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CRISPR/Cas9

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https://www.readbyqxmd.com/read/29917197/crispr-technology-new-paradigm-to-target-the-infectious-disease-pathogens
#1
M A Bakhrebah, M S Nassar, M S Alsuabeyl, W A Zaher, S A Meo
OBJECTIVE: Infectious diseases are one of the prime causes of death worldwide. An innovative sequence specific editing technology "Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)" has been tested on a broad range of microorganisms to target and destroy invading foreign DNA to human cells or tissues. This study aimed to discuss the mechanism and therapeutic usage of CRISPR/Cas9 genome editing technology in the management of various infectious disease pathogens...
June 2018: European Review for Medical and Pharmacological Sciences
https://www.readbyqxmd.com/read/29916068/detection-of-crispr-cas9-induced-genomic-fragment-deletions-in-barley-and-generation-of-homozygous-edited-lines-via-embryogenic-pollen-culture
#2
Eszter Kapusi, Eva Stöger
The CRISPR/Cas9 system from Streptococcus pyogenes is an increasingly popular tool for genome editing due to its ease of application. Here we demonstrate genomic DNA fragment removal using RNA directed Cas9 nuclease in barley. The high mutation frequency confirms the exceptional efficiency of the system and its suitability for generating loss-of-function mutant lines that may be used in functional genetics approaches to study endomembrane trafficking pathways and posttranslational protein modifications. The generation of doubled haploids from genome edited plants allows the recovery of true breeding lines that are instantly homozygous for the edited alleles...
2018: Methods in Molecular Biology
https://www.readbyqxmd.com/read/29915997/synthetic-biology-advances-and-applications-in-the-biotechnology-industry-a-perspective
#3
Leonard Katz, Yvonne Y Chen, Ramon Gonzalez, Todd C Peterson, Huimin Zhao, Richard H Baltz
Synthetic biology is a logical extension of what has been called recombinant DNA (rDNA) technology or genetic engineering since the 1970s. As rDNA technology has been the driver for the development of a thriving biotechnology industry today, starting with the commercialization of biosynthetic human insulin in the early 1980s, synthetic biology has the potential to take the industry to new heights in the coming years. Synthetic biology advances have been driven by dramatic cost reductions in DNA sequencing and DNA synthesis; by the development of sophisticated tools for genome editing, such as CRISPR/Cas9; and by advances in informatics, computational tools, and infrastructure to facilitate and scale analysis and design...
June 18, 2018: Journal of Industrial Microbiology & Biotechnology
https://www.readbyqxmd.com/read/29915899/stable-expression-of-infliximab-in-crispr-cas9-mediated-bak1-deficient-cho-cells
#4
Zhi Miao, Qian Li, Jian Zhao, Peng Wang, Lei Wang, Hong-Peng He, Nan Wang, Hao Zhou, Tong-Cun Zhang, Xue-Gang Luo
OBJECTIVES: To establish stable infliximab-expressing Chinese hamster ovary (CHO) cells with high tolerance to serum-free culture. RESULTS: Bcl-2 antagonist/killer 1 (BAK1), which is a key mediator of the apoptosis pathway, was disrupted, and infliximab, which is a broadly used monoclonal antibody for the treatment of rheumatoid arthritis and other autoimmune diseases, was incorporated into the BAK1 locus of the CHO chromosome using the clustered regularly interspaced short palindromic repeats (CRISPR)/Cas genome-editing technique...
June 18, 2018: Biotechnology Letters
https://www.readbyqxmd.com/read/29914963/do-fitness-costs-explain-the-rapid-spread-of-kelch13-c580y-substitutions-conferring-artemisinin-resistance
#5
Shalini Nair, Xue Li, Grace A Arya, Marina McDew-White, Marco Ferrari, François Nosten, Tim J C Anderson
Fitness costs are key determinants of whether drug resistance alleles establish and how fast they spread within populations. More than 125 different kelch13 alleles, each containing a different amino acid substitution, have arisen in SE Asian malaria parasite ( Plasmodium falciparum ) populations under artemisinin selection over the past 15 years in a dramatic example of a soft selective event. However, just one of these alleles (C580Y) is now outcompeting other alleles in multiple different countries and is spreading towards fixation...
June 18, 2018: Antimicrobial Agents and Chemotherapy
https://www.readbyqxmd.com/read/29914939/transcriptional-up-regulation-of-bag3-a-chaperone-assisted-selective-autophagy-factor-in-animal-models-of-ky-deficient-hereditary-myopathy
#6
Elliot J Jokl, Gideon L Hughes, Tobias Cracknell, Mary E Pownall, Gonzalo Blanco
The importance of kyphoscoliosis peptidase (KY) in skeletal muscle physiology has recently been emphasised by the identification of novel human myopathies associated with KY deficiency. Neither the pathogenic mechanism of KY deficiency nor a specific role for KY in muscle function have been established. However, aberrant localisation of FLNC in muscle fibers has been shown in humans and mice with loss of function mutations in the KY gene. FLNC turnover has been proposed to be controlled by Chaperone Assisted Selective Autophagy (CASA), a client-specific and tension-induced pathway that is required for muscle maintenance...
June 18, 2018: Disease Models & Mechanisms
https://www.readbyqxmd.com/read/29914921/determining-the-pathogenicity-of-a-genomic-variant-of-uncertain-significance-using-crispr-cas9-and-human-induced-pluripotent-stem-cells
#7
Ning Ma, Joe Zhang, Ilanit Itzhaki, Sophia L Zhang, Haodong Chen, Francois Haddad, Tomoya Kitani, Kitchener D Wilson, Lei Tian, Rajani Shrestha, Haodi Wu, Chi Keung Lam, Nazish Sayed, Joseph C Wu
Background -The progression toward low-cost and rapid next-generation sequencing has uncovered a multitude of variants of uncertain significance (VUS) in both patients and asymptomatic "healthy" individuals. A VUS is a rare or novel variant for which disease pathogenicity has not been conclusively demonstrated or excluded, and thus cannot be definitively annotated. VUS, therefore, pose critical clinical interpretation and risk-assessment challenges, and new methods are urgently needed to better characterize their pathogenicity...
June 18, 2018: Circulation
https://www.readbyqxmd.com/read/29914884/tracking-progress-an-update-on-animal-models-for-duchenne-muscular-dystrophy
#8
EDITORIAL
Dominic J Wells
Duchenne muscular dystrophy (DMD) is a progressive, fatal, X-linked monogenic muscle disorder caused by mutations in the DMD gene. In order to test treatments for DMD, a range of natural and engineered animal models have been developed, including mice, rats, dogs and pigs. Sui and colleagues have now added a dystrophic rabbit model to this range using CRISPR/Cas9 to disrupt exon 51 of DMD Rabbits have the advantage of being easier to breed and less costly than dog or pig models, but having clear clinical signs, in contrast to many mouse models...
June 13, 2018: Disease Models & Mechanisms
https://www.readbyqxmd.com/read/29913036/the-2017-ixa-presidential-lecture-recent-developments-in-xenotransplantation
#9
REVIEW
Peter J Cowan
The last few years have seen significant progress in xenotransplantation. Porcine xenograft survival in preclinical models continues to improve, accompanied by the adjustment of immunosuppression to more clinically realistic levels. The rapid uptake of CRISPR/Cas9 technology has accelerated the generation of new knock-in and knockout pigs, including animals null for the endogenous retrovirus PERV. This brief review presents a personal view of recent developments in the field.
May 2018: Xenotransplantation
https://www.readbyqxmd.com/read/29912475/crispr-cas9-mediated-knock-in-of-an-optimized-teto-repeat-for-live-cell-imaging-of-endogenous-loci
#10
Ipek Tasan, Gabriela Sustackova, Liguo Zhang, Jiah Kim, Mayandi Sivaguru, Mohammad HamediRad, Yuchuan Wang, Justin Genova, Jian Ma, Andrew S Belmont, Huimin Zhao
Nuclear organization has an important role in determining genome function; however, it is not clear how spatiotemporal organization of the genome relates to functionality. To elucidate this relationship, a method for tracking any locus of interest is desirable. Recently clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated protein 9 (Cas9) or transcription activator-like effectors were adapted for imaging endogenous loci; however, they are mostly limited to visualization of repetitive regions...
June 15, 2018: Nucleic Acids Research
https://www.readbyqxmd.com/read/29912461/exploiting-crispr-cas9-technology-to-investigate-individual-histone-modifications
#11
Juan-José Vasquez, Carolin Wedel, Raul O Cosentino, T Nicolai Siegel
Despite their importance for most DNA-templated processes, the function of individual histone modifications has remained largely unknown because in vivo mutational analyses are lacking. The reason for this is that histone genes are encoded by multigene families and that tools to simultaneously edit multiple genomic loci with high efficiency are only now becoming available. To overcome these challenges, we have taken advantage of the power of CRISPR-Cas9 for precise genome editing and of the fact that most DNA repair in the protozoan parasite Trypanosoma brucei occurs via homologous recombination...
June 15, 2018: Nucleic Acids Research
https://www.readbyqxmd.com/read/29912402/cutting-edge-genetics-crispr-cas9-editing-of-plant-genomes
#12
Cara L Soyars, Brenda A Peterson, Christian A Burr, Zachary L Nimchuk
The Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)/CRISPR associated nuclease 9 (Cas9) system is a genome editing technology transforming the field of plant biology by virtue of the system's efficiency and specificity. The system has quickly evolved for many diverse applications including multiplex gene mutation, gene replacement, and transcriptional control. As CRISPR/Cas9 is increasingly applied to plants, it is becoming clear that each component of the system can be modified to improve editing results...
April 18, 2018: Plant & Cell Physiology
https://www.readbyqxmd.com/read/29912293/crispr-cas9-in-plants-at-play-in-the-genome-and-at-work-for-crop-improvement
#13
Babar Hussain, Stuart James Lucas, Hikmet Budak
The clustered regularly interspaced short palindromic repeats/CRISPR-associated protein 9 (CRISPR/Cas9) system uses single-guide RNAs for genome editing, making it a simple, robust, powerful tool for targeted gene mutagenesis, knockout and knock-in/replacement, as well as transcriptional regulation. Here, we review the working principles, components and potential modifications of CRISPR/Cas9 for efficient single and multiplex gene editing in plants. We also describe recent work that has used CRISPR/Cas9 to improve economically important traits in crop plants...
April 17, 2018: Briefings in Functional Genomics
https://www.readbyqxmd.com/read/29911105/genetics-of-cardiovascular-disease-fishing-for-causality
#14
REVIEW
Christoph Paone, Federica Diofano, Deung-Dae Park, Wolfgang Rottbauer, Steffen Just
Cardiovascular disease (CVD) is still the leading cause of death in all western world countries and genetic predisposition in combination with traditional risk factors frequently mediates their manifestation. Genome-wide association (GWA) studies revealed numerous potentially disease modifying genetic loci often including several SNPs and associated genes. However, pure genetic association does not prove direct or indirect relevance of the modifier region on pathogenesis, nor does it define within the associated region the exact genetic driver of the disease...
2018: Frontiers in Cardiovascular Medicine
https://www.readbyqxmd.com/read/29910815/-toxoplasma-chinese-1-strain-of-wh3%C3%AE-rop16-i-iii-gra15-ii-genetic-background-contributes-to-abnormal-pregnant-outcomes-in-murine-model
#15
Cong Wang, Weisheng Cheng, Qian Yu, Tian Xing, Shoubin Chen, Lei Liu, Li Yu, Jian Du, Qingli Luo, Jilong Shen, Yuanhong Xu
Toxoplasma gondii infection evokes a strong Th1-type response with interleukin (IL)-12 and interferon (IFN)-γ secretion. Recent studies suggest that the infection of pregnant mice with T. gondii may lead to adverse pregnancy results caused by subversion of physiological immune tolerance at maternofetal interface rather than direct invasion of the parasite. Genotype-associated dense granule protein GRA15II tends to induce classically activated macrophage (M1) differentiation and subsequently activating NK, Th1, and Th17 cells whereas rhoptry protein ROP16I/III drives macrophages to alternatively activated macrophage (M2) polarization and elicits Th2 immune response...
2018: Frontiers in Immunology
https://www.readbyqxmd.com/read/29908711/gene-editing-on-center-stage
#16
REVIEW
Rasmus O Bak, Natalia Gomez-Ospina, Matthew H Porteus
Smithies et al. (1985) and Jasin and colleagues (1994) provided proof of concept that homologous recombination (HR) could be applied to the treatment of human disease and that its efficiency could be improved by the induction of double-strand breaks (DSBs). A key advance was the discovery of engineered nucleases, such as zinc-finger nucleases (ZFNs) and transcription activator-like (TAL) effector nucleases (TALENs), that can generate site-specific DSBs. The democratization and widespread use of genome editing was enabled by the discovery of the clustered regularly interspaced short palindromic repeats (CRISPR)-Cas9 nuclease system...
June 13, 2018: Trends in Genetics: TIG
https://www.readbyqxmd.com/read/29908341/crispr-cas9-based-heritable-targeted-mutagenesis-in-thermobia-domestica-a-genetic-tool-in-an-apterygote-development-model-of-wing-evolution
#17
Takahiro Ohde, Yusuke Takehana, Takahiro Shiotsuki, Teruyuki Niimi
Despite previous developmental studies on basally branching wingless insects and crustaceans, the evolutionary origin of insect wings remains controversial. Knowledge regarding genetic regulation of tissues hypothesized to have given rise to wings would help to elucidate how ancestral development changed to allow the evolution of true wings. However, genetic tools available for basally branching wingless species are limited. The firebrat Thermobia domestica is an apterygote species, phylogenetically related to winged insects...
June 13, 2018: Arthropod Structure & Development
https://www.readbyqxmd.com/read/29907766/efficient-rna-drug-delivery-using-red-blood-cell-extracellular-vesicles
#18
Waqas Muhammad Usman, Tin Chanh Pham, Yuk Yan Kwok, Luyen Tien Vu, Victor Ma, Boya Peng, Yuen San Chan, Likun Wei, Siew Mei Chin, Ajijur Azad, Alex Bai-Liang He, Anskar Y H Leung, Mengsu Yang, Ng Shyh-Chang, William C Cho, Jiahai Shi, Minh T N Le
Most of the current methods for programmable RNA drug therapies are unsuitable for the clinic due to low uptake efficiency and high cytotoxicity. Extracellular vesicles (EVs) could solve these problems because they represent a natural mode of intercellular communication. However, current cellular sources for EV production are limited in availability and safety in terms of horizontal gene transfer. One potentially ideal source could be human red blood cells (RBCs). Group O-RBCs can be used as universal donors for large-scale EV production since they are readily available in blood banks and they are devoid of DNA...
June 15, 2018: Nature Communications
https://www.readbyqxmd.com/read/29907308/advanced-editing-of-the-nuclear-and-plastid-genomes-in-plants
#19
Agnieszka A Piatek, Scott C Lenaghan, C Neal Stewart
Genome editing is a powerful suite of technologies utilized in basic and applied plant research. Both nuclear and plastid genomes have been genetically engineered to alter traits in plants. While the most frequent molecular outcome of gene editing has been knockouts resulting in a simple deletion of an endogenous protein of interest from the host's proteome, new genes have been added to plant genomes and, in several instances, the sequence of endogenous genes have been targeted for a few coding changes. Targeted plant characteristics for genome editing range from single gene targets for agronomic input traits to metabolic pathways to endow novel plant function...
August 2018: Plant Science: An International Journal of Experimental Plant Biology
https://www.readbyqxmd.com/read/29906414/guide-rna-selection-for-crispr-cas9-transfections-in-plasmodium-falciparum
#20
Jose M Ribeiro, Meera Garriga, Nicole Potchen, Anna K Crater, Ankit Gupta, Daisuke Ito, Sanjay A Desai
CRISPR-Cas9 mediated genome editing is addressing key limitations in the transfection of malaria parasites. While this method has already simplified the needed molecular cloning and reduced the time required to generate mutants in the human pathogen Plasmodium falciparum, optimal selection of required guide RNAs and guidelines for successful transfections have not been well characterized, leading workers to use time-consuming trial and error approaches. We used a genome-wide computational approach to create a comprehensive and publicly accessible database of possible guide RNA sequences in the P...
June 12, 2018: International Journal for Parasitology
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