Archive for the ‘Gene Therapy Research’ Category

By Daily Mail Reporter

PUBLISHED: 03:02 EST, 1 October 2012 | UPDATED: 03:05 EST, 1 October 2012

A gene that causes 1,000 babies to be born deaf in Britain each year has been discovered by scientists.

It is hoped the breakthrough will lead to new treatments for profound deafness.

Research published online in Nature Genetics shows babies born deaf have mutated versions of a protein called CIB2.

Two deaf children use sign language: Scientists hope their latest genetic discovery will help with developing new treatments

The gene binds to calcium within a cell and is behind a condition known as Usher syndrome type 1 that causes deafness and 'non-syndromic' hearing loss, the study shows.

Dr Zubair Ahmed, of the University of Cincinnati, said: 'In this study, researchers were able to pinpoint the gene which caused deafness in Usher syndrome type 1 as well as deafness that is not associated with the syndrome through the genetic analysis of 57 humans from Pakistan and Turkey.'

He said these findings could help researchers develop new therapeutic targets for those at risk for this syndrome.

Usher syndrome is a genetic defect that causes deafness, night-blindness and a loss of peripheral vision through the progressive degeneration of the retina.

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Deafness gene discovery could lead to new treatments

Editor's Note: Charles Gersbach can be reached at (919) 684-1129 or charles.gersbach@duke.edu. Lauren Polstein can be reached at (919) 688-9100 or Lauren.polstein@duke.edu. A photograph of the researchers is available, as well as an example of the gene expression.

Durham, NC - Using blue light, Duke University bioengineers have developed a system for ordering genes to produce proteins, an advance they said could prove invaluable in clinical settings as well as in basic science laboratories.

This new approach could greatly improve the ability of researchers and physicians to control gene expression, which is the process by which genes give instructions for the production of proteins key to all living cells.

"We can now, with our method, make gene expression reversible, repeatable, tunable, and specific to different regions of a gene," said Lauren Polstein, a graduate student working in the laboratory of Charles Gersbach, assistant professor of biomedical engineering at Duke's Pratt School of Engineering. "Current methods of getting genes to express can achieve some of those characteristics, but not all at once."

The new system can also control where the genes are expressed in three dimensions, which becomes especially important for researchers attempting to bioengineer living tissues.

"The light-based strategy allows us to regulate gene expression for biotechnology and medical applications, as well as for gaining a better understanding of gene function, interactions between cells, and how tissues develop into particular shapes," Polstein said.

The results of the Duke experiments were published online in the Journal of the American Chemical Society (http://tinyurl.com/brt3plh). The research was supported by a Faculty Early Career Development Award from the National Science Foundation and a Director's New Innovator Award from the National Institutes of Health.

The Duke system, which has been dubbed LITEZ (Light Induced Transcription using Engineered Zinc finger proteins), combines proteins from two diverse sources. The light-sensitive proteins are derived from a common flowering plant (Arabidopsis thaliana).

"We hijacked the specific proteins in plants that allows them to sense the length of the day," Gersbach said.

The second protein is in a class of so-called zinc finger proteins, which can be readily engineered to attach to specific regions of a gene. They are ubiquitous in biomedical research.

More here:
Duke Blue Light Controls Gene Expression

Washington, Oct 1 (IANS) Going deaf? Blame a genetic mutation, linked with Usher syndrome type 1, says the latest finding, which could help develop more effective ways of treating this syndrome.

Usher syndrome is a genetic defect that causes deafness, night-blindness and a loss of peripheral vision through the progressive degeneration of the retina.

Researchers from the University of Cincinnati and Cincinnati Children's Hospital Medical Centre, partnered the study with the National Institute on Deafness and other Communication Disorders (NIDCD), Baylor College of Medicine and the University of Kentucky, the journal Nature Genetics reports.

"Researchers were able to pinpoint the gene which caused deafness in Usher syndrome type 1 as well as deafness that is not associated with the syndrome through the genetic analysis of 57 humans from Pakistan and Turkey," says Zubair Ahmed, assistant professor of ophthalmology from Cincinnati Children's and the study's lead investigator.

Ahmed says that a protein, called CIB2, which binds to calcium within a cell, is associated with deafness in Usher syndrome type 1 and non-syndromic hearing loss. "To date, mutations affecting CIB2 are the most common and prevalent genetic cause of non-syndromic hearing loss in Pakistan," he says, according to a Cincinnati statement.

"With this knowledge, we are one step closer to understanding the mechanism of mechano-electrical transduction and possibly finding a genetic target to prevent non-syndromic deafness as well as that associated with Usher syndrome type 1," Ahmed says.

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Genetic defect plays role in hearing loss too

Public release date: 30-Sep-2012 [ | E-mail | Share ]

Contact: Hilary Glover hilary.glover@biomedcentral.com 44-020-319-22370 BioMed Central

Vibration-induced white finger disease (VWF) is caused by continued use of vibrating hand held machinery (high frequency vibration >50 Hz), and affects tens of thousands of people. New research published in BioMed Central's open access journal Clinical Epigenetics finds that people with a genetic polymorphism (A2191G) in sirtuin1 (SIRT1), a protein involved in the regulation of endothelial NOS (eNOS), are more likely to suffer from vibration-induced white finger disease.

VWF (also known as hand arm vibration syndrome (HAVS)) is a secondary form of Raynaud's disease involving the blood vessels and nerves of arms, fingers and hands. Affected fingers feel stiff and cold and loose sensation for the duration of the attack, which can be very painful. Loss of sensation can make it difficult to carry out manual activities. Initially attacks are triggered by cold temperatures but as the disease progresses attacks can occur at any time.

Little is known about what causes the restriction in blood flow, however researchers from Germany investigated the role of SIRT 1 by looking at polymorphisms (naturally occurring variations in DNA sequence) in people affected by VWF.

SIRT1 regulates activation of other genes by controlling how tightly DNA is wound in the nucleus. Tightly wound DNA cannot be 'read' and consequently cannot be used to make new protein. SIRT1 is known to regulate vasodilation by targeting eNOS, a nitric oxide synthase within the cells lining the inside of blood vessels, which regulates smooth muscle contraction, and hence the diameter of the vessel, and the amount of blood that can flow through it.

Of 113 polymorphisms tested, in the gene coding for SIRT1, only four actually affected the protein, the rest were non-coding or false positives. Of these four, only one was different between people with VWF and unaffected controls. A single nucleotide at position 2191 can either be an A or a G. In the unaffected population 99.7% had the A, but amongst the patients with VWF, almost a third had the G.

Dr Susanne Voelter-Mahlknecht from the University of Tuebingen, who led this study, explained, "While this does not mean that only people with the G version of the gene for SIRT1 will get VWF, it can be used to identify a set of people who would be at risk of VWF if they used vibrating hand held tools. Testing for this variant before starting to work with vibrating machinery could prevent years of pain and disability."

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Media contact

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The genetics of white finger disease

Public release date: 1-Oct-2012 [ | E-mail | Share ]

Contact: Phyllis Edelman pedelman@genetics-gsa.org 301-634-7302 Genetics Society of America

BETHESDA, MD October 1, 2012 With flu season around the corner, getting a seasonal vaccine might be one of the best ways to prevent people from getting sick. These vaccines only work, however, if their developers have accurately predicted which strains of the virus are likely to be active in the coming season because vaccines must be developed in advance of the upcoming flu season. Recently, a team of scientists from Germany and the United Kingdom have improved the prediction methods used to determine which strains of the flu virus to include in the current season's vaccine. The research describing this advance is published in the October 2012 issue of GENETICS (http://www.genetics.org).

"Seasonal influenza kills about half a million people per year, but improved vaccines can curb this number," said Michael Lssig, Ph.D., a researcher involved in the work from the Institute for Theoretical Physics at the University of Cologne in Kln, Germany. "Although this study is some distance from direct applications, it is a necessary step toward improved prediction methods. We hope that it helps yield better vaccines against influenza," Lssig added.

To make this advance, scientists analyzed the DNA sequences of thousands of influenza strains isolated from patients worldwide, dating to 1968. By analyzing this dataset, researchers were able to determine which strains were most successful at expanding into the entire population, and which mutations were least successful in spreading. Using a new statistical method, the researchers found that many more mutations than we thought initially succeed in replicating and surviving. These mutations compete; some make it into the entire population, others die out. This analysis of the virus enables prediction of trends which can help vaccine developers understand the rules of flu virus evolution. This knowledge, in turn, can be used to predict which strains of the virus are most likely to spread through a human population.

"Every year, new concerns emerge about 'super flus' that have the potential to kill many people," said Mark Johnston, Editor-in-Chief of the journal GENETICS. "This research itself will not stop any people from getting sick, but it could give us a heads up to particularly dangerous strains that might be on the horizon. With that information, we may be able to develop increasingly effective vaccines."

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FUNDING: This work was partially supported by the Wellcome Trust [080711/ Z/06] (N.S.) and by Deutsche Forschungsgemeinschaft grant SFB 680 (to M.L.). This work was also supported in part by the National Science Foundation under grant PHY05-51164 during a visit to the Kavli Institute of Theoretical Physics (University of California, Santa Barbara).

CITATION: Natalja Strelkowa and Michael Lssig Clonal Interference in the Evolution of Influenza Genetics October 2012 192:671-682

ABOUT GENETICS: Since 1916, GENETICS (http://www.genetics.org/) has covered high quality, original research on a range of topics bearing on inheritance, including population and evolutionary genetics, complex traits, developmental and behavioral genetics, cellular genetics, gene expression, genome integrity and transmission, and genome and systems biology. GENETICS, a peer-reviewed, peer-edited journal of the Genetics Society of America is one of the world's most cited journals in genetics and heredity.

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Evolutionary analysis improves ability to predict the spread of flu

ScienceDaily (Sep. 28, 2012) Vibration-induced white finger disease (VWF) is caused by continued use of vibrating hand held machinery (high frequency vibration >50 Hz), and affects tens of thousands of people. New research published in BioMed Central's open access journal Clinical Epigenetics finds that people with a genetic polymorphism (A2191G) in sirtuin1 (SIRT1), a protein involved in the regulation of endothelial NOS (eNOS), are more likely to suffer from vibration-induced white finger disease.

VWF (also known as hand arm vibration syndrome (HAVS)) is a secondary form of Raynaud's disease involving the blood vessels and nerves of arms, fingers and hands. Affected fingers feel stiff and cold and loose sensation for the duration of the attack, which can be very painful. Loss of sensation can make it difficult to carry out manual activities. Initially attacks are triggered by cold temperatures but as the disease progresses attacks can occur at any time.

Little is known about what causes the restriction in blood flow, however researchers from Germany investigated the role of SIRT 1 by looking at polymorphisms (naturally occurring variations in DNA sequence) in people affected by VWF.

SIRT1 regulates activation of other genes by controlling how tightly DNA is wound in the nucleus. Tightly wound DNA cannot be 'read' and consequently cannot be used to make new protein. SIRT1 is known to regulate vasodilation by targeting eNOS, a nitric oxide synthase within the cells lining the inside of blood vessels, which regulates smooth muscle contraction, and hence the diameter of the vessel, and the amount of blood that can flow through it.

Of 113 polymorphisms tested, in the gene coding for SIRT1, only four actually affected the protein, the rest were non-coding or false positives. Of these four, only one was different between people with VWF and unaffected controls. A single nucleotide at position 2191 can either be an A or a G. In the unaffected population 99.7% had the A, but amongst the patients with VWF, almost a third had the G.

Dr Susanne Voelter-Mahlknecht from the University of Tuebingen, who led this study, explained, "While this does not mean that only people with the G version of the gene for SIRT1 will get VWF, it can be used to identify a set of people who would be at risk of VWF if they used vibrating hand held tools. Testing for this variant before starting to work with vibrating machinery could prevent years of pain and disability.

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The above story is reprinted from materials provided by BioMed Central Limited.

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The genetics of vibration-induced white finger disease

CAMBRIDGE, Mass. & BOTHELL, Wash.--(BUSINESS WIRE)--

Millennium: The Takeda Oncology Company, a wholly owned subsidiary of Takeda Pharmaceutical Company Limited (TSE:4502), and Seattle Genetics, Inc. (SGEN), today announced preliminary data from one arm of a three arm phase I, open-label, multicenter study designed to determine the safety and activity of sequential and combination treatment approaches of brentuximab vedotin with CHOP1 or CH-P chemotherapy in newly diagnosed patients with CD30-positive mature T- and NK- cell lymphomas. Data were presented from one arm, which evaluates brentuximab vedotin in sequence with CHOP in patients with newly diagnosed systemic anaplastic large cell lymphoma (sALCL), a subtype of mature T- and NK cell lymphomas. The data were reported during an oral presentation at the ESMO 2012 Congress (European Society for Medical Oncology) being held September 28 October 2, 2012 in Vienna, Austria.

Brentuximab vedotin is an antibody-drug conjugate (ADC) directed to CD30, a defining marker of sALCL. Data from the other arms of the study evaluating administration of brentuximab vedotin with CH-P are expected to be presented during 2012.

The preliminary data from the sequential treatment arm of this phase I study show that side effects were manageable and there are also some encouraging data on activity, said Michelle Fanale, M.D., University of Texas MD Anderson Cancer Center, Houston, TX. We look forward to reporting additional data from the combination arms of the study at an upcoming medical congress.

Sequential Therapy with Brentuximab Vedotin in Newly Diagnosed Patients with Systemic Anaplastic Large Cell Lymphoma

The oral presentation featured data from Arm 1 of the study, which evaluated treatment with 1.8milligrams per kilogram of single-agent brentuximab vedotin for two cycles prior to sixcycles of CHOP. Patients who achieved a complete remission (CR) or partial remission (PR) following CHOP were eligible to continue single-agent brentuximab vedotin for up to eight cycles. The primary endpoints for Arm 1 are safety and tolerability. The secondary endpoint is investigator assessment of response using the Revised Response Criteria for Malignant Lymphoma (Cheson 2007). The median age of enrolled patients was 62years (range, 23-81). Among the 13 patients with newly diagnosed sALCL, ten had anaplastic lymphoma kinase (ALK)-negative disease and three had ALK-positive disease.

The companies plan to initiate a phase III clinical trial in patients with mature T-cell lymphomas evaluating brentuximab vedotin in combination with CH-P compared to CHOP in late 2012 to early 2013.

Data, presented by Dr. Fanale, included:

Details of the oral presentation are as follows:

Data on brentuximab vedotin was also described in the following poster session:

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Millennium and Seattle Genetics Highlight Data from ADCETRIS® (Brentuximab Vedotin) Trial in Patients with Newly ...

Newswise Bethesda, MDOctober 1, 2012 Listed below are the selected highlights for the October 2012 issue of the Genetics Society of Americas journal, GENETICS. The October issue is available online at http://www.genetics.org/content/current. Please credit GENETICS, Vol. 192, October 2012, Copyright 2012.

Please feel free to forward to colleagues who may be interested in these articles.

ISSUE HIGHLIGHTS

Energy-dependent modulation of glucagon-like signaling in Drosophila via the AMP-activated protein kinase, pp. 457466 Jason T. Braco, Emily L. Gillespie, Gregory E. Alberto, Jay E. Brenman, and Erik C. Johnson How organisms maintain energetic homeostasis is unclear. These authors show that the actions of a known cellular sensor of energythe AMP-activated protein kinase (AMPK)cause release of a glucagon-like hormone in Drosophila. They further show that AMPK regulates secretion of adipokinetic hormone. This suggests new roles and targets for AMPK and suggests metabolic networks are organized similarly throughout Metazoa.

The relation of codon bias to tissue-specific gene expression in Arabidopsis thaliana, pp. 641649 Salvatore Camiolo, Lorenzo Farina, and Andrea Porceddu This article reports systematic differences in usage of synonymous codons in Arabidopsis thaliana genes whose expression is tissue specific. The authors propose that codon bias evolves as an adaptive response to the different abundances of tRNAs in different tissues. Integrity and function of the Saccharomyces cerevisiae spindle pole body depends on connections between the membrane proteins Ndc1, Rtn1, and Yop1, pp. 441455 Amanda K. Casey, T. Renee Dawson, Jingjing Chen, Jennifer M. Friederichs, Sue L. Jaspersen, and Susan R. Wente Budding yeast face an unusual challenge during cell division: they must segregate their chromosomes while the nuclear envelope remains intact. Consequently, mitosis begins with insertion of the duplicated spindle pole body (a.k.a. centrosome) into the nuclear envelope, a process that parallels the generation of new nuclear pore complexes. These authors report data that suggest new mechanisms for linking nuclear division and transport.

Cellular memory of acquired stress resistance in Saccharomyces cerevisiae, pp. 495505 Qiaoning Guan, Suraiya Haroon, Diego Gonzlez Bravo, Jessica L. Will, and Audrey P. Gasch Cells can retain memory of prior experiences that influence future behaviors. Here, the authors show that budding yeast retains a multifaceted memory of prior stress treatment. Cells pretreated with salt retain peroxide tolerance for several generations after removal of the initial stressor. This is due to long-lived catalase, produced during salt treatment and distributed to daughter cells. These cells also display transcriptional memory dependent on the nuclear pore subunit Nup42 that functions to promote reacquisition of stress tolerance in future stress cycles.

Genomic variation in natural populations of Drosophila melanogaster, pp. 533598 Charles H. Langley, Kristian Stevens, Charis Cardeno, Yuh Chwen G. Lee, Daniel R. Schrider, John E. Pool, Sasha A. Langley, Charlyn Suarez, Russell B. Corbett-Detig, Bryan Kolaczkowski, Shu Fang, Phillip M. Nista, Alisha K. Holloway, Andrew D. Kern, Colin N. Dewey, Yun S. Song, Matthew W. Hahn, and David J. Begun This article greatly extends studies of population genetic variation in natural populations of Drosophila melanogaster, which have played an important role in the development of evolutionary theory. The authors describe genome sequences of 43 individuals taken from two natural populations of D. melanogaster. The genetic polymorphism, divergence, and copy-number variation revealed in these data are presented at several scales, providing unprecedented insight into forces shaping genome polymorphism and divergence.

Estimating allele age and selection coefficient from time-serial data, pp. 599607 Anna-Sapfo Malaspinas, Orestis Malaspinas, Steven N. Evans, and Montgomery Slatkin The relative importance of the four fundamental processes driving evolutiongenetic drift, natural selection, migration, and mutationremains undetermined. These authors propose a new approach to estimate the selection coefficient and the allele age of time serial data. They apply their methodology to ancient sequences of a horse coat color gene and demonstrate that the causative allele existed as a rare segregating variant prior to domestication. This illuminates the debate on the relative importance of new vs. standing variation in adaptation and domestication. DNA replication origin function is promoted by H3K4 di-methylation in Saccharomyces cerevisiae, pp. 371384 Lindsay F. Rizzardi, Elizabeth S. Dorn, Brian D. Strahl, and Jeanette Gowen Cook What defines a DNA replication origin? It is becoming increasingly apparent that post-translational modifications of nucleosomes near replication origins help mark them and control their activity. The genetic analysis presented in this article implicates di-methylated histone H3 lysine 4 (stimulated by histone H2B monoubiquitination) as part of the definition of active replication origins. Since these histone modifications are highly conserved, these findings are relevant to genome organization in other eukaryotes.

Comparative oncogenomics implicates the Neurofibromin 1 gene (NF1) as a breast cancer driver, pp. 385396 Marsha D. Wallace, Adam D. Pfefferle, Lishuang Shen, Adrian J. McNairn, Ethan G. Cerami, Barbara L. Fallon, Vera D. Rinaldi, Teresa L. Southard, Charles M. Perou, and John C. Schimenti This study of a mouse model of genomic instability indicates that NF1 (Neurofibromin 1) deficiency can drive breast cancer. ~ 63,000 people in the United States annually will develop breast cancer with an NF1 deficiency. Together with evidence that NF1 depletion confers resistance of human breast cancer cells to tamoxifen, these findings suggest therapeutic strategies for patients with NF1-deleted tumors.

ABOUT GENETICS: Since 1916, GENETICS (http://www.genetics.org/) has covered high quality, original research on a range of topics bearing on inheritance, including population and evolutionary genetics, complex traits, developmental and behavioral genetics, cellular genetics, gene expression, genome integrity and transmission, and genome and systems biology. GENETICS, a peer-reviewed, peer-edited journal of the Genetics Society of America is one of the world's most cited journals in genetics and heredity.

See the rest here:
GENETICS Journal Highlights for October 2012

Consumer Genetics Conference 2012 Kicks Off This Week in Boston Featuring Preeminent Leaders in Industry, Academia, Medicine and Government to Examine Advancements, Applications, Issues and Challenges in Personal Genomics and 21st Century Healthcare

~Harvards Professor of Genetics George Church to Discuss his New Book During Keynote~ ~X PRIZE Foundation Special Announcement on Day One~ ~Three Days of Presentations, Panel Discussions and Lively Interchange at this Highly Interactive and Unique Forum Expected To Help Shape and Guide This Emerging Industry ~

Newswise Boston, MA, October 1, 2012 -- The 4th Annual Consumer Genetics Conference takes place this week, October 3-5 at the Boston Seaport Hotel. This unique interactive forum, will highlight three major themes regarding this burgeoning industry: Technology (Day 1), Business & Translation (Day 2) and Applications (Day 3). Presentations will specifically cover a variety of topics including: personal genomics, next and third generation sequencing, molecular diagnostics, industry funding and investment, and the current and future applications of genomics in clinical practices and as well as in nutrition, food genetics and cosmetics.

Along with program presentations and panel sessions, there are several notable keynote addresses and a few major news announcements: Day One will open with Lee Silver, Professor of Molecular Biology and Public Affairs of Princeton University presenting: Self-Discovery in the Age of personal Genomics (8:45 a.m.). This presentation will explore how consumer genetics has blossomed from infancy to adolescence with an array of consumer-facing products. It will also examine how this cottage industry is still struggling with growing pains as it faces a mix of regulators, restless innovators, and demanding and empowered patients.

Day One will also highlight: A keynote by George Church, Professor of Genetics and Director of the Center for Computational Genetics at Harvard Medical School and Father of the Personal Genome Project, will be given at the close of Day One (5:20 p.m.) with Professor Church providing a candid assessment of new sequencing technologies, current trends in personal genomics and projections on the future path of genomics in medicine. This address will come in the wake of his new book launch: Regenesis: How Synthetic Biology Will Reinvent Nature and Ourselves. Professor Church will be available for conversations and interviews following his presentation. A welcome reception will take place at 6:00 p.m.

There will also be a special X PRIZE Foundation announcement at the Consumer Genetics Conference about the Archon Genomics X PRIZE presented by Express Scripts in the afternoon at the Conference on October 3rd. This announcement will be consistent with X Prizes overall mission which is to bring about radical breakthroughs for the benefit of humanity. In this case, the news is expected to usher in a new era of personalized medicine through whole genome sequencing. [More information can be obtained by contacting Grant Company, Senior Director & Prize Lead for the Genomics X PRIZE, who will be in attendance at the conference in Boston.]

The featured presentation on Day 2 will be delivered by Marc Salit, Group Leader, National Institute of Standards and Technology, where he will present: Genome-in-a Bottle: Reference Materials and Methods for Confidence in Whole Genome Sequencing. In this presentation, he will explore the need for reference materials, methods and data to measure results for sound, reproducible research and regulated applications of whole genomic sequencing in the clinic.

Also on Day Two there will be two notable panel presentations: VC and Investment Banking panel (10:45 a.m.) focused on the corporate criteria they look for when funding companies involved in genomics, and the metrics that guide their decisions. A Physicians Perspective on deploying genomics and sequencing data in preventative and clinical care (1:45 p.m.)

On Day 3, the keynote will be presented by Kenneth Chahine of Ancestry.com, which has the worlds largest online resource for family history with an extensive collection of over 10 billion historical records. This keynote will focus on their new direct-to-consumer genealogical DNA test that delivers (i) prediction of identity-by-descent and a test that allows customers to find genetic relatives , and (ii) the individuals admixture to provide a predicted genetic ethnicity.

Highlights on Day Three will also include:

More here:
Consumer Genetics Conference 2012 Kicks Off This Week in Boston

Public release date: 1-Oct-2012 [ | E-mail | Share ]

Contact: Phyllis Edelman pedelman@genetics-gsa.org 301-634-7302 Genetics Society of America

Bethesda, MDOctober 1, 2012 Listed below are the selected highlights for the October 2012 issue of the Genetics Society of America's journal, GENETICS. The October issue is available online at http://www.genetics.org/content/current. Please credit GENETICS, Vol. 192, October 2012, Copyright 2012.

Please feel free to forward to colleagues who may be interested in these articles.

ISSUE HIGHLIGHTS

Energy-dependent modulation of glucagon-like signaling in Drosophila via the AMP-activated protein kinase, pp. 457 Jason T. Braco, Emily L. Gillespie, Gregory E. Alberto, Jay E. Brenman, and Erik C. Johnson How organisms maintain energetic homeostasis is unclear. These authors show that the actions of a known cellular sensor of energythe AMP-activated protein kinase (AMPK)cause release of a glucagon-like hormone in Drosophila. They further show that AMPK regulates secretion of adipokinetic hormone. This suggests new roles and targets for AMPK and suggests metabolic networks are organized similarly throughout Metazoa.

The relation of codon bias to tissue-specific gene expression in Arabidopsis thaliana, pp. 641 Salvatore Camiolo, Lorenzo Farina, and Andrea Porceddu This article reports systematic differences in usage of synonymous codons in Arabidopsis thaliana genes whose expression is tissue specific. The authors propose that codon bias evolves as an adaptive response to the different abundances of tRNAs in different tissues.

Integrity and function of the Saccharomyces cerevisiae spindle pole body depends on connections between the membrane proteins Ndc1, Rtn1, and Yop1, pp. 441 Amanda K. Casey, T. Renee Dawson, Jingjing Chen, Jennifer M. Friederichs, Sue L. Jaspersen, and Susan R. Wente Budding yeast face an unusual challenge during cell division: they must segregate their chromosomes while the nuclear envelope remains intact. Consequently, mitosis begins with insertion of the duplicated spindle pole body (a.k.a. centrosome) into the nuclear envelope, a process that parallels the generation of new nuclear pore complexes. These authors report data that suggest new mechanisms for linking nuclear division and transport.

Cellular memory of acquired stress resistance in Saccharomyces cerevisiae, pp. 495 Qiaoning Guan, Suraiya Haroon, Diego Gonzlez Bravo, Jessica L. Will, and Audrey P. Gasch Cells can retain memory of prior experiences that influence future behaviors. Here, the authors show that budding yeast retains a multifaceted memory of prior stress treatment. Cells pretreated with salt retain peroxide tolerance for several generations after removal of the initial stressor. This is due to long-lived catalase, produced during salt treatment and distributed to daughter cells. These cells also display transcriptional memory dependent on the nuclear pore subunit Nup42 that functions to promote reacquisition of stress tolerance in future stress cycles.

Genomic variation in natural populations of Drosophila melanogaster, pp. 533 Charles H. Langley, Kristian Stevens, Charis Cardeno, Yuh Chwen G. Lee, Daniel R. Schrider, John E. Pool, Sasha A. Langley, Charlyn Suarez, Russell B. Corbett-Detig, Bryan Kolaczkowski, Shu Fang, Phillip M. Nista, Alisha K. Holloway, Andrew D. Kern, Colin N. Dewey, Yun S. Song, Matthew W. Hahn, and David J. Begun This article greatly extends studies of population genetic variation in natural populations of Drosophila melanogaster, which have played an important role in the development of evolutionary theory. The authors describe genome sequences of 43 individuals taken from two natural populations of D. melanogaster. The genetic polymorphism, divergence, and copy-number variation revealed in these data are presented at several scales, providing unprecedented insight into forces shaping genome polymorphism and divergence.

See the article here:
Genetics Society of America's GENETICS journal highlights for October 2012

SMi is delighted to welcome: Carlos Camozzi, Vice President & Chief Medical Officer at uniQure, to their inaugural Orphan Drugs & Rare Diseases conference taking place next week on 8th & 9th October 2012 in London. Dr. Camozzi will be joining leading leaders in gene therapy and rare diseases.

(PRWEB UK) 1 October 2012

Jrn Aldag, chief executive of UniQure, says that the announcement from the EMA is an overdue signal to the gene-therapy community that things are changing. It unlocks the potential, he says, quoted in Nature. You will see more investment coming.*

Perhaps as important is UniQures pioneering annuity approach to reimbursement, staggering payments over the five years for which treatment has been proved to work. As highlighted by the Financial Times, such financial innovation may prove as important as the scientific breakthroughs that led to the treatment in the first place**.

Other keynote speakers presenting at this outstanding event include:

Visit http://www.smi-online.co.uk/goto/orphandrugs-event38.asp to secure your place now!

Alternatively, contact Alternatively Contact Fateja Begum on telephone +44 (0) 20 7827 6184 or email on fbegum@smi-online.co.uk

** Healthcare: Uncommon complaints. Financial Times, July 25th, 2012, p.9.

About SMi Group

The SMi Group is a world leader in business to business information. With nearly two decades in the business, thousands of senior executives from blue chip companies have already benefitted from SMis highly targeted conferences, workshops and publications. For more information visit:

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Orphan Drugs & Rare Diseases conference

By Sharmistha Banerjee - September 25, 2012 | Tickers: NBS, OSIR, PSTI | 0 Comments

Sharmistha is a member of The Motley Fool Blog Network -- entries represent the personal opinions of our bloggers and are not formally edited.

Far-reaching accomplishments in the biotechnology sector meet its most ambitious expectations, stem cell therapy. The birth of this new industry has boosted the enthusiasm and energy of investors and has brought unprecedented capability and optimistic predictions. New developments in regenerative medicine are bringing about exciting, novel approaches to create therapies for hard to treat diseases. The biotechnology industry has been soaring in 2012 as companies both large and small have shown impressive growth.

The cell therapy space has seen relatively small companies making strides in the right direction with increased government support. Osiris Therapeutics (NASDAQ: OSIR) a leading stem cell company is currently the only company with an approved cell therapy. The approval is more of a first step in a long walk for Osiris. Reuters reported that shares of Osiris Therapeutics rose 15% on May 30, 2012, after U.S. health regulators said the stem cell technology company's wound treatment was eligible for reimbursement when used in hospitals in out-patient settings or in ambulances. The company carries over a $300 million market capitalization and trades at $9.50 per share, primarily on the strength of a recent Canadian approval for its stem cell drug for graft-versus-host disease. Osiris Therapeutics has a 1-year low of $4.12 and a 1-year high of $14.46. The company has a market cap of $311.3 million and a price-to-earnings ratio of 90.98. Investors are impressed and optimistic with Osiris progress in cell-based therapies. They currently have a $9.75 target price on the stock. Despite having to negotiate a more challenging regulation process the company has continued to show investors strong gains in 2012.With a current ratio of 8.51 and debt equity of (0.00%) the company boasts of a financially secure position in the market.

Pluristem Therapeutics (NASDAQ: PSTI) a small firm with a market cap of less than $180 million has been concentrating on its placenta-based cell therapies, is considered one of the more advanced in the cell therapy arena, and unlike OSIR, its lead candidates treat diseases that could potentially return significant revenue. The upside for PSTI is lower costs, quicker healing time, ease of administration, and most importantly, it can grow vessels and provide the possibility of a cure, which has led to optimism surrounding the stock. Shares of Pluristem Therapeutics are up over 3.98% and most likely headed higher in the days ahead. It has traded higher by 85% during the last three months and is now valued at $200 million. Pluristem may actually beat OSIR in the race to become the first U.S. approved cell therapy with its bone marrow therapy, in which it has recently applied for approval. Pluristem is a company that I think is showing great promise. From the stock's action in the last several months, it is clear investors recognize that Pluristem's unique platform technology has the potential for tremendous value in a lucrative range of medical markets both the very large and the very small. The company wins both ways. Its clinical segment is creating candidates with large revenue potential, with analysts projecting peak sales of $700 million for AMR-001, which treats patients following acute myocardial infarction. The company is reasonably well funded with around $42 million in cash and cash equivalents.

NeoStem (NYSEMKT: NBS) is by far the leader in regards to the manufacturing business, and no other company comes close. In addition, its stock has returned the most over in the last three months, with a 100% gain. NeoStem stocks looks promising as a biotechnology investment. First, the company is focusing on several promising areas of new stem cell treatment development. Second, its contract manufacturing business brings in revenues to offset some of its drug development expenditures. Third, the contract manufacturing business could earn substantial royalties if any of the products on which it works with customers proves to be a commercial success. NeoStem's manufacturing segment which is also known as PCT, is well positioned to return larger gains over the next 24 months with several late stage candidates under development. a $110 million company that has increased in value by 70% during the last three months, In addition to the PCT business, NeoStem's most promising therapy is aimed at preventing major cardiac problems following acute myocardial infarction (AMI), an area that is potentially a multibillion-dollar business. NeoStem's therapy is meeting endpoints never before reached,

The three companies discussed above are showing much potential for growth and each present a significant upward shift in the current stock prices while contributing greatly to the advances of cell therapy.

Osiris is the closest to generating substantial revenue by already having two approvals, and is currently testing its therapy on other diseases, thereby leaving open the possibility of future gains. Pluristem has candidates to treat diseases in potentially large markets, and is expanding with its manufacturing facility. Although Stem Cells is in the early phases of development, it still has a very innovating therapy that, if proven effective, could advance the space even further. NeoStem possesses all the benefits of an innovating technology, a diversified pipeline, and is a candidate with significant revenue potential.

At this point, it appears that the entire space is moving forward and has lifted observers' expectations by making rapid progress. It makes sense that these three stocks would trade with such considerable gains, as investors can now identify the benefits of cell therapies. And as more approvals occur, it could be a space that trades considerably higher regardless of the market's indecisiveness. With the sector growing and maturing, investing in biotech stocks seems a promising choice in future.

SharmisthaB has no positions in the stocks mentioned above. The Motley Fool has no positions in the stocks mentioned above. Try any of our Foolish newsletter services free for 30 days. We Fools may not all hold the same opinions, but we all believe that considering a diverse range of insights makes us better investors. The Motley Fool has a disclosure policy.If you have questions about this post or the Fools blog network, click here for information.

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Stem Cell Therapy—Breakthrough in Health Paradigm

Scientists are hoping recent developments in the usage of stem cell therapy to treat common health problems can be transferred to diabetes.

Researchers from the University of Adelaides Robinson Institute say stem cells harvested from bone marrow have been shown to work magic when injected into inflamed joints.

These cells can convert themselves into a cartridge and give relief to the joint, Professor Andrew Zannattino told 7News.

Stem cells have also been proven to do a similar thing to other bones, even those damaged by severe injury or cancer.

These cells start stimulating the blood vessels to come into those sites and they can form bone itself, Professor Stan Gronthos said.

A trial on heart attack patients has also delivered promising results.

The heart muscle actually becomes re-oxygenated with new blood so it actually repairs the heart, Professor Grontos said.

They say the next step is seeing if stem cell therapy has an effect on diabetes.

It is hoped stem cells will eventually be able to be administered with a simple needle or intravenously, and researchers are hoping a therapy will be readily available in hospitals within five years.

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Hopes stem cell therapy can help diabetes sufferers

WEDNESDAY, Sept. 26 (HealthDay News) -- A colorectal gene database will help further research into the disease, a new study suggests.

The CRCgene database gathers all genetic association studies on colorectal cancer. It allows researchers to accurately interpret the risk factors of the disease and provides insight into the direction of further research, according to Julian Little, with the department of epidemiology and community medicine at the University of Ottawa, and colleagues.

To determine the genetic factors associated with colorectal cancer, they analyzed data from all published genetic association studies on colorectal cancer.

The researchers identified 16 independent gene variants with the strongest links to colorectal cancer, among 23 variants, a number lower than expected. Unfortunately, the researchers say, this reduces the feasibility of combining variants as a profile in a prediction tool to identity people who are at increased risk for colorectal cancer and who should be screened for the disease.

Even so, the analysis "provides a resource for mining available data and puts into context the sample sizes required for the identification of true associations," the researchers wrote in the Sept. 27 issue of the Journal of the National Cancer Institute.

About 950,000 new cases of colorectal cancer are diagnosed each year, according to a journal news release. Risk factors for the disease include age, diet, lifestyle and possibly genetics.

-- Robert Preidt

Copyright 2012 HealthDay. All rights reserved.

SOURCE: Journal of the National Cancer Institute, news release, Sept. 27, 2012

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Colon Cancer Gene Database May Assist Research Efforts

Hyderabad, Sept. 29:

The International Crops Research Institute for Semi-Arid Tropics (ICRISAT) and the Indian Council of Agricultural Research (ICAR) will host the sixth international conference on Legume Genetics and Genomics (ICLGG) here.

The six-day conference will begin on October 2. About 500 delegates from 44 countries are expected to attend the conference to discuss advances in the area of legume genetics and genomics.

William D. Dar, Director-General of ICRISAT; Swapan Datta, Deputy Director-General (Crop Science) of ICAR; and Rick Dixon, Plant Biology Division Director and Senior Vice-President of Noble Foundation, the USA, will address the conference.

This conference is of great significance for India as legumes form an important constituent of the vegetarian diet consumed globally and complement cereal crops as a rich source of dietary protein (ranging from 20-40 per cent), Dar said here in a statement.

India is the largest or one of the largest producers of a majority of pulse crops in the world. It contributes 25 per cent to the global legume production.

Despite being the largest producer for the majority of pulse crops there is a significant difference in its production and consumption. Owing to this huge difference in the production and consumption, India has to spend a significant amount of its foreign currency on import of these legumes to fill the gap. In 2008, India spent about $2 billion on the import of these legumes, an ICRISAT statement said.

Earlier editions of the conference have been held in the US (twice), France, Australia and Mexico.

kurmanath.kanchi@thehindu.co.in

Continued here:
Meet on legume genetics to open in Hyderabad on Oct. 2

By Maureen Salamon HealthDay Reporter

FRIDAY, Sept. 28 (HealthDay News) -- A sophisticated genetic test sometimes used during pregnancy can't always predict if chromosomal abnormalities will cause problems in children, leading some mothers to label the information "toxic knowledge" they wish they hadn't received, a small new study shows.

Researchers from three universities found that expectant mothers receiving bad news about a genetic test called a DNA microarray -- more often used after birth to identify chromosomal problems in children with unexplained delays or defects -- reported mostly negative responses, ranging from feeling blindsided to needing support to digest the information and make critical decisions about their pregnancies.

The women's reactions challenge the notion that knowledge is power, especially when that knowledge pertains to ambiguous information about an unborn baby's health, said study author Barbara Bernhardt, a genetic counselor and clinical professor of medicine at the Hospital of the University of Pennsylvania.

"I think we need to have better information readily available to patients and providers . . . to make the decisions they need to make in a timely manner," Bernhardt said. "We also need additional education of obstetricians and midwives to feel more comfortable talking to patients about it and counseling them about the results."

The study was published online recently in the journal Genetics in Medicine.

Standard DNA testing offered to pregnant women uses tests such as amniocentesis and chorionic villus sampling, which involve "karyotyping" to identify common abnormalities such as Down syndrome. But DNA microarrays can detect smaller-scale chromosomal changes that can signal future problems such as autism or congenital disorders, although the test can't necessarily predict how severe the problem will be or even if a gene variant will produce any discernible conditions in the child.

Some of the 54 study participants, 23 of whom were interviewed at least six months after childbirth or pregnancy termination, had learned from ultrasound or other tests that their fetus had abnormalities. But for those whose prior tests had come back "normal," learning their baby had genetic variants of unknown significance sent some of them into a tailspin.

Bernhardt's team identified five key responses that described the women's experiences, including considering the microarray results toxic knowledge they wish they hadn't learned. Many of the women accepted the testing -- which costs between $1,500 and $3,000 -- because it was offered at no charge to them, which they felt was "an offer too good to pass up."

Unlike children who are tested using DNA microarrays to help diagnose existing problems, children tested prenatally may never develop health or developmental issues arising from their genetic variants. "We discovered in the course of the study that some of the parents have the same [chromosomal] deletion or duplication as their fetus" but never suffered any ill effects, said Bernhardt, also co-director of the Penn Center for the Integration of Genetic Healthcare Technologies.

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Prenatal test presents dilemmas to expectant mothers

September 28, 2012

Image Caption: A Metabiota field staff member collects blood samples from bushmeat in the Democratic Republic of Congo. Credit: Metabiota

John Neumann for redOrbit.com Your Universe Online

An intriguing new virus from the Democratic Republic of Congo (DRC) has been identified as the cause of a deadly outbreak of acute hemorrhagic fever, which killed two people and left one gravely ill in the summer of 2009.

Described this week in the open-access journal PLoS Pathogens the new microbe has been named Bas-Congo virus (BASV) after the province in the southwest corner of the Congo where the three people lived.

The virus was first discovered when a teenager, living in the rural village of Mangala in the DRC, suddenly fell ill and developed symptoms of a hemorrhagic fever, including bleeding from mucous membranes and blood in the vomit. This victim died within three days of the first signs of illness.

A week later, a 13-year-old girl who attended the same school and lived in the same neighborhood came down with a similar illness and also died within three days, writes Nathan D. Wolfe, Joseph Fair, and Charles Chiu for National Geographic.

Known viruses, such as Ebola, HIV and influenza, represent just the tip of the microbial iceberg, explains Joseph Fair, PhD, a co-author and vice president of Metabiota. Identifying deadly unknown viruses, such as Bas-Congo virus, gives us a leg up in controlling future outbreaks.

These are the only three cases known to have occurred, although there could be additional outbreaks from this virus in the future, said Charles Chiu, MD, PhD, an assistant professor of laboratory medicine at UCSF and director of the UCSF-Abbott Viral Diagnostics and Discovery Center, who spearheaded the UCSF effort to identify the virus.

As a first step, Metabiota enlisted the help of close collaborator Dr. Eric Delwart at Blood Systems Research Institute (BSRI). Using sophisticated genetic sequencing techniques, Dr. Delwart detected a fragment of genetic information related to the rhabdovirus family.

Continue reading here:
New Genetic Snooping Technique Finds New Deadly Virus

Featured Article Academic Journal Main Category: Stem Cell Research Also Included In: Biology / Biochemistry Article Date: 28 Sep 2012 - 1:00 PDT

Current ratings for: Purging Stem Cells To Make Therapy Safer

The study appears in a 27 September issue of the journal Stem Cells Translational Medicine.

iPS cells have properties similar to embryonic stem cells, which are "master cells" with an unlimited capacity to differentiate into any type of tissue in the body, such as brain, lung, skin, heart, and liver. Thus their potential in regenerative medicine, where damaged or diseased tissue can be repaired or replaced by growing new tissue, is huge, as senior author Timothy Nelson explains in a press release:

"Pluripotent stem cells show great promise in the field of regenerative medicine; however, the risk of uncontrolled cell growth will continue to prevent their use as a therapeutic treatment."

Nelson is Assistant Professor of Medicine and Pharmacology and works in the General Internal Medicine department and the Transplant Center at the Mayo.

The idea of using iPS cells is for doctors to be able to take some adult tissue, for example skin cells, from the patient who needs the treatment, and then turn the cells from that tissue into iPS cells.

Then, those iPS cells are coaxed to turn into the target type of cell, for instance lung cells. As a result of the coaxing the iPS cells turn into (differentiate) the target tissue type.

But current ways of doing this leaves some iPS cells undifferentiated, so they get transplanted into the patient along with the differentiated cells, leaving the risk that they will differentiate on their own in an uncontrolled way and form tumors.

For their study, Nelson and colleagues used lab mice to show that pretreating iPS cells with a chemotherapy drug, selectively damages the DNA of the stem cells, killing them off so they cannot grow uncontrollably and form tumors. The chemotherapy kills the iPS cells by triggering cell suicide or apoptosis, which is a natural response to DNA damage.

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Purging Stem Cells To Make Therapy Safer

By Ryan Flinn - 2012-09-28T11:30:00Z

Cytori Therapeutics Inc. (CYTX), a biotechnology company with $10 million in annual revenue, won a $4.7 million U.S. government contract to develop a stem cell therapy to treat burns caused by thermal or radioactive bombs.

The two-year contract with the Department of Health and Human Services Biomedical Advanced Research and Development Authority may be worth $106 million over five years if certain milestones are met, San Diego-based Cytori said today in a statement. The company had a net loss last year of $32 million, according to data compiled by Bloomberg.

Cytoris experimental therapy takes adipose tissue, or body fat, from a patient and through its device separates the adult stem and regenerative cells before transferring them to a burn wound. Money from the contract will be used to develop the device and take it through the U.S. regulatory approval process with the Food and Drug Administration, Chief Executive Officer Christopher Calhoun said.

These cells help to facilitate the healing of the injury, he said in a telephone interview. They release growth factors that stimulate new blood flow.

Testing the technology in a clinical trial and getting approval may take five years, Calhoun said. The company is currently testing its therapy for other soft tissue damage, as well as cardiovascular disease.

Once approved, the device will be deployed in hospitals across the country, and can be used for routine burns as well as a treatment for patients in wake of a mass casualty event that could injure 10,000 people, Cytori said in the statement.

Cytori declined 39 cents, or 9.2 percent, to $3.86 at the close yesterday in New York. The companys shares gained 75 percent this year through yesterday.

To contact the reporter on this story: Ryan Flinn in San Francisco at rflinn@bloomberg.net

To contact the editor responsible for this story: Reg Gale at rgale5@bloomberg.net

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Cytori’s Stem Cell Therapy for Burns Wins U.S. Contract

27-09-2012 03:27 Almost £60 million of awards from the Medical Research Council (MRC) will help scientists gain fresh insights into illnesses and inherited disorders. The funding to the University's MRC Human Genetics Unit and the MRC Institute of Genetics and Molecular Medicine (IGMM) will help doctors develop and deliver new tests and therapies for patients. It will boost research into conditions such as schizophrenia, cystic fibrosis and genetic eye disorders including retinitis pigmentosa, coloboma and anophthalmia. Dr Chris Boyd, Dr Alastair Innes and Dr Steve Cunningham tell us about a groundbreaking gene therapy trial for adults and children with cystic fibrosis (CF) - coordinated by the UK Cystic Fibrosis Gene Therapy Consortium (GTC).

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UK's first cystic fibrosis gene therapy trial - Video

ScienceDaily (Sep. 27, 2012) For a cancer patient, over-expression of the MYC oncogene is a bad omen. Scientists have long known that in tumor cells, elevated levels of MYC's protein product, c-Myc, are associated with poor clinical outcomes, including increased rates of metastasis, recurrence, and mortality. Yet decades of research producing thousands of scientific papers on the subject have failed to consistently explain precisely how c-Myc exerts its effects across a broad range of cancer types. Until now, that is.

The prevailing theory emerging from this massive body of research has been that in tumor cells, c-Myc affects the expression of specific genes or sets of genes -- that so-called Myc target genes are being selectively activated or repressed, leading to aberrant cellular behavior. Now, however, researchers in the lab of Whitehead Institute Member Richard Young are dispelling this commonly held notion, showing that elevated expression of c-Myc amplifies the activity of all expressed genes in tumor cells of multiple cancer types. It turns out that high levels of c-Myc send a tumor cell's gene expression program into overdrive. Transcription increases dramatically, allowing malignant cells to overwhelm factors that might normally hamper their growth and proliferation. This surprising finding, published in this week's issue of the journal Cell, provides a simple, elegant explanation for how a single protein can have such profound effect in so many and varied types of cancer. The newly revealed mechanism may also help scientists develop novel therapeutic approaches that disrupt c-Myc's activity.

"MYC is a key driver in most major cancers, but it has been notoriously difficult to drug," says Young, who is also a professor of biology at MIT. "Now that we know the mechanism by which c-Myc acts, we can go after the components of that mechanism as potential drug targets. This research creates an even stronger impetus to find a way to drug the thing."

One potential drawback to thwarting c-Myc's activity is the important role it plays in normal cell division. That role is so powerful that cells co-evolved an emergency death pathway to keep c-Myc expression in check. If c-Myc's production spins out of control in an otherwise normal cell, the cell immediately commits suicide through a process called apoptosis. But in cancer cells in which c-Myc is overproduced, this suicide pathway is compromised, allowing the cell to survive and proliferate.

"MYC is the most deregulated gene in cancer," says Charles Lin, a graduate student in the Young lab and co-author of the Cell paper. "It's been called a bad-boy, a Swiss army knife, and a jack-of-all-trades because, according to previous research, it could do everything under the sun in a cancer cell. But most of the different attributes ascribed to MYC are contradictory or seemingly incompatible."

Propelled by its earlier research that identified c-Myc as an important regulator of transcription in embryonic stem cells, the Young lab began to focus on c-Myc's activity within cancer cells. Lab members found that as the expression of c-Myc increases in these cells, the protein attaches to the promoters and enhancers of all active genes, thereby amplifying the active genes' transcription. The heightened transcription produces cells bloated with excessive RNAs and proteins capable of altering normal cellular functions. Researchers observed this phenomenon in cells from a host of cancers, including Burkitt's lymphoma, small cell lung cancer, multiple myeloma, and glioblastoma multiforme.

"The previous research now makes sense -- finally!" says Jakob Lovn, co-author and postdoctoral researcher in the Young lab. "Our findings provide a way to unify everybody's seemingly conflicting data. I think that's really nice. Instead of saying 'you're all wrong,' we're saying 'you're all right, and here's why.' The model makes a lot of sense in terms of the biology that has been described so far."

With a better understanding of how c-Myc can wreak so much damage, the Young lab is turning its efforts to disrupting c-Myc's activity. Although cancer cells that overproduce c-Myc are associated with poor clinical outcomes, their reliance on c-Myc for survival may represent an Achilles' heel. When these "Myc-addicted" cells are deprived of c-Myc in vitro, even for a short period of time, they quickly die. Research in mice has shown that, Myc-addicted tumors deprived of the protein shrink dramatically. Despite c-Myc's necessary role in normal cell division, particularly in tissues with rapid cell turnover, such as the intestine and blood, these mouse studies have shown that if c-Myc activity is restored after a brief period, normal tissues quickly bounce back, while tumors are unable to regain their footing.

"So what we think now is that potentially, if drugs can tune down the levels of transcription just slightly, this might be catastrophic for the Myc-addicted cancer cells," says Peter Rahl, co-author and postdoctoral researcher in the Young lab. "You wouldn't need to abolish all transcription because that would be toxic to your other cells. So we're hoping that our model will show us ways to create a therapeutic window where the Myc-addicted cells just won't be able to adapt to lower levels of transcripts."

This work was supported by National Institutes of Health (grants HG002668 and CA146445), Swedish Research Council, American Cancer Society, and Damon-Runyon Cancer Research Foundation.

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Aggressive cancer exploits Myc oncogene to amplify global gene activity

BUDAPEST, HUNGARY--(Marketwire - Sep 28, 2012) - Power of the Dream Ventures, Inc. ( OTCBB : PWRV ) is pleased to announce the acquisition of Genetic Immunity, Inc., a Phase III clinical stage biotechnology company with experimental nanomedicines that will lead to the next generation of immunotherapies, in a market that is projected to reach $11.00 billion by 2018.

Genetic Immunity's lead product candidate is an immune boosting drug for HIV, which is now only treated by antiretroviral drugs that decrease the ability of the immune system to fight with the virus. DermaVir HIV-specific Immunotherapy is the first of a new line of curative nanomedicine products developed for the treatment and eradication of HIV. In addition, Genetic Immunity has implemented a Predictive Genomic Biomarker as companion diagnostics to accurately predict potential responder patients to DermaVir treatment. Such innovations towards personalized medicine increase the treatment effect and reduce the cost of pivotal trials in full compliance with the FDA's initiatives to improve products for patients (Driving Biomedical Innovation, 2011). In addition, following a successful DermaVir trial on HIV-infected adults, the US government is sponsoring a Phase II pediatric clinical trial.

DermaVir is the first therapeutic vaccine that consistently boosts broadly directed central memory T-cells in human subjects. This immune response has been correlated with containment of viremia in Elite Controllers. The Phase II randomized, multicenter, placebo controlled trial conducted in Germany established the optimal DermaVir dose and provided data that demonstrates the killing of HIV-infected cells. Therefore, the eradication of HIV or the conversion of progressors to Elite Controllers via DermaVir immunization became a testable hypothesis.

"This acquisition milestone is the result of our collaboration for a common goal to sell stock in Genetic Immunity to the public. The acquisition of a private company by a public one corresponds to a novel IPO, and offers tremendous upside potential for all the shareholders of Genetic Immunity and PWRV. Starting today, financial market participants will have an opportunity to determine the price of our business. We are eager, because comparable technology companies trade at over half a billion dollar valuation. On a more personal note, I believe that Genetic Immunity's platform technology is a once in a lifetime opportunity. For the first time we are truly in reach of eradicating a highly infectious disease. We are proud to be a part of the process whereby the innovations presented by Genetic Immunity can become publicly available," commented Viktor Rozsnyay, CEO of Power of the Dream Ventures.

"Through this highly innovative financial transaction, Genetic Immunity achieves its corporate objective to become a publicly traded company and to retain the control over the business. The financial and technological synergy between the two Companies provides for substantial growth opportunity and high return on investment to our shareholders," said Dr. Julianna Lisziewicz, CEO of Genetic Immunity.

With the acquisition Genetic Immunity becomes a 100% wholly owned subsidiary of Power of the Dream Ventures, Inc.

About PDV Power of the Dream Ventures, Inc. is a leading technology holding company. We identify and harness the unique technological prowess of Hungary's high-tech industry, turning promising ideas and ready to market products/technologies into global industry leaders. We focus on developing, acquiring, or co-developing technologies that originate exclusively in Hungary. For more information, please visit http://www.powerofthedream.com

About Genetic Immunity Genetic Immunity is a clinical stage technology company committed to discovering, developing, manufacturing and commercializing a new class of immunotherapeutic biologic drugs for the treatment of viral infections, cancer and allergies. The Company's two distinguished technology platforms will revolutionize the treatment of these chronic diseases. Our Langerhans' cell targeting nanomedicines are exceptional in both safety and immune modulating activity boosting specific Th1-type central memory T cells. Such immune responses differ from antibodies induced by vaccines. These are essential to eliminate infected cells or cancerous cells, and balance the immune reactivity in response to allergens. Our IT team generated a complex algorithm to match the mechanism of action of our drugs with clinical efficacy. In the future, we will predict the clinical and immunological benefits of our drugs based on the patient's disease and genomic background. The unique mixture of our technologies represents the next generation of personalized but not individualized medicines ensuring a longer and higher economic return.

Genetic Immunity's primary focus is the development of DermaVir that acts to boost the immune system of HIV-infected people to eliminate infected cells that remain in the reservoirs after successful antiretroviral treatment. Three clinical trials conducted in the EU and US showed that DermaVir immunizations were as safe as placebo and only four sequential patch treatments required to reduce the HIV infected cells in the blood within 24 weeks.

In 1988 Drs. Lisziewicz and Lori founded Genetic Immunity in the US after they described the 1st patient whose immune system was boosted to control HIV after treatment interruption (Lisziewicz et al. New England Journal of Medicine 1999) that lead to the invention of DermaVir. The Company's innovative technology team directed by Dr. Lisziewicz, a champion of immune busting therapies, is now headquartered in Budapest, Hungary. For more information please visit http://www.geneticimmunity.com

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Power of the Dream Ventures Acquires Genetic Immunity

Public release date: 27-Sep-2012 [ | E-mail | Share ]

Contact: Jennifer Schutz newsbureau@mayo.edu 507-284-5005 Mayo Clinic

ROCHESTER, Minn. -- Mayo Clinic researchers have found a way to detect and eliminate potentially troublemaking stem cells to make stem cell therapy safer. Induced Pluripotent Stem cells, also known as iPS cells, are bioengineered from adult tissues to have properties of embryonic stem cells, which have the unlimited capacity to differentiate and grow into any desired types of cells, such as skin, brain, lung and heart cells. However, during the differentiation process, some residual pluripotent or embryonic-like cells may remain and cause them to grow into tumors.

"Pluripotent stem cells show great promise in the field of regenerative medicine; however, the risk of uncontrolled cell growth will continue to prevent their use as a therapeutic treatment," says Timothy Nelson, Ph.D., M.D., lead author on the study, which appears in the October issue of STEM CELLS Translational Medicine.

Using mouse models, Mayo scientists overcame this drawback by pretreated stem cells with a chemotherapeutic agent that selectively damages the DNA of the stem cells, efficiently killing the tumor-forming cells. The contaminated cells died off, and the chemotherapy didn't affect the healthy cells, Dr. Nelson says.

"The goal of creating new therapies is twofold: to improve disease outcome with stem cell-based regenerative medicine while also ensuring safety. This research outlines a strategy to make stem cell therapies safer for our patients while preserving their therapeutic efficacy, thereby removing a barrier to translation of these treatments to the clinic," says co-author Alyson Smith, Ph.D.

Stem cell therapies continue to be refined and improved. Researchers are finding that stem cells may be more versatile than originally thought, which means they may be able to treat a wider variety of diseases, injuries and congenital anomalies. Stem cell therapy is an emerging regenerative strategy being studied at Mayo Clinic.

"By harnessing the potential of regenerative medicine, we'll be able to provide more definitive solutions to patients," says Andre Terzic, M.D., Ph.D., co-author and director of Mayo Clinic's Center for Regenerative Medicine.

###

Other members of the Mayo research team included Clifford Folmes, Ph.D., Katherine Hartjes, Natalie Nelson and Saji Oommen, Ph.D. The research was supported by the Todd and Karen Wanek Family Program for Hypoplastic Left Heart Syndrome, National Institutes of Health New Innovator Award OD007015-01, and a Mayo Clinic Center for Regenerative Medicine accelerated research grant.

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Mayo Clinic finds way to weed out problem stem cells, making therapy safer

DUBLIN--(BUSINESS WIRE)--

Research and Markets (http://www.researchandmarkets.com/research/gdkzds/down_syndrome) has announced the addition of Elsevier Science and Technology's new book "Down Syndrome: From Understanding the Neurobiology to Therapy, Vol 197" to their offering.

Down syndrome (DS) is the most common example of neurogenetic aneuploid disorder leading to mental retardation. In most cases, DS results from an extra copy of chromosome 21 (HSA21) producing deregulated gene expression in brain that gives raise to subnormal intellectual functioning. The topic of this volume is of broad interest for the neuroscience community, because it tackles the concept of neurogenomics, that is, how the genome as a whole contributes to a neurodevelopmental cognitive disorders, such as DS, and thus to the development, structure and function of the nervous system.

This volume of Progress in Brain Research discusses comparative genomics, gene expression atlases of the brain, network genetics, engineered mouse models and applications to human and mouse behavioral and cognitive phenotypes. It brings together scientists of diverse backgrounds, by facilitating the integration of research directed at different levels of biological organization, and by highlighting translational research and the application of the existing scientific knowledge to develop improved DS treatments and cures.

- Leading authors review the state-of-the-art in their field of investigation and provide their views and perspectives for future research

- Chapters are extensively referenced to provide readers with a comprehensive list of resources on the topics covered

- All chapters include comprehensive background information and are written in a clear form that is also accessible to the non-specialist

For more information visit http://www.researchandmarkets.com/research/gdkzds/down_syndrome

Source: Elsevier Science and Technology

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Research and Markets: Down Syndrome: From Understanding the Neurobiology to Therapy, Vol 197

Public release date: 27-Sep-2012 [ | E-mail | Share ]

Contact: NHLBI Office of Communications nhlbi_news@nhlbi.nih.gov 301-496-4236 NIH/National Heart, Lung and Blood Institute

Scientists may have discovered why a protein called MYC can provoke a variety of cancers. Like many proteins associated with cancer, MYC helps regulate cell growth. A study carried out by researchers at the National Institutes of Health and colleagues found that, unlike many other cell growth regulators, MYC does not turn genes on or off, but instead boosts the expression of genes that are already turned on.

These findings, which will be published in Cell on Sept. 28, could lead to new therapeutic strategies for some cancers.

"We carried out a highly sophisticated analysis of MYC activity in cells, but came away with a simple rule. MYC is not a power switch but a universal amplifier," said co-lead study author Keji Zhao, Ph.D., director of the Systems Biology Center at the NIH's National Heart, Lung, and Blood Institute (NHLBI). "This discovery offers a unifying idea of how and why abnormal levels of MYC are found in so many different cancer types, such as breast cancer, lung cancer, and several blood cancers."

"MYC is much like the volume control of a music player," added co-lead David Levens, M.D., Ph.D., a senior investigator in the Laboratory of Pathology at the National Cancer Institute (NCI), also part of NIH. "If you're listening to opera, for example, adding more MYC will make the opera louder, but it won't change the program to rap. And if you have only silence, MYC will just give you more silence."

Both researchers noted that this new understanding of MYC function could influence future treatment efforts for MYC-associated tumors. They suggest that trying to limit MYC activity, or turning down the volume just the right amount, would be a better strategy than using targeted chemotherapy to try to eliminate all MYC activity.

MYC aids in cell activation, a process in which cells mature and divide quickly. During an immune response, for example, white blood cells are activated to help fight infections. If activation isn't properly regulated, then cells can start growing out of control and result in cancer. Researchers have known that abnormally high levels of MYC can lead to cancer, but until now, no one had been able to explain how it can lead to so many different cancers.

Zhao, Levens, and their colleagues used a specially designed fluorescent protein that allowed them to track MYC in white blood cells in a lab dish. They chose white blood cells, specifically B cells and T cells that fight infections, because they are frequently affected by abnormal MYC and can transform into lymphoma and myeloma cells.

The team exposed the B and T cells to foreign toxins to stimulate an immune response and activate the fluorescent MYC. The researchers could then examine the cells at different time points and see which genes the MYC proteins seemed to affect.

Read more from the original source:
Major cancer protein amplifies global gene expression, NIH study finds