TABLE OF CONTENTS
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October 11 2012, Volume 5 / Issue 40 |
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 | Analysis Cover Story Translational Notes Targets and Mechanisms
The Distillery: Therapeutics Cancer Cardiovascular disease Endocrine/metabolic disease Hematology Infectious disease Musculoskeletal disease Neurology
The Distillery: Techniques Assays and screens Chemistry Disease models Drug delivery Drug platforms Imaging Markers | |
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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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Ember: warming up to brown fat Michael J. Haas doi:10.1038/scibx.2012.1045
Boston-area researchers have shown that inhibiting a cation channel dubbed TRPV4 induced white fat cells to behave like brown fat, thereby protecting mice from diet-induced obesity and insulin resistance. Ember Therapeutics has options to in-license the findings as a part of a trio of new deals for brown fat–related targets.
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Translational Notes | Top |
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Leukemia team building Chris Cain doi:10.1038/scibx.2012.1046
The Leukemia & Lymphoma Society has announced its first research partnership with a large biopharma company—a four-year, multimillion-dollar deal with Celgene to solicit and fund proposals from academics and smaller biotechs. The not-for-profit organization expects the deal's framework to serve as a template for future collaborations and is in talks with additional big biotech and pharma companies.
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Targets and Mechanisms | Top |
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Closing time for HCV protease Kai-Jye Lou doi:10.1038/scibx.2012.1047
Astex has used its fragment-based drug discovery platform to identify a new allosteric binding site on the full-length HCV NS3/4A protein and thinks compounds that bind the site can inhibit the protein's helicase activity in addition to its proteolytic activity. The result could be molecules with better efficacy than existing HCV drugs that target the protease active site.
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Repurposing naratriptan Lauren Martz doi:10.1038/scibx.2012.1048
Japanese researchers have shown in mice that GlaxoSmithKline's marketed migraine drug Amerge naratriptan could be repurposed to treat spinal and bulbar muscular atrophy. Although the pharma is not saying if it will pursue the new indication for the serotonin (5-HT1D) receptor agonist, the research group is already planning an investigator-led Phase II trial.
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Distillery: Therapeutics |
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Cancer | Top |
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Pyruvate kinase M2 isozyme (PKM2) doi:10.1038/scibx.2012.1049
Cell culture and mouse studies suggest PKM2 activators could help treat cancer.
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Cardiovascular disease | Top |
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Anoctamin 6 (ANO6; TMEM16F) doi:10.1038/scibx.2012.1050
Mouse and cell culture studies suggest inhibiting TMEM16F could help prevent thrombosis.
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Endocrine/metabolic disease | Top |
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α1-Antitrypsin (AAT; A1AT; SERPINA1); histone deacetylase 7 (HDAC7) doi:10.1038/scibx.2012.1051
In vitro studies suggest suberoylanilide hydroxamic acid (SAHA) could help treat AAT deficiency.
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Transient receptor potential vanilloid 4 (TRPV4; VRL2) doi:10.1038/scibx.2012.1052
A study in cell culture and in mice suggests antagonizing TRPV4 could help treat obesity and type 2 diabetes.
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Hematology | Top |
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Factor IX; factor Xa doi:10.1038/scibx.2012.1053
In vitro and monkey studies identified a bifunctional antibody targeting factor IX and factor Xa that could help treat hemophilia A.
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Infectious disease | Top |
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GTP pyrophosphokinase (relA) doi:10.1038/scibx.2012.1054
In vitro studies identified an inhibitor of relA that could help treat Gram-positive bacterial infections.
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HCV NS3/4A protein complex doi:10.1038/scibx.2012.1055
Fragment-based screening using crystals of the HCV NS3/4A holoenzyme identified inhibitors of an allosteric site that could help treat HCV infection.
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Musculoskeletal disease | Top |
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Calcitonin gene-related peptide (CGRP) doi:10.1038/scibx.2012.1056
In vitro and mouse studies suggest reducing CGRP expression could help treat spinal and bulbar muscular atrophy (SBMA).
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Neurology | Top |
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β-Site APP-cleaving enzyme 1 (BACE1); β-amyloid 40 doi:10.1038/scibx.2012.1057
Rodent studies suggest fused bicyclic iminopyrimidinone-based BACE1 inhibitors could help treat neurodegenerative diseases such as AD.
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Epidermal growth factor receptor (EGFR) doi:10.1038/scibx.2012.1058
Drosophila and mouse studies suggest EGFR inhibitors could help treat AD.
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Superoxide dismutase 1 (SOD1) doi:10.1038/scibx.2012.1059
Mouse and worm studies suggest a new class of carbazoles could help treat ALS and PD.
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Not applicable doi:10.1038/scibx.2012.1060
Rat studies suggest ceria nanoparticles could help treat stroke.
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Distillery: Techniques |
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Assays and screens | Top |
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NMR of hyperpolarized fluorine to characterize ligand-protein interactions doi:10.1038/scibx.2012.1061
Hyperpolarized fluorine could improve the sensitivity of fluorine-based NMR for characterizing ligand-protein interactions.
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Chemistry | Top |
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Vascular catheters modified with polysulfobetaine to prevent thrombus formation and microbial attachment doi:10.1038/scibx.2012.1062
Polysulfobetaine surface modification of vascular catheters could help prevent catheter-associated thrombosis and infection.
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Disease models | Top |
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Mice with mesodermal inactivation of Pten (Mmac1; Tep1) as models for alveolar capillary dysplasia (ACD) doi:10.1038/scibx.2012.1063
Mice with mesodermal inactivation of Pten could help identify new treatments for ACD, a congenitally lethal condition characterized by disordered pulmonary vascular development.
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Drug delivery | Top |
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β-Galactosidase–responsive prodrugs of chemotherapeutics targeted to cancer surface receptors doi:10.1038/scibx.2012.1064
In vitro and mouse studies suggest tumor-penetrating, β-galactosidase–responsive prodrugs could help treat cancers.
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Drug platforms | Top |
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Cell or tissue transplantation to restore organ functions doi:10.1038/scibx.2012.1065
Mouse studies suggest transplantation of cells into lymph nodes could rescue organ function.
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Imaging | Top |
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Functionalized multiwalled carbon nanotubes as contrast agents for ultrasound imaging doi:10.1038/scibx.2012.1066
Functionalized multiwalled carbon nanotubes could be used as contrast agents to enhance ultrasound imaging.
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Markers | Top |
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Fibroblast growth factor receptor 1 (FGFR1; CD331), FGFR2 and fibroblast growth factor 19 (FGF19) amplification may help predict sensitivity to FGFR inhibitors in cancer doi:10.1038/scibx.2012.1067
FGFR1, FGFR2 and FGF19 amplification may help predict sensitivity to FGFR inhibitors. In breast, lung and osteosarcoma cell lines and primary human osteosarcoma samples, FGFR1 amplification was associated with sensitivity to the pan-FGFR inhibitor NVP-BGJ398.
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Fibroblast growth factor receptor 4 (FGFR4; CD334) SNP as a response marker for pancreatic neuroendocrine tumors doi:10.1038/scibx.2012.1068
A SNP in FGFR4 could be used as a marker to predict pancreatic neuroendocrine tumor progression and response to mammalian target of rapamycin (mTOR; FRAP; RAFT1) inhibitors.
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Mutations in v-ski sarcoma viral oncogene homolog (SKI) as the cause of Shprintzen-Goldberg syndrome (SGS) with aortic aneurysm doi:10.1038/scibx.2012.1069
Genetic studies suggest mutations in SKI cause SGS with aortic aneurysm, a connective tissue disorder with unknown etiology.
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RNA profiling to determine prognosis in multiple sclerosis (MS) doi:10.1038/scibx.2012.1070
Studies of patient samples identified a transcriptional signature that could help determine MS prognosis.
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