Thursday, 25 July 2013

SciBX: Science-Business eXchange Contents: July 25 2013, Volume 6 / Issue 28

SciBX: Science-Business eXchange


TABLE OF CONTENTS

July 25 2013, Volume 6 / Issue 28

Analysis

Cover Story
Translational Notes
Targets and Mechanisms
Tools

The Distillery: Therapeutics

Autoimmune disease
Cancer
Dermatology
Infectious disease
Neurology
Other

The Distillery: Techniques

Disease models
Drug delivery
Drug platforms
Imaging
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Biopharma Dealmakers
A supplement to Nature Biotechnology and Nature Reviews Drug Discovery 

The May 2013 issue of Biopharma Dealmakers showcases companies with partnering opportunities and contains a special feature on oncology. This week, find out about how you can collaborate with Merck.


SciBX Collections
Macrocycles and constrained peptides

This special collection explores the factors behind the growing interest in macrocycles and constrained peptides over the last five years and their effect on drug discovery. 

Click here to access the collection for free!

Produced with support from:
Aileron Therapeutics, Inc., Polyphor LTD., PolyPeptide Group, Lanthio Pharma B.V.
 

Analysis

Cover Story

Top

MPS IIIA: gene therapy for brain and body
Michael J. Haas
doi:10.1038/scibx.2013.709
Within a year of reporting preclinical proof of concept for a systemically delivered MPS IIIA gene therapy, Autonomous University of Barcelona researchers have substantially improved efficacy in the CNS while still treating peripheral organs via intracerebral delivery. Partner Esteve is now raising funds to enter the clinic.
Full Text | PDF

Translational Notes

Top

MJFF's progress in markers
Michael J. Haas
doi:10.1038/scibx.2013.710
To identify clinical populations for testing new therapies for Parkinson's disease, the Michael J. Fox Foundation made available the first set of data and samples from its LRRK2 Cohort Consortium and presented preliminary data from its Parkinson's Progression Marker Initiative.
Full Text | PDF

Targets and Mechanisms

Top

Silencing neuropathic pain
C. Simone Fishburn
doi:10.1038/scibx.2013.711
Researchers at The Johns Hopkins University School of Medicine have identified a long noncoding RNA that is upregulated by nerve injury associated with neuropathic pain. The molecule silences voltage-gated potassium channel Kv1.2 exclusively in damaged neurons and could thus be a new target for the indication.
Full Text | PDF

Tools

Top

Universal appeal for CARs
Tracey Baas
doi:10.1038/scibx.2013.712
MD Anderson and Sangamo are overcoming the need for autologous cells in CAR-expressing T cell–based immunotherapies. The group has used gene-modifying tools to mask donor-derived T cells from immune surveillance machinery in the recipient and is planning clinical trials with such cells targeting CD19.
Full Text | PDF

Distillery: Therapeutics

Autoimmune disease

Top

Calcium channel N-type; calcium channel P/Q-type
doi:10.1038/scibx.2013.713
Cell-based and mouse studies identified an agonist of N- and P/Q-type calcium channels that could help treat Lambert-Eaton myasthenic syndrome.
Full Text | PDF

Cancer

Top

Ataxia telangiectasia mutated (ATM); DNA-dependent protein kinase (DNA-PK)
doi:10.1038/scibx.2013.714
Mouse and cell culture studies suggest inhibiting the DNA-PK catalytic subunit could help treat ATM-deficient cancers.
Full Text | PDF

Proteasome
doi:10.1038/scibx.2013.715
Cell culture studies suggest a noncompetitive, imidazoline-based proteasome inhibitor could help treat cancer.
Full Text | PDF

Stem cell factor receptor tyrosine kinase (c-Kit; KIT; CD117)
doi:10.1038/scibx.2013.716
In vitro studies suggest quinazoline-pyrazolourea–based KIT inhibitors could help treat Gleevec imatinib–resistant gastrointestinal stromal tumors (GISTs).
Full Text | PDF

E2F1; tumor protein p73 (TP73; p73)
doi:10.1038/scibx.2013.717
Cell culture and mouse studies suggest combining methotrexate with an antifolate prodrug could help treat melanoma.
Full Text | PDF

Mitochondrial oxidative phosphorylation
doi:10.1038/scibx.2013.718
Cell culture and mouse studies suggest inhibiting oxidative phosphorylation could help treat drug-resistant melanoma.
Full Text | PDF

Peroxiredoxin 2 (PRDX2)
doi:10.1038/scibx.2013.719
Mouse studies suggest compounds that mimic PRDX2 activity could help prevent melanoma metastasis.
Full Text | PDF

Aurora kinase A (AURKA; aurora-A); v-myc myelocytomatosis viral related oncogene neuroblastoma derived (MYCN; NMYC)
doi:10.1038/scibx.2013.720
Cell culture and mouse studies suggest AURKA inhibitors that disrupt the AURKA-NMYC complex could help treat neuroblastomas.
Full Text | PDF

Dermatology

Top

Septin 4 (SEPT4; ARTS)
doi:10.1038/scibx.2013.721
In vitro and mouse studies suggest inhibiting SEPT4 could help treat wounds and promote hair follicle regeneration.
Full Text | PDF

Infectious disease

Top

HBV core protein
doi:10.1038/scibx.2013.722
In vitro and mouse studies suggest the arginine-rich domain of HBV core protein could help treat bacterial infections.
Full Text | PDF

Not applicable
doi:10.1038/scibx.2013.723
In vitro and mouse studies suggest silver could be used to potentiate the effects of antibiotics to treat bacterial infections.
Full Text | PDF

Unknown
doi:10.1038/scibx.2013.724
Cell culture studies suggest anthracimycin derivatives could help treat bacterial infections.
Full Text | PDF

Unknown
doi:10.1038/scibx.2013.725
Cell culture and mouse studies suggest selective estrogen receptor modulators (SERMs) could help treat Ebola virus infection.
Full Text | PDF

Not applicable
doi:10.1038/scibx.2013.726
Rodent studies suggest the addition of Baraclude entecavir to plasmid- and adenoviral vector–based vaccination protocols could help treat chronic HBV infection.
Full Text | PDF

Mycobacterium tuberculosis decaprenylphosphoryl-β-D-ribose 2′-oxidase (dprE1); M. tuberculosis molybdopterin biosynthesis protein (moeW)
doi:10.1038/scibx.2013.727
Mouse and cell culture studies suggest an inhibitor of dprE1 and moeW could help treat drug-resistant tuberculosis infection.
Full Text | PDF

Estrogen receptor
doi:10.1038/scibx.2013.728
Cell culture and mouse studies suggest estradiol could help treat UTIs in postmenopausal women.
Full Text | PDF

Neurology

Top

Unknown
doi:10.1038/scibx.2013.729
Cell culture and mouse studies suggest guaifenesin could help treat diabetic neuropathy.
Full Text | PDF

Neurotrophic tyrosine kinase receptor 2 (NTRK2; TrkB)
doi:10.1038/scibx.2013.730
Mouse studies suggest NTRK2 inhibitors could help treat temporal lobe epilepsy.
Full Text | PDF

Purinergic receptor P2Y G protein–coupled 1 (P2RY1; P2Y1)
doi:10.1038/scibx.2013.731
Cell culture studies identified dual-acting antioxidant and P2Y1 receptor agonist nucleotides that could be useful as neuroprotectants.
Full Text | PDF

CREB binding protein (CREBBP; CBP); E1A binding protein p300 (EP300; p300)
doi:10.1038/scibx.2013.732
Mouse and cell culture studies suggest CBP and p300 agonists could be useful for improving cognitive function.
Full Text | PDF

μ-Opioid receptor (OPRM1; MOR); cannabinoid CB1 receptor (CNR1)
doi:10.1038/scibx.2013.733
SAR and mouse studies identified a bivalent dual MOR agonist and CNR1 antagonist that could be useful for treating pain.
Full Text | PDF

Potassium channel Kv1.2 (KCNA2)
doi:10.1038/scibx.2013.734
Rat studies suggest targeting KCNA2 antisense long noncoding RNA (lncRNA) could help treat neuropathic pain.
Full Text | PDF

Other

Top

Solute carrier family 22 organic cation transporter member 2 (SLC22A2; OCT2)
doi:10.1038/scibx.2013.735
Mouse and cell culture studies suggest inhibiting OCT2 could help decrease oxaliplatin-induced neurotoxicity.
Full Text | PDF

Distillery: Techniques

Disease models

Top

Matrix metalloproteinase 9 (MMP9)-deficient mice for modeling pre-eclampsia
doi:10.1038/scibx.2013.736
Mouse fetuses and pregnant mice lacking Mmp9 could be useful in identifying therapies to treat pre-eclampsia.
Full Text | PDF

Organoid model for colon cancer derived from primary intestinal epithelial cells
doi:10.1038/scibx.2013.737
Primary intestinal epithelial cells modified to form tumorigenic organoids in 3D cell culture could provide an in vitro model for colon cancer.
Full Text | PDF

Drug delivery

Top

Retinal delivery of gene therapy via intravitreal injection of an engineered adeno-associated viral (AAV) vector
doi:10.1038/scibx.2013.738
Intravitreal injection of an engineered AAV vector could enable retinal gene therapy.
Full Text | PDF

Serum protease–degradable peptide linkers to control antimicrobial peptide release
doi:10.1038/scibx.2013.739
Customized peptide linkers that are degraded by serum proteases could be used to control the release of therapeutic peptides from pegylated prodrugs.
Full Text | PDF

Drug platforms

Top

CpG-depleted adeno-associated virus (AAV) vectors for improved delivery to skeletal muscle
doi:10.1038/scibx.2013.740
AAV vectors depleted of the toll-like receptor 9 (TLR9) ligand CpG could improve the success of gene therapy for musculoskeletal diseases.
Full Text | PDF

Crystal structure of variable lymphocyte receptor (VLR) VLRB.aGPA.23 with a tumor-associated glycan antigen
doi:10.1038/scibx.2013.741
The crystal structure of VLR bound to a tumor-associated glycan antigen could be useful for designing tumor-targeting VLRs for therapeutic and diagnostic applications.
Full Text | PDF

Imaging

Top

Near-infrared fluorescent proteins (iRFPs) that allow multicolor, in vivo imaging
doi:10.1038/scibx.2013.742
In vitro and mouse studies identified four iRFPs that could be used for noninvasive, multicolor imaging in animals.
Full Text | PDF

Top
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