TABLE OF CONTENTS
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January 31 2013, Volume 6 / Issue 4 |
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 | Analysis Cover Story Translational Notes Targets and Mechanisms Public-Private Interface
The Distillery: Therapeutics Autoimmune disease Cancer Cardiovascular disease Endocrine/metabolic disease Genitourinary disease Infectious disease Inflammation Neurology Various
The Distillery: Techniques Drug platforms Assays and screens Disease models |  | Advertisement |  |  |  | BioPharma Dealmakers A supplement to Nature Biotechnology and Nature Reviews Drug Discovery
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SciBX: Science-Business eXchange Recommend SciBX to your library today
SciBx is a weekly publication that identifies and analyzes the most important translational research articles from over 40 journals. Find out which papers have real scientific and commercial potential, and why. Subscribe to SciBX and you won't miss the next big thing.
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Analysis |
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Cover Story | Top |
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CRISPR genome editing Chris Cain doi:10.1038/scibx.2013.77
Five separate research teams have developed a Cas9 endonuclease–based method for sequence-specific genome modification guided by DNA-RNA base pairing. Although the approach is technically more straightforward than existing methods, such as zinc finger nucleases and transcription activator–like effector nucleases, its specificity has yet to be fully determined.
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Translational Notes | Top |
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Translational globetrotting Steve Edelson doi:10.1038/scibx.2013.78
Six like-minded translational research centers have partnered to help speed the commercialization of academic research. The international cadre, dubbed the Global Alliance of Leading Drug Discovery and Development Centres, comprises more than 400 drug developers working on at least 165 projects.
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Targets and Mechanisms | Top |
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Toll-erating AD Lev Osherovich doi:10.1038/scibx.2013.79
Laval University and GlaxoSmithKline have shown that peripheral administration of the adjuvant MPL can help treat Alzheimer's disease in mice. Although the findings point to a potential immunomodulatory approach for addressing the neurodegenerative disease, the pharma is now focusing on elucidating the mechanism before advancing the program.
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Public-Private Interface | Top |
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Evotec's growing Ivy Kai-Jye Lou doi:10.1038/scibx.2013.80
Evotec is double dipping into the Ivy League, this time partnering with Yale University to move early stage research from the university's labs into late preclinical and IND-ready assets for third parties to license. The Yale collaboration is upstream of the biotech's 2011 deal with Harvard University because the projects are not predefined.
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Distillery: Therapeutics |
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Autoimmune disease | Top |
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Desmoglein 1 (DSG1); DSG3 doi:10.1038/scibx.2013.81
In vitro and mouse studies identified a DSG-binding peptide that could help treat PV.
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Cancer | Top |
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NADP-dependent malic enzyme 1 cytosolic (ME1); NADP-dependent malic enzyme 2 mitochondrial (ME2); p53 doi:10.1038/scibx.2013.82
Cell culture and mouse studies suggest inhibiting ME1 or ME2 may help treat cancer.
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NGFI-A binding protein 2 (EGR1 binding protein 2; NAB2); signal transducer and activator of transcription 6 (STAT6) doi:10.1038/scibx.2013.83
Two separate genetic sequencing studies identified a NAB2-STAT6 gene fusion associated with solitary fibrous tumors (SFTs), which could provide a new therapeutic target.
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BCR-ABL tyrosine kinase doi:10.1038/scibx.2013.84
Cell culture and mouse studies suggest the BCR-ABL tyrosine kinase inhibitor GZD824 could help treat imatinib-resistant cancer.
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VEGF doi:10.1038/scibx.2013.85
Mouse and human studies suggest Votrient pazopanib, a VEGF signaling inhibitor, could help improve the efficacy of paclitaxel in anaplastic thyroid cancer.
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Cardiovascular disease | Top |
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POU class 1 homeobox 1 (POU1F1; PIT1) doi:10.1038/scibx.2013.86
Cell culture and mouse studies suggest spironolactone could help prevent calcification of vascular and other soft tissues, which is seen in patients who have chronic kidney disease (CKD).
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Endocrine/metabolic disease | Top |
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Free fatty acid receptor 1 (FFAR1; GPR40) doi:10.1038/scibx.2013.87
In vitro and mouse studies identified an alkyne series of FFAR1 agonists that could help treat type 2 diabetes.
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Genitourinary disease | Top |
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Prolactin releasing hormone receptor (PRLHR; GPR10) doi:10.1038/scibx.2013.88
Patient sample and mouse studies suggest inhibiting PRLHR could help treat uterine fibroids.
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Infectious disease | Top |
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Lysine-specific demethylase 4 (KDM4; JMJD2) doi:10.1038/scibx.2013.89
Cell culture and explanted mouse ganglia studies suggest JMJD2 inhibitors could be used to treat primary HSV-1 infection and prevent reactivation.
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Influenza A virus matrix protein 2 (M2) doi:10.1038/scibx.2013.90
In vitro studies identified M2 inhibitors that could help treat amantadine-resistant influenza.
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Inflammation | Top |
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Leptin doi:10.1038/scibx.2013.91
Mouse studies suggest anticholinergic compounds could help treat obesity-induced asthma.
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Neurology | Top |
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Histone deacetylase 3 (HDAC3) doi:10.1038/scibx.2013.92
Mouse studies suggest HDAC3 inhibition could help treat cocaine addiction.
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Toll-like receptor 4 (TLR4) doi:10.1038/scibx.2013.93
Cell culture and mouse studies suggest moderately agonizing TLR4 could help treat AD.
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Hypoxia-inducible factor prolyl hydroxylase (HIF-PH; EGLN); hypoxia-inducible factor 1α (HIF1A; HIF1α) doi:10.1038/scibx.2013.94
Human and rabbit studies suggest compounds that inhibit HIF-PH could prevent neurogenesis deficits in preterm infants.
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Various | Top |
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Caspase recruitment domain family member 15 (CARD15; NOD2) doi:10.1038/scibx.2013.95
Mouse studies suggest intestinal microbiota from organisms with functional NOD2 could help protect against colitis and colitis-associated colorectal cancer.
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Distillery: Techniques |
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Assays and screens | Top |
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DNA analysis of Pap smear samples to detect ovarian and endometrial cancer doi:10.1038/scibx.2013.96
DNA sequencing of Pap smear samples could help diagnose ovarian and endometrial cancer.
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Disease models | Top |
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Induced pluripotent stem (iPS) cell–derived models of familial hypertrophic cardiomyopathy (HCM) doi:10.1038/scibx.2013.97
Patient-derived iPS cell models of familial HCM could help identify new treatments for the disease.
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Mouse model for lymphocytic choriomeningitis virus (LCMV) infection that produces hemorrhagic-like disease doi:10.1038/scibx.2013.98
A new mouse model could be used to screen for therapeutics or test vaccines against arenavirus infections that cause hemorrhagic-like disease.
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Transgenic miniature pig model for hypercholesterolemia and atherosclerosis doi:10.1038/scibx.2013.99
A transgenic miniature pig model for hypercholesterolemia and atherosclerosis could aid the development of imaging technologies, intravascular devices and therapeutics.
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Drug platforms | Top |
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Antigen-specific T cells generated from induced pluripotent stem (iPS) cells derived for adoptive immunotherapy doi:10.1038/scibx.2013.100
Two separate groups showed that iPS cells derived from mature CD8+ T cells can be differentiated into antigen-specific T cells that may be useful for overcoming T cell exhaustion during adoptive immunotherapy.
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Clustered, regularly interspaced short palindromic repeats (CRISPR) RNA editing system to modify mammalian DNA doi:10.1038/scibx.2013.101
A CRISPR-derived genome editing system could be used to modify mammalian DNA.
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Sortase-mediated modification of a targeted mAb to improve antigen delivery and presentation doi:10.1038/scibx.2013.102
Sortase-mediated modification of therapeutic mAbs could be useful for developing vaccines and therapeutics to treat viral infections.
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Structure-based design of peptide inhibitors and activators of ubiquitin enzymes doi:10.1038/scibx.2013.103
In vitro studies identified a strategy to develop peptide inhibitors and activators of ubiquitin pathway enzymes that could help treat various diseases.
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Terminally differentiated stem cell–derived cells with negligible immunogenicity doi:10.1038/scibx.2013.104
Terminally differentiated stem cell–derived cells show negligible immunogenicity, suggesting they could be useful for transplant and cell therapy applications.
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