Thursday, January 31, 2013

SciBX: Science-Business eXchange Contents: January 31 2013, Volume 6 / Issue 4

SciBX: Science-Business eXchange

TABLE OF CONTENTS

January 31 2013, Volume 6 / Issue 4

Analysis

Cover Story
Translational Notes
Targets and Mechanisms
Public-Private Interface

The Distillery: Therapeutics

Autoimmune disease
Cancer
Cardiovascular disease
Endocrine/metabolic disease
Genitourinary disease
Infectious disease
Inflammation
Neurology
Various

The Distillery: Techniques

Drug platforms
Assays and screens
Disease models
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Analysis

Cover Story

Top

CRISPR genome editing
Chris Cain
doi:10.1038/scibx.2013.77
Five separate research teams have developed a Cas9 endonuclease–based method for sequence-specific genome modification guided by DNA-RNA base pairing. Although the approach is technically more straightforward than existing methods, such as zinc finger nucleases and transcription activator–like effector nucleases, its specificity has yet to be fully determined.
Full Text | PDF

Translational Notes

Top

Translational globetrotting
Steve Edelson
doi:10.1038/scibx.2013.78
Six like-minded translational research centers have partnered to help speed the commercialization of academic research. The international cadre, dubbed the Global Alliance of Leading Drug Discovery and Development Centres, comprises more than 400 drug developers working on at least 165 projects.
Full Text | PDF

Targets and Mechanisms

Top

Toll-erating AD
Lev Osherovich
doi:10.1038/scibx.2013.79
Laval University and GlaxoSmithKline have shown that peripheral administration of the adjuvant MPL can help treat Alzheimer's disease in mice. Although the findings point to a potential immunomodulatory approach for addressing the neurodegenerative disease, the pharma is now focusing on elucidating the mechanism before advancing the program.
Full Text | PDF

Public-Private Interface

Top

Evotec's growing Ivy
Kai-Jye Lou
doi:10.1038/scibx.2013.80
Evotec is double dipping into the Ivy League, this time partnering with Yale University to move early stage research from the university's labs into late preclinical and IND-ready assets for third parties to license. The Yale collaboration is upstream of the biotech's 2011 deal with Harvard University because the projects are not predefined.
Full Text | PDF

Distillery: Therapeutics

Autoimmune disease

Top

Desmoglein 1 (DSG1); DSG3
doi:10.1038/scibx.2013.81
In vitro and mouse studies identified a DSG-binding peptide that could help treat PV.
Full Text | PDF

Cancer

Top

NADP-dependent malic enzyme 1 cytosolic (ME1); NADP-dependent malic enzyme 2 mitochondrial (ME2); p53
doi:10.1038/scibx.2013.82
Cell culture and mouse studies suggest inhibiting ME1 or ME2 may help treat cancer.
Full Text | PDF

NGFI-A binding protein 2 (EGR1 binding protein 2; NAB2); signal transducer and activator of transcription 6 (STAT6)
doi:10.1038/scibx.2013.83
Two separate genetic sequencing studies identified a NAB2-STAT6 gene fusion associated with solitary fibrous tumors (SFTs), which could provide a new therapeutic target.
Full Text | PDF

BCR-ABL tyrosine kinase
doi:10.1038/scibx.2013.84
Cell culture and mouse studies suggest the BCR-ABL tyrosine kinase inhibitor GZD824 could help treat imatinib-resistant cancer.
Full Text | PDF

VEGF
doi:10.1038/scibx.2013.85
Mouse and human studies suggest Votrient pazopanib, a VEGF signaling inhibitor, could help improve the efficacy of paclitaxel in anaplastic thyroid cancer.
Full Text | PDF

Cardiovascular disease

Top

POU class 1 homeobox 1 (POU1F1; PIT1)
doi:10.1038/scibx.2013.86
Cell culture and mouse studies suggest spironolactone could help prevent calcification of vascular and other soft tissues, which is seen in patients who have chronic kidney disease (CKD).
Full Text | PDF

Endocrine/metabolic disease

Top

Free fatty acid receptor 1 (FFAR1; GPR40)
doi:10.1038/scibx.2013.87
In vitro and mouse studies identified an alkyne series of FFAR1 agonists that could help treat type 2 diabetes.
Full Text | PDF

Genitourinary disease

Top

Prolactin releasing hormone receptor (PRLHR; GPR10)
doi:10.1038/scibx.2013.88
Patient sample and mouse studies suggest inhibiting PRLHR could help treat uterine fibroids.
Full Text | PDF

Infectious disease

Top

Lysine-specific demethylase 4 (KDM4; JMJD2)
doi:10.1038/scibx.2013.89
Cell culture and explanted mouse ganglia studies suggest JMJD2 inhibitors could be used to treat primary HSV-1 infection and prevent reactivation.
Full Text | PDF

Influenza A virus matrix protein 2 (M2)
doi:10.1038/scibx.2013.90
In vitro studies identified M2 inhibitors that could help treat amantadine-resistant influenza.
Full Text | PDF

Inflammation

Top

Leptin
doi:10.1038/scibx.2013.91
Mouse studies suggest anticholinergic compounds could help treat obesity-induced asthma.
Full Text | PDF

Neurology

Top

Histone deacetylase 3 (HDAC3)
doi:10.1038/scibx.2013.92
Mouse studies suggest HDAC3 inhibition could help treat cocaine addiction.
Full Text | PDF

Toll-like receptor 4 (TLR4)
doi:10.1038/scibx.2013.93
Cell culture and mouse studies suggest moderately agonizing TLR4 could help treat AD.
Full Text | PDF

Hypoxia-inducible factor prolyl hydroxylase (HIF-PH; EGLN); hypoxia-inducible factor 1α (HIF1A; HIF1α)
doi:10.1038/scibx.2013.94
Human and rabbit studies suggest compounds that inhibit HIF-PH could prevent neurogenesis deficits in preterm infants.
Full Text | PDF

Various

Top

Caspase recruitment domain family member 15 (CARD15; NOD2)
doi:10.1038/scibx.2013.95
Mouse studies suggest intestinal microbiota from organisms with functional NOD2 could help protect against colitis and colitis-associated colorectal cancer.
Full Text | PDF

Distillery: Techniques

Assays and screens

Top

DNA analysis of Pap smear samples to detect ovarian and endometrial cancer
doi:10.1038/scibx.2013.96
DNA sequencing of Pap smear samples could help diagnose ovarian and endometrial cancer.
Full Text | PDF

Disease models

Top

Induced pluripotent stem (iPS) cell–derived models of familial hypertrophic cardiomyopathy (HCM)
doi:10.1038/scibx.2013.97
Patient-derived iPS cell models of familial HCM could help identify new treatments for the disease.
Full Text | PDF

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Mouse model for lymphocytic choriomeningitis virus (LCMV) infection that produces hemorrhagic-like disease
doi:10.1038/scibx.2013.98
A new mouse model could be used to screen for therapeutics or test vaccines against arenavirus infections that cause hemorrhagic-like disease.
Full Text | PDF

Transgenic miniature pig model for hypercholesterolemia and atherosclerosis
doi:10.1038/scibx.2013.99
A transgenic miniature pig model for hypercholesterolemia and atherosclerosis could aid the development of imaging technologies, intravascular devices and therapeutics.
Full Text | PDF

Drug platforms

Top

Antigen-specific T cells generated from induced pluripotent stem (iPS) cells derived for adoptive immunotherapy
doi:10.1038/scibx.2013.100
Two separate groups showed that iPS cells derived from mature CD8+ T cells can be differentiated into antigen-specific T cells that may be useful for overcoming T cell exhaustion during adoptive immunotherapy.
Full Text | PDF

Clustered, regularly interspaced short palindromic repeats (CRISPR) RNA editing system to modify mammalian DNA
doi:10.1038/scibx.2013.101
A CRISPR-derived genome editing system could be used to modify mammalian DNA.
Full Text | PDF

Sortase-mediated modification of a targeted mAb to improve antigen delivery and presentation
doi:10.1038/scibx.2013.102
Sortase-mediated modification of therapeutic mAbs could be useful for developing vaccines and therapeutics to treat viral infections.
Full Text | PDF

Structure-based design of peptide inhibitors and activators of ubiquitin enzymes
doi:10.1038/scibx.2013.103
In vitro studies identified a strategy to develop peptide inhibitors and activators of ubiquitin pathway enzymes that could help treat various diseases.
Full Text | PDF

Terminally differentiated stem cell–derived cells with negligible immunogenicity
doi:10.1038/scibx.2013.104
Terminally differentiated stem cell–derived cells show negligible immunogenicity, suggesting they could be useful for transplant and cell therapy applications.
Full Text | PDF

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