SciBx is a weekly publication that identifies and analyzes the most important translational research articles from over 40 journals. Find out which papers have real scientific and commercial potential, and why. Subscribe to SciBX and you won't miss the next big thing.
mAb about FGF21 Chris Cain doi:10.1038/scibx.2013.1 Amgen researchers have developed an antibody that mimics fibroblast growth factor 21 and has antidiabetic effects in monkeys. The findings cap a year of advances that have greatly increased understanding of the protein's tissue-specific actions and are informing drug development by at least six companies. Full Text | PDF
DUX4 partnership Lauren Martz doi:10.1038/scibx.2013.2 Fred Hutchinson Cancer Research Center researchers have identified a mutation of double homeobox 4 that could be targeted in facioscapulohumeral muscular dystrophy. The team has partnered with GlaxoSmithKline to develop small molecule inhibitors against DUX4 or its downstream effectors. Full Text | PDF
Targeting inflammation in AD Lev Osherovich doi:10.1038/scibx.2013.3 German researchers have implicated the inflammasome as a key player in Alzheimer's disease. The findings provide additional evidence that AD could be treated with anti-inflammatory agents that act upstream of β-amyloid deposition. Full Text | PDF
Scaling up iPS cells Joanne Kotz doi:10.1038/scibx.2013.4 A Memorial Sloan-Kettering team has conducted one of the first large-scale screens of a small molecule library in neuronal cells obtained by differentiating patient-derived iPS cells. Separately, a partnership led by Roche and the University of Oxford is aiming to establish a repository of iPS cells from 500 patients with diabetes or various neurological diseases. Full Text | PDF
Sirtuin 6 (SIRT6) doi:10.1038/scibx.2013.5 Patient tissue and mouse studies suggest activating SIRT6 could help modulate tumor metabolism to treat cancer. Full Text | PDF
β-Catenin (CTNNB1); yes-associated protein 1 (YAP1); v-yes-1 Yamaguchi sarcoma viral oncogene homolog 1 (YES1; Yes) doi:10.1038/scibx.2013.6 Studies in cancer cell lines suggest inhibiting YES1 could help treat CTNNB1-driven colon cancers. Full Text | PDF
Enhancer of zeste homolog 2 (EZH2) doi:10.1038/scibx.2013.7 In vitro studies identified an EZH2 inhibitor that could help treat DLBCL. Full Text | PDF
Myeloid-lymphoid or mixed-lineage leukemia (MLL; HRX); WD repeat domain 5 (WDR5) doi:10.1038/scibx.2013.8 In vitro studies identified inhibitors of the protein-protein interaction between WDR5 and MLL protein-protein interactions that could help treat leukemias. Full Text | PDF
Neurofibromin 1 (NF1); MEK doi:10.1038/scibx.2013.9 Two studies in mice suggest MEK inhibitors could help treat cancers with mutations in NF1. Full Text | PDF
DNA methyltransferase doi:10.1038/scibx.2013.10 In vitro and mouse studies suggest hypomethylating agents could help treat pancreatic ductal adenocarcinoma (PDAC). Full Text | PDF
Enhancer of zeste homolog 2 (EZH2); protein kinase B (PKB; PKBA; AKT; AKT1) doi:10.1038/scibx.2013.11 Cell culture studies suggest inhibiting the histone methyltransferase EZH2 could help treat prostate cancer. Full Text | PDF
G protein–coupled receptor 119 (GPR119) doi:10.1038/scibx.2013.13 Mouse studies identified a series of indolines that act as selective GPR119 agonists and could help treat type 2 diabetes. Full Text | PDF
Protozoan signal peptide peptidase (SPP) doi:10.1038/scibx.2013.14 Cell culture studies suggest inhibiting protozoan SPP could help treat parasitic infections. Full Text | PDF
CC chemokine receptor 5 (CCR5; CD195) doi:10.1038/scibx.2013.15 Cell culture and mouse studies suggest inhibiting CCR5 could help treat Staphylococcus aureus infections. Full Text | PDF
Dystrophin (DMD) doi:10.1038/scibx.2013.16 In vitro and mouse studies suggest the ryanodine receptor (RyR) inhibitor dantrolene could enhance the effects of antisense oligonucleotides to treat Duchenne muscular dystrophy. Full Text | PDF
Transient receptor potential vanilloid 1 (TRPV1; VR1) doi:10.1038/scibx.2013.17 In vitro and mouse studies suggest activating TRPV1 in skeletal muscle could help treat or prevent muscular atrophy. Full Text | PDF
MicroRNA-214 (miR-214) doi:10.1038/scibx.2013.18 In vitro and mouse studies suggest inhibiting miR-214 could help treat osteoporosis. Full Text | PDF
GABAA receptor doi:10.1038/scibx.2013.19 In vitro and mouse studies suggest GABAA receptor agonists could help treat motor dysfunctions associated with Angelman syndrome, a neurogenetic disorder caused by deletion or inactivation of genes on chromosome 15. Full Text | PDF
Inhibitor of κ-light polypeptide gene enhancer in B cells kinase complex–associated protein (IKBKAP); adrenergic receptor α2 (ADRA2) doi:10.1038/scibx.2013.20 A study in neural cells derived from patients with familial dysautonomia (FD) suggests antagonizing ADRA2 could help treat the disease. Full Text | PDF
Unknown doi:10.1038/scibx.2013.21 Mouse studies suggest lithium could help treat Down syndrome. Full Text | PDF
Assays to monitor leucine-rich repeat kinase 2 (LRRK2) activity doi:10.1038/scibx.2013.22 Cell-based and pharmacodynamic assays for monitoring LRRK2 activity could help identify compounds to treat Parkinson's disease (PD). Full Text | PDF
Natural product–based fragment libraries for ligand discovery doi:10.1038/scibx.2013.23 Natural product–based fragment libraries could be used to identify ligands against targets that are difficult to drug. Full Text | PDF
Glycosylated triterpene–mediated transdermal delivery of macromolecules doi:10.1038/scibx.2013.24 Ex vivo studies suggest glycosylated triterpenes are capable of transdermal transport and could help mediate therapeutic macromolecule delivery. Full Text | PDF
Probe for functional imaging of legumain (LGMN) doi:10.1038/scibx.2013.25 An activity-based fluorescent probe could be used to study the activity of LGMN, a protein associated with cancer and inflammation. Full Text | PDF
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