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Cas9 driven by an optimal promoter improves gene editing in eukaryotic cell lines when paired with synthetic crRNA and tracrRNA
Amanda Haupt, Emily Anderson, Žaklina Strezoska, Hidevaldo Machado, Shawn McClelland, Maren Mayer, Adam Rocker, Annaleen Vermeulen, Amanda Birmingham, Melissa Kelley, Anja Smith

Presented here are results on the efficiency of using synthetic crRNA and tracrRNA to introduce gene editing events when co-transfected with a plasmid expressing Cas9. We explored the use of antibiotic and fluorescence activated cell sorting (FACS) methods for enrichment of cells that have undergone gene editing, and the use of multiple promoters to increase efficiency of gene editing with Cas9 and synthetic tracrRNA and crRNAs.

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Specificity and functionality of microRNA inhibitors
Barbara Robertson, Andrew Dalby, Jon Karpilow, Anastasia Khvorova, Devin Leake and Annaleen Vermeulen

Our findings indicate that features important for natural miRNA target recognition also appear to be important for inhibitor specificity. Understanding the specificity of inhibitors allows for better interpretation of inhibitor activity in endogenous systems.

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Identification of microRNA targets using microRNA modulation techniques and gene expression arrays
Emily M. Anderson, Maren Mayer, Kevin Sullivan, Barbara Robertson, Žaklina Strezoska, Annaleen Vermeulen, and Devin Leake

By examining the overlap of messages down-regulated by miRNA mimics and up-regulated by miRNA inhibitors, we robustly identify miRNA-regulated messages, many of which have canonical seed matches and some which are not identied by standard target prediction programs.

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siRNA Screening: Development of Hit Stratification Strategies
Žaklina Strezoska, Annaleen Vermeulen, Emily M. Anderson, Anja Smith, Devin Leake

This poster compares different approaches to hit stratification and validation after an initial screen. Standard siRNA reagents deconvoluted from a pooled set of four were compared to a pooled set of four specificity enhanced reagents. High confidence hits were similar. To explore the validity of low confidence hits, a chimeric approach was used whereby a gene-specific seed sequence was introduced into a non-targeting siRNA scaffold. This work resulted in new hit stratification strategies.

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The Chemical Synthesis of Long and Highly Modified RNA using 2'-ACE Chemistry
Xiaoqin Cheng, Kristina Larson, Letitia Kwok, David Mierzejewski, Shawn Begay, Randy Rauen, Kelly Grimsley, Kaizhang He, Michael Delaney, David Kitchen, Amanda Haas, Melissa Kelley, Anja van Brabant Smith

Dharmacon has previously developed a novel RNA synthesis chemistry making RNA synthesis as reliable, accessible and of comparable quality as routinely observed in DNA synthesis.

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Droplet-on-Demand Platform for Biochemical Screening & Drug Discovery
L.D. van Vliet1*, F. Gielen1, A. Sinha2, B.T. Koprowski3, J.B. Edel4, X.Niu5, A.J. deMello3, F. Hollfelder1, & J. Motschman2

To demonstrate droplet on demand applications towards study of biological entities encapsulated in nanoliter droplets. Interfacing a droplet on demand platform with microfluidic chips allows for merging and dilution of droplets. This feature is applied to encapsulate yeast cells (S. cerevisiae) and multicellular organisms (C. elegans).

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Direct Targets Identification of a Bioactive Compound
Sylvain Blanc, Paul Bradley, Marie-Edith Gourdel, Michael Cholay, Gisèle Guimèse, Mike Mckenzie, George Nasi, Jean-Christophe and Barbara Ruggiero

Identifying protein partners of a small bioactive molecule is of great
interest in many aspects of life sciences and specifically in the drug
discovery and development process cycle. It is a support to (i) decipher
the mechanism of action after for example a “High Content” screening,
(ii) study “off-target” effects, (iii) adjust therapeutic indications and
clinical regimens of a drug and (iv) consider drug repositioning.

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3D-Tissue/ Whole-blood Co-culture Models Combined with Multi-Analyte Profile (MAP) Analyses for In-vivo-like Immunopharmacology
Stein GM, Joos T, Schmolz M

Human Organotypic Test (HOT) Systems aim at in-vivo like substance characterisation of all preparations meant to act on the human immune system.

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A multiplexed amplicon sequencing technology for FFPE and circulating, cell-free DNA
Laurie Kurihara, Catherine Couture, Julie Laliberte, Sukhinder Sandhu, Jonathan Irish, Tim Harkins and Vladimir Makarov

A novel amplicon approach allowing for hundreds of amplicons to be multiplexed in a single tube with a two workflow from sample to sequencer.

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Scientific News
Oxitec ‘Self-Limiting Gene’ Offers Hope for Controlling Invasive Moth
A new pesticide-free and environmentally-friendly way to control insect pests has moved ahead with the publication of results showing that Oxitec diamondback moths (DBM) with a ‘self-limiting gene’ can dramatically reduce populations of DBM.
Teeth Reveal Lifetime Exposures to Metals, Toxins
Researchers have identified dental biomarkers to reveal links between early iron exposure and late life brain diseases.
Scientists Identify Schizophrenia’s “Rosetta Stone” Gene
Scientists have identified a critical function of what they believe to be schizophrenia’s “Rosetta Stone” gene that could hold the key to decoding the function of all genes involved in the disease.
Could a simple saliva test detect Alzheimer's?
Researchers have presented findings suggesting that a simple, non-invasive diagnostic for Alzheimer's could be within reach.
New Research Advances Genetic Studies in Wildlife Conservation
‘Next-gen’ DNA sequencing of non-invasively collected hair expands field of conservation genetics.
AncestryDNA and Calico to Research the Genetics of Human Lifespan
Collaboration will analyze family history and genetics to facilitate development of cutting-edge therapeutics.
Gene Variation Identified for Teen Binge-Eating
Researchers have identified a gene variant which can lead to teenage binge eating, they hope that their work will inform the development of future preventative measures.
Adaptimmune's Novel Cancer Therapeutics Show Positive Clinical Trial Results
The company has announced that positive data from its Phase I/II study of its affinity enhanced T-cell receptor (TCR) therapeutic targeting the NY-ESO-1 cancer antigen in patients with multiple myeloma has been published.
Gene Testing Now Allows Precision Medicine for Thoracic Aneurysms
Researchers at the Aortic Institute at Yale have tested the genomes of more than 100 patients with thoracic aortic aneurysms, a potentially lethal condition, and provided genetically personalized care.
Facebook for the Proteome
Researchers have developed a network for describing protein-protein interactions that can then be used to examine protein interactions that may have biological or clinical significance.
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