Thursday, May 10, 2012

SciBX: Science-Business eXchange Contents: May 10 2012, Volume 5 / Issue 18

SciBX: Science-Business eXchange

TABLE OF CONTENTS

May 10 2012, Volume 5 / Issue 19

Analysis

Cover Story
Targets and Mechanisms
Tools

The Distillery: Therapeutics

Autoimmune disease
Cancer
Endocrine/metabolic disease
Hepatic disease
Inflammation
Musculoskeletal disease
Transplantation
Various

The Distillery: Techniques

Assays and screens
Computational models
Disease models
Drug delivery
Drug platforms
Markers
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Analysis

Cover Story

Top

A mind for precompetitive collaboration
Chris Cain
doi:10.1038/scibx.2012.483
Although pharmas are cutting back on internal neuroscience R&D programs, the number of precompetitive consortia backed by industry continues to rise. Most initiatives are focused on developing better disease models and discovering new biomarkers. The challenge going forward will be to push the boundaries of the precompetitive space to advance the discovery of new disease-modifying targets and mechanisms.
Full Text | PDF

Targets and Mechanisms

Top

Mitochondrial gene therapy
Lev Osherovich
doi:10.1038/scibx.2012.484
A University of Miami team has used mitochondrion-targeted gene therapy to restore functional ND4 levels in Leber's hereditary optical neuropathy. Next steps include optimizing the approach to cover several variants of the disease and determining the therapy's potential advantages over pharmacological strategies in development.
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PPAR's new course
Steve Edelson
doi:10.1038/scibx.2012.485
California researchers have shown that agonizing PPARδ could help stave off fibrosis in a variety of chronic liver diseases. The findings could open new therapeutic real estate for the handful of biotechs working on PPARδ modulators.
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Tools

Top

Dendrimers get cerebral
Tim Fulmer
doi:10.1038/scibx.2012.486
U.S. researchers have developed a dendrimer-based therapy that reduced brain inflammation and improved the motor function of newborn rabbits with cerebral palsy. The team now needs to determine the therapy's treatment window and its efficacy in the multiple types of cerebral palsy.
Full Text | PDF

Distillery: Therapeutics

Autoimmune disease

Top

Dedicator of cytokinesis 2 (DOCK2)
doi:10.1038/scibx.2012.487
In vitro and mouse studies identified a small molecule DOCK2 inhibitor that could help treat autoimmune disease.
Full Text | PDF

MicroRNA-301a (miR-301a)
doi:10.1038/scibx.2012.488
A study in mice suggests antagonizing miR-301a could help treat MS.
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Cancer

Top

Hedgehog pathway; P glycoprotein (MDR1; ABCB1; P-gp; CD243)
doi:10.1038/scibx.2012.489
Mouse studies suggest P-gp inhibitors could help reverse resistance to saridegib in medulloblastoma.
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Autophagy
doi:10.1038/scibx.2012.490
In vitro and mouse studies suggest dimeric chloroquine analogs could help treat cancer more effectively than hydroxychloroquine.
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Glucose-6-phosphate dehydrogenase (G6PD)
doi:10.1038/scibx.2012.491
An SAR study identified G6PD inhibitors that could be useful for treating solid tumors.
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Tumor necrosis factor-α (TNF-α)
doi:10.1038/scibx.2012.492
Mouse studies suggest low-dose, recombinant TNF-α could be useful as an adjuvant for cancer immunotherapy.
Full Text | PDF

Endocrine/metabolic disease

Top

CD3; insulin (INS; proinsulin); IL-10
doi:10.1038/scibx.2012.493
Studies in mice suggest Lactococcus lactis engineered to secrete both IL-10 and proinsulin could help treat type 1 diabetes.
Full Text | PDF

Hepatic disease

Top

Peroxisome proliferation–activated receptor-δ (PPARD; PPARd)
doi:10.1038/scibx.2012.494
Cell culture and mouse studies suggest PPARδ agonists could help treat liver fibrosis.
Full Text | PDF

Inflammation

Top

High mobility group box 1 (HMGB1); IL-1β; tumor necrosis factor-α (TNF-α)
doi:10.1038/scibx.2012.495
Rat and cell culture studies identified gold complexes of 6-substituted purine derivatives that could help treat inflammation.
Full Text | PDF

Musculoskeletal disease

Top

Peroxisome proliferation–activated receptor-γ (PPARG; PPARGγ)
doi:10.1038/scibx.2012.496
Mouse studies suggest PPARγ inhibitor–treated human mesenchymal stem cells (MSCs) and MSC-derived extracellular matrix (ECM) could help repair bone.
Full Text | PDF

D4Z4 binding element transcript (DBE-T)
doi:10.1038/scibx.2012.497
Cell culture studies suggest inhibiting DBE-T could help treat facioscapulohumeral dystrophy (FSHD), which is a common autosomal-dominant type of muscular dystrophy.
Full Text | PDF

MicroRNA-206 (miR-206)
doi:10.1038/scibx.2012.498
Mouse studies suggest agonizing miR-206 could be useful for treating muscle injury and Duchenne muscular dystrophy (DMD).
Full Text | PDF

Transplantation

Top

Not applicable
doi:10.1038/scibx.2012.499
Mouse studies suggest antibiotics could be useful for treating GvHD.
Full Text | PDF

Various

Top

Bromodomain containing 4 (BRD4)
doi:10.1038/scibx.2012.500
An in vitro study suggests the epigenetic target BRD4 also has kinase activity, which could be inhibited to treat cancer or inflammation.
Full Text | PDF

Distillery: Techniques

Assays and screens

Top

Aptamer-based assay against secreted proteins for the identification of new cancer biomarkers
doi:10.1038/scibx.2012.501
An aptamer-based assay of secreted proteins may help identify new cancer biomarkers.
Full Text | PDF

Paper-based assay for reporting blood type as text
doi:10.1038/scibx.2012.502
A paper-based assay was used to identify and display blood type as text.
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Computational models

Top

Algorithm for creating charged antibodies to improve stability and preserve activity
doi:10.1038/scibx.2012.503
In silico and in vitro studies identified a strategy to create positively charged antibodies that have better stability and lower aggregation than unmodified counterparts.
Full Text | PDF

Disease models

Top

Dopamine D2 receptor expression as a measure of Huntington's disease (HD) pathology
doi:10.1038/scibx.2012.504
Measuring dopamine D2 receptor expression in mouse models of HD could aid the study of therapeutics to treat the disease.
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Mice with working memory deficits as models of dementia-related delirium
doi:10.1038/scibx.2012.505
Mice with short-term deficits in working memory could be useful models of dementia-related delirium and could aid the discovery of new treatments.
Full Text | PDF

SH3 and multiple ankyrin repeat domains 2 (SHANK2)-deficient mouse model of autism spectrum disorder (ASD)
doi:10.1038/scibx.2012.506
Mice lacking Shank2 could be used as models to study ASD and develop new treatments for the condition.
Full Text | PDF

Drug delivery

Top

Acyclic cucurbit[n]urils as molecular containers to improve drug solubility
doi:10.1038/scibx.2012.507
Acyclic cucurbit[n]uril compounds may be useful as molecular containers to increase the solubility of poorly soluble drugs.
Full Text | PDF

Drug platforms

Top

Gene therapy for Leber's hereditary optical neuropathy (LHON)
doi:10.1038/scibx.2012.508
Cell culture and mouse studies suggest a mitochondria-targeted transgene could be useful for treating LHON.
Full Text | PDF

Markers

Top

Epigenomic enhancer variants as biomarkers for colorectal cancer
doi:10.1038/scibx.2012.509
In vitro studies identified epigenomic markers that could be used as biomarkers for colorectal cancer.
Full Text | PDF

Genomic and expression analysis–derived breast cancer subgroups to predict response to therapy
doi:10.1038/scibx.2012.510
Genomic and expression analysis–derived subgroups of breast cancer may help predict response to therapy.
Full Text | PDF

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