Thursday, September 13, 2012

SciBX: Science-Business eXchange Contents: September 13 2012, Volume 5 / Issue 36

SciBX: Science-Business eXchange

TABLE OF CONTENTS

September 13 2012, Volume 5 / Issue 36

Analysis

Cover Story
Targets and Mechanisms
Tools

The Distillery: Therapeutics

Autoimmune disease
Cancer
Endocrine/metabolic disease
Infectious disease
Neurology
Ophthalmic disease

The Distillery: Techniques

Assays and screens
Disease models
Drug delivery
Drug platforms
Markers
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Analysis

Cover Story

Top

VEGF reflects on itself
Tim Fulmer
doi:10.1038/scibx.2012.943
North American researchers have chemically synthesized a d-protein ligand that blocked the binding of VEGF to its receptor in vitro. Reflexion has licensed the compound and will optimize it further before doing head-to-head comparisons versus Lucentis and Avastin in animal models of AMD.
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Targets and Mechanisms

Top

This is (a diagnostic) spinal tap
Michael J. Haas
doi:10.1038/scibx.2012.944
A team led by KineMed has shown that measuring the kinetics of CNS proteins in cerebrospinal fluid could help diagnose Parkinson's disease and amyotrophic lateral sclerosis. The company is now exploring the utility of the approach to diagnose multiple neurodegenerative diseases and to monitor treatment responses.
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Tools

Top

Cracking ENCODE
Lev Osherovich
doi:10.1038/scibx.2012.945
ENCODE, a massive effort to characterize the functional elements of the human genome, went public with its findings last week. For industry, the data are likely to spark a re-examination of genomewide association study results. Companies best positioned to take rapid advantage of the findings are those with knockdown technologies able to screen for biological effects of genes the consortium flagged as key disease players.
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Probing for a point-of-care TB test
Kai-Jye Lou
doi:10.1038/scibx.2012.946
Researchers at Stanford University and Texas A&M University have synthesized highly sensitive class A β-lactamase–specific fluorogenic probes that could enable the development of a fast and simple point-of-care diagnostic for tuberculosis. Global BioDiagnostics has in-licensed the technology.
Full Text | PDF

Distillery: Therapeutics

Autoimmune disease

Top

p38 Mitogen-activated protein kinase (p38 MAPK; MAPK14)
doi:10.1038/scibx.2012.947
In vitro and rat studies identified a p38 MAPK inhibitor that could help treat autoimmune diseases.
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Cancer

Top

Core-binding factor β-subunit (CBFB; CBFβ); runt-related transcription factor 1 (RUNX1)
doi:10.1038/scibx.2012.948
In vitro and mouse studies suggest inhibiting the interaction between RUNX1 and CBFβ could help treat core-binding factor leukemias, which have mutations that affect RUNX1 or CBFβ and account for about 24% of adult AML and 25% of pediatric ALL cases.
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R-Spondin 2 (RSPO2); RSPO3
doi:10.1038/scibx.2012.949
Human genomic studies suggest antagonizing RSPO2 and RSPO3 gene fusion proteins could be useful for treating colorectal cancer.
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Multiple endocrine neoplasia I (MEN1; menin)
doi:10.1038/scibx.2012.950
Mouse studies suggest adenovirus-mediated MEN1 gene replacement therapy could help treat pituitary tumors in multiple endocrine neoplasia type 1, an autosomal dominant disorder characterized by neuroendocrine tumors.
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Endocrine/metabolic disease

Top

Unknown
doi:10.1038/scibx.2012.951
Rat studies suggest antidiabetic prodrugs engineered to release nitric oxide (NO) could reduce cardiovascular risks associated with treatment.
Full Text | PDF

Infectious disease

Top

ATPase Ca++ transporting plasma membrane 4 (ATP2B4; PMCA4); MARVEL domain containing 3 (MARVELD3)
doi:10.1038/scibx.2012.952
Genomewide association studies identified two loci associated with risk of severe malaria that could become drug targets.
Full Text | PDF

Neurology

Top

β-Amyloid 42; prion protein (PRNP; PrP; CD230)
doi:10.1038/scibx.2012.953
Cell culture studies suggest soluble PrP could help treat AD.
Full Text | PDF

c-Jun N-terminal kinase 3 (JNK3; MAPK10); β-amyloid 42
doi:10.1038/scibx.2012.954
In vitro and mouse studies suggest JNK3 inhibition could help treat AD.
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Meprin Aβ (MEP1B)
doi:10.1038/scibx.2012.955
In vitro studies suggest inhibiting MEP1B could help treat AD.
Full Text | PDF

Huntingtin (HTT)
doi:10.1038/scibx.2012.956
In vitro and mouse studies suggest allele-selective single-strand small interfering RNA (ss-siRNA) could help treat HD.
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Amyloid precursor protein (APP); chromogranin B (CHGB); neuregulin 1 (NRG1); α-synuclein (SNCA)
doi:10.1038/scibx.2012.957
Mouse and human studies suggest measuring the transport rates of CNS proteins into and out of cerebrospinal fluid (CSF) could help diagnose PD and ALS.
Full Text | PDF

Calcium channel voltage-dependent N type-α 1B subunit (CACNA1B; CaV2.2); collapsin response mediator protein-2 (DPYSL2; CRMP-2)
doi:10.1038/scibx.2012.958
In vitro and rat studies suggest inhibiting the interaction between CaV2.
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Neuronal pentraxin 1 (NPTX1; NP1)
doi:10.1038/scibx.2012.959
Rodent studies suggest inhibiting NP1 could help treat neuropathic pain.
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Ryanodine receptor 2 (RyR2)
doi:10.1038/scibx.2012.960
Mouse studies suggest stabilizing RyR2 could help prevent post-traumatic stress disorder.
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Ophthalmic disease

Top

VEGF-A
doi:10.1038/scibx.2012.961
An in vitro study identified an antagonist of VEGF-A that could be useful for treating AMD. The d-stereoisomer of VEGF-A was synthesized and used to screen for d-protein ligands of VEGF-A.
Full Text | PDF

Distillery: Techniques

Assays and screens

Top

Class A β-lactamase (blaC)-specific detection of Mycobacterium tuberculosis
doi:10.1038/scibx.2012.962
Fluorescent probes specific for M. tuberculosis blaC could be useful for detecting M. tuberculosis in clinical samples.
Full Text | PDF

Human blood metabolite–based timetable
doi:10.1038/scibx.2012.963
A human blood metabolite–based timetable could help estimate internal body time with just two blood samples.
Full Text | PDF

Mass spectrometric, antibody-free quantification of proteins in biofluids
doi:10.1038/scibx.2012.964
A mass spectrometry–based method could be useful for quantifying proteins in biofluids with ELISA-like sensitivity.
Full Text | PDF

Disease models

Top

Conditional fukutin (Fktn) knockout mice as a model for secondary dystroglycanopathies
doi:10.1038/scibx.2012.965
Conditional Fktn knockout mice could be useful models for secondary dystroglycanopathies, a family of muscular dystrophies caused by mutations in multiple glycosyltransferase genes.
Full Text | PDF

Drug delivery

Top

Lipid nanoparticle formulation of self-amplifying RNAs for vaccination
doi:10.1038/scibx.2012.966
Self-amplifying RNA presented in a lipid nanoparticle formulation could be a viable nonviral alternative to viral delivery–based RNA vaccination technologies.
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Polyethylene glycol (PEG) coating to improve delivery of drug-loaded nanoparticles across the blood brain barrier (BBB)
doi:10.1038/scibx.2012.967
Densely coating drug-loaded nanoparticles with PEG could help improve their delivery to the brain.
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Drug platforms

Top

High throughput flow cytometry for small molecule screening
doi:10.1038/scibx.2012.968
High throughput, multiplexed flow cytometry could provide a way to rapidly screen small molecules in immune cells.
Full Text | PDF

Markers

Top

Map of disease gene regulation by noncoding DNA
doi:10.1038/scibx.2012.969
A genomic study identified noncoding DNA sequences that regulate the expression of disease-related genes and might provide targets that affect disease progression.
Full Text | PDF

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