TABLE OF CONTENTS |
July 19 2012, Volume 5 / Issue 28 |
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The Distillery: Therapeutics Autoimmune disease Cancer Gastrointestinal disease Hematology Infectious disease Musculoskeletal disease Neurology Ophthalmic disease Various
The Distillery: Techniques Assays and screens Computational models Disease models Drug delivery Drug platforms
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Analysis |
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Cover Story | Top |
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PI3Kδ turns schizophrenic Tim Fulmer doi:10.1038/scibx.2012.718
NIH researchers have linked phosphoinositide 3-kinase-δ to neuregulin signaling and shown that inhibiting the kinase improved behavior in rodent models of schizophrenia. The findings could offer a way to finally target the neuregulin pathway.
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Targets and Mechanisms | Top |
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OutFOXing tumors Tracey Baas doi:10.1038/scibx.2012.719
Mount Sinai and Case Western researchers have found a new use for the dopamine receptor antagonist trifluoperazine: restoring the sensitivity of tumors to EGFR inhibitors. The key is trifluoperazine's off-target activity inhibiting the nuclear export of the tumor suppressor FOXO1.
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Getting AGRP on obesity Kai-Jye Lou doi:10.1038/scibx.2012.720
New York researchers have identified GPR17 as a potentially druggable target on AGRP neurons, which are known to regulate factors relevant to obesity. The findings open up a new pathway to mine for therapeutic strategies against a cell population for which earlier targeting strategies have had little success.
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Tools | Top |
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SEAing change in R&D Kai-Jye Lou doi:10.1038/scibx.2012.721
UCSF, SeaChange and Novartis have developed a computational approach for large-scale, automated prediction of binding interactions between small molecules and targets that have been associated with adverse drug reactions. The method could help companies improve R&D productivity by pointing to safety signals and helping prioritize candidates in silico.
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Distillery: Therapeutics |
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Autoimmune disease | Top |
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Guanine nucleotide binding protein-like 3 (GNL3; nucleostemin) doi:10.1038/scibx.2012.722
Genomewide association studies identified mutations in the GNL3 gene that could help predict susceptibility to osteoarthritis.
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Cancer | Top |
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Death effector domain containing (DEDD) doi:10.1038/scibx.2012.723
Mouse and patient studies suggest enhancing DEDD signaling could help treat and prevent cancer metastasis.
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Signal transducer and activator of transcription 3 (STAT3) doi:10.1038/scibx.2012.724
In vitro and mouse studies suggest inhibiting STAT3 could enhance antitumor immunity to help treat MCL.
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AXL receptor tyrosine kinase (AXL; UFO) doi:10.1038/scibx.2012.725
In vitro, patient sample and mouse studies suggest inhibiting AXL could help treat epidermal growth factor receptor (EGFR) inhibitor–resistant NSCLC.
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BRAF doi:10.1038/scibx.2012.726
Patient and cell culture studies suggest NSCLC patients with inactivating BRAF mutations may respond to Sprycel dasatinib.
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Jun (AP1) proto-oncogene doi:10.1038/scibx.2012.727
Mouse studies identified a Jun mRNA–targeting DNAzyme that could help treat SCC and BCC.
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Gastrointestinal disease | Top |
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Epidermal growth factor receptor (EGFR) doi:10.1038/scibx.2012.728
Mouse and infant studies suggest amniotic fluid could be used to prevent necrotizing enterocolitis (NEC).
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Hematology | Top |
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CD3e molecule-ε CD3-TCR complex (CD3E) doi:10.1038/scibx.2012.729
Mouse studies suggest anti-CD3E mAbs could help treat Omenn syndrome, a form of severe combined immunodeficiency that is associated with mutations in recombination activating genes (RAGs).
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Infectious disease | Top |
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Plasmodium falciparum lysyl-tRNA synthetase (Pfkrs1) doi:10.1038/scibx.2012.730
In vitro and cell culture studies suggest inhibiting Pfkrs1 could help treat liver- and blood-stage malarial infections.
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Musculoskeletal disease | Top |
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Bone morphogenetic protein 2 (BMP2); BMP4; bone morphogenetic protein receptor type IA (BMPR1A) doi:10.1038/scibx.2012.731
In vitro and mouse studies suggest a BMPR1A-Fc fusion protein could help treat bone-related disorders such as osteoporosis.
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Neurology | Top |
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Caspase-6 (CASP6; MCH2) doi:10.1038/scibx.2012.732
In vitro and cell culture studies identified a peptide-based inhibitor of CASP6, which is associated with neurodegenerative diseases such as AD and HD.
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Neuritin (NRN) doi:10.1038/scibx.2012.733
Rat studies suggest increasing NRN levels could help treat depression.
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Epoxide hydrolase doi:10.1038/scibx.2012.734
Rat studies suggest inhibitors of soluble epoxide hydrolase could help treat pain associated with diabetic neuropathy.
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Protein kinase B (PKB; PKBA; AKT; AKT1) doi:10.1038/scibx.2012.735
In vitro and mouse studies identified an AKT activator that could help treat neurological conditions including stroke.
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μ-Opioid receptor (OPRM1; MOR) doi:10.1038/scibx.2012.736
Mouse studies identified MOR-activating endomorphin-1 analogs that could help treat pain.
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Phosphoinositide 3-kinase-δ (PI3Kδ) doi:10.1038/scibx.2012.737
In vitro, rat and patient genetic studies suggest inhibitors of PI3Kd could help treat schizophrenia.
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Ophthalmic disease | Top |
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Opticin (OPTC) doi:10.1038/scibx.2012.738
Mouse studies suggest OPTC could decrease neovascularization to help treat retinopathy.
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Various | Top |
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Delta-like 4 (DLL4) doi:10.1038/scibx.2012.739
Mouse studies suggest antagonizing DLL4 could help treat atherosclerosis and metabolic diseases.
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Distillery: Techniques |
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Assays and screens | Top |
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In vitro blood brain barrier (BBB) with pluripotent stem cells doi:10.1038/scibx.2012.740
In vitro studies suggest human pluripotent stem cells could be used to screen for BBB-modulating compounds.
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Computational models | Top |
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Correction and assembly of single-molecule sequencing reads doi:10.1038/scibx.2012.741
A computational approach for correcting errors in long-read, single-molecule sequencing could aid in developing applications of the technology for biomedical discovery and guiding treatment.
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Disease models | Top |
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Induced pluripotent stem (iPS) cells derived from patients with Huntington's disease (HD) doi:10.1038/scibx.2012.742
In vitro studies suggest iPS cell lines generated from patients with HD could help identify new therapeutics.
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Mouse model of acrodermatitis enteropathica doi:10.1038/scibx.2012.743
A mouse model of acrodermatitis enteropathica could help identify therapeutics for the rare lethal metabolic disorder that impairs zinc uptake.
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Drug delivery | Top |
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Adeno-associated virus (AAV) expression of an anti-nicotine antibody doi:10.1038/scibx.2012.744
Persistent expression of an anti-nicotine antibody using an AAV vector could help prevent nicotine addiction.
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Drug platforms | Top |
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Genetically corrected induced pluripotent stem (iPS) cell–derived mesoangioblasts for limb-girdle muscular dystrophy doi:10.1038/scibx.2012.745
Genetically corrected mesoangioblasts derived from iPS cells could be useful for treating limb-girdle muscular dystrophy, which is caused by mutations in the sarcoglycan-α (SGCA) gene.
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