Готові списки джерел за темами / Gene Editing (CRISPR/Cas9)

Добірка наукової літератури з теми "Gene Editing (CRISPR/Cas9)"

Автор: Grafiati
Опубліковано: 7 липня 2024

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Статті в журналах з теми "Gene Editing (CRISPR/Cas9)":

1

Paul Strickland, Skylar. "CRISPR-Cas9: Gene Editing." International Journal of Science and Research (IJSR) 12, no. 6 (June 5, 2023): 2439–42. http://dx.doi.org/10.21275/sr23624231215.

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2

Yang, Jiayi. "Applications of the CRISPR-Cas9 system in cancer models." Theoretical and Natural Science 21, no. 1 (December 20, 2023): 28–33. http://dx.doi.org/10.54254/2753-8818/21/20230804.

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Cancer has a high mortality and prevalence rate in the world. CRISPR-Cas9 is one of the novel and most common gene-editing techniques. Compared with the first two generations of gene-editing technologies, CRISPR-Cas9 system has the advantages of easy design, low cost, high efficiency and so on. sgRNA guides Cas9 to the site of the targeted gene, and Cas9 cuts the DNA strand at that site, triggering the NHEJ or HDR mechanism so as to achieve the purpose of deletion or insertion. CRISPR-Cas9 can be combined with other factors for other purposes, such as CRISPRa, CRISPRi, and base editing. The CRISPR system now has been used extensively for research into biological mechanisms and disease treatments. Since cancer is controlled by genes, a number of researchers in recent years have looked at using the CRISPR system to treat cancer. The CRISPR technology has greatly improved our understanding of cancer and the factors that affect it, and has had a major impact on the study and treatment of cancer. CRISPR gene editing can quickly and efficiently generate gene knockouts and regulate gene expression to identify relevant genes that influence cancer growth. This review systematically introduces CRISPR-Cas9 and its application methods, delivery modes, and discusses some studies using cell lines and organoids in vitro and animal models for cancer therapy in vivo.
3

Isachenko, Nadya, Gayane Aleksanyan, Paul Diehl, and Donato Tedesco. "Abstract 2950: CRISPR/saCas9 and CRISPR/spCas9 systems for combinatorial genetic screens (CRISPR-KO, CRISPRa, CRISPRi)." Cancer Research 84, no. 6_Supplement (March 22, 2024): 2950. http://dx.doi.org/10.1158/1538-7445.am2024-2950.

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Abstract This study explores the utilization of orthogonal CRISPR-based gene editing/modulation systems for combinatorial genetic screens using CRISPR knockout (CRISPR-KO), CRISPR activation (CRISPRa), and CRISPR interference (CRISPRi) functionalities. S. aureus (sa)Cas9 is an alternative nuclease to S. pyogenes (sp)Cas9 in scenarios where the latter cannot be used, or when multiple independent CRISPR systems need to be simultaneously expressed in the same cell. In this study we set out to explore the feasibility of utilizing different combinations of saCas9 and spCas9 CRISPR systems to achieve the simultaneous inactivation (via CRISPR-KO or CRISPRi) and transactivation (via CRISPRa) of different target genes in the same host cell. For this purpose, a complete set of tools for CRISPR/saCas9 gene editing and gene modulation was developed, compatible with CRISPR/spCas9 co-expression. Specifically, we developed and validated optimized saCas9 and sg(sa)RNA lentiviral and AAV vectors, dual expression (sp)/(sa)sgRNA lentiviral library vectors, as well as (sa)CRISPR-KO, (sa)CRISPRi, (sa)CRISPRa fluorescence-based activity kits for the functional validation of saCas9 expressing cell lines. Results demonstrating the feasibility of the orthogonal screens will be presented, with different combinations of CRISPR-KO, CRISPRa, and CRISPRi systems in multiple cell lines. Citation Format: Nadya Isachenko, Gayane Aleksanyan, Paul Diehl, Donato Tedesco. CRISPR/saCas9 and CRISPR/spCas9 systems for combinatorial genetic screens (CRISPR-KO, CRISPRa, CRISPRi) [abstract]. In: Proceedings of the American Association for Cancer Research Annual Meeting 2024; Part 1 (Regular Abstracts); 2024 Apr 5-10; San Diego, CA. Philadelphia (PA): AACR; Cancer Res 2024;84(6_Suppl):Abstract nr 2950.
4

Gong, Chongzhi, Shengchan Huang, Rentao Song, and Weiwei Qi. "Comparative Study between the CRISPR/Cpf1 (Cas12a) and CRISPR/Cas9 Systems for Multiplex Gene Editing in Maize." Agriculture 11, no. 5 (May 10, 2021): 429. http://dx.doi.org/10.3390/agriculture11050429.

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Although the clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated protein 9 (Cas9) system has been proved to be an efficient multiplex gene editing system in maize, it was still unclear how CRISPR/Cpf1 (Cas12a) system would perform for multiplex gene editing in maize. To this end, this study compared the CRISPR/Cpf1 system and CRISPR/Cas9 system for multiplex gene editing in maize. The bZIP transcription factor Opaque2 (O2) was used as the target gene in both systems. We found that in the T0 and T1 generations, the CRISPR/Cpf1 system showed lower editing efficiency than the CRISPR/Cas9 system. However, in the T2 generation, the CRISPR/Cpf1 system generated more types of new mutations. While the CRISPR/Cas9 system tended to edit within the on-target range, the CRISPR/Cpf1 system preferred to edit in between the targets. We also found that in the CRISPR/Cpf1 system, the editing efficiency positively correlated with the expression level of Cpf1. In conclusion, the CRISPR/Cpf1 system offers alternative choices for target-site selection for multiplex gene editing and has acceptable editing efficiency in maize and is a valuable alternative choice for gene editing in crops.
5

Dowdy, Steven F. "Controlling CRISPR-Cas9 Gene Editing." New England Journal of Medicine 381, no. 3 (July 18, 2019): 289–90. http://dx.doi.org/10.1056/nejmcibr1906886.

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6

Wu, Yirui. "The Development of Gene Editing Technology and Controversial Issues: A Discussion." Highlights in Science, Engineering and Technology 91 (April 15, 2024): 123–30. http://dx.doi.org/10.54097/6gj0tk11.

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The fields of genetics and biomedicine stand to benefit greatly from the innovative technique known as gene editing technology, especially with the potential uses of CRISPR-Cas9.the creation of crops resistant to disease, the treatment of genetic illnesses, and the advancement of biomedical research in fields like sickle-cell anemia and rice gene editing are all made possible by CRISPR-Cas9's capacity to precisely pinpoint and edit specific genes. Despite being beneficial to humanity, the quick development of human gene editing technology has also brought up ethical, legal, societal, and technical issues. Its management, informed consent, privacy, and social equality are among the issues that require considerable attention.In an effort to give the scientific community something to think about, this article examines the state of gene editing today, its potential for the future, and the debates surrounding its application. It concentrates on the way the CRISPR/Cas system works, how CRISPER/Cas9 gene editing is used, and the range of problems it has brought up.
7

Yang, Lan, Hao Li, Yao Han, Yingjie Song, Mingchen Wei, Mengya Fang, and Yansong Sun. "CRISPR/Cas9 Gene Editing System Can Alter Gene Expression and Induce DNA Damage Accumulation." Genes 14, no. 4 (March 27, 2023): 806. http://dx.doi.org/10.3390/genes14040806.

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Clustered regularly interspaced short palindromic repeats (CRISPR) and the associated protein (Cas) gene editing can induce P53 activation, large genome fragment deletions, and chromosomal structural variations. Here, gene expression was detected in host cells using transcriptome sequencing following CRISPR/Cas9 gene editing. We found that the gene editing reshaped the gene expression, and the number of differentially expressed genes was correlated with the gene editing efficiency. Moreover, we found that alternative splicing occurred at random sites and that targeting a single site for gene editing may not result in the formation of fusion genes. Further, gene ontology and KEGG enrichment analysis showed that gene editing altered the fundamental biological processes and pathways associated with diseases. Finally, we found that cell growth was not affected; however, the DNA damage response protein—γH2AX—was activated. This study revealed that CRISPR/Cas9 gene editing may induce cancer-related changes and provided basic data for research on the safety risks associated with the use of the CRISPR/Cas9 system.
8

Zhou, Junming, Xinchao Luan, Yixuan Liu, Lixue Wang, Jiaxin Wang, Songnan Yang, Shuying Liu, Jun Zhang, Huijing Liu, and Dan Yao. "Strategies and Methods for Improving the Efficiency of CRISPR/Cas9 Gene Editing in Plant Molecular Breeding." Plants 12, no. 7 (March 28, 2023): 1478. http://dx.doi.org/10.3390/plants12071478.

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Following recent developments and refinement, CRISPR-Cas9 gene-editing technology has become increasingly mature and is being widely used for crop improvement. The application of CRISPR/Cas9 enables the generation of transgene-free genome-edited plants in a short period and has the advantages of simplicity, high efficiency, high specificity, and low production costs, which greatly facilitate the study of gene functions. In plant molecular breeding, the gene-editing efficiency of the CRISPR-Cas9 system has proven to be a key step in influencing the effectiveness of molecular breeding, with improvements in gene-editing efficiency recently becoming a focus of reported scientific research. This review details strategies and methods for improving the efficiency of CRISPR/Cas9 gene editing in plant molecular breeding, including Cas9 variant enzyme engineering, the effect of multiple promoter driven Cas9, and gRNA efficient optimization and expression strategies. It also briefly introduces the optimization strategies of the CRISPR/Cas12a system and the application of BE and PE precision editing. These strategies are beneficial for the further development and optimization of gene editing systems in the field of plant molecular breeding.
9

Desai, Devam, Hiral Panchal, Shivani Patel, and Ketul Nayak. "CRISPR - CAS9 GENE EDITING: A REVIEW." International Journal of Advanced Research 8, no. 10 (October 31, 2020): 1127–32. http://dx.doi.org/10.21474/ijar01/11943.

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CRISPR is an RNA guided genome editing technique of genetic engineering which works like genetic scissors. Based on simplified version of bacterial CRISPR-Cas9 antiviral defense system. It is more accurate, faster and cost efficient than other genome editing methods. There are two components in this system: First component includes a single guide RNA (sgRNA) of system which will identify target sequence in genome and Second component will include Cas9 nuclease of system which will act as a pair of scissors to spilt the double strands of DNA. CRISPR has promising therapeutic applications. This current review focuses on mechanism, therapeutic applications, delivery systems, limitations and different approaches used for gene editing using CRISPR.
10

Preece, Roland, and Christos Georgiadis. "Emerging CRISPR/Cas9 applications for T-cell gene editing." Emerging Topics in Life Sciences 3, no. 3 (April 2, 2019): 261–75. http://dx.doi.org/10.1042/etls20180144.

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Abstract Gene editing tools are being rapidly developed, accelerating many areas of cell and gene therapy research. Each successive gene editing technology promises increased efficacy, improved specificity, reduced manufacturing cost and design complexity; all of which are currently epitomised by the clustered regularly interspaced short palindromic repeats (CRISPR) and CRISPR-associated protein (Cas9) platform. Since its conceptualisation, CRISPR-based gene editing has been applied to existing methodologies and has further allowed the exploration of novel avenues of research. Implementation of CRISPR/Cas9 has been instrumental to recent progress in the treatment of cancer, primary immunodeficiency, and infectious diseases. To this end, T-cell therapies have attempted to harness and redirect antigen recognition function, and through gene editing, broaden T-cell targeting capabilities and enhance their potency. The purpose of this review is to provide insights into emerging applications of CRISPR/Cas9 in T-cell therapies, to briefly address concerns surrounding CRISPR-mediated indel formation, and to introduce CRISPR/Cas9 base editing technologies that hold vast potential for future research and clinical translation.
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Статті в журналах Дисертації Книги

Дисертації з теми "Gene Editing (CRISPR/Cas9)":

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

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 vitro efficacy of selected endochin-like quinolones (ELQs) against B. besnoiti and N. caninum tachyzoites on a 3-day proliferation inhibition assay, long-term experiment with the duration of 20 days, and ultrastructural changes induced by ELQs were evaluated in N. caninum. The second part of the report consists of a monography reviewing the CRISPR/Cas9 gene editing technology applied to a targeted sag1 gene knock-out in N. caninum assay; Resumo: Os parasitas do filo Apicomplexa estão entre os agentes patogénicos causadores de morbilidade mais prevalentes no mundo, representando sérios desafios para a saúde pública e animal. Neospora caninum e Besnoitia besnoiti são agentes etiológicos da neosporose e besnoitiose. Até hoje, não existem opções de tratamento e prevenção disponíveis para estas parasitoses, tornando-se urgente investir no desenvolvimento de métodos para o diagnóstico, prevenção e tratamento destes protozoários. A presente dissertação está dividido em duas partes. A primeira parte relativa a três ensaios focados no desenvolvimento de medicamentos, testa a eficácia in vitro de endoquinas tipo quinolonas contra taquizoítos de B. besnoiti e N. caninum num ensaio inibiçãoproliferação de três dias, numa experiência de tratamento de longo-curso e através de microscopia de transmissão de eletrões para avaliar alterações ultraestruturais. A segunda parte consiste numa monografia sobre a tecnologia de edição genómica CRISPR/Cas9 aplicada ao knock-out do gene sag1 em N. caninum.
3

Cui, Xiucheng. "Targeted Gene Editing Using CRISPR/Cas9 in a Wheat Protoplast System." Thesis, Université d'Ottawa / University of Ottawa, 2017. http://hdl.handle.net/10393/36543.

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The clustered regularly interspaced short palindromic repeats (CRISPR)/Cas9 system has become a promising tool for targeted gene editing in a variety of organisms including plants. In this system, a 20 nt sequence on a single guide RNA (sgRNA) is the only gene-specific information required to modify a target gene. Fusarium head blight (FHB) is a devastating disease in wheat caused by the fungus Fusarium graminearum. The trichothecene it produces, deoxynivalenol (DON), is a major mycotoxin contaminant causing food production loss both in quality and yield. In this project, we used the CRISPR/Cas9 system to modify three wheat genes identified in previous experiments, including an ABC transporter (TaABCC6), and the Nuclear Transcription Factor X box-binding-Like 1 (TaNFXL1), both associated with FHB susceptibility, and a non-specific Lipid Transfer Protein (nsLTP) named TansLTP9.4 which correlates with FHB resistance. Two sgRNAs were designed to target each gene and were shown in an in vitro CRISPR/Cas9 assay to guide the sequence-specific cleavage with high efficiency. Another assay for CRISPR/Cas9 was established by the optimization of a wheat protoplast isolation and transformation system. Using a construct expressing a green fluorescent protein (GFP) as a positive control, estimated transformation efficiencies of about 60% were obtained with different batches of protoplasts. High-throughput sequencing of PCR amplicons from protoplasts transformed with editing constructs clearly showed that the three genes have been successfully edited with efficiencies of up to 42.2%. In addition, we also characterized by RT-qPCR the expression pattern of 10 genes in DON-treated protoplasts; seven of the genes were induced by DON in the protoplasts, consistent with their previously identified DON induction in treated wheat heads, while three genes expressed differentially between DON-treated wheat heads and protoplasts. Preliminary bioinformatics analyses showed that these differentially expressed genes are involved in different plant defense mechanisms.
4

Croci, Susanna. "CRISPR-Cas9 gene editing: a new promising treatment for Rett syndrome." Doctoral thesis, Università di Siena, 2020. http://hdl.handle.net/11365/1120546.

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Rett syndrome (RTT) is a neurodevelopmental disorder affecting the central nervous system and is one of the most common causes of intellectual disability in girls, resulting in severe cognitive and physical disabilities. Mutations in MECP2 and FOXG1 genes cause the classic form and the congenital variant of Rett syndrome, respectively. Both genes are transcriptional regulators and both under- and over-expression of these gene cause disease in humans. To characterize the biological mechanisms implicated in disease pathogenesis, we established and characterized a human neuronal model based on genetic reprogramming of patient fibroblasts into induced Pluripotent Stem Cells (iPSCs). Functional analyses performed in MECP2 iPSC-derived neurons demonstrated that these cells closely mimic the impairment of molecular pathway characterizing the disease revealing defects in GABAergic system and cytoskeleton dynamics. Furthermore, we explored the possibility to use iPSC-derived neurons to develop and study a new treatment for RTT patients. Effective therapies are not currently available and the need for tight regulation of MeCP2 and FOXG1 expression for proper brain functioning makes gene replacement therapy risky. Therefore, gene editing would be much more effective. Gene editing based on CRISPR/Cas9 technology and Homology Directed Repair appears an appealing option for the development of new therapeutic approaches. We have engineered a two-plasmid system to correct FOXG1 (c.688C>T (p(Arg230Cys)); C.765G>A (p.Trp255Ter)) and MECP2 (c.473C>T-p.Thr158Met) variants.. Mutation-specific sgRNAs and donor DNAs have been selected and cloned together with an mCherry/GFP reporter system. Cas9 flanked by sgRNA recognition sequences for auto-cleaving has been cloned in a second plasmid. The system has been designed to be ready for in vivo delivery via Adeno-Associated Viral (AAV) vectors. NGS analysis of corrected cells from MECP2 and FOXG1 patients demonstrated an high editing efficiency, ranging from 20 to 80 % of HDR and confirmed that this correction strategy is feasible in neurons. Functional analyses in edited cells confirm the correction of molecular defects due to the mutation. Based on the use of AAV viruses and their capacity to cross the Blood Brain Barrier (BBB) following intravenous injection these experiments will allow us to demonstrate the full potential of gene editing as a therapeutic option for RTT and for other neurodevelopmental disorders currently lacking an effective treatment.
5

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 été soulevées vis-à-vis de la génotoxicité pouvant être induite par la Cas9. Une littérature émergente pointe le risque de génotoxicité au site ciblé. Le travail de thèse présentée ici s’inscrit dans cette thématique. La première partie de l’étude a eu pour objectif de décrire la génotoxicité induite par une unique cassure double-brin faite par la Cas9. La caractérisation des effets a été faite à la fois à l’échelle nucléotidique, par le suivi de la balance HDR / InDels, mais également à l’échelle du chromosome. Le suivi de l’intégrité chromosomique a permis de mettre en lumière un nouveau risque de génotoxicité encore non-caractérisé. Un système de détection sensible et spécifique de ce risque a été mis au point pour continuer de le caractériser. Le second objectif a été de répondre aux limites soulevées par la génotoxicité non-voulus, en mettant au point une méthode d’édition génique plus sûre et aussi efficace, via l’utilisation d’une unique cassure simple-brin par la Cas9D10A -nickase
Gene therapy is a promising therapeutic strategy for the monogenic diseases treatment. If the first approaches, called additive, have relied on the use of viral vectors, a growing share is now turning to gene editing. Less than a decade after its characterization, the CRISPR-Cas9 system has moved gene editing to a clinical stage. However, in the same period of time, several questions have been raised regarding the genotoxicity that can be induced by Cas9. An emerging literature points to the risk of genotoxicity at the targeted site. The thesis work presented here is part of this theme. The first part of the study aimed to describe the genotoxicity induced by a single double-stranded break made by Cas9. Characterization of the effects was done both at the nucleotide level, by monitoring the HDR / InDels balance, but also at the chromosome scale. The monitoring of chromosomal integrity has brought to light a new risk of genotoxicity that was not characterized. A sensitive and specific detection system for this risk has been developed to further characterize it. The second objective was to address the limitations of unwanted genotoxicity by developing a safer and more efficient gene editing method through the use of a single single-stranded breakage by Cas9D10A-nickase
6

Giada, Beligni. "Application of the CRISPR-Cas9 genome editing approach for the correction of the p.Gly2019Ser (c.6055G>A) LRRK2 variant in Parkinson Disease." Doctoral thesis, Università di Siena, 2022. https://hdl.handle.net/11365/1220257.

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Parkinson’s disease (PD) is one of the most common long-term degenerative disorders that affect the nervous system. Clinical symptoms are bradykinesia, resting tremor and postural imbalance due to the loss of dopaminergic neurons in the substantia nigra pars compacta. Heterozygous mutations in the Leucine Rich Repeat Kinase 2 gene (LRRK2) have been identified both in familial and sporadic cases of PD. The most common variant is the p.Gly2019Ser substitution (c.6055G>A). To date there is no effective treatment available. The genome editing tool CRISPR/Cas9 has recently transformed the field of biotechnology and biomedical discovery, posing the basis for the development of innovative treatments. Using CRISPR/Cas9 technology and Homology Directed Repair, our project aims to validate gene editing as an alternative therapeutic approach for PD through the genetic correction of the pathogenic p.Gly2019Ser LRRK2 mutation restoring the wild-type sequence both in human and mouse models. Specifically, we tested various strategies, based on the CRISPR/Cas9-based genome editing technique, for the correction of LRRK2 p.Gly2019Ser (c.6055G>A) variant in primary mouse and human fibroblasts with promising results. If the correction experiments in in vitro models will confirm the good efficiency of the approach, these experiments will represent a fundamental step for the subsequent evaluation of the potential of gene therapy for the treatment of PD as well as other brain disorders for which no therapy is currently available.
7

Poggi, Lucie. "Gene editing approaches of microsatellite disorders : shortening expanded repeats." Electronic Thesis or Diss., Sorbonne université, 2020. http://www.theses.fr/2020SORUS412.

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Les maladies à triplet sont dues à des expansions de trinucléotides dans l’ADN. Aucun traitement n’existe pour les soigner. Le but de cette thèse est de mettre au point de nouvelles approches de thérapie génique pour supprimer les expansions pathologiques dans le génome humain. Dans une première partie, un système expérimental dans la levure a été construit afin d’évaluer l’efficacité de différentes nucléases associées au système CRISPR sur des microsatellites. La seconde partie est concentrée sur une maladie à triplet en particulier ; la dystrophie myotonique de type 1 (DM1), qui est due à une expansion d’une répétition de triplets CTG dans la région 3’UTR du gène DMPK. Une nucléase, TALENCTG , construite pour induire une cassure double-brin dans les répétitions CTG en 3’UTR du gène DMPK, induit de manière très efficace des contractions de triplets CTG dans la levure. Des événements de contraction ont été observés lorsque cette nucléase est exprimée. Des expériences in vivo dans un modèle de souris contenant un fragment d’ADN génomique humain de patient contenant 1000 CTG ont été menées. Des particules virales AAV recombinantes portant le gène de la TALEN ont été produites. Après injection intramusculaire, les cellules musculaires expriment la nucléase, mais dû à une toxicité ou immunogénicité de la protéine, l’expression est perdue. Enfin, le système mis au point dans la levure a été transposé dans une lignée cellulaire humaine établie, les HEK293FS. Ce système pourra servir à sélectionner des nucléases actives dans les cellules humaines
Microsatellite disorders are a specific class of human diseases that are due to the expansion of repeated sequences above pathological thresholds. These disorders have varying symptoms and pathogenic mechanisms, caused by the expanded repeat. No cure exists for any of these dramatic conditions. This thesis is investigating new gene editing approaches to remove pathological expansions in the human genome. In a first part, a yeast-based screen was constructed to identify potent CRISPR-associated nucleases that can cut these microsatellites. The second part focuses on myotonic dystrophy type 1 (DM1), which is due to and expanded CTG repeat tract located at the 3’UTR of the DMKP gene. A nuclease, TALENCTG was designed to induce a double strand break into the CTG repeats. It was previously shown to be active in yeast cells, inducing contractions of CTG repeats from a DM1 patient integrated into the yeast genome. The TALEN was tested in DM1 patient cells. The nuclease was found to trigger some contraction events in patient cells. In vivo experiments were carried out in a mouse model of myotonic dystrophy type 1 containing a human genomic fragment from a patient and 1000 CTG. Intramuscular injections of recombinant AAV encoding the TALENCTG revealed that the nuclease is toxic and/or immunogenic in muscle cells in the tested experimental conditions. Finally, the reporter assay integrated in yeast to screen nucleases was transposed in HEK293FS cell line. The integrated cassette contains a CTG expansion from a myotonic dystrophy type 1 patient flanked by two halves of GFP genes. This system would enable to find nucleases active in human cells
8

Waghulde, Harshal B. "Mapping and CRISPR/Cas9 Gene Editing for Identifying Novel Genomic Factors Influencing Blood Pressure." University of Toledo Health Science Campus / OhioLINK, 2016. http://rave.ohiolink.edu/etdc/view?acc_num=mco1470402637.

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9

Jayavaradhan, Rajeswari. "Optimization of Gene Editing Approaches for Human Hematopoietic Stem Cells." University of Cincinnati / OhioLINK, 2019. http://rave.ohiolink.edu/etdc/view?acc_num=ucin1543919940219677.

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Ryu, Junghyun. "The direct injection of CRISPR/Cas9 system into porcine zygotes for genetically modified pig production." Diss., Virginia Tech, 2019. http://hdl.handle.net/10919/101763.

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The pig has similar features to the human in aspects such as physiology, immunology, and organ size. Because of these similarities, genetically modified pigs have been generated for xenotransplantation. Also, when using the pig as a model for human diseases (e.g. cystic fibrosis transmembrane conductance regulator), the pig exhibited similar symptoms to those that human patients present. The main goal of this work was to examine the efficacy of direct injection of the CRISPR/Cas9 system (clustered regularly interspaced short palindromic repeats/ CRISPR associated protein 9) in pigs and to overcome shortcomings that resulted after direct injection into the cytoplasm of developing zygotes. By using direct injection of CRISPR/Cas9 into developing zygotes, we successfully generated fetuses and piglets containing 9 different mutations. The total number of aborted fetuses was 20 and of live piglets was 55. Moreover, one issue that was encountered during the production of mutated pigs was that insertion or deletion (indel) mutations did not always introduce a premature stop codon because it did not interfere with the codon read. As a result of these triplet indel(s) mutations, a hypomorphic phenotype was presented; consequently, the mutated gene was partially functional. To prevent this hypomorphic phenotype, we introduced two sgRNAs to generate an intended deletion that would remove a DNA fragment on the genome by causing two double-strand breaks (DSB) during non-homologous end joining (NHEJ). The injection of two sgRNAs successfully generated the intended deletion on the targeted genes in embryos and live piglets. Results after using intended deletions, in IL2RG mutation pigs, did not show hypomorphic phenotypes even when a premature stop codon was not present. After using the intended deletion approach, function of the targeted genes was completely disrupted regardless of the presence or absence of a premature stop codon. Our next aim was to introduce (i.e. knock-in) a portion of exogenous (donor) DNA sequence into a specific locus by utilizing the homology direct repair (HDR) pathway. Because of the cytotoxicity of the linear form of the donor DNA, the concentration of the injected donor DNA was adjusted. After concentration optimization, four different donor DNA fragments targeting four different genes were injected into zygotes. Efficiency of knock-in was an average of 35%. Another donor DNA was used in this study which is IL2RG-IA donor DNA carried 3kb of exogenous cassette. It showed 15.6% of knock-in efficiency. IL2RG-IA Donor DNA injected embryos were transferred into surrogates, and a total of 7 pigs were born from one surrogate, but none of the 7 were positive for the knock-in. Future experiments need to be developed to optimize this approach. Overall, the direct injection of CRISPR/Cas9 is advantageous in cost, time, and efficiency for large animal production and for biomedical research. However, there are still unsolved challenges (off-targeting effects, low efficiency of knock-in, and monoallelic target mutation) that need to be elucidated for future application in humans and other species.
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Книги з теми "Gene Editing (CRISPR/Cas9)":

1

Little, Jamie. Using Genomic Transgenes and the CRISPR/Cas9 Gene Editing System to Understand How Hedgehog Signaling Regulates Costal2 and Cubitus Interruptus in Drosophila melanogaster. [New York, N.Y.?]: [publisher not identified], 2017.

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Luo, Yonglun, ed. CRISPR Gene Editing. New York, NY: Springer New York, 2019. http://dx.doi.org/10.1007/978-1-4939-9170-9.

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María Vaschetto, Luis. CRISPR-/Cas9 Based Genome Editing for Treating Genetic Disorders and Diseases. New York: CRC Press, 2021. http://dx.doi.org/10.1201/9781003088516.

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4

Isaacson, Walter. Code Breaker: Jennifer Doudna, Gene Editing, and the Future of the Human Race. New York, USA: Simon & Schuster, 2021.

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5

Service, Congressional. Advanced Gene Editing: CRISPR-Cas9. Independently Published, 2019.

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6

Kozubek, Jim. Modern Prometheus: Editing the Human Genome with Crispr-Cas9. Cambridge University Press, 2018.

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7

CRISPR/Cas9: Einschneidende Revolution in der Gentechnik. Berlin, Germany: Springer, 2018.

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8

CRISPR/Cas9: Einschneidende Revolution in der Gentechnik. Springer Verlag, 2018.

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9

Yamamoto, Takashi. Targeted Genome Editing Using Site-Specific Nucleases: ZFNs, TALENs, and the CRISPR/Cas9 System. Springer, 2016.

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10

Yamamoto, Takashi. Targeted Genome Editing Using Site-Specific Nucleases: ZFNs, TALENs, and the CRISPR/Cas9 System. Springer, 2015.

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Частини книг з теми "Gene Editing (CRISPR/Cas9)":

1

Gopika, Boro Arthi, Arumugam Vijaya Anand, Natchiappan Senthilkumar, Senthil Kalaiselvi, and Santhanu Krishnapriya. "Gene Editing Using CRISPR/Cas9 System." In CRISPR and Plant Functional Genomics, 258–70. Boca Raton: CRC Press, 2024. http://dx.doi.org/10.1201/9781003387060-15.

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2

Bao, Aili, Lam-Son Phan Tran, and Dong Cao. "CRISPR/Cas9-Based Gene Editing in Soybean." In Legume Genomics, 349–64. New York, NY: Springer US, 2020. http://dx.doi.org/10.1007/978-1-0716-0235-5_19.

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3

García-Caparrós, Pedro. "Breeding for Yield Quality Parameters and Abiotic Stress in Tomato Using Genome Editing." In A Roadmap for Plant Genome Editing, 395–409. Cham: Springer Nature Switzerland, 2023. http://dx.doi.org/10.1007/978-3-031-46150-7_23.

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AbstractGrowing tomatoes is an important aspect of agriculture around the world because of the positive effects it has on people’s health and the economy. Tomato breeders and growers have always been inspired by the market’s insatiable desire for high-yielding and high-quality tomatoes. Crop production, yield, and quality are all negatively affected by abiotic stress, which includes factors like drought, salinity, heat, and cold. As climate change alters weather patterns throughout the world, farmers around the world are increasingly worried about the effects of abiotic stress on their tomato crops. The CRISPR/Cas9 gene-editing tool has attracted attention as an alternative for solving the need for high-yield and superior-quality tomatoes, as well as for managing abiotic stress in tomato plants. This method of gene editing offers new possibilities for the development of stress-tolerant tomato varieties. The present book chapter provides a comprehensive review of the current knowledge on CRISPR/Cas9 and its potential implications in tomato agriculture, with a particular emphasis on enhancing yield quality and conferring resistance to abiotic stresses. The CRISPR/Cas9 technology has the potential to enhance the taste, appearance, and nutritional value of tomatoes by accurately altering the genes responsible for flavor, color, aroma, and nutrition. The previously mentioned condition could end up in the cultivation of tomatoes that exhibit heightened levels of sweetness, as well as elevated concentrations of crucial vitamins, minerals, and antioxidants. The application of CRISPR/Cas9-mediated modifications has the possibility to augment the plant’s capacity to endure abiotic stress conditions through the introduction of genes implicated in different pathways that contribute to enhanced resilience to such challenging surroundings. In conclusion, the use of CRISPR/Cas9 offers an intriguing chance for improving tomato farming through the enhancement of crop quality and yield, as well as the strengthening of tomato plants against adverse abiotic conditions.
4

Ito, Takeshi, Hiroshi Yamatani, Takashi Nobusawa, and Makoto Kusaba. "Development of a CRISPR-Cas9-Based Multiplex Genome-Editing Vector and Stay-Green Lettuce." In Gene Editing in Plants, 405–14. Singapore: Springer Nature Singapore, 2024. http://dx.doi.org/10.1007/978-981-99-8529-6_15.

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5

Erol, Nihal Öztolan. "Soybean Improvement and the Role of Gene Editing." In A Roadmap for Plant Genome Editing, 271–89. Cham: Springer Nature Switzerland, 2023. http://dx.doi.org/10.1007/978-3-031-46150-7_17.

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AbstractSoybean is a major agricultural crop that is used for food, feed, and industrial products. However, soybean production is facing several challenges, including pests, diseases, and environmental factors. In recent years, there has been a growing interest in using gene editing technologies to improve soybean traits. Gene editing technologies offer a promising new approach to improving soybean production and quality.Gene editing technologies can be used to precisely alter the soybean genome. There are a number of different gene editing technologies that can be used to improve soybeans. One of the most commonly used technologies is CRISPR/Cas9, which uses a protein called Cas9 to cut DNA at a specific location. This can be used to insert, delete, or modify genes. Other gene editing technologies include zinc finger nucleases (ZFNs), and transcription activator-like effector nucleases (TALENs). Gene editing technologies have the potential to revolutionize soybean breeding. This can be used to introduce new traits, such as resistance to pests and diseases, or to improve existing traits, such as yield and oil content.The use of gene editing technologies in soybean improvement is still in its early stages, but the potential benefits are significant. Gene editing technologies offer a more precise and efficient way to improve soybean production than traditional breeding methods. They also offer the potential to create new varieties of soybeans that are better able to meet the challenges of a changing world.
6

Reem, Nathan T., and Joyce Van Eck. "Application of CRISPR/Cas9-Mediated Gene Editing in Tomato." In Methods in Molecular Biology, 171–82. New York, NY: Springer New York, 2019. http://dx.doi.org/10.1007/978-1-4939-8991-1_13.

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7

Everman, Jamie L., Cydney Rios, and Max A. Seibold. "Primary Airway Epithelial Cell Gene Editing Using CRISPR-Cas9." In Methods in Molecular Biology, 267–92. New York, NY: Springer New York, 2018. http://dx.doi.org/10.1007/978-1-4939-7471-9_15.

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8

Liao, Xiaofeng, and Liwu Li. "CRISPR-Cas9-Induced Gene Editing in Primary Human Monocytes." In Methods in Molecular Biology, 189–93. New York, NY: Springer US, 2024. http://dx.doi.org/10.1007/978-1-0716-3754-8_15.

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9

Singh, Surender, Roni Chaudhary, Siddhant Chaturvedi, and Siddharth Tiwari. "Deciphering the Role of CRISPR/Cas9 in the Amelioration of Abiotic and Biotic Stress Conditions." In Gene Editing in Plants, 193–226. Singapore: Springer Nature Singapore, 2024. http://dx.doi.org/10.1007/978-981-99-8529-6_8.

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10

Razzaq, Ali, Ghulam Mustafa, Muhammad Amjad Ali, Muhammad Sarwar Khan, and Faiz Ahmad Joyia. "CRISPR-mediated genome editing in maize for improved abiotic stress tolerance." In Molecular breeding in wheat, maize and sorghum: strategies for improving abiotic stress tolerance and yield, 405–20. Wallingford: CABI, 2021. http://dx.doi.org/10.1079/9781789245431.0023.

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Abstract This chapter discusses the applications of CRISPR-mediated genome editing to improve the abiotic stress tolerance (such as drought, heat, waterlogging and cold tolerance) of maize. CRISPR/Cas9 has great potential for maize genome manipulation at desired sites. By using CRISPR/Cas9-mediated genome editing, numerous genes can be targeted to produce elite maize cultivars that minimize the challenges of abiotic stresses. In the future, more precise and accurate variants of the CRISPR/Cas9 toolbox are expected to be used for maize yield improvement.

Тези доповідей конференцій з теми "Gene Editing (CRISPR/Cas9)":

1

Stacey, Minviluz. "Utility of CRISPR/Cas in accelerating gene discovery in soybean." In 2022 AOCS Annual Meeting & Expo. American Oil Chemists' Society (AOCS), 2022. http://dx.doi.org/10.21748/rzne1660.

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The use of CRISPR/Cas9 has been successfully applied in various plant species to induce targeted genome editing, including soybean. Soybean is recalcitrant to transformation and thus, plants with stable CRISPR gene edits are costly and take a long time to produce. Moreover, soybean is allotetraploid and editing paralogous genes are often necessary to obtain observable phenotype(s). For each gene target, we designed two gRNAs to increase editing efficiency and allow rapid genotyping by PCR. We also tested the CRISPR reagents in transient hairy root transformation to determine if the Cas9 and gRNAs would perform properly in transgenic soybean plants. Our results showed that we can indeed obtain highly efficient, cost-effective CRISPR/Cas editing in soybean to generate novel genotypes for gene discovery and downstream field propagation and breeding efforts. Examples of CRISPR-edited genes and their associated seed traits will be discussed.
2

Wang, Chihan. "Applications of CRISPR/Cas9 gene-editing technology in cancer." In Third International Conference on Biological Engineering and Medical Science (ICBioMed2023), edited by Alan Wang. SPIE, 2024. http://dx.doi.org/10.1117/12.3013218.

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3

Zhiyang, Gan. "Applications and challenges for CRISPR/Cas9-mediated gene editing." In 7TH INTERNATIONAL CONFERENCE ON MATHEMATICS: PURE, APPLIED AND COMPUTATION: Mathematics of Quantum Computing. AIP Publishing, 2022. http://dx.doi.org/10.1063/5.0115407.

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4

Li, Ling. "CRISPR/Cas9-based editing of OsNF-YC4/GmNF-YC4 promoter yields high-protein crops." In 2022 AOCS Annual Meeting & Expo. American Oil Chemists' Society (AOCS), 2022. http://dx.doi.org/10.21748/qsgt8379.

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Genome editing is a new breeding technology widely touted for transgene-free crop improvement; however, to date, the majority of derived traits are created through gene knockout. We describe a novel approach using gene editing to upregulate gene expression by removing negative repressor binding motifs. Our previous work demonstrated that ectopic expression of NF-YC4 increases protein content of leaves and seeds at the expense of carbohydrates. We detected several conserved motifs predicted to be bound by repressors in the promoter of rice and soybean NF-YC4 genes. Using CRISPR/Cas9 to edit the promoters of rice and soybean NF-YC4 genes, we deleted promoter fragments harboring repressor binding motifs. Those deletions resulted in decreased repressor binding, increased NF-YC4 expression, increased protein and decreased carbohydrates. Gene-edited plants showed up to 48% higher leaf protein and 15% increased seed protein. Moreover, we exemplify a general approach for upregulating gene expression through targeted genomic deletions.
5

Cheng, Qiming. "The application of CRISPR/Cas9 technology in plant gene editing." In Third International Conference on Biological Engineering and Medical Science (ICBioMed2023), edited by Alan Wang. SPIE, 2024. http://dx.doi.org/10.1117/12.3012857.

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6

Kershanskaya, O. I., Zh Kuli, A. Maulenbay, D. Nelidova, S. N. Nelidov, and J. Stephens. "NEW CRISPR/CAS9 GENE EDITING TECHNOLOGY FOR DEVELOPMENT OF AGRICULTURAL BIOTECHNOLOGY." In The All-Russian Scientific Conference with International Participation and Schools of Young Scientists "Mechanisms of resistance of plants and microorganisms to unfavorable environmental". SIPPB SB RAS, 2018. http://dx.doi.org/10.31255/978-5-94797-319-8-1434-1437.

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7

Murillo, Alvaro, Meghan Larin, Emma L. Randall, Alysha Taylor, Mariah Lelos, and Vincent Dion. "I05 CRISPR-Cas9 nickase-mediated gene editing to treat Huntington’s disease." In EHDN 2022 Plenary Meeting, Bologna, Italy, Abstracts. BMJ Publishing Group Ltd, 2022. http://dx.doi.org/10.1136/jnnp-2022-ehdn.231.

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8

Li, Xi, Wanbing Tang, Chenjie Zhou, Yulin Yang, Zhengang Peng, Wenrong Zhou, Qunsheng Ji, and Yong Cang. "Abstract 785: Application of CRISPR/Cas9 gene editing to primary T cells." In Proceedings: AACR Annual Meeting 2018; April 14-18, 2018; Chicago, IL. American Association for Cancer Research, 2018. http://dx.doi.org/10.1158/1538-7445.am2018-785.

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9

Zuckermann, Marc, Britta Ismer, Volker Hovestadt, Christiane B. Knobbe-Thomsen, Marc Zapatka, Paul A. Northcott, Martine F. Roussel, et al. "Abstract 5109: Somatic CRISPR/Cas9-mediated gene editing enables versatile brain tumor modeling." In Proceedings: AACR Annual Meeting 2018; April 14-18, 2018; Chicago, IL. American Association for Cancer Research, 2018. http://dx.doi.org/10.1158/1538-7445.am2018-5109.

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Sagawa, Cintia. "Identification of HLB Susceptibility Genes in a Citrus Population Generated Using Multiplexed CRISPR/Cas9 Gene Editing." In IS-MPMI Congress. IS-MPMI, 2023. http://dx.doi.org/10.1094/ismpmi-2023-6.

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Citrus greening or Huanglongbing (HLB) disease is caused by the bacteria Candidatus Liberibacter spp. which is a major threat to the citrus industry worldwide. This has led to the generation of a large number of genomic databases characterizing HLB at the molecular level. Based on the genomic information of 71 published transcriptomic and proteomic datasets we selected 1,200 potential susceptibility genes associated with HLB in citrus. By using CRISPR/Cas9 technology combined with a multiplex approach, we generated 300 constructs targeting a combination of four of those genes in each construct. We currently have a population of over 3,000 transformed lines of Carrizo, a commercial rootstock hybrid (Citrus sinensis 'Washington' sweet orange X Poncirus trifoliata) and population of Valencia sweet orange lines with a diversified combination of mutations. To aid in genotyping, our group is performing a whole genome sequencing of the hybrid Carrizo. Biallelic/homozygous mutants were confirmed by Sanger sequencing of target sites and alignment with respective genomes. Confirmed mutants are currently being tested for bacterial resistance. Identification and stacking of susceptibility gene mutations will be valuable in developing tolerance to HLB and these mutations can subsequently be introduced into other economically significant citrus cultivars and evaluated in the field.

Звіти організацій з теми "Gene Editing (CRISPR/Cas9)":

1

Young, Erin, Cem Kuscu, Christine Watkins, and Murat Dogan. Using CRISPR Gene Editing to Prevent Accumulation of Lipids in Hepatocytes. University of Tennessee Health Science Center, January 2022. http://dx.doi.org/10.21007/com.lsp.2022.0007.

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CRISPR gene editing is a molecular technology that can be used to silence gene expression. In this experiment, genes that are known to play a role in lipid accumulation in hepatocytes were targeted. Specifically, levels of fatty acid transport proteins 2 and 5 (FATP2 & 5) have been shown to be elevated in cases of non-alcoholic fatty liver disease. The goal of this experiment was to reduce expression of these genes by using a dead Cas9 (dCas9) protein with an attached inhibitory domain (KRAB) that acts on the promotor region. When measuring the mRNA expression, it was determined that the levels of the CRISPR-modified gene products were significantly reduced compared to the control. However, the same extent of inhibition was not consistently observed when conducting flow cytometry. Current work is aimed at discovering why lipid accumulation is not inhibited to the expected degree based on the results of mRNA expression.
2

Morin, S., L. L. Walling, Peter W. Atkinson, J. Li, and B. E. Tabashnik. ets for CRISPR/Cas9-mediated gene drive in Bemisia tabaci. Israel: United States-Israel Binational Agricultural Research and Development Fund, 2021. http://dx.doi.org/10.32747/2021.8134170.bard.

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The goal of our BARD proposal was to build both the necessary infrastructure and knowledge for using the CRISPR/Cas9-based gene drive system to control the whitefly Bemisia tabaci. Our research focused on achieving three main goals: (1) establishing a CRISPR/Cas9 gene-editing system for producing genetically-edited B. tabaci; (2) generating and testing CRISPR/Cas9-mediated mutations targeting genes that represent two gene drive strategies: population replacement and population suppression; (3) using computer modeling to optimize strategies for applying CRISPR/Cas9 to control B. tabaci populations in the field. CRISPR gene drive is one of the most promising strategies for diminishing the negative impacts of harmful insects. This technique can introduce mutations into wild populations of pests that reduce their ability to cause damage, reduce their population size, or both. In principle, this can be selfsustaining because mutations carried by relatively few insects can increase in frequency and spread quickly throughout wild populations. Because of this sustainability and the potential benefits to society, agricultural gene-drive systems are most likely to be funded by government agencies, foundations, and grower associations; as with sterile insect releases and most biocontrol programs. Although gene drives have received intensive study in Drosophila and mosquito vectors of human disease, we were one of the first teams pursuing this approach for crop pests. Our project was also one of the first to address CRISPR gene drive in the Hemiptera, an insect order that includes hundreds of pest species. We focused on developing and implementing CRISPR gene drive to reduce the massive damage caused by B. tabaci. This haplodiploid insect is one of the world's most devastating crop pests. Whereas extensive work by others explored CRISPR in diploid species, our project pioneered application of this revolutionary technology to haplodiploids, which have a distinct system of inheritance that presents special challenges and opportunities. Our project has achieved several breakthroughs, including publication of the first paper analyzing CRISPR gene drive in haplodiploids (Li et al. 2020, see next section). Our modeling results from this landmark study demonstrate that CRISPR gene drive can work in haplodiploids, especially if fitness costs associated with the driver allele are low or nil. Our paper was the first to provide a conceptual framework for evaluating and optimizing CRISPR gene drive strategies for managing B. tabaci and other haplodiploid pests. Our breakthroughs in the laboratory have created the infrastructure needed to develop CRISPR for controlling B. tabaci. We established a microinjection system enabling us to introduce CRISPR-derived mutations into B. tabaci embryos. We have used this system to generate and track inherited eye-color mutants of B. tabaci. We have identified and cloned germline promoters, and demonstrated their function in transgenic B. tabaci embryos and other hemipteran insects. We have also developed a tool to easily identify B. tabaci harboring CRISPR-mediated mutations by tagging target genes using a transgenic fluorescent marker. The successful completion of our project provides all the knowledge and infrastructure essential for developing a novel genetic approach for B. tabaci control, which can serve as a non-chemical "green" alternative for managing this global pest. We predict that our discoveries will accelerate the development of the CRISPR gene drive technique for reducing the numbers of this pest and the damage it causes. Still, realization of the benefits of gene-drive technology for pest control will require sustained attention to potential environmental and societal impacts, as well as regulatory and implementation challenges. Given the great promise of this technology and the urgent need for better control methods, we expect that guidance documents and regulations will be in place to allow the scientific community to safely move gene drives for pest control from the laboratory to field trials.
3

Zarate, Sebastian, Ilaria Cimadori, Maria Mercedes Roca, Michael S. Jones, and Katie Barnhill-Dilling. Assessment of the Regulatory and Institutional Framework for Agricultural Gene Editing via CRISPR-based Technologies in Latin America and the Caribbean. Inter-American Development Bank, May 2023. http://dx.doi.org/10.18235/0004904.

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Genome editing tools have promised tremendous opportunities in agriculture for breeding crops and livestock across the food supply chain. Potentially addressing issues associated with a growing global population, sustainability concerns, and possibly helping address the effects of climate change (Kuiken, Barrangou, and Grieger 2021). These promises come alongside environmental, cultural, and socio-economic risks. Including concerns that governance systems are not keeping pace with technological developments and are ill-equipped, or not well suited to evaluate risks new genome editing tools may introduce. Understanding these complex, dynamic interactions across the LAC region is important to inform appropriate and acceptable regional governance and investment strategies. The power and promise of gene editing, CRISPR specifically, were first realized with the discovery of CRISPR loci in the 1980s (Anzalone, Koblan, and Liu 2020). Since that time, CRISPR-Cas systems have been further developed enabling genome editing in virtually all organisms across the tree of life (Anzalone, Koblan, and Liu 2020). Gene editing is not a singular technology or technique; it refers most often to a set of techniques that enable the manipulation of a genome with greater precision than previous iterations of genetic engineering (Shukla-Jones, Friedrichs, and Winickoff 2018b). The Inter-American Development Bank partnered with North Carolina State Universitys Genetic Engineering and Society (GES) Center to assess the regulatory and institutional frameworks surrounding gene-editing via CRISPR-based technologies in the Latin America and Caribbean (LAC) regions. The project studied the following core components: Current Policy Evaluation: Understanding what the future may hold requires a critical examination of the current status of the regulatory landscape. Analysis of the existing regulatory systems for agricultural biotechnologies throughout Latin America and how they included considerations for novel biotechnology strategies such as gene editing through CRISPR technologies were done. Forecasting and Future Policy Scenario Analysis: Potential products created through gene editing may face very different situations on the ground, depending on countries diverse regulations and market structures. To clarify the potential impacts of regulatory reforms, we included concrete case studies in our analysis. Identifying investment priorities: The diversity of the region naturally means that countries will have unique priorities and needs with respect to investment in agricultural biotechnology development and regulatory infrastructure. The document evaluates the accomplishments of the region in the development of gene edited products, highlighting both private and public sector innovations.
4

Kuiken, Todd, and Jennifer Kuzma. Genome Editing in Latin America: Regional Regulatory Overview. Inter-American Development Bank, July 2021. http://dx.doi.org/10.18235/0003410.

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The power and promise of genome editing, CRISPR specifically, was first realized with the discovery of CRISPR loci in the 1980s.3 Since that time, CRISPR-Cas systems have been further developed enabling genome editing in virtually all organisms across the tree of life.3 In the last few years, we have seen the development of a diverse set of CRISPR-based technologies that has revolutionized genome manipulation.4 Enabling a more diverse set of actors than has been seen with other emerging technologies to redefine research and development for biotechnology products encompassing food, agriculture, and medicine.4 Currently, the CRISPR community encompasses over 40,000 authors at 20,000 institutions that have documented their research in over 20,000 published and peer-reviewed studies.5 These CRISPR-based genome editing tools have promised tremendous opportunities in agriculture for the breeding of crops and livestock across the food supply chain. Potentially addressing issues associated with a growing global population, sustainability concerns, and possibly help address the effects of climate change.4 These promises however, come along-side concerns of environmental and socio-economic risks associated with CRISPR-based genome editing, and concerns that governance systems are not keeping pace with the technological development and are ill-equipped, or not well suited, to evaluate these risks. The Inter-American Development Bank (IDB) launched an initiative in 2020 to understand the complexities of these new tools, their potential impacts on the LAC region, and how IDB may best invest in its potential adoption and governance strategies. This first series of discussion documents: “Genome Editing in Latin America: Regulatory Overview,” and “CRISPR Patent and Licensing Policy” are part of this larger initiative to examine the regulatory and institutional frameworks surrounding gene editing via CRISPR-based technologies in the Latin America and Caribbean (LAC) regions. Focusing on Argentina, Bolivia, Brazil, Colombia, Honduras, Mexico, Paraguay, Peru, and Uruguay, they set the stage for a deeper analysis of the issues they present which will be studied over the course of the next year through expert solicitations in the region, the development of a series of crop-specific case studies, and a final comprehensive regional analysis of the issues discovered.
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Bagley, Margo. Genome Editing in Latin America: CRISPR Patent and Licensing Policy. Inter-American Development Bank, July 2021. http://dx.doi.org/10.18235/0003409.

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The power and promise of genome editing, CRISPR specifically, was first realized with the discovery of CRISPR loci in the 1980s.i Since that time, CRISPR-Cas systems have been further developed enabling genome editing in virtually all organisms across the tree of life.i In the last few years, we have seen the development of a diverse set of CRISPR-based technologies that has revolutionized genome manipulation.ii Enabling a more diverse set of actors than has been seen with other emerging technologies to redefine research and development for biotechnology products encompassing food, agriculture, and medicine.ii Currently, the CRISPR community encompasses over 40,000 authors at 20,000 institutions that have documented their research in over 20,000 published and peer-reviewed studies.iii These CRISPR-based genome editing tools have promised tremendous opportunities in agriculture for the breeding of crops and livestock across the food supply chain. Potentially addressing issues associated with a growing global population, sustainability concerns, and possibly help address the effects of climate change.i These promises however, come along-side concerns of environmental and socio-economic risks associated with CRISPR-based genome editing, and concerns that governance systems are not keeping pace with the technological development and are ill-equipped, or not well suited, to evaluate these risks. The Inter-American Development Bank (IDB) launched an initiative in 2020 to understand the complexities of these new tools, their potential impacts on the LAC region, and how IDB may best invest in its potential adoption and governance strategies. This first series of discussion documents: “Genome Editing in Latin America: Regulatory Overview,” and “CRISPR Patent and Licensing Policy” are part of this larger initiative to examine the regulatory and institutional frameworks surrounding gene editing via CRISPR-based technologies in the Latin America and Caribbean (LAC) regions. Focusing on Argentina, Bolivia, Brazil, Colombia, Honduras, Mexico, Paraguay, Peru, and Uruguay, they set the stage for a deeper analysis of the issues they present which will be studied over the course of the next year through expert solicitations in the region, the development of a series of crop-specific case studies, and a final comprehensive regional analysis of the issues discovered.
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Paran, Ilan, and Allen Van Deynze. Regulation of pepper fruit color, chloroplasts development and their importance in fruit quality. United States Department of Agriculture, January 2014. http://dx.doi.org/10.32747/2014.7598173.bard.

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Pepper exhibits large natural variation in chlorophyll content in the immature fruit. To dissect the genetic and molecular basis of this variation, we conducted QTL mapping for chlorophyll content in a cross between light and dark green-fruited parents, PI 152225 and 1154. Two major QTLs, pc1 and pc10, that control chlorophyll content by modulation of chloroplast compartment size in a fruit-specific manner were detected in chromosomes 1 and 10, respectively. The pepper homolog of GOLDEN2- LIKE transcription factor (CaGLK2) was found as underlying pc10, similar to its effect on tomato fruit chloroplast development. A candidate gene for pc1was found as controlling chlorophyll content in pepper by the modulation of chloroplast size and number. Fine mapping of pc1 aided by bulked DNA and RNA-seq analyses enabled the identification of a zinc finger transcription factor LOL1 (LSD-One-Like 1) as a candidate gene underlying pc1. LOL1 is a positive regulator of oxidative stress- induced cell death in Arabidopsis. However, over expression of the rice ortholog resulted in an increase of chlorophyll content. Interestingly, CaAPRR2 that is linked to the QTL and was found to affect immature pepper fruit color in a previous study, did not have a significant effect on chlorophyll content in the present study. Verification of the candidate's function was done by generating CRISPR/Cas9 knockout mutants of the orthologues tomato gene, while its knockout experiment in pepper by genome editing is under progress. Phenotypic similarity as a consequence of disrupting the transcription factor in both pepper and tomato indicated its functional conservation in controlling chlorophyll content in the Solanaceae. A limited sequence diversity study indicated that null mutations in CaLOL1 and its putative interactorCaMIP1 are present in C. chinensebut not in C. annuum. Combinations of mutations in CaLOL1, CaMIP1, CaGLK2 and CaAPRR2 are required for the creation of the extreme variation in chlorophyll content in Capsicum.
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Gilkeson, Luke. CRISPR-Cas9 Gene Therapy Review: A Novel Way to Treat Genetic Disease. Ames (Iowa): Iowa State University, May 2024. http://dx.doi.org/10.31274/cc-20240624-452.

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8

Podlevsky, Joshua. Cas9 Protein Post-translational Modifications (PTMs): A Potential Biomarker of Gene-editing. Office of Scientific and Technical Information (OSTI), October 2019. http://dx.doi.org/10.2172/1571552.

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9

Heo, Y., Y. Xu, X. Quan, Y. Seong, N. Kim, and J. Kim. CRISPR/Cas9 nuclease-mediated gene knock-in in bovine pluripotent stem cells and embryos. Cold Spring Harbor Laboratory, May 2014. http://dx.doi.org/10.1101/005421.

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10

Research, Gratis. The Mystery behind Bacterial Retrons. Gratis Research, December 2020. http://dx.doi.org/10.47496/gr.blog.05.

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Retron-mediated cell killing serves as a defensive strategy to prevent the spreading of phage infection in bacteria and the combined action of retron and CRISPR-based gene editing appear to be a potent gene-editing tool.

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