiglia di micotossine che colpisce mangimi e alimenti considerata cancerogena per l'uomo e gli animali. Sono stati condotti diversi studi per identificare i geni del mais associati alla resistenza della pianta ospite all'infezione da Fv e l'accumulo di fumonisina. È noto che le ossilipine regolano la difesa contro i patogeni e che il cross-talk lipidico ospite-patogeno influenza la patogenesi. A questo proposito, i mutanti di mais trasposonici del gene ZmLOX4, la linea suscettibi

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BORRELLI, VIRGINIA MARIA GRAZIA. "Caratterizzazione del gene LIPOSSIGENASI 4 e approccio CRISPR-Cas9 per aumentare la resistenza alla fusariosi di mais." Doctoral thesis, Università Cattolica del Sacro Cuore, 2018. http://hdl.handle.net/10280/53792.

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Il Fusarium verticillioides (Fv) causa il marciume rosa della spiga e contamina le cariossidi con fumonisine, una famiglia di micotossine che colpisce mangimi e alimenti considerata cancerogena per l'uomo e gli animali. Sono stati condotti diversi studi per identificare i geni del mais associati alla resistenza della pianta ospite all'infezione da Fv e l'accumulo di fumonisina. È noto che le ossilipine regolano la difesa contro i patogeni e che il cross-talk lipidico ospite-patogeno influenza la patogenesi. A questo proposito, i mutanti di mais trasposonici del gene ZmLOX4, la linea suscettibi

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Ibraheim, Raed R. "Genome Engineering Goes Viral: Repurposing of Adeno-associated Viral Vectors for CRISPR-mediated in Vivo Genome Engineering." eScholarship@UMMS, 2020. https://escholarship.umassmed.edu/gsbs_diss/1114.

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One of the major challenges facing medicine and drug discovery is the large number of genetic diseases caused by inherited mutations leading to a toxic gain-of-function, or loss-of-function of the disease protein. Microbiology offered a new glimpse of hope to address those disorders with the adaptation of the bacterial CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) defense system as a genome editing tool. Cas9 is a unique CRISPR-associated endonuclease protein that can be easily programmed with an RNA [a single-guide RNA (sgRNA)] that is complementary to nearly any DNA locu

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Janin, Grajcarek. "Genome-wide microhomologies enable precise template-free editing of biologically relevant deletion mutations." Kyoto University, 2020. http://hdl.handle.net/2433/253215.

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Foster, Robert Graham. "Development of a modular in vivo reporter system for CRISPR-mediated genome editing and its therapeutic applications for rare genetic respiratory diseases." Thesis, University of Edinburgh, 2018. http://hdl.handle.net/1842/33040.

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Rare diseases, when considered as a whole, affect up to 7% of the population, which would represent 3.5 million individuals in the United Kingdom alone. However, while 'personalised medicine' is now yielding remarkable results using recent sequencing technologies in terms of diagnosing genetic conditions, we have made much less headway in translating this patient information into therapies and effective treatments. Even with recent calls for greater research into personalised treatments for those affected by a rare disease, progress in this area is still severely lacking, in part due to the as

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Marco, Giménez Andrés 1993. "Generation and validation of a CRISPR platform for rapid and inducible genome editing in human pluripotent stem cells and kidney organoids." Doctoral thesis, TDX (Tesis Doctorals en Xarxa), 2022. http://hdl.handle.net/10803/673798.

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Our current knowledge about the function of human genes is mostly based on data from genetic studies using animal models. However, divergence among species may hamper the understanding of genetic mechanisms underlying human specific traits. Nowadays, an alternative to animal models stands on the generation of organ-like cultures derived from human pluripotent stem cells (hPSCs), the so called organoids. In the last years, the organoids have proved to recapitulate, in a high extent, the development, multicellular architecture, and physiology of human organs. To perform genetic studies in

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Hahn, Florian [Verfasser], Andreas P. M. [Gutachter] Weber, and Peter [Gutachter] Westhoff. "Genome editing and establishment of efficient gene targeting approaches in Arabidopsis using the CRISPR/Cas9 system / Florian Hahn ; Gutachter: Andreas P. M. Weber, Peter Westhoff." Düsseldorf : Universitäts- und Landesbibliothek der Heinrich-Heine-Universität Düsseldorf, 2018. http://d-nb.info/1159373612/34.

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Sürün, Duran [Verfasser], Beatrix [Akademischer Betreuer] Süß, M. Cristina [Akademischer Betreuer] Cardoso, and Harald von [Akademischer Betreuer] Melchner. "High Efficiency Gene Correction in Hematopoietic Cells by Donor Template-free CRISPR/Cas9 Genome Editing / Duran Sürün ; Beatrix Süß, M. Cristina Cardoso, Harald von Melchner." Darmstadt : Universitäts- und Landesbibliothek Darmstadt, 2018. http://d-nb.info/1153546396/34.

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Pröbsting, Michael Verfasser], Daguang [Akademischer Betreuer] [Cai, and Christian [Gutachter] Jung. "Application of CRISPR-Cas9 genome editing systems for improving oilseed rape (Brassica napus) disease resistance against Verticillium longisporum / Michael Pröbsting ; Gutachter: Christian Jung ; Betreuer: Daguang Cai." Kiel : Universitätsbibliothek Kiel, 2020. http://nbn-resolving.de/urn:nbn:de:gbv:8-mods-2020-00084-7.

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Pröbsting, Michael [Verfasser], Daguang [Akademischer Betreuer] Cai, and Christian [Gutachter] Jung. "Application of CRISPR-Cas9 genome editing systems for improving oilseed rape (Brassica napus) disease resistance against Verticillium longisporum / Michael Pröbsting ; Gutachter: Christian Jung ; Betreuer: Daguang Cai." Kiel : Universitätsbibliothek Kiel, 2020. http://d-nb.info/120658887X/34.

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Carayon, Alexandre. "Mise en place de l'identité musculaire durant la myogenèse embryonnaire chez la drosophile." Thesis, Toulouse 3, 2018. http://www.theses.fr/2018TOU30107.

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La diversité morphologique des muscles squelettiques permet la précision et la coordination des mouvements propres à chaque espèce animale. L'établissement du patron musculaire a lieu au cours du développement embryonnaire durant le processus de myogenèse. Il a été décomposé en quatre étapes chez la drosophile : la spécification de groupes de myoblastes équivalents (groupes promusculaires) à des positions précises du mésoderme, la sélection d'une ou plusieurs cellules progéniteurs à partir de chaque groupe, la division asymétrique des progéniteurs en cellules fondatrices des muscles, et enfin,

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Youssef, Divana. "Recherches de méthodes innovantes issues des biotechnologies pour l'amélioration génétique du blé tendre (Triticum aestivum L.)." Thesis, Université Clermont Auvergne‎ (2017-2020), 2017. http://www.theses.fr/2017CLFAC054.

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L’amélioration génétique du blé tendre (Triticum aestivum L.), une des trois céréales les plus cultivées, représente un intérêt stratégique pour la sécurité alimentaire de la population mondiale. Cette amélioration génétique va nécessiter une meilleure compréhension des mécanismes moléculaires et physiologiques mis en jeu, et va aussi réclamer une efficacité accrue dans notre capacité à intervenir finement sur le génome. Les avancées majeures réalisées dans le domaine des biotechnologies ces dernières années permettent d’envisager de nouveaux champs d’action pour appréhender le fonctionnement

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Stens, Cassandra, Isabella Enoksson, and Sara Berggren. "The CRISPR-Cas system." Thesis, Linköpings universitet, Institutionen för fysik, kemi och biologi, 2020. http://urn.kb.se/resolve?urn=urn:nbn:se:liu:diva-171997.

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Derived from and inspired by the adaptive immune system of bacteria, CRISPR has gone from basic biology knowledge to a revolutionizing biotechnological tool, applicable in many research areas such as medicine, industry and agriculture. The full mechanism of CRISPR-Cas9 was first published in 2012 and various CRISPR-Cas systems have already passed the first stages of clinical trials as new gene therapies. The immense research has resulted in continuously growing knowledge of CRISPR systems and the technique seems to have the potential to greatly impact all life on our planet. Therefore, this li

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Rabai, Aymen. "Correction de l'ADN in vitro et in vivo comme thérapie personnalisée pour les myopathies congénitales." Thesis, Strasbourg, 2018. http://www.theses.fr/2018STRAJ117.

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L’édition du génome utilisant CRISPR/Cas9 est récemment apparue comme une stratégie thérapeutique potentielle des maladies génétiques. Pour les mutations dominantes de type gain de fonction, la correction allèle-spécifique pourrait être l'approche la plus appropriée. Ici, nous avons testé l'inactivation ou la correction d'une mutation hétérozygote du gène de la dynamine 2 (DNM2) causant la forme autosomique dominante de la myopathie centronucléaire (CNM). Des ARN-guides tronqués ciblant spécifiquement l'allèle muté ont été testés sur des cellules de patients et des myoblastes d'un modèle murin

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