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Bringing on the BRAF Tracey Baas doi:10.1038/scibx.2012.140 Independent findings from European and U.S. teams suggest combining BRAF inhibitors with EGFR inhibitors could treat colorectal cancers resistant to monotherapy with either type of drug. Both groups are now planning clinical trials with such combinations. Full Text | PDF
Going viral in Parkinson's disease Kai-Jye Lou doi:10.1038/scibx.2012.141 U.K. researchers have generated a peptide-RNA conjugate that can be delivered noninvasively to the brain and has a therapeutic effect in Parkinson's disease models. The group is now elucidating the neuroprotective mechanism and determining the smallest RNA domain needed for the effect. Full Text | PDF
HUS and them Lauren Martz doi:10.1038/scibx.2012.142 Two groups have identified new strategies to treat or prevent hemolytic uremic syndrome, a potentially lethal complication of Escherichia coli infection. One method involves repurposing Sanofi's Mozobil CXCR4 antagonist, and the other uses the natural metal ion manganese to neutralize the toxin that causes HUS. Full Text | PDF
Doubling down on dopamine Lev Osherovich doi:10.1038/scibx.2012.143 A Scripps Florida team has pinpointed a receptor complex in the hypothalamus that may play a role in the increase in appetite caused by dopamine receptor antagonists used to treat schizophrenia. The challenge now is to design next-generation antipsychotics without the appetite-related side effects. Full Text | PDF
Not applicable doi:10.1038/scibx.2012.144 In vitro studies suggest gold-containing indole analogs could help treat cancer. Full Text | PDF
Tryptophan 2,3-dioxygenase (TDO2; TDO) doi:10.1038/scibx.2012.145 Mouse studies suggest inhibiting TDO could help treat cancer. Full Text | PDF
Janus kinase-2 (JAK-2); signal transducer and activator of transcription 5 (STAT5) doi:10.1038/scibx.2012.146 Cell culture and mouse studies suggest JAK-2 inhibitors exert anti-CML effects via off-target activity at STAT5. Full Text | PDF
BRAF; epidermal growth factor receptor (EGFR) doi:10.1038/scibx.2012.147 Two independent studies in mice suggest combining EGFR inhibitors with BRAF inhibitors could help treat BRAF-mutant colorectal cancers that are unresponsive to BRAF inhibitors. Full Text | PDF
Myeloid-lymphoid or mixed-lineage leukemia (MLL; HRX); multiple endocrine neoplasia I (MEN1; Menin) doi:10.1038/scibx.2012.148 In vitro studies identified thienopyrimidine-based MEN1 inhibitors that could help treat leukemias. Full Text | PDF
WEE1 tyrosine kinase (WEE1) doi:10.1038/scibx.2012.149 Patient sample and cell culture studies suggest combining WEE1 inhibitors with cytarabine could help treat leukemia. Full Text | PDF
Anaplastic lymphoma kinase (ALK); epidermal growth factor receptor (EGFR); stem cell factor receptor tyrosine kinase (c-Kit; KIT; CD117) doi:10.1038/scibx.2012.150 Patient and cell culture studies suggest targeting EGFR and KIT signaling pathways could help treat lung cancers resistant to ALK inhibitors. Full Text | PDF
Guanine nucleotide binding protein β-polypeptide 2-like 1 (GNB2L1; RACK1) doi:10.1038/scibx.2012.151 Patient sample and mouse studies suggest inhibiting RACK1 could help treat NSCLC. Full Text | PDF
Aldo-keto reductase family 1 member C3 (AKR1C3) doi:10.1038/scibx.2012.152 In vitro studies identified specific AKR1C3 inhibitors that could help treat castration-resistant prostate cancer. Full Text | PDF
O-Linked N-acetylglucosamine transferase (OGT) doi:10.1038/scibx.2012.153 Mouse studies suggest inhibiting OGT could help treat prostate cancer. Full Text | PDF
Eukaryotic translation initiation factor 2α kinase 4 (EIF2AK4; GCN2) doi:10.1038/scibx.2012.154 Studies in mice suggest activation of the amino acid starvation response could protect against surgical ischemia/reperfusion injury. Full Text | PDF
Diacylglycerol O-acyltransferase-1 (DGAT1) doi:10.1038/scibx.2012.155 Rodent and cell culture studies suggest the DGAT1 inhibitor ABT-046 could help treat dyslipidemia. Full Text | PDF
Membrane-bound transcription factor peptidase site-1 (MBTPS1; SKI-1) doi:10.1038/scibx.2012.156 Cell culture studies suggest inhibiting SKI-1 could help treat HCV infection. Full Text | PDF
Pleckstrin homology domain containing family O member 1 (PLEKHO1; CKIP-1) doi:10.1038/scibx.2012.157 Rat studies suggest bone formation–targeting liposomes loaded with PLEKHO1 small interfering RNA could help treat osteoporosis. Full Text | PDF
N-Acetyltransferase 8 (NAT8); NAT8B doi:10.1038/scibx.2012.158 Human brain tissue and cell culture studies suggest NAT8 and NAT8B could be antagonized to treat AD. Full Text | PDF
Solute carrier family 12 potassium-chloride transporter member 2 (SLC12A2, NKCC1); p75 neurotrophin receptor (p75 NTR) doi:10.1038/scibx.2012.159 Studies in mice suggest that bumetanide could help treat nerve injury. Full Text | PDF
Regulator of G-protein signaling 4 (RGS4) doi:10.1038/scibx.2012.160 Mouse studies suggest inhibiting RGS4 could help treat PD. Full Text | PDF
Neurotrophic tyrosine kinase receptor 2 (NTRK2; TrkB) doi:10.1038/scibx.2012.161 Mouse studies suggest TrkB agonists could help improve breathing dysfunction in patients with Rett syndrome. Full Text | PDF
Dopamine D2 receptor; (GHSR) doi:10.1038/scibx.2012.162 Studies in cell culture and in mice suggest a heterodimeric complex of the dopamine D2 receptor and the ghrelin receptor may be involved in hyperphagia caused by antipsychotic therapy. Full Text | PDF
Retinitis pigmentosa GTPase regulator (RPGR) doi:10.1038/scibx.2012.163 Canine studies suggest gene therapy could be used to correct defects in RPGR that lead to retinitis pigmentosa, a hereditary form of vision loss. Full Text | PDF
Cystic fibrosis transmembrane conductance regulator (CFTR) doi:10.1038/scibx.2012.164 In vitro studies suggest stabilizing two distinct steps of mutant ΔF508 CFTR protein folding could help treat CF. Full Text | PDF
In vitro transmigration assay to model the blood brain barrier (BBB) doi:10.1038/scibx.2012.165 An in vitro transmigration assay could be a useful BBB model to help identify new targets and compounds to treat CNS disorders like multiple sclerosis (MS). Full Text | PDF
Mouse model of intrahepatic cholangiocarcinoma doi:10.1038/scibx.2012.166 A mouse model of cholangiocarcinoma could be used to screen for treatments of the disease. Full Text | PDF
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