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https://www.readbyqxmd.com/read/29769662/novel-internal-regulators-and-candidate-mirnas-within-mir-379-mir-656-mirna-cluster-can-alter-cellular-phenotype-of-human-glioblastoma
#1
Subhashree Nayak, Meghali Aich, Anupam Kumar, Suman Sengupta, Prajakta Bajad, Parashar Dhapola, Deepanjan Paul, Kiran Narta, Suvendu Purkrait, Bharati Mehani, Ashish Suri, Debojyoti Chakraborty, Arijit Mukhopadhyay, Chitra Sarkar
Clustered miRNAs can affect functioning of downstream pathways due to possible coordinated function. We observed 78-88% of the miR-379/miR-656 cluster (C14MC) miRNAs were downregulated in three sub-types of diffuse gliomas, which was also corroborated with analysis from The Cancer Genome Atlas (TCGA) datasets. The miRNA expression levels decreased with increasing tumor grade, indicating this downregulation as an early event in gliomagenesis. Higher expression of the C14MC miRNAs significantly improved glioblastioma prognosis (Pearson's r = 0...
May 16, 2018: Scientific Reports
https://www.readbyqxmd.com/read/29769640/hypoxia-induced-microrna-191-contributes-to-hepatic-ischemia-reperfusion-injury-through-the-zonab-cyclin-d1-axis
#2
Wenming Pan, Lin Wang, Xiao-Fei Zhang, Hongji Zhang, Jinxiang Zhang, Guoliang Wang, Peng Xu, Yunwei Zhang, Ping Hu, Xiao-Dong Zhang, Run-Lei Du, Hui Wang
Hepatic ischemia/reperfusion injury (IRI) is a common cause of morbidity and mortality in liver transplantation settings and involves severe cell death and inflammatory responses. MicroRNA-191 has recently been reported to be abnormally expressed in hepatocellular carcinoma and other liver diseases in the regulation of important cellular processes. However, little is known about its function and molecular mechanism in IRI. Here, we demonstrate that miR-191 is significantly upregulated in a cultured cell line during hypoxia/reperfusion (H/R) and in liver tissue during IRI in mice...
May 16, 2018: Cell Death and Differentiation
https://www.readbyqxmd.com/read/29766242/inactivation-of-deubiquitinase-cyld-enhances-therapeutic-antibody-production-in-chinese-hamster-ovary-cells
#3
Yafang Lu, Qin Zhou, Qianqian Han, Pengfei Wu, Lanlan Zhang, Lin Zhu, David T Weaver, Changzhi Xu, Buchang Zhang
Chinese hamster ovary (CHO) cells are promising host engineering cells for industry manufacturing of therapeutic antibodies. However, cell death due to apoptosis remains a huge challenge to augment antibody production, and developing CHO cells with enhanced anti-apoptosis and proliferation ability is fundamental for cell line development and high-yielding bioprocesses. Deubiquitinase cylindromatosis (CYLD) has been proved to be a tumor suppressor by negatively regulating NF-κB and Wnt/β-catenin signaling pathways...
May 15, 2018: Applied Microbiology and Biotechnology
https://www.readbyqxmd.com/read/29760161/ctcf-boundary-remodels-chromatin-domain-and-drives-aberrant-hox-gene-transcription-in-acute-myeloid-leukemia
#4
Huacheng Luo, Fei Wang, Jie Zha, Haoli Li, Bowen Yan, Qinghua Du, Fengchun Yang, Amin Sobh, Christopher Vulpe, Leylah Drusbosky, Christopher Cogle, Iouri Chepelev, Bing Xu, Stephen D Nimer, Jonathan Licht, Yi Qiu, Baoan Chen, Mingjiang Xu, Suming Huang
HOX gene dysregulation is a common feature of acute myeloid leukemia (AML). The molecular mechanisms underlying aberrant HOX gene expression and associated AML pathogenesis remain unclear. The nuclear protein CCCTC-binding factor (CTCF), when bound to insulator sequences, constrains temporal HOX gene expression patterns within confined chromatin domains for normal development. Here, we employed targeted pooled CRISPR-Cas9 knockout library screening to interrogate the function of CTCF boundaries in the HOX gene loci...
May 14, 2018: Blood
https://www.readbyqxmd.com/read/29759937/identification-of-epigenetic-regulators-of-dux4-fl-for-targeted-therapy-of-facioscapulohumeral-muscular-dystrophy
#5
Charis L Himeda, Takako I Jones, Ching-Man Virbasius, Lihua Julie Zhu, Michael R Green, Peter L Jones
Facioscapulohumeral muscular dystrophy (FSHD) is caused by epigenetic de-repression of the disease locus, leading to pathogenic misexpression of the DUX4 gene in skeletal muscle. While the factors and pathways involved in normal repression of the FSHD locus in healthy cells have been well characterized, very little is known about those responsible for the aberrant activation of DUX4-fl in FSHD myocytes. Reasoning that DUX4-fl activators might represent useful targets for small molecule inhibition, we performed a highly targeted, candidate-based screen of epigenetic regulators in primary FSHD myocytes...
April 26, 2018: Molecular Therapy: the Journal of the American Society of Gene Therapy
https://www.readbyqxmd.com/read/29756770/versatile-high-throughput-fluorescence-assay-for-monitoring-cas9-activity
#6
Kyle Seamon, Yooli K Light, Edwin Saada, Joseph S Schoeniger, Brooke Harmon
The RNA-guided DNA nuclease Cas9 is now widely used for the targeted modification of genomes of human cells and various organisms. Despite the extensive use of CRISPR systems for genome engineering and the rapid discovery and engineering of new CRISPR-associated nucleases, there are no high-throughput assays for measuring enzymatic activity. The current laboratory and future therapeutic uses of CRISPR technology have a significant risk of accidental exposure or clinical off-target effects, under-scoring the need for therapeutically-effective inhibitors of Cas9...
May 14, 2018: Analytical Chemistry
https://www.readbyqxmd.com/read/29755309/targeted-delivery-of-crispr-cas9-mediated-cancer-gene-therapy-via-liposome-templated-hydrogel-nanoparticles
#7
Zeming Chen, Fuyao Liu, Yanke Chen, Jun Liu, Xiaoying Wang, Ann T Chen, Gang Deng, Hongyi Zhang, Jie Liu, Zhangyong Hong, Jiangbing Zhou
Due to its simplicity, versatility, and high efficiency, the clustered regularly interspaced short palindromic repeat (CRISPR)/Cas9 technology has emerged as one of the most promising approaches for treatment of a variety of genetic diseases, including human cancers. However, further translation of CRISPR/Cas9 for cancer gene therapy requires development of safe approaches for efficient, highly specific delivery of both Cas9 and single guide RNA to tumors. Here, novel core-shell nanostructure, liposome-templated hydrogel nanoparticles (LHNPs) that are optimized for efficient codelivery of Cas9 protein and nucleic acids is reported...
December 8, 2017: Advanced Functional Materials
https://www.readbyqxmd.com/read/29752937/lncrna-thor-promotes-human-renal-cell-carcinoma-cell-growth
#8
Xue-Ting Ye, Hang Huang, Wei-Ping Huang, Wei-Lie Hu
BACKGROUND: Recent studies have characterized a novel but extremely conserved long non-coding RNA (LncRNA) THOR. THOR directly associates with insulin-like growth factor 2 mRNA-binding protein 1 (IGF2BP1) to promote mRNA stabilization of key pro-cancerous genes. RESULTS: Here, we show that THOR is expressed in human renal cell carcinoma (RCC) tissues and established/primary human RCC cells. It was not detected in normal renal tissues nor in HK-2 and primary human renal epithelial cells...
May 9, 2018: Biochemical and Biophysical Research Communications
https://www.readbyqxmd.com/read/29751014/a-versatile-tool-for-the-quantification-of-crispr-cas9-induced-genome-editing-events-in-human-hematopoietic-cell-lines-and-hematopoietic-stem-progenitor-cells
#9
Rajeswari Jayavaradhan, Devin Pillis, Punam Malik
The efficient site-specific DNA double-strand breaks (DSB) created by CRISPR/Cas9 has revolutionized genome engineering, and has great potential for editing hematopoietic stem/progenitor cells (HSPC). However, detailed understanding of the variables that influence choice of DNA-DSB repair (DDR) pathways by HSPC is required for therapeutic levels of editing in these clinically relevant cells. We developed a hematopoietic-reporter system that rapidly quantifies the three major DDR pathways utilized at the individual DSB created by CRISPR/Cas9: NHEJ, MMEJ and HDR; and show its applicability in evaluating the different DDR outcomes utilized by human hematopoietic cell lines and primary human HSPC...
May 8, 2018: Journal of Molecular Biology
https://www.readbyqxmd.com/read/29748615/specific-targeting-of-point-mutations-in-egfr-l858r-positive-lung-cancer-by-crispr-cas9
#10
Alvin Ho-Kwan Cheung, Chit Chow, Jinglin Zhang, Yuhang Zhou, Tingting Huang, Kayla Ching-Kei Ng, Terry Cho-Tsun Or, Yoyo Yao Yao, Yujuan Dong, Jackie Mei-Wah Fung, Lei Xiong, Aden Ka-Yin Chan, Wai-Ming Raymond Lung, Wei Kang, Ka-Fai To
Cancer cells are defined genetically by the mutations they harbor, commonly single nucleotide substitutions. Therapeutic approaches which specifically target cancer cells by recognizing these defining genetic aberrations are expected to exhibit minimal side-effects. However, current protein-based targeted therapy is greatly limited by the range of genes that can be targeted, as well as by acquired resistance. We hypothesized that a therapeutic oligonucleotide-based strategy may address this need of specific cancer targeting...
May 10, 2018: Laboratory Investigation; a Journal of Technical Methods and Pathology
https://www.readbyqxmd.com/read/29748595/crispr-lbcpf1-prevents-choroidal-neovascularization-in-a-mouse-model-of-age-related-macular-degeneration
#11
Taeyoung Koo, Sung Wook Park, Dong Hyun Jo, Daesik Kim, Jin Hyoung Kim, Hee-Yeon Cho, Jeungeun Kim, Jeong Hun Kim, Jin-Soo Kim
LbCpf1, derived from Lachnospiraceae bacterium ND2006, is a CRISPR RNA-guided endonuclease and holds promise for therapeutic applications. Here we show that LbCpf1 can be used for therapeutic gene editing in a mouse model of age-related macular degeneration (AMD). The intravitreal delivery of LbCpf1, targeted to two angiogenesis-associated genes encoding vascular endothelial growth factor A (Vegfa) and hypoxia inducing factor 1a (Hif1a), using adeno-associated virus, led to efficient gene disruption with no apparent off-target effects in the retina and retinal pigment epithelium (RPE) cells...
May 10, 2018: Nature Communications
https://www.readbyqxmd.com/read/29746416/crispr-genome-surgery-in-the-retina-in-light-of-off-targeting
#12
Galaxy Y Cho, Kellie A Schaefer, Alexander G Bassuk, Stephen H Tsang, Vinit B Mahajan
PURPOSE: Recent concerns regarding the clinical utilization of clustered regularly interspaced short palindromic repeats (CRISPR) involve uncertainties about the potential detrimental effects that many arise due to unintended genetic changes, as in off-target mutagenesis, during CRISPR genome surgery. This review gives an overview of off-targeting detection methods and CRISPR's place in the clinical setting, specifically in the field of ophthalmology. RESULTS: As CRISPR utilization in the laboratory setting has increased, knowledge regarding CRISPR mechanisms including its off-target effects has also increased...
May 7, 2018: Retina
https://www.readbyqxmd.com/read/29741611/crispr-cas9-editing-in-human-pluripotent-stem-cell-cardiomyocytes-highlights-arrhythmias-hypocontractility-and-energy-depletion-as-potential-therapeutic-targets-for-hypertrophic-cardiomyopathy
#13
Diogo Mosqueira, Ingra Mannhardt, Jamie R Bhagwan, Katarzyna Lis-Slimak, Puspita Katili, Elizabeth Scott, Mustafa Hassan, Maksymilian Prondzynski, Stephen C Harmer, Andrew Tinker, James G W Smith, Lucie Carrier, Philip M Williams, Daniel Gaffney, Thomas Eschenhagen, Arne Hansen, Chris Denning
Aims: Sarcomeric gene mutations frequently underlie hypertrophic cardiomyopathy (HCM), a prevalent and complex condition leading to left ventricle thickening and heart dysfunction. We evaluated isogenic genome-edited human pluripotent stem cell-cardiomyocytes (hPSC-CM) for their validity to model, and add clarity to, HCM. Methods and results: CRISPR/Cas9 editing produced 11 variants of the HCM-causing mutation c.C9123T-MYH7 [(p.R453C-β-myosin heavy chain (MHC)] in 3 independent hPSC lines...
May 8, 2018: European Heart Journal
https://www.readbyqxmd.com/read/29735912/towards-resolving-the-pro-and-anti-tumor-effects-of-the-aryl-hydrocarbon-receptor
#14
Supraja Narasimhan, Elizabeth Stanford Zulick, Olga Novikov, Ashley J Parks, Jennifer J Schlezinger, Zhongyan Wang, Fabrice Laroche, Hui Feng, Francesca Mulas, Stefano Monti, David H Sherr
We have postulated that the aryl hydrocarbon receptor (AHR) drives the later, more lethal stages of some cancers when chronically activated by endogenous ligands. However, other studies have suggested that, under some circumstances, the AHR can oppose tumor aggression. Resolving this apparent contradiction is critical to the design of AHR-targeted cancer therapeutics. Molecular (siRNA, shRNA, AHR repressor, CRISPR-Cas9) and pharmacological (AHR inhibitors) approaches were used to confirm the hypothesis that AHR inhibition reduces human cancer cell invasion (irregular colony growth in 3D Matrigel cultures and Boyden chambers), migration (scratch wound assay) and metastasis (human cancer cell xenografts in zebrafish)...
May 7, 2018: International Journal of Molecular Sciences
https://www.readbyqxmd.com/read/29734705/a-crispr-cas9-based-toolkit-for-fast-and-precise-in-vivo-genetic-engineering-of-bacillus-subtilis-phages
#15
Tobias Schilling, Sascha Dietrich, Michael Hoppert, Robert Hertel
Phages are currently under discussion as a solution for the antibiotic crisis, as they may cure diseases caused by multi-drug-resistant pathogens. However, knowledge of phage biology and genetics is limited, which impedes risk assessment of therapeutic applications. In order to enable advances in phage genetic research, the aim of this work was to create a toolkit for simple and fast genetic engineering of phages recruiting Bacillus subtilis as host system. The model organism B. subtilis represents a non-pathogenic surrogate of its harmful relatives, such as Bacillus anthracis or Bacillus cereus ...
May 4, 2018: Viruses
https://www.readbyqxmd.com/read/29732484/crispr-cas9-the-powerful-new-genome-editing-tool-for-putative-therapeutics-in-obesity
#16
REVIEW
María José Franco-Tormo, Mireille Salas-Crisostomo, Nuno Barbosa Rocha, Henning Budde, Sérgio Machado, Eric Murillo-Rodríguez
The molecular technology known as clustered regularly interspaced palindromic repeats (CRISPR)/CRISPR-associated protein (Cas) is revolutionizing the field of medical research and deepening our understanding of numerous biological processes. The attraction of CRISPR/Cas9 lies in its ability to efficiently edit DNA or modulate gene expression in living eukaryotic cells and organisms, a technology that was once considered either too expensive or scientifically risky. CRISPR/Cas9 has been successfully applied in agriculture to develop the next generation of disease-resistant plants...
May 7, 2018: Journal of Molecular Neuroscience: MN
https://www.readbyqxmd.com/read/29731717/innovative-therapeutic-strategies-for-cystic-fibrosis-moving-forward-to-crispr-technique
#17
REVIEW
Michele Marangi, Giuseppa Pistritto
One of the most revolutionary technologies in recent years in the field of molecular biology is CRISPR-Cas9. CRISPR technology is a promising tool for gene editing that provides researchers the opportunity to easily alter DNA sequences and modify gene function. Its many potential applications include correcting genetic defects, treating and preventing the spread of diseases. Cystic fibrosis (CF) is one of the most common lethal genetic diseases caused by mutations in the CF transmembrane conductance regulator (CFTR) gene...
2018: Frontiers in Pharmacology
https://www.readbyqxmd.com/read/29731431/large-scale-expansion-of-human-ipsc-derived-skeletal-muscle-cells-for-disease-modeling-and-cell-based-therapeutic-strategies
#18
Erik van der Wal, Pablo Herrero-Hernandez, Raymond Wan, Mike Broeders, Stijn L M In 't Groen, Tom J M van Gestel, Wilfred F J van IJcken, Tom H Cheung, Ans T van der Ploeg, Gerben J Schaaf, W W M Pim Pijnappel
Although skeletal muscle cells can be generated from human induced pluripotent stem cells (iPSCs), transgene-free protocols include only limited options for their purification and expansion. In this study, we found that fluorescence-activated cell sorting-purified myogenic progenitors generated from healthy controls and Pompe disease iPSCs can be robustly expanded as much as 5 × 1011 -fold. At all steps during expansion, cells could be cryopreserved or differentiated into myotubes with a high fusion index...
April 28, 2018: Stem Cell Reports
https://www.readbyqxmd.com/read/29725684/systemic-delivery-of-crispr-cas9-with-peg-plga-nanoparticles-for-chronic-myeloid-leukemia-targeted-therapy
#19
Yang Liu, Gui Zhao, Cong-Fei Xu, Ying-Li Luo, Zi-Dong Lu, Jun Wang
Chronic myeloid leukemia (CML), which is characterized by the Philadelphia translocation, which fuses breakpoint cluster region (BCR) sequences from chromosome 22 upstream of the Abelson murine leukemia viral oncogene homolog (ABL) on chromosome 9, requires specific and efficient treatment. The CRISPR/Cas9 system, with its mechanism of specific DNA complementary recognition by engineered guide RNA (gRNA), allows the development of novel therapeutics for CML. To achieve targeted therapy of CML with the CRISPR/Cas9 system, we encapsulated a CRISPR/Cas9 plasmid (pCas9) expressing gRNA targeting the overhanging fusion region of the BCR-ABL gene (pCas9/gBCR-ABL) with poly(ethylene glycol)-b-poly(lactic acid-co-glycolic acid) (PEG-PLGA)-based cationic lipid-assisted polymeric nanoparticles (CLANs), which specifically disrupted the CML-related BCR-ABL gene while sparing the BCR and ABL genes in normal cells...
May 4, 2018: Biomaterials Science
https://www.readbyqxmd.com/read/29723758/optimization-of-lipid-assisted-nanoparticle-for-disturbing-neutrophils-related-inflammation
#20
Yang Liu, Zhi-Ting Cao, Cong-Fei Xu, Zi-Dong Lu, Ying-Li Luo, Jun Wang
Inflammation is closely related to the development of many diseases and is commonly characterized by abnormal infiltration of immune cells, especially neutrophils. The current therapeutics of inflammatory diseases give little attention to direct modulation of these diseases with respect to immune cells. Nanoparticles are applied for efficient drug delivery into the disease-related immune cells, but their performance is significantly affected by their surface properties. In this study, to optimize the properties of nanoparticles for modulating neutrophils-related inflammation, we prepared a library of poly(ethylene glycol)-b-poly(lactide-co-glycolide) (PEG-b-PLGA)-based cationic lipid-assisted nanoparticles (CLANs) with different surface PEG density and surface charge...
April 26, 2018: Biomaterials
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