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https://www.readbyqxmd.com/read/29149602/specification-of-physiologic-and-disease-states-by-distinct-proteins-and-protein-conformations
#1
REVIEW
Daniel F Jarosz, Vikram Khurana
Protein conformational states-from intrinsically disordered ensembles to amyloids that underlie the self-templating, infectious properties of prion-like proteins-have attracted much attention. Here, we highlight the diversity, including differences in biophysical properties, that drive distinct biological functions and pathologies among self-templating proteins. Advances in chemical genomics, gene editing, and model systems now permit deconstruction of the complex interplay between these protein states and the host factors that react to them...
November 16, 2017: Cell
https://www.readbyqxmd.com/read/29148872/disease-context-and-gene-editing
#2
Clive E Bowman
No abstract text is available yet for this article.
November 2017: Journal of the Royal Society of Medicine
https://www.readbyqxmd.com/read/29141166/designing-ethical-trials-of-germline-gene-editing
#3
Bryan Cwik
The recent announcement that researchers have successfully used the CRISPR (clustered regularly interspaced short palindromic repeats) gene-editing technique to correct a mutation that leads to hypertrophic cardiomyopathy in human embryos is the latest reminder of the urgency of the social and..
November 16, 2017: New England Journal of Medicine
https://www.readbyqxmd.com/read/29141136/systemic-mrna-delivery-to-the-lungs-by-functional-polyester-based-carriers
#4
Yunfeng Yan, Hu Xiong, Xinyi Zhang, Qiang Cheng, Daniel John Siegwart
Messenger RNA (mRNA) has recently come into focus as an emerging therapeutic class with great potential for protein replacement therapy, cancer immunotherapy, regenerative medicine, vaccines, and gene editing. However, the lack of effective and safe delivery methods impedes the broad application of mRNA-based therapeutics. Here, we report a robust approach to develop efficient polymeric delivery carriers for mRNA. Lead polyesters were identified by in vitro screening of a 480-member combinatorially modified poly(trimethylolpropane allyl ether-co-suberoyl chloride) library for the delivery of luciferase encoding mRNA (Luc mRNA) to IGROV1 cells...
November 15, 2017: Biomacromolecules
https://www.readbyqxmd.com/read/29140568/crispr-cas9-based-safe-harbor-gene-editing-in-rhesus-ipscs
#5
Ravi Chandra Yada, John W Ostrominski, Ilker Tunc, So Gun Hong, Jizhong Zou, Cynthia E Dunbar
NHP iPSCs provide a unique opportunity to test safety and efficacy of iPSC-derived therapies in clinically relevant NHP models. To monitor these cells in vivo, there is a need for safe and efficient labeling methods. Gene insertion into genomic safe harbors (GSHs) supports reliable transgene expression while minimizing the risk the modification poses to the host genome or target cell. Specifically, this protocol demonstrates targeting of the adeno-associated virus site 1 (AAVS1), one of the most widely used GSH loci in the human genome, with CRISPR/Cas9, allowing targeted marker or therapeutic gene insertion in rhesus macaque induced pluripotent stem cells (RhiPSCs)...
November 15, 2017: Current Protocols in Stem Cell Biology
https://www.readbyqxmd.com/read/29138405/ca-2-signals-initiate-at-immobile-ip3-receptors-adjacent-to-er-plasma-membrane-junctions
#6
Nagendra Babu Thillaiappan, Alap P Chavda, Stephen C Tovey, David L Prole, Colin W Taylor
IP3 receptors (IP3Rs) release Ca(2+) from the ER when they bind IP3 and Ca(2+). The spatial organization of IP3Rs determines both the propagation of Ca(2+) signals between IP3Rs and the selective regulation of cellular responses. Here we use gene editing to fluorescently tag endogenous IP3Rs, and super-resolution microscopy to determine the geography of IP3Rs and Ca(2+) signals within living cells. We show that native IP3Rs cluster within ER membranes. Most IP3R clusters are mobile, moved by diffusion and microtubule motors...
November 15, 2017: Nature Communications
https://www.readbyqxmd.com/read/29137643/efficient-gene-editing-in-corynebacterium-glutamicum-using-the-crispr-cas9-system
#7
Feng Peng, Xinyue Wang, Yang Sun, Guibin Dong, Yankun Yang, Xiuxia Liu, Zhonghu Bai
BACKGROUND: Corynebacterium glutamicum (C. glutamicum) has traditionally been used as a microbial cell factory for the industrial production of many amino acids and other industrially important commodities. C. glutamicum has recently been established as a host for recombinant protein expression; however, some intrinsic disadvantages could be improved by genetic modification. Gene editing techniques, such as deletion, insertion, or replacement, are important tools for modifying chromosomes...
November 14, 2017: Microbial Cell Factories
https://www.readbyqxmd.com/read/29133817/transgenic-cavendish-bananas-with-resistance-to-fusarium-wilt-tropical-race-4
#8
James Dale, Anthony James, Jean-Yves Paul, Harjeet Khanna, Mark Smith, Santy Peraza-Echeverria, Fernando Garcia-Bastidas, Gert Kema, Peter Waterhouse, Kerrie Mengersen, Robert Harding
Banana (Musa spp.) is a staple food for more than 400 million people. Over 40% of world production and virtually all the export trade is based on Cavendish banana. However, Cavendish banana is under threat from a virulent fungus, Fusarium oxysporum f. sp. cubense tropical race 4 (TR4) for which no acceptable resistant replacement has been identified. Here we report the identification of transgenic Cavendish with resistance to TR4. In our 3-year field trial, two lines of transgenic Cavendish, one transformed with RGA2, a gene isolated from a TR4-resistant diploid banana, and the other with a nematode-derived gene, Ced9, remain disease free...
November 14, 2017: Nature Communications
https://www.readbyqxmd.com/read/29133816/disruption-of-diphthamide-synthesis-genes-and-resulting-toxin-resistance-as-a-robust-technology-for-quantifying-and-optimizing-crispr-cas9-mediated-gene-editing
#9
Tobias Killian, Steffen Dickopf, Alexander K Haas, Claudia Kirstenpfad, Klaus Mayer, Ulrich Brinkmann
We have devised an effective and robust method for the characterization of gene-editing events. The efficacy of editing-mediated mono- and bi-allelic gene inactivation and integration events is quantified based on colony counts. The combination of diphtheria toxin (DT) and puromycin (PM) selection enables analyses of 10,000-100,000 individual cells, assessing hundreds of clones with inactivated genes per experiment. Mono- and bi-allelic gene inactivation is differentiated by DT resistance, which occurs only upon bi-allelic inactivation...
November 13, 2017: Scientific Reports
https://www.readbyqxmd.com/read/29130152/combining-engineered-nucleases-with-adeno-associated-viral-vectors-for-therapeutic-gene-editing
#10
Benjamin E Epstein, David V Schaffer
With the recent advent of several generations of targeted DNA nucleases, most recently CRISPR/Cas9, genome editing has become broadly accessible across the biomedical community. Importantly, the capacity of these nucleases to modify specific genomic loci associated with human disease could render new classes of genetic disease, including autosomal dominant or even idiopathic disease, accessible to gene therapy. In parallel, the emergence of adeno-associated virus (AAV) as a clinically important vector raises the possibility of integrating these two technologies towards the development of gene editing therapies...
2017: Advances in Experimental Medicine and Biology
https://www.readbyqxmd.com/read/29130151/viral-vectors-engineered-cells-and-the-crispr-revolution
#11
James E DiCarlo, Anurag Deeconda, Stephen H Tsang
Over the past few decades the ability to edit human cells has revolutionized modern biology and medicine. With advances in genome editing methodologies, gene delivery and cell-based therapeutics targeted at treatment of genetic disease have become a reality that will become more and more essential in clinical practice. Modifying specific mutations in eukaryotic cells using CRISPR-Cas systems derived from prokaryotic immune systems has allowed for precision in correcting various disease mutations. Furthermore, delivery of genetic payloads by employing viral tropism has become a crucial and effective mechanism for delivering genes and gene editing systems into cells...
2017: Advances in Experimental Medicine and Biology
https://www.readbyqxmd.com/read/29129659/generation-of-conditional-acvrl1-knockout-mice-by-crispr-cas9-mediated-gene-targeting
#12
Ming Xu, Hongzhi Xu, Jian Chen, Chunjui Chen, Feng Xu, Zhiyong Qin
OBJECTIVES: This study aimed to generate mutant mice containing the Acvrl1 gene flanked with LoxP sequences to allow conditional deletion ofAcvrl1 by the LoxP/Cre system. Such mice may facilitate the development of brain arteriovenous malformation (BAVM) models. METHODS: The CRISPR/Cas9 technique was used to edit Acvrl1. Two single guide RNAs (sgRNAs) with recognition sites on intron 3 and 8 and a donor vector that was homologous with the targeted gene and contained two LoxP sequences were designed and constructed...
November 9, 2017: Molecular and Cellular Probes
https://www.readbyqxmd.com/read/29128367/nemo-links-nuclear-factor-%C3%AE%C2%BAb-to-human-diseases
#13
REVIEW
Gunter Maubach, Michael Naumann
The nuclear factor (NF)-κB essential modulator (NEMO) is a key regulator in NF-κB-mediated signaling. By transmitting extracellular or intracellular signals, NEMO can control NF-κB-regulated genes. NEMO dysfunction is associated with inherited diseases such as incontinentia pigmenti (IP), ectodermal dysplasia, anhidrotic, with immunodeficiency (EDA-ID), and some cancers. We focus on molecular studies, human case reports, and mouse models emphasizing the significance of NEMO molecular interactions and modifications in health and diseases...
November 8, 2017: Trends in Molecular Medicine
https://www.readbyqxmd.com/read/29124278/targeted-gene-editing-in-human-pluripotent-stem-cells-using-site-specific-nucleases
#14
Sylvia Merkert, Ulrich Martin
Introduction of induced pluripotent stem cell (iPSC) technology and site-directed nucleases brought a major breakthrough in the development of regenerative therapies and biomedical research. With the advancement of ZFNs, TALENs, and the CRISPR/Cas9 technology, straightforward and precise manipulation of the genome of human pluripotent stem cells (PSC) became possible, allowing relatively easy and fast generation of gene knockouts, integration of transgenes, or even introduction of single nucleotide changes for correction or introduction of disease-specific mutations...
November 10, 2017: Advances in Biochemical Engineering/biotechnology
https://www.readbyqxmd.com/read/29124055/regulatory-and-scientific-advancements-in-gene-therapy-state-of-the-art-of-clinical-applications-and-of-the-supporting-european-regulatory-framework
#15
REVIEW
Marta Carvalho, Bruno Sepodes, Ana Paula Martins
Advanced therapy medicinal products (ATMPs) have a massive potential to address existing unmet medical needs. Specifically, gene therapy medicinal products (GTMPs) may potentially provide cure for several genetic diseases. In Europe, the ATMP regulation was fully implemented in 2009 and, at this point, the Committee for Advanced Therapies was created as a dedicated group of specialists to evaluate medicinal products requiring specific expertise in this area. To date, there are three authorized GTMPs, and the first one was approved in 2012...
2017: Frontiers in Medicine
https://www.readbyqxmd.com/read/29123113/talen-mediated-functional-correction-of-human-ipsc-derived-macrophages-in-context-of-hereditary-pulmonary-alveolar-proteinosis
#16
Alexandra Kuhn, Mania Ackermann, Claudio Mussolino, Toni Cathomen, Nico Lachmann, Thomas Moritz
Hereditary pulmonary alveolar proteinosis (herPAP) constitutes a rare, life threatening lung disease characterized by the inability of alveolar macrophages to clear the alveolar airspaces from surfactant phospholipids. On a molecular level, the disorder is defined by a defect in the CSF2RA gene coding for the GM-CSF receptor alpha-chain (CD116). As therapeutic options are limited, we currently pursue a cell and gene therapy approach aiming for the intrapulmonary transplantation of gene-corrected macrophages derived from herPAP-specific induced pluripotent stem cells (herPAP-iPSC) employing transcriptional activator-like effector nucleases (TALENs)...
November 9, 2017: Scientific Reports
https://www.readbyqxmd.com/read/29122734/gene-editing-of-mps-i-human-fibroblasts-by-co-delivery-of-a-crispr-cas9-plasmid-and-a-donor-oligonucleotide-using-nanoemulsions-as-nonviral-carriers
#17
Roselena Silvestri Schuh, Talita Giacomet de Carvalho, Roberto Giugliani, Ursula Matte, Guilherme Baldo, Helder Ferreira Teixeira
Mucopolysaccharidosis type I (MPS I) is an inherited disease caused by the deficiency of alpha-L-iduronidase (IDUA). This study shows the use of nanoemulsions co-complexed with the plasmid of CRISPR/Cas9 system and a donor oligonucleotide aiming at MPS I gene editing in vitro. Nanoemulsions composed of MCT, DOPE, DOTAP, DSPE-PEG, and water were prepared by high-pressure homogenization. The DNA was complexed by adsorption or encapsulation of preformed DNA/DOTAP complexes with nanoemulsions at +4/ -1 charge ratio...
November 6, 2017: European Journal of Pharmaceutics and Biopharmaceutics
https://www.readbyqxmd.com/read/29122551/crispr-mediated-deletion-of-the-pecam-1-cytoplasmic-domain-increases-receptor-lateral-mobility-and-strengthens-endothelial-cell-junctional-integrity
#18
Danying Liao, Heng Mei, Yu Hu, Debra K Newman, Peter J Newman
AIMS: PECAM-1 is an abundant endothelial cell surface receptor that becomes highly enriched at endothelial cell-cell junctions, where it functions to mediate leukocyte transendothelial migration, sense changes in shear and flow, and maintain the vascular permeability barrier. Homophilic interactions mediated by the PECAM-1 extracellular domain are known to be required for PECAM-1 to perform these functions; however, much less is understood about the role of its cytoplasmic domain in these processes...
November 6, 2017: Life Sciences
https://www.readbyqxmd.com/read/29121043/the-poverty-related-neglected-diseases-why-basic-research-matters
#19
Peter J Hotez
Together, malaria and the neglected tropical diseases (NTDs) kill more than 800,000 people annually, while creating long-term disability in millions more. International support for mass drug administration, bed nets, and other preventive measures has resulted in huge public health gains, while support for translational research is leading to the development of some new neglected disease drugs, diagnostics, and vaccines. However, funding for basic science research has not kept up, such that we are missing opportunities to create a more innovative pipeline of control tools for parasitic and related diseases...
November 2017: PLoS Biology
https://www.readbyqxmd.com/read/29121022/public-views-on-gene-editing-and-its-uses
#20
George Gaskell, Imre Bard, Agnes Allansdottir, Rui Vieira da Cunha, Peter Eduard, Juergen Hampel, Elisabeth Hildt, Christian Hofmaier, Nicole Kronberger, Sheena Laursen, Anna Meijknecht, Salvör Nordal, Alexandre Quintanilha, Gema Revuelta, Núria Saladié, Judit Sándor, Júlio Borlido Santos, Simone Seyringer, Ilina Singh, Han Somsen, Winnie Toonders, Helge Torgersen, Vincent Torre, Márton Varju, Hub Zwart
No abstract text is available yet for this article.
November 9, 2017: Nature Biotechnology
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