TABLE OF CONTENTS |
June 20 2013, Volume 6 / Issue 24 |
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The Distillery: Therapeutics Autoimmune disease Cancer Cardiovascular disease Dermatology Endocrine/metabolic disease Inflammation Neurology Pulmonary disease
The Distillery: Techniques Disease models Drug platforms Markers
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Analysis |
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Cover Story | Top |
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Interfacing with Ras Amy Donner doi:10.1038/scibx.2013.588
A German team has identified a compound that disrupts a protein-protein interaction that localizes K-Ras to the cell membrane, thus selectively inhibiting tumor growth. The interface provides a new small molecule binding site for the handful of companies and academics working on ways to tackle the previously undruggable Ras family.
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Targets and Mechanisms | Top |
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Rationalizing CF combos Chris Cain doi:10.1038/scibx.2013.589
A McGill University–led team has shown that CFTR corrector compounds target only one of two sequential steps required for proper folding of the mutated protein. The findings suggest there is a clear rationale for developing combinations of correctors to treat patients with cystic fibrosis.
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FGF9 for baldness Lauren Martz doi:10.1038/scibx.2013.590
A University of Pennsylvania team has found that increasing FGF9 levels in wounded skin can promote the growth of hair follicles in mice. Follica has licensed the findings and plans to test the effects of FGF9 in hair growth indications.
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Tools | Top |
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Transferring flu protection Tracey Baas doi:10.1038/scibx.2013.591
Two independent studies provide proof of concept that gene transfer could be used to establish broad protection against influenza. Both groups are working to move their influenza A virus hemagglutinin–expressing vectors into humans.
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Distillery: Therapeutics |
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Autoimmune disease | Top |
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Tyrosine kinase 2 (TYK2) doi:10.1038/scibx.2013.592
In vitro and rodent studies suggest selectively inhibiting TYK2 could help treat IBD and psoriasis.
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Cancer | Top |
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Apolipoprotein A-1 (APOA1) doi:10.1038/scibx.2013.593
Mouse studies suggest APOA1 could help treat solid tumors.
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Eukaryotic translation elongation factor 2 kinase (EEF2K) doi:10.1038/scibx.2013.594
Patient, mouse and cell culture studies suggest inhibiting EEF2K could help improve the efficacy of nutrient deprivation therapy for cancer.
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Neuropilin 2 (NRP2) doi:10.1038/scibx.2013.595
Patient sample and mouse studies suggest inhibiting NRP2 could help prevent cancer metastasis.
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Ninjurin 1 (NINJ1) doi:10.1038/scibx.2013.596
Cell culture studies suggest inhibiting NINJ1 could help treat cancer.
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BRAF doi:10.1038/scibx.2013.597
Patient studies suggest Zelboraf vemurafenib could help treat BRAF V600E mutant MM.
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Cardiovascular disease | Top |
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Dickkopf homolog 1 (DKK1) doi:10.1038/scibx.2013.598
Cell culture studies suggest inhibiting DKK1 could help treat atherosclerosis.
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Complement component 3a receptor 1 (C3AR1; C3AR) doi:10.1038/scibx.2013.599
Mouse studies suggest C3AR agonists could help treat ischemia/reperfusion injury.
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NADH dehydrogenase subunit 3 (ND3; MT-ND3) doi:10.1038/scibx.2013.600
In vitro and mouse studies suggest S-nitrosylation of MT-ND3 could help prevent ischemia/reperfusion injury.
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Dermatology | Top |
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B-type natriuretic peptide (BNP; NPPB); natriuretic peptide receptor A (NPR1; NPRA) doi:10.1038/scibx.2013.601
Mouse studies suggest inhibiting NPPB or NPRA could help treat itch.
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Endocrine/metabolic disease | Top |
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Solute carrier family 10 sodium-dependent bile acid transporter member 2 (SLC10A2; ASBT; IBAT) doi:10.1038/scibx.2013.602
In vitro and rodent studies identified benzothiazepine ASBT inhibitors that could help treat diabetes.
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Branched chain ketoacid dehydrogenase kinase (BCKDK; BDK) doi:10.1038/scibx.2013.603
In vitro and mouse studies identified a BDK inhibitor that could help treat diseases associated with accumulation of branched-chain amino acids (BCAAs), including the genetic disorder branched-chain ketoaciduria, obesity and diabetes.
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Inflammation | Top |
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Leukotriene B4 (LTB4); complement 5 (C5) doi:10.1038/scibx.2013.604
In vitro and mouse studies suggest inhibitors of both C5 and LTB4 could help treat inflammatory diseases.
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Neurology | Top |
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Glyceraldehyde-3-phosphate dehydrogenase (GAPDH) doi:10.1038/scibx.2013.605
Mouse studies suggest omigapil and other inhibitors of GAPDH nitrosylation could help treat cocaine addiction or overdose.
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Semaphorin 4D (SEMA4D) doi:10.1038/scibx.2013.606
Cell and tissue culture studies suggest SEMA4D-Fc could be used to treat epilepsy.
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Dopamine D2 receptor; dopamine D3 receptor; serotonin (5-HT1A) receptor; serotonin (5-HT1B) receptor doi:10.1038/scibx.2013.607
In vitro and rodent studies suggest coumarin piperazine derivatives could be useful for treating psychosis.
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Pulmonary disease | Top |
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Mucin 5B oligomeric mucus/gel-forming (MUC5B) doi:10.1038/scibx.2013.608
Patient studies suggest the rs35705950 SNP in the MUC5B promoter region could be a marker of interstitial lung disease risk.
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Distillery: Techniques |
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Disease models | Top |
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A microchip platform to model heart failure doi:10.1038/scibx.2013.609
A microchip platform could be used to model failing myocardium and identify molecules to prevent myocardial infarction (MI) or hypertension.
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Drug platforms | Top |
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Live, attenuated influenza A virus with suppressed hemagglutinin (HA) and neuraminidase (NA) expression as a vaccine doi:10.1038/scibx.2013.610
Live, attenuated influenza A virus with suppressed HA and NA expression could be used to develop vaccines.
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mAb antagonists against glucose-dependent insulinotropic polypeptide receptor (GIPR) doi:10.1038/scibx.2013.611
mAb antagonists against GIPR could be useful as tools to develop improved inhibitors and study receptor function in human disease.
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Self-assembling influenza nanoparticle vaccines doi:10.1038/scibx.2013.612
Self-assembling influenza nanoparticles could be used as vaccines to induce broadly neutralizing antibodies.
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Markers | Top |
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Nuclear localization status of nuclear factor (erythroid-derived 2)-like 2 (NFE2L2; NRF2) to distinguish essential thrombocythemia (ET) and primary myelofibrosis (MF) doi:10.1038/scibx.2013.613
The nuclear localization status of NRF2 could help differentiate between the myeloproliferative disorders ET and primary MF.
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Peptidyl arginine deiminase type III (PADI3; PAD3) autoantibodies as a prognostic marker for rheumatoid arthritis (RA) doi:10.1038/scibx.2013.614
Patient sample studies suggest autoantibodies that recognize PAD3 and activate PAD4 (PADI4) could be used to identify patients at risk for radiographic progression in RA.
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