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Dissertations / Theses on the topic 'CRISPR'

Author: Grafiati
Published: 4 June 2021
Last updated: 26 July 2025

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1

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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2

TAVELLA, SARA. "WEAPONIZING CRISPR/CAS9." Doctoral thesis, Università degli Studi di Milano, 2022. http://hdl.handle.net/2434/908993.

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One of the major limits of current therapies against cancer and viral infections is the nonspecific toxicity that they often cause on healthy tissues because of their impact on important cellular mechanisms shared, to different extents, between diseased and healthy cells. For this reason, there is an unmet need for more specific and more effective therapies. Wherefore, the aim of my project is the development of a novel strategy, with potential for therapy, that allows the induction of sequence-specific DNA lesions (DNA double-strand break, DSB), by the use of the CRISPR/Cas9 system targ
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3

Demozzi, Michele. "Identification of novel active Cas9 orthologs from metagenomic data." Doctoral thesis, Università degli studi di Trento, 2022. http://hdl.handle.net/11572/337709.

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CRISPR-Cas is the state-of-the-art biological tool that allows precise and fast manipulation of the genetic information of cellular genomes. The translation of the CRISPR-Cas technology from in vitro studies into clinical applications highlighted a variety of limitations: the currently available systems are limited by their off-target activity, the availability of a Cas-specific PAM sequence next to the target and the size of the Cas protein. In particular, despite high levels of activity, the size of the CRISPR-SpCas9 editing machinery is not compatible with an all-in-one AAV delivery system
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4

Stachler, Aris-Edda [Verfasser]. "Das CRISPR-Cas-System von Haloferax volcanii: CRISPRi und Autoimmunität / Aris-Edda Stachler." Ulm : Universität Ulm, 2017. http://d-nb.info/1140118145/34.

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5

Vyhovskyi, Danylo. "In vivo studies of CRISPR adaptation mechanism and specificity." Electronic Thesis or Diss., Sorbonne université, 2023. http://www.theses.fr/2023SORUS729.

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Cette thèse examine les mécanismes de l'immunité adaptative CRISPR-Cas chez les procaryotes, en utilisant principalement le système de type I-E chez Escherichia coli, en se concentrant sur le processus d'acquisition de spacers et la spécificité du système. Elle éclaire la dynamique de génération de spacers et leur intégration dans les CRISPR-arrays, en comparant les modes d'adaptation naïve et primed. L'étude révèle qu'une séquence proximale à un PAM (protospacer adjacent motif) particulier entrave l'acquisition de spacers en mode primed, fournissant un identifiant distinct pour les spacers ac
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6

Cullot, Grégoire. "Génotoxicité des systèmes CRISPR-Cas9." Thesis, Bordeaux, 2019. http://www.theses.fr/2019BORD0344.

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La thérapie génique est une stratégie thérapeutique prometteuse pour le traitement des maladies monogéniques. Si les premières approches, dites additives, ont reposées sur l’utilisation de vecteurs viraux, une part grandissante se tourne désormais vers l’édition génique. Celle-ci est permise par la mise au point de nouvelles générations d’endonucléases, et en particulier le système CRISPR-Cas9. Moins d’une décennie après sa caractérisation, le système CRISPR-Cas9 a permis de faire passer l’édition génique à un stade clinique. Toutefois, dans le même laps de temps, plusieurs interrogations ont
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7

Chew, Wei Leong. "Postnatal Genome Editing With CRISPR." Thesis, Harvard University, 2016. http://nrs.harvard.edu/urn-3:HUL.InstRepos:33493352.

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Targeted genome editing holds tremendous promise for permanent correction of many genetic diseases. The recently developed CRISPR/Cas9 genome-editing tool exhibits facile programmability and robust gene-editing efficiency, and has been applied in cell cultures and animal tissues. However, multi-organ gene-editing in live mammals has not been examined or achieved. This study demonstrates genetic modification in multiple organs of postnatal mice by systemic delivery of CRISPR with adeno-associated viruses (AAVs). I resolved the AAV payload limitation by splitting Cas9 and reconstituting the nati
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8

Medvedeva, Sofia. "Natural Diversity of CRISPR Spacers." Electronic Thesis or Diss., Sorbonne université, 2019. http://www.theses.fr/2019SORUS538.

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Le système CRISPR-Cas est un système immunitaire procaryote de type interférence ARN dirigé contre des éléments génétiques mobiles, tels que les virus et les plasmides. Le système consiste en un ou plusieurs loci CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats ; courtes répétitions palindromiques groupées et régulièrement espacées) associés à des protéines Cas (CRISPR-associated proteins) dont ils sont séparés par une séquence dite leader. Toutes les protéines Cas peuvent être fonctionnellement attribuées à des modules d'adaptation, d'expression et d'interférence. L’analyse d
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9

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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10

ageely, Eman. "Chemical Tools for Potential Therapeutic Applications of CRISPR Systems." OpenSIUC, 2020. https://opensiuc.lib.siu.edu/dissertations/1831.

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Clustered regularly interspaced short palindromic repeats (CRISPR) are derived from a bacterial and archaeal adaptive immune system. The core enzymes of CRISPR are RNA-guided endonucleases that sequence-specifically cleave foreign double-stranded DNA. Improving and controling the properties of the CRISPR system is a crucial step in advancing the therapeutic potential of CRISPR technology. Several classes of these enzymes exist and are being adapted for biotechnology, such as genome engineering. Cas12a (Cpf1) is a Type V CRISPR-associated (Cas) enzyme that naturally uses only one guide RNA, in
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11

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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12

Reeks, Judith. "Structural studies of CRISPR-associated proteins." Thesis, University of St Andrews, 2013. http://hdl.handle.net/10023/3965.

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Clustered regularly interspaced short palindromic repeats (CRISPRs) act to prevent viral infection and horizontal gene transfer in prokaryotes. The genomic CRISPR array contains short sequences (“spacers”) that are derived from foreign genetic elements. The CRISPR array is transcribed and processed into CRISPR RNAs (crRNAs) used in the sequence-specific degradation of foreign nucleic acids. This process is called interference and is mediated by CRISPR-associated (Cas) proteins. This thesis has focused on the structural and functional characterisation of four Cas proteins from the CRISPR/Cas sy
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13

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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14

Sánchez-Rivera, Francisco J. (Francisco Javier). "Constructing and deconstructing cancer using CRISPR-Cas9." Thesis, Massachusetts Institute of Technology, 2016. http://hdl.handle.net/1721.1/103164.

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Thesis: Ph. D., Massachusetts Institute of Technology, Department of Biology, 2016.<br>This electronic version was submitted by the student author. The certified thesis is available in the Institute Archives and Special Collections.<br>Cataloged from student-submitted PDF version of thesis. Vita.<br>Includes bibliographical references.<br>Cancer is a genetic disease that arises through the sequential acquisition of genetic and epigenetic alterations in oncogenes and tumor suppressor genes. Large-scale efforts to re-sequence protein-coding genes from human cancer cell lines and tumor biopsies
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15

Sokolowski, Richard D. "CRISPR RNA biogenesis by a Cas6 nuclease." Thesis, University of St Andrews, 2015. http://hdl.handle.net/10023/6861.

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Clustered regularly interspaced short palindromic repeats (CRISPRs) and associated (Cas) proteins form the basis of a prokaryotic adaptive immune system. Acquired sections of viral DNA are stored within the host genome as ‘spacers' flanked by ‘repeat' sequences. The CRISPR arrays are transcribed and processed to release mature CRISPR RNAs (crRNAs) – containing a single, intact spacer sequence – that are used by effector complexes to base-pair with matching hostile genetic elements and silence future infections. crRNA-biogenesis is thus an essential step within the defence pathway. Within Type
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16

Junttila, A. (Aino). "CRISPR/Cas9-geenieditointitekniikka HIV-1-infektioiden hoidossa." Bachelor's thesis, University of Oulu, 2018. http://urn.fi/URN:NBN:fi:oulu-201804281568.

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HI-viruksen aiheuttama AIDS on ollut 1980-luvulta asti globaalisti merkittävä uhka terveydelle. Vielä nykyäänkin AIDS-liitännäisiin sairauksiin kuolee vuosittain tuhansia ihmisiä, eikä täydellistä parannuskeinoa ole onnistuttu löytämään. Uusi CRISPR/Cas9-geenieditointitekniikka voisi olla tähän ratkaisu, sillä sen soveltuvuutta HIV-1-infektioiden hoidossa on tutkittu ahkerasti positiivisin tuloksin. CRISPR/Cas9 on alunperin bakteerisoluissa adaptiivisen immuunijärjestelmän tavoin toimiva tekniikka, joka on viime aikoina yleistynyt geeniteknologian parissa nopeasti. Tekniikkana CRISPR/Cas9 on e
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17

Lee, Jooyoung. "Anti-CRISPR Proteins: Applications in Genome Engineering." eScholarship@UMMS, 2020. https://escholarship.umassmed.edu/gsbs_diss/1091.

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Clustered, regularly interspaced, short palindromic repeats and CRISPR-associated proteins (CRISPR-Cas) constitute a bacterial and archaeal adaptive immune system. The ongoing arms race between prokaryotic hosts and their invaders such as phages led to the emergence of anti-CRISPR proteins as countermeasures against the potent antiviral defense. Since the first examples of anti-CRISPRs were shown in a subset of CRISPR-Cas systems, we endeavored to uncover these naturally-occurring inhibitors that inactivate different types of CRISPR-Cas systems. In the first part of my thesis, we have identifi
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18

Chen, Pin-Yi. "Resource competition in CRISPR-mediated gene regulation." Thesis, Massachusetts Institute of Technology, 2020. https://hdl.handle.net/1721.1/127156.

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Thesis: S.M., Massachusetts Institute of Technology, Department of Mechanical Engineering, May, 2020<br>Thesis: S.M., Massachusetts Institute of Technology, Department of Electrical Engineering and Computer Science, May, 2020<br>Cataloged from the official PDF of thesis.<br>Includes bibliographical references (pages 77-79).<br>CRISPR-mediated gene regulation is known for its ability to control multiple targets simultaneously due to its modular nature: the same dCas9 effector can target different genes simply by changing the associated gRNA. However, multiplexing requires the sharing of limited
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19

Maikova, Anna. "The CRISPR-Cas system of human pathogen Clostridium difficile : function and regulation." Thesis, Université de Paris (2019-....), 2019. http://www.theses.fr/2019UNIP7091.

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Clostridium difficile (nouveau nom Clostridioides difficile) est une bactérie à Gram-positif, sporulante, anaérobie stricte, présente dans le sol et les environnements aquatiques, ainsi que dans le tractus intestinal des mammifères. C. difficile est l’un des principaux clostridies pathogènes. Cette bactérie est devenue un vrai problème de santé publique associé à l'antibiothérapie dans les pays industrialisés. La diarrhée associée à C. difficile est actuellement la diarrhée nosocomiale la plus fréquente en Europe et dans le monde. Depuis la dernière décennie, la proportion de formes d’infectio
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20

Garneau, Josiane. "Caractérisation du système CRISPR-CAS chez Streptococcus thermophilus." Thesis, Université Laval, 2009. http://www.theses.ulaval.ca/2009/26441/26441.pdf.

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21

Ng, Sheng Rong. "CRISPR-mediated interrogation of small cell lung cancer." Thesis, Massachusetts Institute of Technology, 2018. http://hdl.handle.net/1721.1/117782.

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Thesis: Ph. D., Massachusetts Institute of Technology, Department of Biology, 2018.<br>This electronic version was submitted by the student author. The certified thesis is available in the Institute Archives and Special Collections.<br>Cataloged student-submitted from PDF version of thesis. Vita.<br>Includes bibliographical references.<br>Small cell lung cancer (SCLC) is a highly aggressive neuroendocrine lung carcinoma that remains among the most lethal of solid tumor malignancies. Despite decades of research, treatment outcomes for SCLC remain very poor, highlighting the need for novel appr
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22

Dufour, Josiane. "CRISPR/Cas9 pour traiter la maladie de Huntington." Master's thesis, Université Laval, 2020. http://hdl.handle.net/20.500.11794/66699.

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La maladie de Huntington (MH) est une maladie neurodégénérative caractérisée par plusieurs symptômes moteurs, cognitifs et psychiatriques, causés par une expansion du trinucléotide CAG présent dans le gène huntingtine. L’expression de la protéine correspondante mène éventuellement à un dysfonctionnement et une mort cellulaire. OBJECTIFS: À ce jour, il n’existe pas de thérapies efficaces afin de traiter ou ralentir la MH, mais les essais actuels suggèrent qu’une réduction de la quantité de protéines mutantes pourrait être bénéfique. Dans cette étude, il a été question d’utiliser le système CRIS
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23

Wagner, Dimitrios Laurin [Verfasser]. "CRISPR-Cas9 and T cells / Dimitrios Laurin Wagner." Berlin : Medizinische Fakultät Charité - Universitätsmedizin Berlin, 2021. http://d-nb.info/1234984946/34.

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24

Eckerbert, My. "CRISPR i cancerimmunologin : Kliniska prövningar, utmaningar och framtid." Thesis, Linnéuniversitetet, Institutionen för kemi och biomedicin (KOB), 2019. http://urn.kb.se/resolve?urn=urn:nbn:se:lnu:diva-85390.

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Att förstå olika tumörers biologi är en viktig förutsättning för att kunna utveckla nya cancerbehandlingsmetoder. Ett nytt verktyg inom cancerterapin, både för att förstå tumörers uppkomst samt hitta nya läkemedelsmål och behandlingar, är det mycket potenta genredigeringsverktyget Clustered Regularly Interspaced Short Palindromic Repeats med CRISPR-Associerade proteiner, CRISPR-Cas9. Det är ett adaptivt immunförsvar funnet hos prokaryoter. CRISPR är ett programmerbart RNA-guidat system som har DNA som mål. Tekniken går att tillämpa inom cancerimmunologin genom att t ex man
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25

Jakimo, Noah Michael. "Precise and expansive genomic positioning for CRISPR edits." Thesis, Massachusetts Institute of Technology, 2019. https://hdl.handle.net/1721.1/123626.

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Thesis: Ph. D., Massachusetts Institute of Technology, School of Architecture and Planning, Program in Media Arts and Sciences, 2019<br>Cataloged from PDF version of thesis.<br>Includes bibliographical references (pages 91-105).<br>The recent harnessing of microbial adaptive immune systems, known as CRISPR, has enabled genome-wide engineering across all domains of life. A new generation of gene-editing tools has been fashioned from the natural DNA/RNA-targeting ability of certain CRISPR-associated (Cas) proteins and their guide RNA, which work together to recognize and defend against infectiou
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Ambrosini, Chiara. "Translational modulation through CRISPR-Cas-mediated genome editing." Doctoral thesis, Università degli studi di Trento, 2021. http://hdl.handle.net/11572/323819.

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More than 300 human conditions, ranging from cancer predisposition to developmental and neurological mendelian disorders, are caused by haploinsufficiency (HI), a genetic condition by which mutational inactivation of a single allele leads to reduced protein levels and is enough to produce the disease phenotype. Therefore, translational enhancement of the spare allele could exert a therapeutic effect. Here we propose a novel approach for the potential rescue of haploinsufficiency disease loci based on the insertion of specific single nucleotide changes in the Kozak sequence. Since this sequenc
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BARILLA', CRISTINA. "imprinting disease modelling by iPSCs and CRISPR/Cas9." Doctoral thesis, Università degli studi di Pavia, 2018. http://hdl.handle.net/11571/1214798.

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Chromosomal rearrangement occurs in 1.8% of pregnancies (Forabosco A et al., 2009) and often lead to pregnancy interruption or early death of the newborn. The Kagami-Ogata syndrome is caused by rearrangements of a region on the maternal chromosome 14 that contains a cluster of genes regulated by imprinting mechanism. The Kagami-Ogata syndrome is often characterized by a severe phenotype that can, in some cases, cause the death of the patient in early ages. Even though the genes involved in the rearranged region are known, the molecular mechanisms related with the Kagami-Ogata syndrome have not
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28

Lacombe, Laurie. "CRISPR-Cas9-based strategies for enhanced targeted integration." Electronic Thesis or Diss., université Paris-Saclay, 2024. http://www.theses.fr/2024UPASL047.

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La transplantation de cellules souches et progénitrices hématopoïétiques (CSPH) corrigées autologues est une stratégie prometteuse pour traiter les troubles génétiques sanguins, immunitaires et métaboliques en raison de leur capacité d'autorenouvellement et de différenciation en cellules sanguines variées. Une des méthodes les plus utilisées pour modifier les CSPH repose sur les outils d'édition de gènes comme les nucléases CRISPR/Cas9. L'intégration ciblée via CRISPR-Cas9 permet l'insertion précise de séquences thérapeutiques, offrant une source durable de cellules corrigées La cassure double
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Bouchareb, Mohamed Amine. "Identification de nouveaux miARNs ovariens et analyse fonctionnelle de mir202 chez le médaka (Oryzias latipes)." Thesis, Rennes 1, 2016. http://www.theses.fr/2016REN1B027.

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Les microARNs (miARNs), petits ARNs non codants, sont des fins régulateurs de l’expression des gènes. Les miARNs jouent des rôles essentiels dans les processus biologiques physiologiques mais aussi pathologiques. Cependant, leurs rôles durant l’ovogenèse chez les vertébrés demeurent peu documentés. D’une manière similaire aux gènes ovocytes-spécifiques, nous avons proposé l’hypothèse que les miARNs ovaire-prédominants joueraient des rôles essentiels durant l’ovogenèse et/ou le développement embryonnaire précoce. L’objectif de ma thèse était, dans un premier temps, d’identifier les miARNs ovair
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Bernheim, Aude. "The distribution of CRISPR-Cas systems is affected by interactions with DNA repair pathways." Thesis, Sorbonne Paris Cité, 2017. http://www.theses.fr/2017USPCB070/document.

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Les systèmes CRISPR-Cas confèrent aux bactéries une immunité adaptative contre les éléments génétiques mobiles jouant ainsi un rôle important dans l’évolution bactérienne. Cependant, moins de la moitié des génomes bactériens encodent des systèmes CRISPR-Cas ; cela, malgré la protection qu’ils confèrent et leur haut taux de transfert horizontal. Des hypothèses telles que le coût des phénomènes d’auto-immunité ou de posséder des défenses adaptatives plutôt qu’innées ont été mises en avant pour expliquer ce paradoxe. Je propose une nouvelle hypothèse complémentaire : le contexte génétique jouerai
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Parrot, Camila. "Création d'un système rapporteur pour l'étude de mutations de p53." Thesis, Bordeaux, 2016. http://www.theses.fr/2016BORD0198.

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Le cancer est responsable de plus de 15% des décès. L’activation d’oncogènes et l’inactivation de gènes suppresseurs de tumeur contribuent à la transformation des cellules. Dans 50% des cas de cancers le gène TP53 est muté. C’est pourquoi comprendre les conséquences de ces mutations est indispensable pour développer des tests permettant de cibler p53 dans le cadre de thérapies. Dans cette étude nous avons utilisé la nouvelle technique de modification de génomes, CRISPR-Cas9. Cette technique a été utilisée dans le but d’introduire des mutations spécifiques de TP53 dans le génome de fibroblastes
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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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33

KARTJE, ZACHARY. "GUIDE RNA MODIFICATION AND STRUCTURE-FUNCTION OF CRISPR-CAS9." OpenSIUC, 2018. https://opensiuc.lib.siu.edu/dissertations/1610.

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CRISPR (clustered regularly interspaced short palindromic repeats)-guided nucleases such as Cas9 remain atop the most exciting biological systems in gene editing and therapeutic potential. The modern adaptation of the Streptococcus pyogenes (Spy) Cas9 machinery for directed DNA editing relies on two major components; a Cas9 protein, and an RNA guide. This study aims at modification of the guide RNA to probe structural requirements and modification tolerance while studying the biochemical properties of the ribonucleoprotein complex. We find that the SpyCas9-RNP is not only capable of heavy subs
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34

Chatterjee, Pranam. "A novel CRISPR-Cas9 platform with divergent targeting capabilities." Thesis, Massachusetts Institute of Technology, 2018. http://hdl.handle.net/1721.1/119080.

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Thesis: S.M., Massachusetts Institute of Technology, School of Architecture and Planning, Program in Media Arts and Sciences, 2018.<br>Cataloged from PDF version of thesis.<br>Includes bibliographical references (pages 47-49).<br>RNA-guided DNA endonucleases of the CRISPR-Cas system are widely used for genome engineering and thus have numerous applications in a wide variety of fields. The range of sequences that CRISPR endonucleases can recognize, however, is constrained by the need for a specific protospacer adjacent motif (PAM) flanking the target site. In this thesis, we demonstrate the nat
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Ellis, Donald Christian. "Genetic screens in vivo using the CRISPR/Cas9 system." Thesis, Massachusetts Institute of Technology, 2017. http://hdl.handle.net/1721.1/109640.

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Thesis: S.M., Massachusetts Institute of Technology, Department of Biology, 2017.<br>Cataloged from PDF version of thesis.<br>Includes bibliographical references (pages 82-87).<br>An unmet and paramount need in the field of cancer research is to rapidly translate basic biological findings to clinically relevant therapeutics for cancer patients. Recent technological advances have generated many innovative applications to cancer biology and in a short time have yielded a wealth of information about putative vulnerabilities across a range of cancers. The proposed work involves the development of
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Perli, Samuel David. "An integrated CRISPR-Cas toolkit for engineering human cells." Thesis, Massachusetts Institute of Technology, 2015. http://hdl.handle.net/1721.1/99781.

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Thesis: Ph. D., Massachusetts Institute of Technology, Department of Electrical Engineering and Computer Science, 2015.<br>This electronic version was submitted by the student author. The certified thesis is available in the Institute Archives and Special Collections.<br>Cataloged from student-submitted PDF version of thesis.<br>Includes bibliographical references (pages 145-158).<br>Natively functioning Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)-associated (Cas) system is a prokaryotic adaptive immune system that confers resistance to foreign genetic elements includin
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Sousa, Maria Cristina Ferreira de. "Targeted gene editing in Neospora caninum using CRISPR/Cas9." Master's thesis, Universidade de Évora, 2021. http://hdl.handle.net/10174/29205.

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Apicomplexa are amongst the most prevalent and morbidity-causing pathogen agents worldwide, representing serious challenges to animal and public health. Neospora caninum and Besnoitia besnoiti are causing agents of neosporosis and besnoitiosis. Until today, there are no effective treatment options against these parasitosis. Therefore, it is urgent to invest in the development of methods for diagnosis, prevention, control, and treatment against these protozoan pathogens. The present dissertation is divided in two parts. The first part summarizes three assays on drug development, testing the in
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Bai, Haimeng. "Targeting Zebrafish Hair Bundle-Related Genes Using CRISPR/Cas9." Case Western Reserve University School of Graduate Studies / OhioLINK, 2017. http://rave.ohiolink.edu/etdc/view?acc_num=case1497019240515581.

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39

Jäghagen, Linnea. "Deletion av intronsegment med CRISPR/Cas9 i Arabidopsis thaliana." Thesis, Umeå universitet, Institutionen för klinisk mikrobiologi, 2021. http://urn.kb.se/resolve?urn=urn:nbn:se:umu:diva-184731.

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40

Seletsky, Alexandra East. "Molecular mechanisms and applications of RNA targeting CRISPR endonucleases." Thesis, University of California, Berkeley, 2017. http://pqdtopen.proquest.com/#viewpdf?dispub=10279364.

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<p> Evolutionary pressure to protect against phage-induced lysis and rampant horizontal gene transfer has created a wide repertoire of defensive pathways in bacteria. CRISPR-Cas (<u>c</u>lustered <u>r</u>egularly <u> i</u>nterspaced <u>s</u>hort <u>p</u>alindromic <u> r</u>epeats, <u>C</u>RISPR-<u>a</u>ssociated) systems are adaptive immune pathways that use RNA-guided nucleases to direct cleavage of invading nucleic acids. The programmable nature of these enzymes has enabled a revolution for DNA-targeting applications including gene editing, transcriptional control, and genomic imaging. In ad
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41

Maule, Giulia. "Harnessing CRISPR technology for the treatment of cystic fibrosis." Doctoral thesis, Università degli studi di Trento, 2020. http://hdl.handle.net/11572/268920.

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Cystic fibrosis is an autosomal recessive disease caused by mutations in the CFTR gene. A significant number of mutations (~13%) alter the correct splicing of the CFTR gene, causing the transcription of aberrant transcripts resulting in the production of a non-functional CFTR channel. We focus our research on two intronic CF causing mutations, 3272-26A&gt;G and 3849+10kbC&gt;T that create a new acceptor and donor splice site, respectively, generating in the inclusion of intronic portions into the mRNA. We developed a new genome editing approach to permanently correct the abovementioned mutati
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Maule, Giulia. "Harnessing CRISPR technology for the treatment of cystic fibrosis." Doctoral thesis, Università degli studi di Trento, 2020. http://hdl.handle.net/11572/268920.

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Cystic fibrosis is an autosomal recessive disease caused by mutations in the CFTR gene. A significant number of mutations (~13%) alter the correct splicing of the CFTR gene, causing the transcription of aberrant transcripts resulting in the production of a non-functional CFTR channel. We focus our research on two intronic CF causing mutations, 3272-26A&gt;G and 3849+10kbC&gt;T that create a new acceptor and donor splice site, respectively, generating in the inclusion of intronic portions into the mRNA. We developed a new genome editing approach to permanently correct the abovementioned mutati
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43

Tiroille, Victor. "Ingénierie génétique d'organoïdes à l'aide de nanoblades et étude du rôle d'UBTD1 comme modulateur de la force d'adhésion cellulaire dans les organoïdes de prostate." Electronic Thesis or Diss., Université Côte d'Azur, 2021. http://theses.univ-cotedazur.fr/2021COAZ6039.

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Au cours de ma thèse, j'ai travaillé sur le modèle d'organoïde 3D en suivant deux objectifs principaux : i) développer des outils génétiques pour modifier le génome des organoïdes et ii) déchiffrer le rôle de la ubiquitin domain-containing protein 1 (UBTD1) dans le développement des organoïdes de la prostate.L'ingénierie du génome est devenue ces dernières années plus accessible grâce aux endonucléases programmables par ARN telles que le système CRISPR-Cas9. Cependant, l'utilisation de cette technologie d'édition dans des organes synthétiques appelés "organoïdes" reste très inefficace. Ceci es
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Mazed, Fetta. "Etude des mécanismes de résistance aux inhibiteurs de FLT3 dans les leucémies aigues myéloïdes." Thesis, Sorbonne Paris Cité, 2019. http://www.theses.fr/2019USPCC089.

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Les leucémies aiguës myéloïdes (LAM) constituent un groupe hétérogène d’hémopathies malignes résultant de la prolifération clonale d’un progéniteur myéloïde bloqué dans sa différenciation. De pronostic globalement défavorable, dépend de l’âge et de facteurs cytogénétiques et moléculaires. La mutation du gène FLT3 de type duplication en tandem du domaine juxta-membranaire (ITD : internal tandem duplication) est détectée dans 30% des échantillons de LAM, corrèle à une fréquence accrue de rechutes et à un pronostic défavorable. La mutation ITD conduit à une activation dérégulée et constitutive de
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Raas, Quentin. "Inactivation génique des transporteurs ABC peroxysomaux ABCD1 et ABCD2 dans les cellules microgliales BV-2 : étude de la physiopathogenèse de l’adrénoleucodystrophie liée à l’X." Thesis, Bourgogne Franche-Comté, 2018. http://www.theses.fr/2018UBFCI011/document.

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L’adrénoleucodystrophie liée à l’X (X-ALD) est une maladie neurodégénérative sévère caractérisée par une accumulation d’acides gras à très longue chaîne (AGTLC), conséquence d’un défaut de β-oxydation peroxysomale. La maladie est associée à l’absence de la protéine ABCD1, transporteur ABC du peroxysome qui, tout comme son homologue le plus proche, ABCD2, participe à l’import des AGTLC-CoA au sein du peroxysome, l’unique site de leur dégradation par β-oxydation. La compréhension des mécanismes physiopathologiques est aujourd’hui limitée par le manque de modèles expérimentaux pertinents, cellula
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Krossa, Imene. "Étude des mécanismes moléculaires impliqués dans le mélanome oculaire : recherche de cibles thérapeutiques." Electronic Thesis or Diss., Université Côte d'Azur, 2023. http://www.theses.fr/2023COAZ6047.

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Le mélanome uvéal représente le cancer intraoculaire le plus fréquent chez l’adulte. Malgré un traitement réussi des tumeurs primaires par chirurgie ou proton thérapie, les métastases se développent chez 50% des patients principalement au foie. Les mélanomes uvéaux métastatiques sont hautement réfractaires aux thérapies existantes. Seule l’immunothérapie par tebentafusp permet d’augmenter la survie des patients avec un mélanome uvéal métastatique, mais ce traitement est limité aux patients HLA-A*02:01 positifs. Il faut donc trouver un traitement efficace pour la majorité des patients. Au stade
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Gentil, Whisnayder. "Estudos in vitro da inibição da atividade da telomerase de Leishmania major." Botucatu, 2018. http://hdl.handle.net/11449/181150.

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Orientador: Maria Cano<br>Resumo: As leishmanioses são doenças tropicais negligenciadas, causadas por protozoários do gênero Leishmania. Elas são consideradas pela Organização Mundial da Saúde como uma doença endêmica em muitos países, incluindo o Brasil. Assim, a busca por novas metodologias de combate e controle dessas doenças parasitárias se faz necessária. Uma das abordagens refere-se ao estudo da biologia molecular dos parasitos causadores da doença, como o estudo dos telômeros, os quais são repetições em tandem associadas a proteínas e localizadas nas extremidades dos cromossomos da maio
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48

Oliveira, Vanessa Cristina de. "Edição do gene TFAM pela engenharia CRISPR Cas9 em modelo bovino." Universidade de São Paulo, 2016. http://www.teses.usp.br/teses/disponiveis/10/10132/tde-20032017-162120/.

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O fator de transcrição A mitocondrial (TFAM) é um membro da subfamília HMGB que se liga a promotores do DNA mitocondrial (mtDNA). É um gene importante para a manutenção do mtDNA, pois regula o número de cópias e é essencial para inicialização da replicação e transcrição do mtDNA. Recentemente técnicas de edição gênica vêm sendo utilizada como uma ferramenta bastante eficaz na manipulação genômica. A nova tecnologia chamada de CRISPR/ Cas9 (Regulary interspaced clustered short palindromic repeats) utiliza um RNA guia (gRNA) curto que contém 20 nucleotídeos complementares a sequência de DNA. Qua
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Rose, Elaine. "Identifying Mechanisms of Resistance to Oncolytic Virotherapy in Acute Leukemia Through a Genome-wide CRISPR Screen." Thesis, Université d'Ottawa / University of Ottawa, 2018. http://hdl.handle.net/10393/38112.

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Approximately half of all adults diagnosed with acute leukemia (AL) relapse after standard chemotherapy, highlighting the need for alternative treatment options. We have previously shown that vaccination with irradiated autologous tumour cells infected with an oncolytic virus (OV) can elicit a durable, tumour specific, T cell- mediated response in a mouse model of AL. In the context of this AL infected cell vaccine (ICV) model, infection of autologous cells ex vivo with an OV is essential for stimulating a lasting immune response. While the murine AL line L1210 can be robustly infected with Ma
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50

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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