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https://www.readbyqxmd.com/read/28926587/functional-characterization-of-zebrafish-orthologs-of-the-human-beta-3-glucosyltransferase-b3glct-gene-mutated-in-peters-plus-syndrome
#1
Eric Weh, Hideyuki Takeuchi, Sanaa Muheisen, Robert S Haltiwanger, Elena V Semina
Peters Plus Syndrome (PPS) is a rare autosomal recessive disease characterized by ocular defects, short stature, brachydactyly, characteristic facial features, developmental delay and other highly variable systemic defects. Classic PPS is caused by loss-of-function mutations in the B3GLCT gene encoding for a β3-glucosyltransferase that catalyzes the attachment of glucose via a β1-3 glycosidic linkage to O-linked fucose on thrombospondin type 1 repeats (TSRs). B3GLCT was shown to participate in a non-canonical ER quality control mechanism; however, the exact molecular processes affected in PPS are not well understood...
2017: PloS One
https://www.readbyqxmd.com/read/28925369/generation-of-a-gene-corrected-isogenic-control-ipsc-line-from-cystic-fibrosis-patient-specific-ipscs-homozygous-for-p-phe508del-mutation-mediated-by-talens-and-ssodn
#2
Sylvia Merkert, Christien Bednarski, Gudrun Göhring, Toni Cathomen, Ulrich Martin
Cystic fibrosis (CF) is a monogenetic disease caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, which affects multiple organs. Human induced pluripotent stem cells (iPSCs) derived from CF patients and the generation of isogeneic gene-corrected control cell lines enable disease modelling, drug discovery or toxicological studies and therefore the development of CF patient-specific therapies. We have previously generated a hiPSC line from a CF patient homozygous for the p...
August 2017: Stem Cell Research
https://www.readbyqxmd.com/read/28919449/genome-editing-in-fishes-and-their-applications
#3
Bo Zhu, Wei Ge
There have been revolutionary progresses in genome engineering in the past few years. The newly-emerged genome editing technologies including zinc-finger nuclease (ZFN), transcription activator-like effector nuclease (TALEN) and clustered regularly interspaced short palindromic repeats associated with Cas9 (CRISPR/Cas9) have enabled biological scientists to perform efficient and precise targeted genome editing in different species. Fish represent the largest group of vertebrates with many species having values for both scientific research and aquaculture industry...
September 14, 2017: General and Comparative Endocrinology
https://www.readbyqxmd.com/read/28913801/targeted-mutagenesis-in-hexaploid-bread-wheat-using-the-talen-and-crispr-cas-systems
#4
Yanpeng Wang, Yuan Zong, Caixia Gao
The use of sequence-specific transcription activator-like effector nucleases (TALENs) and the clustered regularly interspaced short palindromic repeats-associated system (CRISPR/Cas9) have provided powerful reverse genetic approaches to the targeted modification of genomes in numerous organisms. Both systems have been employed to generate loss-of-function alleles in bread wheat, by targeting multiple and single copies of genes. Here we present protocols for modifying the wheat genome using the two systems. The protocols include the design of TALEN and CRISPR/Cas9 target sites and their construction, evaluation of their activities in protoplasts, transformation of plants, and mutation screening...
2017: Methods in Molecular Biology
https://www.readbyqxmd.com/read/28913702/impaired-fertility-and-motor-function-in-a-zebrafish-model-for-classic-galactosemia
#5
Jo M Vanoevelen, M Estela Rubio-Gozalbo, Britt van Erven, Jörgen Bierau, Xiaoping Huang, Gerard T Berry, Rein Vos, Ana I Coelho
Classic galactosemia is a genetic disorder of galactose metabolism, caused by severe deficiency of galactose-1-phosphate uridylyltransferase (GALT) enzyme activity due to mutations of the GALT gene. Its pathogenesis is still not fully elucidated, and a therapy that prevents chronic impairments is lacking. In order to move research forward, there is a high need for a novel animal model, which allows organ studies throughout development and high-throughput screening of pharmacologic compounds. Here, we describe the generation of a galt knockout zebrafish model and present its phenotypical characterization...
September 14, 2017: Journal of Inherited Metabolic Disease
https://www.readbyqxmd.com/read/28911205/a-zebrafish-model-of-x-linked-adrenoleukodystrophy-recapitulates-key-disease-features-and-demonstrates-a-developmental-requirement-for-abcd1-in-oligodendrocyte-patterning-and-myelination
#6
Lauren R Strachan, Tamara J Stevenson, Briana Freshner, Matthew D Keefe, D Miranda Bowles, Joshua L Bonkowsky
X-linked adrenoleukodystrophy (ALD) is a devastating inherited neurodegenerative disease caused by defects in the ABCD1 gene and affecting peripheral and central nervous system myelin. ABCD1 encodes a peroxisomal transmembrane protein required for very long chain fatty acid (VLCFA) metabolism. We show that zebrafish (Danio rerio) Abcd1 is highly conserved at the amino acid level with human ABCD1, and during development is expressed in homologous regions including the central nervous system and adrenal glands...
September 15, 2017: Human Molecular Genetics
https://www.readbyqxmd.com/read/28905511/talen-mediated-targeted-mutagenesis-of-more-than-100-comt-copies-alleles-in-highly-polyploid-sugarcane-improves-saccharification-efficiency-without-compromising-biomass-yield
#7
Baskaran Kannan, Je Hyeong Jung, Geoffrey W Moxley, Sun-Mi Lee, Fredy Altpeter
Sugarcane is the world's most efficient feedstock for commercial production of bioethanol due to its superior biomass production and accumulation of sucrose in stems. Integrating first and second generation ethanol conversion processes will enhance the biofuel yield per unit area by utilizing both sucrose as well as cell wall bound sugars for fermentation. RNAi suppression of the lignin biosynthetic gene caffeic acid O-methyltransferase (COMT) has been demonstrated to improve bioethanol production from lignocellulosic biomass...
September 14, 2017: Plant Biotechnology Journal
https://www.readbyqxmd.com/read/28904745/genome-editing-of-the-hiv-co-receptors-ccr5-and-cxcr4-by-crispr-cas9-protects-cd4-t-cells-from-hiv-1-infection
#8
Zhepeng Liu, Shuliang Chen, Xu Jin, Qiankun Wang, Kongxiang Yang, Chenlin Li, Qiaoqiao Xiao, Panpan Hou, Shuai Liu, Shaoshuai Wu, Wei Hou, Yong Xiong, Chunyan Kong, Xixian Zhao, Li Wu, Chunmei Li, Guihong Sun, Deyin Guo
BACKGROUND: The main approach to treat HIV-1 infection is combination antiretroviral therapy (cART). Although cART is effective in reducing HIV-1 viral load and controlling disease progression, it has many side effects, and is expensive for HIV-1 infected patients who must remain on lifetime treatment. HIV-1 gene therapy has drawn much attention as studies of genome editing tools have progressed. For example, zinc finger nucleases (ZFN), transcription activator like effector nucleases (TALEN) and clustered regularly interspaced short palindromic repeats (CRISPR)-Cas9 have been utilized to successfully disrupt the HIV-1 co-receptors CCR5 or CXCR4, thereby restricting HIV-1 infection...
2017: Cell & Bioscience
https://www.readbyqxmd.com/read/28903419/development-of-zebrafish-medulloblastoma-like-pnet-model-by-talen-mediated-somatic-gene-inactivation
#9
Jaegal Shim, Jung-Hwa Choi, Moon-Hak Park, Hyena Kim, Jong Hwan Kim, Seon-Young Kim, Dongwan Hong, Sunshin Kim, Ji Eun Lee, Cheol-Hee Kim, Jeong-Soo Lee, Young-Ki Bae
Genetically engineered animal tumor models have traditionally been generated by the gain of single or multiple oncogenes or the loss of tumor suppressor genes; however, the development of live animal models has been difficult given that cancer phenotypes are generally induced by somatic mutation rather than by germline genetic inactivation. In this study, we developed somatically mutated tumor models using TALEN-mediated somatic gene inactivation of cdkn2a/b or rb1 tumor suppressor genes in zebrafish. One-cell stage injection of cdkn2a/b-TALEN mRNA resulted in malignant peripheral nerve sheath tumors with high frequency (about 39%) and early onset (about 35 weeks of age) in F0 tp53(e7/e7) mutant zebrafish...
August 15, 2017: Oncotarget
https://www.readbyqxmd.com/read/28903395/tgf-%C3%AE-2-induced-angptl4-expression-promotes-tumor-progression-and-osteoclast-differentiation-in-giant-cell-tumor-of-bone
#10
Bo Li, Ming Qian, Hao Cao, Qi Jia, Zhipeng Wu, Xinghai Yang, Tianyi Ma, Haifeng Wei, Tianrui Chen, Jianru Xiao
Although emerging studies have implicated that Aiopoietin-like 4 Protein (ANGPTL4) is related to the aggressiveness and metastasis of many tumors, the role of ANGPLT4 in giant cell tumor (GCT) of bone was rarely investigated. The mechanism of ANGPLT4 in tumor-induced osteoclastogenesis still remains unclear. In this study, we first demonstrated that ANGPTL4 was highly expressed in GCT compared to normal tissues, while we showed that TGF-β2 released by osteoclasts induced bone resorption could increase the expression of ANGPTL4 in GCTSCs...
August 15, 2017: Oncotarget
https://www.readbyqxmd.com/read/28899668/the-sp7-gene-is-required-for-maturation-of-osteoblast-linage-cells-in-medaka-oryzias-latipes-vertebral-column-development
#11
Yuki Azetsu, Keiji Inohaya, Yoshiro Takano, Masato Kinoshita, Mai Tasaki, Akira Kudo
Sp7 is a zinc finger transcription factor that is essential for osteoblast differentiation in mammals. To verify the characteristic features of osteoblast-lineage cells in teleosts, we established medaka sp7 mutants using a transcription activator-like effector nuclease (TALEN) genome editing system. These mutants showed severe defects in the formation of skeletal structures. In particular, the neural and the hemal arches were not formed, although the chordal centra were formed. Analysis of the transgenic medaka revealed that sp7 mutant had normal distribution of type X collagen a1 a (col10a1a)-positive osteoblast-like cells around the centrum and at the proximal region of the vertebral arch...
September 9, 2017: Developmental Biology
https://www.readbyqxmd.com/read/28878801/crispr-cas9-a-practical-approach-in-date-palm-genome-editing
#12
REVIEW
Muhammad N Sattar, Zafar Iqbal, Muhammad N Tahir, Muhammad S Shahid, Muhammad Khurshid, Abdullatif A Al-Khateeb, Suliman A Al-Khateeb
The genetic modifications through breeding of crop plants have long been used to improve the yield and quality. However, precise genome editing (GE) could be a very useful supplementary tool for improvement of crop plants by targeted genome modifications. Various GE techniques including ZFNs (zinc finger nucleases), TALENs (transcription activator-like effector nucleases), and most recently clustered regularly interspaced short palindromic repeats (CRISPR)/Cas9 (CRISPR-associated protein 9)-based approaches have been successfully employed for various crop plants including fruit trees...
2017: Frontiers in Plant Science
https://www.readbyqxmd.com/read/28873625/combination-of-different-antifungal-agents-in-oil-in-water-emulsions-to-control-strawberry-jam-spoilage
#13
Susana Ribes, Ana Fuentes, Pau Talens, Jose Manuel Barat
The combination of antifungal agents (cinnamon bark oil, zinc gluconate and trans-ferulic acid) in oil-in-water emulsions to control the fungal spoilage of strawberry jams, minimising essential oil's sensory impact, was evaluated in this work. The in vitro assays of free antifungal agents were performed against five fungal strains; meanwhile, the emulsions assays were conducted against Aspergillus niger given its strong resistance and its relevance in strawberry products. The emulsion formulated with 0.08mg/g of essential oil was able to inhibit mould growth after the incubation period...
January 15, 2018: Food Chemistry
https://www.readbyqxmd.com/read/28854818/recent-advances-in-preclinical-developments-using-adenovirus-hybrid-vectors
#14
Eric Ehrke-Schulz, Wenli Zhang, Jian Gao, Anja Ehrhardt
Adenovirus (Ad) based vectors are efficient gene transfer vehicles to deliver foreign DNA into living organisms offering large cargo capacity low immunogenicity and genotoxicity. As Ad shows low integration rates of their genomes into host chromosomes, vector derived gene expression decreases due to continuous cell cycling in regenerating tissues and dividing cell populations. To overcome this hurdle adenoviral delivery can be combined with mechanisms leading to maintenance of therapeutic DNA and long-term effects of the desired treatment...
August 30, 2017: Human Gene Therapy
https://www.readbyqxmd.com/read/28834068/reversible-loading-of-nanoscale-elements-on-a-multicomponent-supramolecular-polymer-system-by-using-dna-strand-displacement
#15
Willem E M Noteborn, Victorio Saez Talens, Roxanne E Kieltyka
Nucleic acids are excellent building blocks to enable switchable character in supramolecular polymer materials because of their inherent dynamic character and potential for orthogonal self-assembly. Herein, DNA-grafted squaramide bola-amphiphiles are used in a multicomponent supramolecular polymer system and it is shown that they can be addressed by DNAlabeled gold nanoparticles (5 and 15 nm) through sequence complementarity. These nanoparticles can be selectively erased or rewritten on-demand by means of DNA-strand displacement...
August 21, 2017: Chembiochem: a European Journal of Chemical Biology
https://www.readbyqxmd.com/read/28825645/development-of-the-moosy4-enose-iot-for-sulphur-based-voc-water-pollution-detection
#16
Enric Climent, Jose Pelegri-Sebastia, Tomas Sogorb, J B Talens, Jose Chilo
In this paper, we describe a new low-cost and portable electronic nose instrument, the Multisensory Odor Olfactory System MOOSY4. This prototype is based on only four metal oxide semiconductor (MOS) gas sensors suitable for IoT technology. The system architecture consists of four stages: data acquisition, data storage, data processing, and user interfacing. The designed eNose was tested with experiment for detection of volatile components in water pollution, as a dimethyl disulphide or dimethyl diselenide or sulphur...
August 20, 2017: Sensors
https://www.readbyqxmd.com/read/28815851/basics-of-genome-editing-technology-and-its-application-in-livestock-species
#17
REVIEW
Bjoern Petersen
In the last decade, the research community has witnessed a blooming of targeted genome editing tools and applications. Novel programmable DNA nucleases such as zinc finger nucleases (ZFNs), transcription activator-like endonucleases (TALENs) and the clustered regularly interspaced short palindromic repeats/Cas9 system (CRISPR/Cas9) possess long recognition sites and are capable of cutting DNA in a very specific manner. These DNA nucleases mediate targeted genetic alterations by enhancing the DNA mutation rate via induction of double-strand breaks at a predetermined genomic site...
August 2017: Reproduction in Domestic Animals, Zuchthygiene
https://www.readbyqxmd.com/read/28815495/ligand-controlled-site-specific-recombination-in-zebrafish
#18
Avinash Chekuru, Veronika Kuscha, Stefan Hans, Michael Brand
Cre-mediated site-specific recombination has emerged as an indispensable tool for the precise manipulation of genomes allowing lineage-tracing studies, temporal and spatial misexpressions, and in particular the generation of conditional knockout alleles. Previously, we and others showed that Cre and its ligand-inducible variant CreER(T2) are also highly efficient in the developing and adult zebrafish. The number of Cre driver and effector lines is currently still limited in zebrafish. However, the recent advent of novel genome editing tools such as TALEN and CRISPR/Cas will significantly increase interest in the conditional Cre/lox-technology in this organism...
2017: Methods in Molecular Biology
https://www.readbyqxmd.com/read/28810059/targeted-genome-editing-in-caenorhabditis-elegans-using-crispr-cas9
#19
REVIEW
Behnom Farboud
Utilization of programmable nucleases to generate DNA lesions at precise endogenous sequences has transformed the ability to edit genomes from microbes to plants and animals. This is especially true in organisms that previously lacked the means to engineer precise genomic changes, like Caenorhabditis elegans. C. elegans is a 1 mm long free-living, nonparasitic, nematode worm, which is easily cultivated in a laboratory. Its detailed genetic map and relatively compact genome (~100 megabases) helped make it the first metazoan to have its entire genome sequenced...
August 15, 2017: Wiley Interdisciplinary Reviews. Developmental Biology
https://www.readbyqxmd.com/read/28806883/genome-and-epigenome-editing-to-treat-disorders-of-the-hematopoietic-system
#20
Claudio Mussolino, Jamal Alzubi, Valentina Pennucci, Giandomenico Turchiano, Toni Cathomen
The possibility to edit complex genomes in a targeted fashion has revolutionized basic research as well as biomedical and biotechnological applications in the last five years. The targeted introduction of genetic changes allowed researchers to create smart model systems for basic research, bio-engineers to genetically modify crops and farm animals, and translational scientists to develop novel treatment approaches for inherited and acquired disorders for which curative treatment options are not yet available...
August 15, 2017: Human Gene Therapy
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