SciBX: Science-Business eXchange Contents: September 4 2014, Volume 7 / Issue 34

If you are unable to see the message below, click here to view.

TABLE OF CONTENTS September 4 2014, Volume 7 / Issue 34 Analysis Cover Story Translational Notes Targets and Mechanisms The Distillery: Therapeutics Cancer Cardiovascular disease Endocrine/metabolic disease Infectious disease Neurology Ophthalmic disease The Distillery: Techniques Chemistry Disease models Drug platforms Markers   Analysis Cover Story Top Selectively splicing SMN2 Kai-Jye Lou doi:10.1038/scibx.2014.1001 A three-way collaboration between PTC, Roche and the Spinal Muscular Atrophy Foundation has resulted in orally available, blood brain barrier–penetrant, selective small molecule modulators of SMN2 that are advancing in the clinic to treat spinal muscular atrophy. Full Text | PDF Translational Notes Top Funding forgotten proteins Chris Cain doi:10.1038/scibx.2014.1002 A new NIH program is looking to fund understudied members of druggable protein classes by supporting exploratory projects at risk of being passed over because of a lack of preliminary data. Full Text | PDF Meeting of the minds Tracey Baas doi:10.1038/scibx.2014.1003 Allied Minds has partnered with Bristol-Myers Squibb to form Allied-Bristol Life Sciences LLC and can now add therapeutics to its portfolio of academia-based startups. Full Text | PDF Targets and Mechanisms Top Destabilizing data in CF Benjamin Boettner doi:10.1038/scibx.2014.1004 In vitro data from two academic labs show combinations of Kalydeco ivacaftor and lumacaftor destabilize the CFTR channel. Although the new findings are at odds with Vertex's positive clinical results, they are prompting the design of next-generation, more synergistic compounds for cystic fibrosis. Full Text | PDF Distillery: Therapeutics Cancer Top Leukemia-induced noncoding activator RNA (LUNAR1); insulin-like growth factor-1 receptor (IGF1R; CD221) doi:10.1038/scibx.2014.1005 In vitro and mouse studies suggest depleting the LUNAR1 long noncoding RNA (lncRNA) could help treat T cell ALL (T-ALL). Full Text | PDF Galectin-1 (LGALS1) doi:10.1038/scibx.2014.1006 Rodent studies suggest inhibiting LGALS1 could help treat brain tumors. Full Text | PDF DEAD box polypeptide 20 (DDX20; DP103) doi:10.1038/scibx.2014.1007 Studies in patient samples, mice and cell culture suggest inhibiting DP103 could help prevent breast cancer metastasis. Full Text | PDF Cathepsin S doi:10.1038/scibx.2014.1008 Studies in patient samples and mice suggest inhibiting cathepsin S could help prevent cancer metastasis to the brain. Full Text | PDF Fibroblast growth factor receptor (FGFR); IL-6; JAK kinase (JAK); phosphoinositide 3-kinase (PI3K); signal transducer and activator of transcription 3 (STAT3) doi:10.1038/scibx.2014.1009 Mouse and in vitro studies suggest inhibiting STAT3 signaling could help circumvent cancer resistance to tyrosine kinase inhibitors. Full Text | PDF Focal adhesion kinase (FAK) doi:10.1038/scibx.2014.1010 Mouse studies suggest endothelial cell–specific inhibition of FAK could help sensitize tumors to chemotherapy. Full Text | PDF Phosphatidylinositol-4 phosphate 5-kinase type 1α (PIP5K1A) doi:10.1038/scibx.2014.1011 Studies in mice and in human tissue samples suggest inhibiting PIP5K1A could help treat prostate cancer. Full Text | PDF Cardiovascular disease Top Phosphoinositide 3-kinase-γ (PI3Kγ) doi:10.1038/scibx.2014.1012 Rodent studies suggest inhibiting PI3Kγ could help prevent intimal hyperplasia following vascular injury. Full Text | PDF Endocrine/metabolic disease Top Not applicable doi:10.1038/scibx.2014.1013 Mouse and human tissue studies suggest inducing transdifferentiation of α cells to β cells could help treat type 1 diabetes (T1D). Full Text | PDF IL-22 doi:10.1038/scibx.2014.1014 Mouse studies suggest increasing IL-22 signaling could help treat metabolic disorders. Full Text | PDF IL-1; IL-1α; IL-1β doi:10.1038/scibx.2014.1015 Mouse studies suggest inhibiting IL-1 signaling could improve the success of fertility treatments. Full Text | PDF Infectious disease Top Not applicable doi:10.1038/scibx.2014.1016 In vitro and mouse studies suggest 8-hydroxyquinoline (8HQ) analogs could increase intracellular copper concentration to help treat bacterial and fungal infections. Full Text | PDF RSV F protein doi:10.1038/scibx.2014.1017 In vitro studies have identified resistance mechanisms to inhibitors targeting RSV entry via the RSV F protein. Full Text | PDF Neurology Top Serotonin (5-HT7) receptor doi:10.1038/scibx.2014.1018 In vitro and mouse studies suggest selectively blocking the 5-HT7 receptor could enable rapid treatment of depression. Full Text | PDF Aminoadipate aminotransferase (AADAT; KAT2) doi:10.1038/scibx.2014.1019 Rat and nonhuman primate studies suggest KAT2 inhibitors could help treat schizophrenia. Full Text | PDF Survival of motor neuron 2 centromeric (SMN2) doi:10.1038/scibx.2014.1020 Cell culture and mouse studies have identified orally available, CNS-penetrant small molecule SMN2 splice modifiers that could help treat SMA. Full Text | PDF Ophthalmic disease Top Gap junction protein δ2 36 kDa (GJD2; CX36; connexin-36); gap junction protein γ1 45 kDa (GJC1; CX45) doi:10.1038/scibx.2014.1021 In vitro and mouse studies suggest inhibiting gap junction components CX36 or CX45 could help treat neurodegenerative ocular disorders, including ischemic retinopathy and glaucoma. Full Text | PDF Distillery: Techniques Chemistry Top Total synthesis of mannopeptimycin aglycone to enable derivatization doi:10.1038/scibx.2014.1022 A method to synthesize mannopeptimycin aglycone could aid the development of derivatives of the cyclic hexapeptide for use against drug-resistant bacterial infections. Full Text | PDF Disease models Top Induced pluripotent stem (iPS) cell–derived hepatocyte-like cells in micropatterned coculture (MPCC) to model HBV infection doi:10.1038/scibx.2014.1023 In vitro studies suggest an MPCC system using iPS cell–derived hepatocyte-like cells and fibroblasts can be used to model HBV infection. Full Text | PDF Transgenic mouse model of Alzheimer's disease (AD) expressing human β-site APP cleaving enzyme 1 (BACE1) doi:10.1038/scibx.2014.1024 Mice with forebrain-specific expression of human BACE1 could be useful as models for evaluating new treatments for AD. Full Text | PDF Drug platforms Top Asymmetric triplex metallohelices for treating cancer doi:10.1038/scibx.2014.1025 Asymmetric triplex metallohelices could be useful for treating cancer. Full Text | PDF Crystal structure of the β-ketoacyl reductase (KR) domain of fatty acid synthase (FASN; FAS)–small molecule inhibitor complex doi:10.1038/scibx.2014.1026 The crystal structure of the KR domain of FAS could aid the rational design of new small molecule FAS inhibitors to treat cancer. Full Text | PDF Glial Schwann cell precursors as a source for dental stem cells doi:10.1038/scibx.2014.1027 Mouse studies suggest glia cell precursors could be developed as a source for tooth-regenerating stem cells. Full Text | PDF Markers Top Circulating microRNA signature to diagnose acetaminophen poisoning doi:10.1038/scibx.2014.1028 An 11-miRNA signature could be used to identify acetaminophen poisoning earlier and with better sensitivity than measuring alanine aminotransferase (ALT) levels. Full Text | PDF Excision repair cross-complementation group 2 (ERCC2) mutations as a predictive marker of cisplatin sensitivity in urothelial carcinomas doi:10.1038/scibx.2014.1029 Studies in patient samples and cell culture suggest loss-of-function mutations in ERCC2 could help predict urothelial carcinoma sensitivity to cisplatin. Full Text | PDF Top

You have been sent this Table of Contents Alert because you have opted in to receive it. You can change or discontinue your e-mail alerts at any time, by modifying your preferences on your nature.com account at: www.nature.com/myaccount (You will need to log in to be recognised as a nature.com registrant) For further technical assistance, please contact our registration department For print subscription enquiries, please contact our subscription department For other enquiries, please contact our customer feedback department Nature Publishing Group | 75 Varick Street, 9th Floor | New York | NY 10013-1917 | USA Nature Publishing Group's worldwide offices: London - Paris - Munich - New Delhi - Tokyo - Melbourne San Diego - San Francisco - Washington - New York - Boston Macmillan Publishers Limited is a company incorporated in England and Wales under company number 785998 and whose registered office is located at Brunel Road, Houndmills, Basingstoke, Hampshire RG21 6XS. © 2014 Nature Publishing Group, a division of Macmillan Publishers Limited. All Rights Reserved.