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Relevant bibliographies by topics / CRISPRko Screening / Dissertations / Theses

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Dissertations / Theses on the topic 'CRISPRko Screening'

Author: Grafiati

Published: 1 April 2023

Last updated: 31 July 2025

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1

PERSICO, ILARIA. "DEALING WITH THE MAIN CHALLENGES OF FANCONI ANEMIA MOLECULAR DIAGNOSIS AND THERAPY." Doctoral thesis, Università degli Studi di Trieste, 2023. https://hdl.handle.net/11368/3042318.

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L’anemia di Fanconi (Fanconi anemia, FA) è una sindrome genetica rara causata da un difetto nella riparazione del danno al DNA con ampia eterogeneità genetica, numerose mutazioni private ed elevato tasso di mosaicismo. Tali fattori compromettono tuttora la formulazione di una diagnosi molecolare in caso di varianti di difficile caratterizzazione o significato patogenetico incerto. La FA è caratterizzata da aplasia midollare e un’aumentata suscettibilità allo sviluppo di tumori ematologici e solidi. Dato che i trattamenti attualmente disponibili si rivolgono ai soli difetti ematopoietici, è ess

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2

Erard, Nicolas Pascal Jean. "Optimization of molecular tools for high-throughput genetic screening." Thesis, University of Cambridge, 2018. https://www.repository.cam.ac.uk/handle/1810/271895.

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Forward genetic screening allows for the identification of any genes important for a particular biological process or phenotype. While the power of this approach is broadly agreed on, the efficacy of currently available tools limits the strength of conclusions drawn from these experiments. This thesis describes a method to optimize molecular tools for high-throughput screening, both for shRNA and sgRNA based reagents. Using large shRNA efficacy datasets, we first designed an algorithm predicting the potency of shRNAs based on sequence determinants. Combined with a novel shRNA backbone that fur

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3

Sheel, Ankur. "Identification of Essential Genes in Hepatocellular Carcinomas using CRISPR Screening." eScholarship@UMMS, 2019. https://escholarship.umassmed.edu/gsbs_diss/1039.

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Hepatocellular carcinoma (HCC) is an aggressive subtype of liver cancer with a poor prognosis. Currently, prognosis for HCC patients remains poor as few therapies are available. The clinical need for more effective HCC treatments remains unmet partially because HCC is genetically heterogeneous and HCC driver genes amenable to targeted therapy are largely unknown. Mutations in the TP53 gene are found in ~30% of HCC patients and confer poor prognosis to patients. Identifying genes whose depletion can inhibit HCC growth, and determining the mechanisms involved, will aid the development of targete

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4

Rubanova, Natalia. "MasterPATH : network analysis of functional genomics screening data." Thesis, Sorbonne Paris Cité, 2018. http://www.theses.fr/2018USPCC109/document.

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Dans ce travail nous avons élaboré une nouvelle méthode de l'analyse de réseau à définir des membres possibles des voies moléculaires qui sont important pour ce phénotype en utilisant la « hit-liste » des expériences « omics » qui travaille dans le réseau intégré (le réseau comprend des interactions protéine-protéine, de transcription, l’acide ribonucléique micro-l’acide ribonucléique messager et celles métaboliques). La méthode tire des sous-réseaux qui sont construit des voies de quatre types les plus courtes (qui ne se composent des interactions protéine-protéine, ayant au minimum une inter

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5

Rousset, Francois. "CRISPRi screens in bacterial genomics." Electronic Thesis or Diss., Sorbonne université, 2020. http://www.theses.fr/2020SORUS373.

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La génomique chez les bactéries a connu un véritable essor au cours de la dernière décennie grâce aux progrès des méthodes de séquençage de l'ADN. De nouvelles techniques expérimentales sont nécessaires afin de mieux comprendre la fonction des gènes. La découverte des systèmes immunitaires adaptatifs CRISPR-Cas chez les bactéries a conduit au développement de nombreuses technologies pour cibler un acide nucléique de manière séquence-spécifique. En particulier, l’enzyme dCas9 peut être guidée vers une séquence d’ADN par un court ARN nommé sgRNA afin d'inhiber l'expression d'un gène de manière s

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6

Li, Meng. "Genetic dissection of the exit of pluripotency in mouse embryonic stem cells by CRISPR-based screening." Thesis, University of Cambridge, 2018. https://www.repository.cam.ac.uk/handle/1810/277552.

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The ground state naive pluripotency is established in the epiblast of the blastocyst and can be captured by culturing mouse embryonic stem cells (mESCs) with MEK and GSK3 inhibitors (2i). The transcription network that maintains pluripotency has been extensively studied with the indispensable core factors being Oct4, Sox2 and Nanog, together with other ancillary factors reinforcing the network. However, how this network is dissolved at the onset of differentiation is still not fully understood. To identify genes required for differentiation in an unbiased fashion, I conducted a genome-wide CRI

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7

Kaemena, Daniel Fraser. "CRISPR/Cas9 genome-wide loss of function screening identifies novel regulators of reprogramming to pluripotency." Thesis, University of Edinburgh, 2018. http://hdl.handle.net/1842/31184.

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In 2006, Kazutoshi Takahashi and Shinya Yamanaka demonstrated the ability of four transcription factors; Oct4, Sox2, Klf4 and c-Myc to 'reprogram' differentiated somatic cells to a pluripotent state. This technology holds huge potential in the field of regenerative medicine, but reprogramming also a model system by which to the common regulators of all forced cell identity changes, for example, transdifferentiation. Despite this, the mechanism underlying reprogramming remains poorly understood and the efficiency of induced pluripotent stem cell (iPSC) generation, inefficient. One powerful meth

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8

Petrucci, Teresa. "Building a platform for flexible and scalable testing of genetic editors." Doctoral thesis, Università di Siena, 2021. http://hdl.handle.net/11365/1143160.

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Cell-free systems allow to perform in-vitro transcription-translation reactions without requiring living organisms, revolutionising scientific research over the last decade. This allows to easily synthesise a variety of molecular components for genetic editing applications without requiring expensive and time-consuming procedures such as cell culture, animal maintenance etc. In this work, I aimed to develop a high-throughput platform for the rapid, flexible and scalable in-vitro testing of various genetic editors, such as those part of the CRISPR/Cas repertoire. I used the commercially avail

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9

Sczakiel, Henrike Lisa [Verfasser]. "Identifizierung Pathogenese-relevanter Kandidatengene im Hodgkin-Lymphom durch CRISPR/Cas9-basiertes knockout-Screening / Henrike Lisa Sczakiel." Berlin : Medizinische Fakultät Charité - Universitätsmedizin Berlin, 2021. http://d-nb.info/1228859523/34.

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10

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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11

Cresson, Marie. "Study of chikungunya virus entry and host response to infection." Thesis, Lyon, 2019. http://www.theses.fr/2019LYSE1050.

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Les alphavirus sont un groupe de virus enveloppés à ARN simple brin positif retrouvés sur la totalité du globe et responsables de nombreuses maladies humaines et animales. Durant la dernière décennie, une réémergence du virus du chikungunya (CHIKV) a été observée causant de nombreuses épidémies sur tous les continents. Malgré les nombreuses études, les mécanismes moléculaires de réplication du CHIKV et les interactions hôte-virus restent peu caractérisées. L’objectif de mon travail était de mieux comprendre et caractériser l’entrée du virus du chikungunya et les facteurs de l’hôte impliqués da

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12

Mohammad, Jiyan Mageed. "Therapeutic Potential of Piperlongumine for Pancreatic Ductal Adenocarcinoma." Diss., North Dakota State University, 2019. https://hdl.handle.net/10365/31347.

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Pancreatic ductal adenocarcinoma (PDAC) is among the most lethal malignancies because it is often diagnosed at a late disease stage and has a poor response rate to currently available treatments. Therefore, it is critical to develop new therapeutic approaches that will enhance the efficacy and reduce the toxicity of currently used therapies. Here we aimed to evaluate the therapeutic potential and mechanisms of action for piperlongumine (PL), an alkaloid from long pepper, in PDAC models. We postulated that PL causes PDAC cell death through oxidative stress and complements the therapeutic effica

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13

Fracassi, Giulia. "CRISPR/Cas9 screenings and in silico investigations nominate low-frequency alterations in DNA repair genes as biomarkers for castration-resistant prostate cancers." Doctoral thesis, Università degli studi di Trento, 2023. https://hdl.handle.net/11572/364383.

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PARP inhibitors (PARPi) have received regulatory approval for the treatment of multiple tumor types, including prostate cancer (PCa), and are associated with therapeutic response in metastatic castration-resistant prostate cancer (mCRPC) patients characterized by defects in homologous recombination repair genes. Clinical trials results suggest that further improvements are possible, for instance in terms of patients’ enrolment criteria. Only 50% of BRCA1/2-deficient patients (TRITON2 trial) respond to therapy, variable antitumor activity is observed for the covered non-BRCA DNA repair gene (DR

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14

Lembo, Gaia. "Substrate targeting and inhibition of editing deaminases." Doctoral thesis, Università di Siena, 2021. http://hdl.handle.net/11365/1144295.

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Identification of small molecules against APOBEC3B The APOBECs are deaminases that act on DNA and RNA to restrict exogenous nucleic acids. Yet, the signature of their mutagenic activity –especially that of APOBEC3A and APOBEC3B- has been observed in the cancer genomes and their ability to increase the genetic heterogeneity of tumours has been linked to the onset of drug resistance in cancer. As such inhibition of their enzymatic activity represents a potential target for anticancer therapies. During my PhD I worked at the identification of APOBEC3B small-molecule inhibitors. To this aim, I us

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15

Merenda, Alessandra. "Development of a new screening system for the identification of RNF43-related genes and characterisation of other PA-RING family members." Thesis, University of Cambridge, 2017. https://www.repository.cam.ac.uk/handle/1810/267982.

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The E3 ubiquitin ligase RNF43 (RING finger protein 43) is an important negative modulator of the WNT signalling pathway that acts at the plasma membrane by targeting Frizzled and its co-receptor LRP for degradation. In the small intestine, this prevents uncontrolled expansion of the stem cell compartment and so it is essential to the maintenance of normal tissue homeostasis. However, despite its crucial role in fine-tuning the WNT pathway and its role as a tumour suppressor, it is unclear whether RNF43 has further binding partners and what their functional relevance is to the modulation of WNT

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16

SPATARO, CLARISSA. "IDENTIFICATION OF NEW MYC DEPENDENCIES AMONG RNA-BINDING PROTEINS." Doctoral thesis, Università degli Studi di Milano, 2022. http://hdl.handle.net/2434/909490.

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Increased expression and activity of the MYC protein is a widespread cancer hallmark and renders tumor cells addicted to sustained activation of a variety of other gene products. Identification of those dependencies can offer new therapeutic approaches against MYC-driven tumors. Previous studies showed that RNA processing events have a critical role in MYC-induced tumorigenesis and survival. Moreover, we and others observed that multiple genes encoding RNABinding Proteins (RBPs) were positively regulated upon MYC activation in various cell types. Hence, we hypothesized that the activity

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17

Basso, Pauline. "Exolysine, un facteur de virulence majeur de Pseudomonas aeruginosa." Thesis, Université Grenoble Alpes (ComUE), 2017. http://www.theses.fr/2017GREAV063/document.

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Pseudomonas aeruginosa est un pathogène opportuniste responsable d’infections nosocomiales sévères associées à un taux élevé de mortalité. Le système de sécrétion de Type III (SST3) et les effecteurs qu’il injecte sont considérés comme des facteurs de virulence prépondérants de P. aeruginosa. Récemment nous avons caractérisé, un groupe de souches ne possédant pas les gènes du SST3, mais dont la virulence repose sur la sécrétion d’une nouvelle toxine de 172 kDa, nommée Exolysine (ExlA) qui provoque la perméabilisation de la membrane des cellules hôtes. ExlA est sécrétée dans le milieu par une p

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18

Weber, Julia Maria [Verfasser], Radu Roland [Akademischer Betreuer] Rad, Heinrich [Gutachter] Leonhardt, Radu Roland [Gutachter] Rad, and Angelika [Gutachter] Schnieke. "Transposon- and CRISPR-based tools for tumour suppressor gene screening in vivo / Julia Maria Weber ; Gutachter: Heinrich Leonhardt, Radu Roland Rad, Angelika Schnieke ; Betreuer: Radu Roland Rad." München : Universitätsbibliothek der TU München, 2021. http://d-nb.info/1236342879/34.

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19

ALFRED, VICTOR IFEOLUWA. "GENETIC SCREENING TO IDENTIFY INTERACTORS OF ESCRT-II SUBUNIT, VPS25, AND PRELIMINARY CHARACTERISATION OF CANDIDATES." Doctoral thesis, Università degli Studi di Milano, 2018. http://hdl.handle.net/2434/560382.

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ESCRT (Endosomal Sorting Complex Required for Transport) proteins regulate cell surface receptor degradation by sorting and packaging ubiquitinated cargoes into the intraluminal vesicles of multivesicular bodies (MVBs). A range of human diseases including cancer, and neurodegeneration display altered expression or are caused by mutations of ESCRT subunits. Studies have shown that Drosophila tissues lacking ESCRTs display neoplastic-like features like overproliferation and polarity defects, partly due to aberrant signalling including Notch signalling. To understand ESCRT-regulated processes in

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20

Mathews, Bobby. "A zebrafish model system for drug screening in diabetes." Thesis, 2019. http://urn.kb.se/resolve?urn=urn:nbn:se:his:diva-17847.

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GWAS (Genome wide association studies) have aided in the discovery of various novel variants associated with diabetes. However, a detailed study is required to uncover the role of these genes and to determine how their dysfunction affects pathophysiology. Previous work in the lab has been successful in establishing zebrafish as an efficient model to characterise the effects of these candidate genes. Consequently, efforts have been also made to establish zebrafish as an efficient model system for drug screening as well. The current POP (Proof of principle) study aims to find whether treatment w

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21

Singh, V. "Applying bioinformatic tools to better understand eye diseases." Thesis, 2020. https://eprints.utas.edu.au/35302/1/Singh_whole_thesis.pdf.

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The highly specialized cells of the eye function in concert to produce clear vision, one of the most valued senses. Visual impairment or blindness can occur as a result of disease or trauma. Age-related macular degeneration, glaucoma, cataracts and diabetic retinopathy are common causes of vision loss in older individuals. This thesis explores the bioinformatics approaches based on the central dogma as a model for exploring the experimental models for human eye diseases including quality control of stem cells to detect chromosomal abnormalities; epigenetic age prediction of ocular tissues; ide

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22

Wu, Yi-Hsin, and 吳以新. "Establishing CRISPR interference-based genome-wide screening platform for identification of novel genes in macrophage alternative polarization." Thesis, 2019. http://ndltd.ncl.edu.tw/handle/m99x3g.

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碩士<br>國立臺灣大學<br>分子醫學研究所<br>107<br>Macrophages are crucial players in immune regulation. They have a wide spectrum of activation states depend on the diverse surrounding stimuli they receive. Classical activation (M1) and alternative activation (M2) are described as two extremes of their polarized states, which elicit pro-inflammatory responses and anti-inflammatory responses respectively to maintain tissue homeostasis. Regnase-1 is a ribonuclease essential in controlling immune responses by regulating mRNA decay of proinflammatory cytokines, and it is reported to be important in promoting macr

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23

Yang, Miao-Chia, and 楊苗佳. "Using CRISPR/Cas9-Mediated GLA-null Cell Lines as An In Vitro Drug Screening Model for Fabry Disease." Thesis, 2016. http://ndltd.ncl.edu.tw/handle/49926843078539991979.

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碩士<br>國立陽明大學<br>藥理學研究所<br>104<br>Fabry disease is a hereditary, X-linked lysosomal storage disease resulting from deficient activity of the lysosomal α-galactosidase A. It leads to progressive accumulation of glycosphingolipids particularly globotriaosylceramide (GL-3) in lysosomes of the heart, kidneys, skin and various tissues. Regular administration of recombinant human alpha Gal A (rh-α-GLA), termed enzyme replacement therapy (ERT) is currently available as the only effective treatment for the Fabry patients with GL-3 accumulation. However, the rh-α-GLA driven GL-3 clearance has the limita

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24

Simon, Laura. "Use of chemogenomic approaches to characterize RUNX1-mutated Acute Myeloid Leukemia and dissect sensitivity to glucocorticoids." Thesis, 2020. http://hdl.handle.net/1866/24841.

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RUNX1 est un facteur de transcription essentiel pour l’hématopoïèse et joue un rôle important dans la fonction immunitaire. Des mutations surviennent dans ce gène chez 5 à 13% des patients atteints de leucémie myéloïde aiguë (LMA) (RUNX1mut) et définissent un sous-groupe particulier de LMA associé à un pronostic défavorable. En conséquence, il est nécessaire de procéder à une meilleure caractérisation génétique et de concevoir des stratégies thérapeutiques plus efficaces pour ce sousgroupe particulier de LMA. Bien que la plupart des mutations trouvées dans le gène RUNX1 dans la LMA soient supp

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25

Cloutier, Véronique. "Criblage génétique et caractérisation fonctionnelle des mutations dans le gène CHD2 associé à l’épilepsie dans un modèle de poisson zèbre." Thèse, 2018. http://hdl.handle.net/1866/21388.

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