Thursday, February 20, 2014

SciBX: Science-Business eXchange Contents: February 20 2014, Volume 7 / Issue 7

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SciBX: Science-Business eXchange

TABLE OF CONTENTS

February 20 2014, Volume 7 / Issue 7

Analysis

Cover Story
Translational Notes
Targets and Mechanisms
Tools

The Distillery: Therapeutics

Autoimmune disease
Cancer
Endocrine/metabolic disease
Hematology
Infectious disease
Neurology

The Distillery: Techniques

Assays and screens
Drug platforms
Markers
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Cover Story

Top

Europe's upwelling
Steven Edelson and Kai-Jye Lou
doi:10.1038/scibx.2014.188
SciBX's third annual comprehensive analysis of public-private partnerships and early stage venture financing activity shows that Europe's PPP activity in 2013 surpassed that of the U.S. in dramatic fashion.
Full Text | PDF

Translational Notes

Top

BAND-aid for PD and AD
Michael J. Haas
doi:10.1038/scibx.2014.189
Evidence for overlaps between PD and AD have led three foundations to launch the BAND grant program to fund cross-disease research. The studies will use outputs from both the Parkinson's Progression Markers Initiative and the Alzheimer's Disease Neuroimaging Initiative.
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Targets and Mechanisms

Top

Preserving the oocyte reserve
Tracey Baas
doi:10.1038/scibx.2014.190
A Cornell team has preserved fertility in mice by knocking out checkpoint kinase 2. The target offers companies a new opportunity for preventing chemotherapy- and radiotherapy-induced premature menopause and infertility.
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Tools

Top

Degradation from within
Lev Osherovich
doi:10.1038/scibx.2014.191
A Cornell group has proof of concept for proteasomal degradation of intracellular proteins using designer binding proteins to deliver ubiquitin to their targets. The challenge for Ubiquizyme, a newco formed around this concept, is to deliver its molecules in a therapeutic setting.
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Distillery: Therapeutics

Autoimmune disease

Top

Invariant NK T (iNKT) cells
doi:10.1038/scibx.2014.192
In vitro and mouse studies suggest Bacteroides fragilis–derived glycosylceramide sphingolipids (GL-Cers) could help treat IBD by inhibiting activation of iNKT cells.
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Cancer

Top

SWI/SNF-related matrix-associated actin-dependent regulator of chromatin subfamily a member 4 (SMARCA4; BRG1)
doi:10.1038/scibx.2014.193
Studies in cell culture and mice suggest inhibiting BRG1 could help treat AML.
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TANK-binding kinase 1 (TBK1); estrogen receptor
doi:10.1038/scibx.2014.194
In vitro, mouse and patient sample studies suggest inhibiting TBK1 could help treat tamoxifen-resistant breast cancer and that measuring TBK1 expression could help predict response to tamoxifen.
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Procaspase-3
doi:10.1038/scibx.2014.195
In vitro and mouse studies suggest combining compounds that activate procaspase-3 by different mechanisms could help treat cancer.
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Stage-specific embryonic antigen-4 (SSEA-4)
doi:10.1038/scibx.2014.196
Human sample, in vitro and mouse studies suggest an anti-SSEA-4 mAb could be used to treat glioblastoma.
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Spleen tyrosine kinase (SYK)
doi:10.1038/scibx.2014.197
Primary tumor studies suggest measuring levels of two SYK isoforms could help predict treatment outcomes in patients with HCC.
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SWI/SNF-related matrix-associated actin-dependent regulator of chromatin subfamily a member 4 (SMARCA4; BRG1)
doi:10.1038/scibx.2014.198
Cell culture studies suggest inhibiting BRG1 could help treat MYC associated factor X (MAX) mutant small cell lung cancer (SCLC).
Full Text | PDF

Endocrine/metabolic disease

Top

Checkpoint kinase 2 (Chk2; CHEK2)
doi:10.1038/scibx.2014.199
Mouse studies suggest inhibiting CHK2 could prevent premature ovarian failure after radiotherapy or chemotherapy.
Full Text | PDF

α-l-iduronidase (IDUA)
doi:10.1038/scibx.2014.200
In vitro and mouse studies suggest megakaryocyte-targeted gene therapy could help treat mucopolysaccharidosis I (MPS I; Hurler syndrome).
Full Text | PDF

Hematology

Top

Hepcidin
doi:10.1038/scibx.2014.201
In vitro and mouse studies identified modified forms of heparin that could help treat chronic anemia.
Full Text | PDF

Infectious disease

Top

IgA
doi:10.1038/scibx.2014.202
Mouse studies suggest secretory IgA could help prevent bacterial infections in formula-fed neonates.
Full Text | PDF

Triggering receptor expressed on myeloid cells 1 (TREM1)
doi:10.1038/scibx.2014.203
Mouse studies suggest inhibiting the innate immune receptor TREM1 could help reduce inflammation caused by infection.
Full Text | PDF

Mycobacterium tuberculosis transmembrane serine/threonine-protein kinase B (pknB)
doi:10.1038/scibx.2014.204
Cell culture studies suggest inhibiting M. tuberculosis pknB could be useful for treating tuberculosis.
Full Text | PDF

Neurology

Top

Nuclear factor (erythroid-derived 2)-like 2 (NFE2L2; NRF2); heme oxygenase decycling 1 (HMOX1; HO-1; Hsp32)
doi:10.1038/scibx.2014.205
In vitro and mouse studies identified NRF2 pathway activators that could help treat PD.
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Distillery: Techniques

Assays and screens

Top

Diagnosing and monitoring treatment responses in patients with Niemann-Pick disease type C1 (NPC1) using fluorescence-based volumetric measurement of lysosomes
doi:10.1038/scibx.2014.206
A fluorescence-based assay in B cells could help monitor treatment responses and diagnose patients with NPC1.
Full Text | PDF

Screening antimetastatic compounds using microfluidic-based tracking of cell migration
doi:10.1038/scibx.2014.207
A microfluidic device that monitors cell migration could be used to screen antimetastatic compounds.
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Drug platforms

Top

Bone marrow niche–sensitizing chemotherapy to enhance antibody-based acute lymphoblastic leukemia (ALL) elimination
doi:10.1038/scibx.2014.208
Mouse and human sample studies suggest targeting tumor type–specific factors that suppress antitumor immunity could help eliminate residual ALL.
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Improved brain delivery of anti–transferrin receptor (TFRC; TFR)-containing antibodies by reducing TFRC affinity
doi:10.1038/scibx.2014.209
Mouse studies suggest lowering anti-TFRC antibody affinity could increase brain uptake of blood brain barrier (BBB)-penetrant, bispecific antibodies.
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Inducing multipotent progenitor cells from keratinocytes by depleting the tumor protein p63 (TP63; p63) ΔNp63 isoform or DGCR8 microprocessor complex subunit (DGCR8)
doi:10.1038/scibx.2014.210
In vitro studies suggest depleting ΔNp63 or DGCR8 in keratinocytes could induce their conversion into multipotent stem cells.
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Intracellularly expressed antibodies linked to the E3 ubiquitin ligase domain of STIP1 homology and U-box containing protein 1 (STUB1; CHIP) to enable targeted protein degradation
doi:10.1038/scibx.2014.211
In vitro and cell culture studies suggest intracellular expression of antibodies linked to the E3 ubiquitin ligase domain of CHIP could promote ubiquitin-mediated degradation of target proteins.
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Retinal progenitor cell therapy for retinal degeneration
doi:10.1038/scibx.2014.212
Rat studies suggest transplantation of retinal progenitor cells could help treat or prevent retinal degeneration.
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Viral insertion of inducible aptazymes to improve the safety of oncolytic viral therapy
doi:10.1038/scibx.2014.213
In vitro studies suggest inducible aptazymes that regulate viral gene expression could improve the safety of oncolytic viruses.
Full Text | PDF

Markers

Top

A cytosine methylation signature that predicts overall survival in acute myelogenous leukemia (AML)
doi:10.1038/scibx.2014.214
Studies of human samples suggest a cytosine methylation signature can predict overall survival of patients with AML.
Full Text | PDF

Top

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