SciBX: Science-Business eXchange Contents: December 19 2013, Volume 6 / Issue 48

TABLE OF CONTENTS December 19 2013, Volume 6 / Issue 48 Analysis Cover Story Translational Notes Targets and Mechanisms The Distillery: Therapeutics Cancer Cardiovascular disease Endocrine/metabolic disease Hepatic disease Infectious disease Neurology Renal disease Various The Distillery: Techniques Assays and screens Chemistry Disease models Drug delivery Drug platforms Markers Advertisement BioPharma Dealmakers A supplement to Nature Biotechnology and Nature Reviews Drug Discovery The November 2013 issue of BioPharma Dealmakers showcases companies with partnering opportunities. This week, find out about how you can collaborate with ENDECE Neural and Innate Pharma   Analysis Cover Story Top Oral nanoparticles Lauren Martz doi:10.1038/scibx.2013.1371 A U.S. research team has developed targeted nanoparticles that could enable the oral delivery of biologics. Translation of the approach will require identifying a suitable therapeutic payload and fine-tuning the pharmacology of the system. Full Text | PDF Translational Notes Top Translational tidbits C. Simone Fishburn, Lev Osherovich and Kai-Jye Lou doi:10.1038/scibx.2013.1372 GSK opens its doors to an academic consortium and sets up an R&D outpost in San Diego. Also, a roundup of public-private partnerships led by a trio of new consortia funded by the EU's FP7. Full Text | PDF Targets and Mechanisms Top Lipid kinase enters the malaria stage Benjamin Boettner doi:10.1038/scibx.2013.1373 A Novartis-UCSD–led consortium has identified a new class of antimalarials that, unlike marketed drugs, eliminates Plasmodium at all stages of its infection cycle. The pharma is developing derivatives of the lead inhibitor with improved drug-like properties. Full Text | PDF Balancing act in liver fibrosis Michael J. Haas doi:10.1038/scibx.2013.1374 U.S. researchers have shown that CXCR7 and CXCR4 play proregenerative and profibrotic roles following liver injury, making the proteins therapeutic targets to prevent or treat liver fibrosis. Full Text | PDF Distillery: Therapeutics Cancer Top Toll-like receptor 9 (TLR9); signal transducer and activator of transcription 3 (STAT3) doi:10.1038/scibx.2013.1375 Mouse studies suggest simultaneously activating TLR9 and inhibiting STAT3 could help treat AML. Full Text | PDF MicroRNA-17-92 (miR-17-92) doi:10.1038/scibx.2013.1376 Cell culture and mouse studies suggest inhibiting miR-17-92 could help treat medulloblastoma. Full Text | PDF Phosphatidylinositol-5-phosphate 4-kinase type IIα (PIP4K2A); PIP4K2B doi:10.1038/scibx.2013.1377 Mouse and cell culture studies suggest inhibiting PIP4K2A and PIP4K2B could help treat p53− breast cancer. Full Text | PDF c-Ros proto-oncogene 1 receptor tyrosine kinase (ROS1) doi:10.1038/scibx.2013.1378 Mouse and cell culture studies suggest foretinib could help treat cancers harboring oncogenic ROS1 fusions and point mutations. Full Text | PDF K-Ras (KRAS) G12C doi:10.1038/scibx.2013.1379 In vitro and cell culture studies identified a covalent inhibitor of mutant KRAS G12C that could help treat cancer. Full Text | PDF Not applicable doi:10.1038/scibx.2013.1380 In vitro and mouse studies suggest combining copper chelators with glycolysis inhibitors could help treat cancer. Full Text | PDF Not applicable doi:10.1038/scibx.2013.1381 Mouse studies suggest antibiotics could inhibit the potency of DNA-alkylating agents. Full Text | PDF Smoothened (SMO) doi:10.1038/scibx.2013.1382 Structural and cell culture studies suggest oxysterol-based inhibitors could help treat cancers driven by hedgehog signaling. Full Text | PDF Parathyroid hormone (PTH) doi:10.1038/scibx.2013.1383 In vitro and mouse studies suggest PTH could help treat CML. Full Text | PDF Retinoid X receptor-γ (RXRG; RXRγ) doi:10.1038/scibx.2013.1384 In vitro and mouse studies suggest spironolactone or RXRγ agonists could help treat colon cancer. Full Text | PDF BRAF; MEK; microphthalmia-associated transcription factor (MITF); histone deacetylase (HDAC) doi:10.1038/scibx.2013.1385 Cell culture and mouse studies suggest combining MEK and HDAC inhibitors could help treat BRAF-mutant melanoma. Full Text | PDF Plectin (PLEC) doi:10.1038/scibx.2013.1386 In vitro and mouse studies suggest inhibiting PLEC could help treat pancreatic cancer. Full Text | PDF Cardiovascular disease Top SMT3 suppressor of mif two 3 homolog 1 (SUMO1); ATPase Ca++ transporting cardiac muscle slow twitch 2 (ATP2A2; SERCA2A) doi:10.1038/scibx.2013.1387 Pig studies suggest SUMO1 gene therapy could help treat heart failure. Full Text | PDF Factor Xa; thrombin (factor IIa; F2) doi:10.1038/scibx.2013.1388 In vitro and rat studies identified a dual factor Xa and thrombin inhibitor that could help treat thrombosis. Full Text | PDF Endocrine/metabolic disease Top Glucagon receptor (GCGR) doi:10.1038/scibx.2013.1389 In vitro and mouse studies identified an allosteric GCGR antibody that could help treat type 2 diabetes. Full Text | PDF Glucagon-like peptide-1 receptor (GLP-1R; GLP1R); glucagon receptor (GCGR); glucose-dependent insulinotropic polypeptide receptor (GIP receptor) doi:10.1038/scibx.2013.1390 Mouse studies suggest peptides that agonize GLP1R, GCGR and GIP receptor could be used to treat obesity and diabetes. Full Text | PDF Hepatic disease Top CXC chemokine receptor 4 (CXCR4; NPY3R); CXCR7 doi:10.1038/scibx.2013.1391 Mouse studies suggest agonizing CXCR7 or inhibiting CXCR4 could help prevent or treat liver fibrosis. Full Text | PDF Infectious disease Top Cleavage and polyadenylation specific factor 6 (CPSF6); cyclophilin A (CYPA; PPIA) doi:10.1038/scibx.2013.1392 Cell culture studies suggest blocking interaction between HIV capsid protein and host factors CPSF6 or CYPA could help prevent viral replication. Full Text | PDF Plasmodium phosphatidylinositol 4-kinase IIIβ (Plasmodium PI4KIIIβ) doi:10.1038/scibx.2013.1393 In vitro and in vivo studies suggest targeting Plasmodium PI4KIIIβ could help treat malaria. Full Text | PDF Neurology Top Fusion (involved in t(12;16) in malignant liposarcoma) (FUS; TLS) doi:10.1038/scibx.2013.1394 Cell culture studies suggest oligonucleotides that induce FUS exon seven skipping could help prevent the accumulation of mutant FUS protein in ALS motor neurons. Full Text | PDF NMDAR doi:10.1038/scibx.2013.1395 In vitro and mouse studies suggest 24(S) hydroxycholesterol (24(S)-HC) could help treat cognitive dysfunction. Full Text | PDF Protein phosphatase 2 (PPP2CA; PP2A) doi:10.1038/scibx.2013.1396 In vitro and mouse studies suggest inhibiting PPP2CA in the brain could help treat Opitz syndrome, an inherited neurological disorder. Full Text | PDF Histone deacetylase 6 (HDAC6) doi:10.1038/scibx.2013.1397 Studies in mice suggest HDAC6 inhibitors could be useful for treating ischemic stroke. Full Text | PDF Transient receptor potential cation channel subfamily M member 2 (TRPM2); NMDA receptor NR2A subtype (GRIN2A; NR2A); GRIN2B (NR2B) doi:10.1038/scibx.2013.1398 Mouse studies suggest inhibiting TRPM2 could help prevent ischemic damage to neurons. Full Text | PDF Renal disease Top Transient receptor potential cation channel subfamily C member 5 (TRPC5) doi:10.1038/scibx.2013.1399 In vitro and mouse studies suggest inhibiting the calcium channel TRPC5 could help treat proteinuria. Full Text | PDF Various Top c-Mer proto-oncogene tyrosine kinase (MERTK) doi:10.1038/scibx.2013.1400 In vitro studies suggest a new class of MERTK inhibitors could help treat cancer or thrombosis. Full Text | PDF Uromodulin (UMOD; THP); solute carrier family 12 potassium-chloride transporter member 1 (SLC12A1; NKCC2) doi:10.1038/scibx.2013.1401 In vitro and mouse studies suggest inhibiting UMOD could help treat hypertension and chronic kidney disease. Full Text | PDF Diacylglycerol lipase-α (DAGLA) doi:10.1038/scibx.2013.1402 In vitro studies identified a selective inhibitor of DAGLA that could help treat obesity or neurodegenerative diseases. Full Text | PDF Distillery: Techniques Assays and screens Top Monitoring cell-free DNA in plasma to predict organ transplant rejection doi:10.1038/scibx.2013.1403 Cell-free DNA in plasma could be used to quantify viral load and predict risk of transplant rejection. Full Text | PDF Chemistry Top Modifications to the siRNA guide strand 5′ end that improve therapeutic potency doi:10.1038/scibx.2013.1404 Structure-guided computational screening identified modifications to the guide strand that could improve the therapeutic properties of siRNA therapeutics. Full Text | PDF Disease models Top PTEN (MMAC1; TEP1)-mutant mouse model of autism doi:10.1038/scibx.2013.1405 Mice with a heterozygous, autism-linked PTEN deletion could be used to screen therapeutics for autism spectrum disorder. Full Text | PDF Drug delivery Top Fc fragment of IgG receptor transporter-α (FCGRT; FCRN)-targeted nanoparticles for oral nanoparticle delivery doi:10.1038/scibx.2013.1406 In vitro and mouse studies suggest FCRN-targeted nanoparticles could be used for oral drug delivery. Full Text | PDF Drug platforms Top Lin-28 homolog A (LIN28A)-dependent metabolic reprogramming for tissue repair doi:10.1038/scibx.2013.1407 Mouse studies suggest elevating LIN28A activity could help promote tissue regeneration after injury or in advanced age. Full Text | PDF Olfactory ensheathing cells (OECs) to increase myelination doi:10.1038/scibx.2013.1408 Cell culture studies suggest OECs might make better transplant cells for spinal injury repair than Schwann cells. Full Text | PDF Spherical nucleic acid (SNA) nanoparticle conjugates to knock down oncogene expression doi:10.1038/scibx.2013.1409 Systemic delivery of SNAs conjugated to siRNAs could be used to treat glioma. Full Text | PDF Staurosporine analogs (staralogs) with improved selectivity toward analog-sensitive kinases doi:10.1038/scibx.2013.1410 Staralogs could expand approaches to study kinase function using analog-sensitive kinase variants. Full Text | PDF Markers Top Using innate immune markers to detect early stage tumor recurrence doi:10.1038/scibx.2013.1411 Mouse studies suggest markers of the innate immune response could help detect early tumor recurrence. Full Text | PDF Top

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