At the forefront of medicine, Gene Therapy brings you the latest research into genetic and cell-based technologies to treat disease. It also publishes Progress & Prospects reviews and News and Commentary articles, which highlight the cutting edge of the field.

Volume 20, No 10 October 2013 ISSN: 0969-7128 EISSN: 1476-5462

2012 Impact Factor 4.321* 70/290 Biochemistry & Molecular Biology 22/159 Biotechnology & Applied Microbiology 33/161 Genetics & Heredity 25/121 Medicine, Research & Experimental

Editors: J Glorioso, USA N Lemoine, UK

*2012 Journal Citation Reports Science Edition (Thomson Reuters, 2013)

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Gene Therapy - Nature Publishing Group : science journals, jobs ...

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Gene therapy attempts to treat genetic diseases at the molecular level by correcting what is wrong with defective genes. Clinical research into gene therapys safety and effectiveness has just begun. No one knows if gene therapy will work, or for what diseases. If gene therapy is successful, it could work by preventing a protein from doing something that causes harm, restoring the normal function of a protein, giving proteins new functions, or enhancing the existing functions of proteins. How Do You Do It? Gene therapy relies on finding a dependable delivery system to carry the correct gene to the affected cells. The gene must be delivered inside the target cells and work properly without causing adverse effects. Delivering genes that will work correctly for the long term is the greatest challenge of gene therapy.

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Gene Therapy - A Revolution in Progress: Human Genetics and ...

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Please choose from the following list of questions for information about gene therapy, an experimental technique that uses genetic material to treat or prevent disease.

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Gene therapy is an experimental technique that uses genes to treat or prevent disease. In the future, this technique may allow doctors to treat a disorder by inserting a gene into a patients cells instead of using drugs or surgery. Researchers are testing several approaches to gene therapy, including:

Replacing a mutated gene that causes disease with a healthy copy of the gene.

Inactivating, or knocking out, a mutated gene that is functioning improperly.

Introducing a new gene into the body to help fight a disease.

Although gene therapy is a promising treatment option for a number of diseases (including inherited disorders, some types of cancer, and certain viral infections), the technique remains risky and is still under study to make sure that it will be safe and effective. Gene therapy is currently only being tested for the treatment of diseases that have no other cures.

MedlinePlus from the National Library of Medicine offers a list of links to information about genes and gene therapy.

Educational resources related to gene therapy are available from GeneEd.

The Genetic Science Learning Center at the University of Utah provides an interactive introduction to gene therapy.

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Gene Therapy - Genetics Home Reference - National Institutes of Health

Recommendation and review posted by Bethany Smith

Gene therapy is an experimental technique that uses genes to treat or prevent disease. In the future, this technique may allow doctors to treat a disorder by inserting a gene into a patients cells instead of using drugs or surgery. Researchers are testing several approaches to gene therapy, including:

Replacing a mutated gene that causes disease with a healthy copy of the gene.

Inactivating, or knocking out, a mutated gene that is functioning improperly.

Introducing a new gene into the body to help fight a disease.

Although gene therapy is a promising treatment option for a number of diseases (including inherited disorders, some types of cancer, and certain viral infections), the technique remains risky and is still under study to make sure that it will be safe and effective. Gene therapy is currently only being tested for the treatment of diseases that have no other cures.

MedlinePlus from the National Library of Medicine offers a list of links to information about genes and gene therapy.

Educational resources related to gene therapy are available from GeneEd.

The Genetic Science Learning Center at the University of Utah provides an interactive introduction to gene therapy.

The Centre for Genetics Education provides an introduction to gene therapy, including a discussion of ethical and safety considerations.

Next: How does gene therapy work?

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What is gene therapy? - Genetics Home Reference

Recommendation and review posted by Bethany Smith

Gene therapy is the use of DNA as a pharmaceutical agent to treat disease. It derives its name from the idea that DNA can be used to supplement or alter genes within an individual's cells as a therapy to treat disease. The most common form of gene therapy involves using DNA that encodes a functional, therapeutic gene to replace a mutated gene. Other forms involve directly correcting a mutation, or using DNA that encodes a therapeutic protein drug (rather than a natural human gene) to provide treatment. In gene therapy, DNA that encodes a therapeutic protein is packaged within a "vector", which is used to get the DNA inside cells within the body. Once inside, the DNA becomes expressed by the cell machinery, resulting in the production of therapeutic protein, which in turn treats the patient's disease.

Gene therapy was first conceptualized in 1972, with the authors urging caution before commencing gene therapy studies in humans. The first FDA-approved gene therapy experiment in the United States occurred in 1990, when Ashanti DeSilva was treated for ADA-SCID.[1] Since then, over 1,700 clinical trials have been conducted using a number of techniques for gene therapy.[2]

Although early clinical failures led many to dismiss gene therapy as over-hyped, clinical successes since 2006 have bolstered new optimism in the promise of gene therapy. These include successful treatment of patients with the retinal disease Leber's congenital amaurosis,[3][4][5][6]X-linked SCID,[7] ADA-SCID,[8]adrenoleukodystrophy,[9]chronic lymphocytic leukemia (CLL),[10]acute lymphocytic leukemia (ALL),[11]multiple myeloma[12] and Parkinson's disease.[13] These recent clinical successes have led to a renewed interest in gene therapy, with several articles in scientific and popular publications calling for continued investment in the field.[14][15]

In 2012, Glybera became the first gene therapy treatment to be approved for clinical use in either Europe or the United States after its endorsement by the European Commission.[16][17]

Scientists have taken the logical step of trying to introduce genes directly into human cells, focusing on diseases caused by single-gene defects, such as cystic fibrosis, haemophilia, muscular dystrophy, thalassemia, and sickle cell anemia. However, this has proven more difficult than genetically modifying bacteria, primarily because of the problems involved in carrying large sections of DNA and delivering them to the correct site on the gene. Today, most gene therapy studies are aimed at cancer and hereditary diseases linked to a genetic defect. Antisense therapy is not strictly a form of gene therapy, but is a related, genetically mediated therapy.

The most common form of genetic engineering involves the insertion of a functional gene at an unspecified location in the host genome. This is accomplished by isolating and copying the gene of interest, generating a construct containing all the genetic elements for correct expression, and then inserting this construct into a random location in the host organism. Other forms of genetic engineering include gene targeting and knocking out specific genes via engineered nucleases such as zinc finger nucleases, engineered I-CreI homing endonucleases, or nucleases generated from TAL effectors. An example of gene-knockout mediated gene therapy is the knockout of the human CCR5 gene in T-cells to control HIV infection.[18] This approach is currently being used in several human clinical trials.[19]

Gene therapy may be classified into the two following types:

In somatic gene therapy, the therapeutic genes are transferred into the somatic cells (non sex-cells), or body, of a patient. Any modifications and effects will be restricted to the individual patient only, and will not be inherited by the patient's offspring or later generations. Somatic gene therapy represents the mainstream line of current basic and clinical research, where the therapeutic DNA transgene (either integrated in the genome or as an external episome or plasmid) is used to treat a disease in an individual.

In germ line gene therapy, germ cells (sperm or eggs), are modified by the introduction of functional genes, which are integrated into their genomes. Germ cells will combine to form a zygote which will divide to produce all the other cells in an organism and therefore if a germ cell is genetically modified then all the cells in the organism will contain the modified gene. This would allow the therapy to be heritable and passed on to later generations. Although this should, in theory, be highly effective in counteracting genetic disorders and hereditary diseases, some jurisdictions, including Australia, Canada, Germany, Israel, Switzerland, and the Netherlands[20] prohibit this for application in human beings, at least for the present, for technical and ethical reasons, including insufficient knowledge about possible risks to future generations[20] and higher risk than somatic gene therapy (e.g. using non-integrative vectors).[21] The USA has no federal legislation specifically addressing human germ-line or somatic genetic modification (beyond the usual FDA testing regulations for therapies in general).[20]

Gene therapy utilizes the delivery of DNA into cells, which can be accomplished by a number of methods. The two major classes of methods are those that use recombinant viruses (sometimes called biological nanoparticles or viral vectors) and those that use naked DNA or DNA complexes (non-viral methods).

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Gene therapy - Wikipedia, the free encyclopedia

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PORT ST. LUCIE, Fla.--(BUSINESS WIRE)--

Scientists from the medical university Karolinska Institutet (KI), Stockholm, Sweden met with researchers at the Vaccine & Gene Therapy Institute of Florida (VGTI Florida) in Port St. Lucie, Florida for a three-day visit this October. Karolinska Institutet and VGTI scientists discussed opportunities for the development of collaborations between the two organizations to further both institutes research programs.

During the in-depth meetings, the researchers focused on how their respective basic and clinical research capabilities in inflammation, cancer, cardiovascular, and infectious disease research could complement and enhance each other, potentially leading to the development of novel therapeutics for difficult human diseases. Of particular interest is the impact of aging on the functioning of the immune system on disease, and the responses to therapy.

Participants noted that there were many parallels to discuss during the in-depth meetings, and VGTI Florida anticipates return visit from the Karolinska Institutet for a scientific and research topic summit in early February 2014.

We are looking forward to continuing our planning of the upcoming joint symposium we will hold with VGTI Florida, said Karl-Henrik Grinnemo, M.D., Ph.D., Cardiothoracic Surgeon, Department of Molecular Medicine and Surgery at Karolinska Institutet. During the meeting several collaborative projects were discussed, and dialogue regarding KI's presence at VGTI was initiated.

VGTI Florida shared its own considerable expertise in basic science, and described the rapidly growing research capacities located here in South Florida, commented VGTI Director Richard Jove, Ph.D. We also had the opportunity to share the local area and what it can offer with our guests from the Karolinska Institutet. The Karolinska Institutet is one of the worlds leading medical universities.

About VGTI Florida:

The Vaccine & Gene Therapy Institute of Florida (VGTI Florida) is a non-profit 501(c)(3) biomedical research institute dedicated to understanding the roles of our immune system and our genes in disease, as well as the development of innovative treatments. We are in an expansion phase recruiting leading scientists from around the globe to join VGTI Florida where they can work side-by-side targeting infectious disease, cancer, and the impact of an aging immune system. For more information, please visit http://www.VGTIFL.org. VGTI Florida and Translating Research into Health are Registered Trademarks of the Vaccine & Gene Therapy Institute of Florida.

About Karolinska Institutet:

With an overriding mission to contribute to the improvement of human health through research and education, Karolinska Institutet provides more than 40 percent of the medical academic research conducted in Sweden and offers the country s broadest range of education in medicine and health sciences. Many of the discoveries made at Karolinska Institutet have been of great significance, including the pacemaker, the gamma knife, the sedimentation reaction, the Seldinger technique and the preparation of chemically pure insulin. Since 1901 the Nobel Assembly at Karolinska Institutet has selected the Nobel laureates in Physiology or Medicine.

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VGTI Florida Hosts Scientific Delegates from Sweden’s Karolinska Institutet

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Continue reading page |1|2

Five children with a genetic disease that wipes out their immune system have successfully been treated with gene therapy

Editorial: "Gene therapy needs a hero to live up to the hype"

MOST parents dream of a 5-week-old baby who sleeps through the night, but Aga Warnell knew something was wrong. Her baby, Nina, just wasn't hungry in the way her other daughters had been.

Within weeks, Nina became very ill, says her father, Graeme. She was admitted to hospital with a rotavirus infection. Then she picked up pneumonia.

It turned out Nina had a condition called severe combined immunodeficiency (SCID). She had been born without an immune system due to a genetic defect. It is also known as "bubble boy" disease, since people affected have to live in a sterile environment. "The doctors said 'you need to prepare yourself for the fact that Nina probably isn't going to survive'," says Graeme.

A year-and-a-half later, Nina is a happy little girl with a functioning immune system. She has gene therapy and its latest improvements to thank for it.

SCID was the first condition to be treated with gene therapy more than 20 years ago. A virus was used to replace a faulty gene with a healthy one. But in subsequent trials, four young patients were diagnosed with leukaemia two years after receiving a similar treatment. An 18-year-old also died following a reaction to a virus used in gene therapy for a liver condition. It was the start of a rocky road (see "Trials and tribulations of gene therapy").

Gene therapy has come a long way since, and Nina's case, along with others, mark a turning point: researchers seem to have found a safer way of manipulating our genes.

Preliminary results for the first two children to receive the improved SCID gene therapy 18 months ago were presented at the European Society of Gene and Cell Therapy conference in Madrid, Spain, last week. The children's immune systems have continued to improve since receiving the treatment, says Bobby Gaspar of Great Ormond Street Hospital in London, who led the trial.

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'Bubble kid' success puts gene therapy back on track

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A modified version of the virus that causes AIDS could be the unlikely saviour of a promising treatment for a host of deadly diseases

IN TECHNOLOGY, it is called the hype cycle: what initially seems a promising breakthrough leads to inflated expectations until it becomes clear that a great deal of time, money and effort will be needed to realise that promise. Disillusionment sets in until the first real successes are reported, and then the hype is on again.

So it has gone with gene therapy. When, in the late 1980s, the genes for debilitating inherited diseases began to be identified, many believed that cures were within reach, by replacing the faulty genes with working ones. But getting the right gene into the right place without doing more harm than good proved tricky. Now, 23 years after the first gene therapy trial for a rare immune disease called ADA-SCID, researchers finally have some successes to report (see "'Bubble kid' success puts gene therapy back on track").

Still, a major barrier remains: cost. The first gene therapy drug to be approved for clinical use, to treat a pancreatic disease, is also the world's most expensive drug. At the moment, the production of modified viruses the vectors used to shuttle genes into a person's cells is prohibitively expensive, meaning only a handful of those with the diseases in question can be treated.

Pharmaceutical companies may have the means and know-how to scale up production, but inherited genetic diseases are not common. So the industry has been reluctant to invest in treatments for them, preferring instead to channel cash towards bigger killers like cancer.

By a stroke of fortune, a promising form of cancer treatment relying on immunotherapy uses the same viral vector that gene therapists are working on to treat diseases like SCID: a modified version of the virus that causes HIV. Some 700 trials using this kind of safer vector are under way, treating a range of degenerative and immune disorders.

It may seem ironic that a virus that has killed so many holds the potential to yield a cure for a host of other deadly diseases, but such is scientific progress: it comes from unexpected places. That should give fresh grounds for the pharma industry to look again at gene therapy. With a bit of ingenuity and effort, gene therapy might finally live up to the hype.

Correction: When this article was first published on 30 October 2013, the strap and standfirst confused HIV and AIDS.

This article appeared in print under the headline "Live up to the hype"

If you would like to reuse any content from New Scientist, either in print or online, please contact the syndication department first for permission. New Scientist does not own rights to photos, but there are a variety of licensing options available for use of articles and graphics we own the copyright to.

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Gene therapy needs a hero to live up to the hype

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By Chris Reidy/Globe Staff/October 31, 2013

Fidelity Biosciences, a venture capital firm that is a subsidiary of the parent company of Fidelity Investments, and REGENX Biosciences announced the formation of Dimension Therapeutics, a Cambridge-based gene therapy company focused on developing novel treatments for rare diseases such as hemophilia.

Dimension has completed an undisclosed Series A financing led by Fidelity Biosciences.

In conjunction with its launch, Dimension has entered into an exclusive license and collaboration with REGENX. Through that arrangement, Dimension has acquired preferred access toNAVvector technology and rights in REGENX product programs in multiple rare disease indications.

Gene therapy is a fundamental method of disease intervention, changing a patients genetic code to treat genetic disease, and in some cases providing a potential lifelong benefit following a single treatment, Thomas R. Beck, MD, executive partner at Fidelity Biosciences and interim chief executive of Dimension Therapeutics, said in a statement. A core challenge for gene therapy has been the development of safe, efficient vectors to enable delivery of the replacement gene to the correct cells and tissues of the patient to yield benefit. We believe REGENXNAVvectors are the most promising approach forin vivogene therapy and represent the potential for transformative therapy for patients.

Copyright 2013 Globe Newspaper Company.

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Fidelity Biosciences helps launch company focused on gene therapy products

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CAMBRIDGE, MASS. & WASHINGTON--(BUSINESS WIRE)--

Fidelity Biosciences and REGENX Biosciences today announced the formation of Dimension Therapeutics, a gene therapy company focused on developing novel treatments for rare diseases. Dimension will focus on advancing its platform of gene therapy programs in rare diseases through clinical development, starting with lead programs in hemophilia, and building out a world-class product engine for AAV therapeutics. Dimension has completed an undisclosed Series A financing led by Fidelity Biosciences.

In conjunction with its launch, Dimension has entered into an exclusive license and collaboration with REGENX. REGENX holds exclusive rights to a portfolio of over 100 patents and patent applications pertaining to its NAV vector technology that includes novel AAV vectors such as rAAV7, rAAV8, rAAV9, and rAAVrh10. Through its license and collaboration with REGENX, Dimension has acquired preferred access to NAV vector technology and rights in REGENX product programs in multiple rare disease indications.

Gene therapy is a fundamental method of disease intervention, changing a patients genetic code to treat genetic disease, and in some cases providing a potential lifelong benefit following a single treatment, said Thomas R. Beck, M.D., executive partner at Fidelity Biosciences and interim chief executive officer of Dimension Therapeutics. A core challenge for gene therapy has been the development of safe, efficient 'vectors' to enable delivery of the replacement gene to the correct cells and tissues of the patient to yield benefit. We believe REGENX NAV vectors are the most promising approach for in vivo gene therapy and represent the potential for transformative therapy for patients.

Dimension has assembled a team of leaders in the areas of rare disease and gene therapy as well as industry veterans and experienced entrepreneurs. The company has appointed Dr. Beck as interim chief executive officer and Sam Wadsworth, Ph.D., as chief scientific officer. Dr. Wadsworth was previously head of gene therapy research and development at Genzyme, where he led preclinical development for multiple rare disease and gene therapy programs.

The companys scientific advisors are leading experts in the field of gene therapy and rare disease. Dr. James Wilson, director of the gene therapy program at the University of Pennsylvania and the scientific founder of REGENX, will chair the companys Scientific and Technical Advisory Board. NAV vector technology was discovered in the laboratory of Dr. Wilson at the University of Pennsylvania. Other advisors to Dimension include Emil D. Kakkis, M.D., Ph.D., president and chief executive officer of Ultragenyx, a leading rare disease company, and former chief medical officer of Biomarin.

Ben Auspitz, partner at Fidelity Biosciences, has been appointed chairman of the Board of Dimension, and will be joined by directors Allan M. Fox, founding and managing partner of FOXKISER, the entrepreneurial force behind REGENX; Donald J. Hayden, an experienced pharmaceutical executive and the chairman of REGENX; and Dr. Beck.

In parallel to the formation of Dimension, Fidelity Biosciences has also made a direct investment into REGENX, with Mr. Auspitz joining the REGENX Board.

We are pleased to work with Fidelity to establish a new best-in-class company in AAV gene therapy that has the opportunity to invest in the focused development of multiple important rare diseases, including hemophilia, said Ken Mills, president and chief executive officer of REGENX. We view the formation of Dimension as important in the evolution of REGENXs mission to enable access to NAV vector technology through partnership and licensing to create successful new AAV therapeutics.

About Fidelity Biosciences

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Fidelity Biosciences and REGENX Biosciences Launch Dimension Therapeutics to Develop and Commercialize Novel AAV Gene ...

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Dimension Therapeutics wants to develop a lifetime fix for hemophilia using gene therapy.

On Thursday, another gene therapy start-up announced its launch. Dimension Therapeutics hopes to develop virus-delivered gene treatments for rare diseases and its first target is the blood-clotting disorder hemophilia.

The announcement comes just a week after the launch of another gene therapy start up, Spark Therapeutics (see New Gene Therapy Company Launches). One reason that the dashed hopes of gene therapy seem to be mending is that researchers have improved the technologies for delivering genetic fixes. Functional copies of genes are carried by modified viruses, or vectors, into the cells of patients who have missing or dysfunctional copies of those genes. Many groups use vectors based on adeno-associated viruses, or AAVs, which live in most of our bodies already to no ill effect.

Dimension has licensed AAV technology from Washington, D.C.,-based Regenx Biosciences, a company founded by gene therapy pioneer James Wilson. Wilson headed the University of Pennsylvania institute that oversaw a gene therapy trial in 1999 that ended with the death of Jesse Gelsinger, an 18-year-old trial volunteer (see The Glimmering Promise of Gene Therapy). Gelsingers death was blamed on an immune reaction to the experimental therapys viral vector.

That trial used a different kind of virus and since its tragic end, Wilson had searched for better vectors, which he found in AAVs. According to Wired, Wilsons original AAV, AAV1, was the basis for the first gene therapy to be approved in a Western market (see Gene Therapy on the Mend as Treatment Gets Western Approval). Spark Therapeutics is also using a type of AAV to deliver its treatments.

Wilson and his team have since discovered and developed hundreds of modified AAVs, which can target different organs in the body but have been stripped of their ability to replicate. Regenx licensed several vectors to Dimension. A release announcing Dimensions launch suggests that it was Regenx technology that inspired confidence from venture capital firm Fidelity Biosciences to fund the new company:

A core challenge for gene therapy has been the development of safe, efficient vectors to enable delivery of the replacement gene to the correct cells and tissues of the patient to yield benefit, said Fidelity partner and interim CEO of Dimension Thomas Beck. We believe Regenex [vectors] are the most promising approach for in vivo gene therapy.

An early-stage trial of a Regenx vector carrying the gene missing from certain hemophilia patients showed it could correct the disorder (four of the six trial participants were able to quit taking their prophylactic clotting medication) with few side-effects, reported researchers in 2011. The modified virus vectors can still attract the attention of the immune system, but the medical researchers were able to control the immune reaction with immunosuppressive drugs.

While many gene therapy researchers and companies use AAV technology, there are some exceptions. Bluebird Bio, for instance, uses an attenuated version of an HIV viruses that cannot replicate. Bluebird is recruiting patients for alate-stage trial of a gene therapy for a hereditary form of childhood neurodegeneration.

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Improved Virus Technology Spurs New Gene Therapy Startups

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DUBLIN--(BUSINESS WIRE)--

Research and Markets (http://www.researchandmarkets.com/research/kn8z7q/china_food_safety) has announced the addition of the "China Food Safety Testing Industry Report, 2013-2015" report to their offering.

China food safety testing industry started from testing of agricultural and livestock products in the early 1900s. With the issuance of Chinese food safety related laws and regulations as well as the enhancement of food safety supervision, China food safety testing industry has developed rapidly. In 2009-2012, China food safety testing market grew at the average annual growth rate of 20%. In 2012, the market value hit RMB4.01 billion, reflecting a year-on-year increase of 11.1%. And the figure is expected to be RMB4.411 billion in 2013.

Key Topics Covered

1 Overview of Food Safety Testing Industry

1.1 Definition and Classification

1.2 Industry Chain

2 Operating Environments of Chinese Food Safety Testing

2.1 Policy

2.2 International Market

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Research and Markets: China Food Safety Testing Industry Report, 2013-2015

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PUBLIC RELEASE DATE:

29-Oct-2013

Contact: Dr. Hongbin Zhang hbz7049@tamu.edu 979-862-2244 Texas A&M AgriLife Communications

COLLEGE STATION A new study by Texas A&M University cotton researchers and breeders will take advantage of new high-throughput sequencing technology to rapidly advance cotton genetics research and breeding.

Their goal: maintain U.S. cotton's competitiveness in the world cotton market, according to Dr. Hongbin Zhang, professor of plant genomics and systems biology and director of the Laboratory for Plant Genomics and Molecular Genetics in College Station.

The three-year, $500,000 National Institute for Food and Agriculture-funded study, will be conducted by Zhang, along with Dr. Meiping Zhang, Texas A&M AgriLife Research associate research scientist; Dr. C. Wayne Smith, Texas A&M professor of cotton breeding and soil and crop sciences associate department head, and Dr. Steve Hague, associate professor of cotton genetics and breeding in the Texas A&M AgriLife Research Cotton Improvement Lab.

"Cotton is the leading textile fiber and a major bioenergy oilseed crop in Texas and the U.S., with an annual economic impact of about $120 billion in the U.S.," Zhang said.

"In our previous studies, we have already constructed the first genome-wide physical map of Upland cotton, which accounts for more than 90 percent of the cotton in Texas and the U.S." he said. "We are also using the physical map as a platform to sequence the cotton genome."

Also, they previously developed a population of 1,172 recombinant inbred lines that are essential to fine map the cotton genome and genes of economic importance for fiber and oilseed production, Zhang said.

They phenotyped seven of the traits important for fiber quality and yield in 200 of those lines and their parents using three replicated field trials for three years at College Station. The researchers then sequenced and profiled the gene expressions in the developing fibers of those lines, Zhang said.

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New Texas A&M gene study aimed at enhanced cotton fiber breeding, toolkits

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PUBLIC RELEASE DATE:

30-Oct-2013

Contact: Vicki Cohn vcohn@liebertpub.com 914-740-2100 x2156 Mary Ann Liebert, Inc./Genetic Engineering News

New Rochelle, NY, October 30, 2013The repair of ruptured tendons often requires the use of a graft to bridge gaps between the torn tendon and bone. A tissue-engineered collagen graft can reduce the complications associated with other types of tendon grafts, but it may not be able to support full load bearing until integrated into the surrounding tissue. A new suture technique designed to support this tissue-engineered tendon is described in BioResearch Open Access, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers. The article is available free on the BioResearch Open Access website.

The article "Development of a Surgically Optimized Graft Insertion Suture Technique to Accommodate a Tissue-Engineered Tendon In Vivo" presents an innovative interlocking suture technique that distributes suture tension away from the cut end of the injured tendon provides adequate mechanical strength to allow for weight bearing as healing progresses.

Coauthors Prasad Sawadkar et al., University College London and University of Manchester, UK, describe the suture technique and present the results of mechanical stress tests and image analysis of tendons repaired using either standard graft insertion methods or their novel suture technique. "We now have ex vivo proof of concept that this suture technique is suitable for testing in vivo, and this will be the next stage of our research," state the authors.

"Advances in tendon repair and bioengineering are essential for improved management and outcomes of tendon injuries," says BioResearch Open Access Editor Jane Taylor, PhD, MRC Centre for Regenerative Medicine, University of Edinburgh, Scotland. "This article shows exciting 'proof of concept' ex vivo data, which will be useful for improving current tendon repair techniques."

###

About the Journal

BioResearch Open Access is a bimonthly peer-reviewed open access journal led by Editor-in-Chief Robert Lanza, MD, Chief Scientific Officer, Advanced Cell Technology, Inc. and Editor Jane Taylor, PhD. The Journal provides a new rapid-publication forum for a broad range of scientific topics including molecular and cellular biology, tissue engineering and biomaterials, bioengineering, regenerative medicine, stem cells, gene therapy, systems biology, genetics, biochemistry, virology, microbiology, and neuroscience. All articles are published within 4 weeks of acceptance and are fully open access and posted on PubMedCentral. All journal content is available on the BioResearch Open Access website.

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Novel technique for suturing tissue-engineered collagen graft improves tendon repair

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October 30, 2013 Sophie Langley

Using genetic engineering, scientists at the University of Washington have identified a population of neurons that tell the brain to shut off appetite.

The study, published on 13 October 2013 in the journal Nature, considered what might make an animal lose its appetite. Researchers said there are a number of natural reasons, including infection, nausea, pain or simply having eaten too much.

Nerves within the gut that are distressed or insulated send information to the brain through the vagus nerve. Appetite is suppressed when these messages activate specific neurons ones that contain calcitonin gene-related peptide (CGRP) in a region of the brain called parabrachial nucleus.

Study method

In mouse trials, the researchers used genetic techniques and viruses to introduce light-activatable proteins into CGRP neurons. Activation of these proteins excites the cells to transmit chemical signals to other regions of the brain. When they activated the CGRP neurons with a laser, the hungry mice immediately lost their appetite and walked away from their liquid diet; when the laser turned off, the mice resumed drinking the liquid diet.

These results demonstrate that activation of the CGRP-expressing neurons regulates appetite, said Richard Palmiter, Professor of Biochemistry at the University of Washington and Investigator of the Howard Hughes Medical Institute. This is a nice example of how the brain responds to unfavourable conditions in the body, such as nausea caused by food poisoning, he said.

Using a similar approach, neurons in other brain regions have been identified that can stimulate the appetite of mice that are not hungry. Researchers said they hoped to identify the complete neural circuit (wiring diagram) in the brain that regulates feeding behaviour. By identifying these neural circuits, researchers said scientists may be able to design therapies that promote or decrease appetite.

The study was conducted by Matthew E. Carter in Richard Palmiters laboratory and Marta E. Soden in Larry S. Zweifels (Assistant Professor of Pharmacology at the University of Washington) laboratory. Funding for the research was provided by the Davis Foundation, the Klarman Family Foundation, the Howard Hughes Institute and the National Institutes of Health.

Brain circuit responsible for appetite suppression identified

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Scientists discover neural circuit responsible for appetite suppression

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PUBLIC RELEASE DATE:

30-Oct-2013

Contact: Jim Fessenden james.fessenden@umassmed.edu 508-856-2000 University of Massachusetts Medical School

WORCESTER, MA New insights into how human cytomegalovirus (CMV), the leading cause of birth defects associated with infection spreads from pregnant mother to fetus and from organ to organ in newborns provides translational researchers an exciting new avenue for investigation that may lead to the development of therapeutic interventions. Using next generation sequencing and population genetic modeling, scientists at the University of Massachusetts Medical School (UMMS) and the Ecole Polytechnique Fdrale de Lausanne (EPFL) have found that CMV evolves rapidly and dramatically in humans. These findings, published in PLoS Genetics, provide new genetic targets that could impede the evolution of CMV and prevent its spread.

"These findings have important implications for how we think about and develop new therapeutic treatments for CMV," said Timothy F. Kowalik, PhD, associate professor of microbiology and physiological systems and senior author of the study. "Although CMV is able to infect a wide variety of organs throughout the body, there are a substantial number of genetic changes that occur before the virus can spread and replicate efficiently in different anatomic niches. If these genetic changes can be prevented, it may be possible to isolate and block the spread of CMV."

CMV is a ubiquitous virus that infects most of the human population and can move throughout the body from organ to organ. Infection is usually asymptomatic in healthy hosts, but may cause severe symptoms for patients with a compromised immune system, such as organ transplant recipients, HIV-infected persons, newborn infants or the fetus during gestation.

Congenital CMV infection, which is passed from a pregnant mother to fetus, is a significant cause of birth defects, and remains a high priority for vaccine development according to the nonprofit, Institute of Medicine. An estimated 30,000 infants per year in the U.S. are diagnosed with congenital CMV infection, and nearly 20 percent exhibit permanent neurologic effects such as hearing loss or developmental delay.

To better understand how CMV evolves in fetuses and newborns during symptomatic congenital infection, researchers at UMMS and the University of Minnesota Medical School collected samples from the plasma and urine of five congenitally infected infants during the first year after birth. Using next generation DNA sequencing, Kowalik and colleagues studied the diversity and changes in viral DNA sequences over time and between organs. Though the DNA sequences from viruses taken from the same type of sample (e.g. plasma) were similar to each other, the study's authors found dramatic differences between the sequences collected from viruses in the plasma and urine of the same infant. Surprisingly, the plasma and urine sequences from the same infant were as different as sequences from two unrelated infants.

These results suggest that CMV is able to evolve very quickly as the differences between the plasma and urine sequences likely occurred in the short period between the initial, in utero infection, and the first year after birth. However, the mechanism driving this phenomenon remained unclear.

To answer this question, researchers used mathematical modeling and statistical inference to uncover evidence that population bottlenecks and expansions may play a significant role in the virus' evolution after infection. Characterized by a substantial reduction in viral copies followed by a quick rebound, population bottlenecks and expansions can lead to dramatic changes in DNA sequences that result in two related populations quickly becoming dissimilar. In the case of CMV infection, this phenomenon appeared to coincide with the virus moving from the mother to the fetus and later migrating from the plasma to the kidneys.

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Research points to potential window for treating CMV and preventing mother-to-child transmission

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Newswise In the first molecular genetic study of families with a history of both language impairment and autism, scientists may have uncovered a shared origin for the two conditions, an important step toward explaining why some cases of autism are accompanied by language difficulties and others are not. The study, a collaboration of The Research Institute at Nationwide Childrens Hospital with experts at Rutgers University, indicates that a disorder called specific language impairmentone of the most common developmental delays in childrenmay be caused by the same genetic variants that lead to language difficulties in some children with autism. The findings are published Oct. 30 in the American Journal of Psychiatry.

As many as two-thirds of individuals with an Autism Spectrum Disorder (ASD) also have language impairments, which can range from mild limitations to complete non-verbal behavior. However, the remaining third may have normal or even above average language abilities. To investigate whether specific language impairment and language-impaired autism cases are caused by the same genetic variants, researchers examined the genetic code of 79 families with a history of both conditions.

Using a genome-wide scan and a series of language tests, the researchers identified two new genetic links for language impairment in these families: 15q23-26 and 16p12. Each of these new links is jointly related to language-impaired ASD and non-ASD related specific language impairment, suggesting a single cause for both issues.

A genetic cause of language impairment may help explain why some kids with ASD have language impairments and others dont, as well as why some members of a family have language impairment only and others have ASD as well, says Christopher W. Bartlett, PhD, principal investigator in the Battelle Center for Mathematical Medicine at Nationwide Childrens and lead author of the study. The research is part of a long-term collaboration between scientists at Nationwide Childrens and Rutgers, initiated by a grant from the National Institute of Mental Health to Linda M. Brzustowicz, MD, professor of the Department of Genetics at Rutgers and senior investigator on the project.

Language impairment is not part of the diagnostic definition of ASD. And according to Dr. Bartlett, this study raises the question of whether language impairment is actually a dissociable trait in at least some forms of ASD.

There is nothing about autism that dictates that language impairment has to occur, says Dr. Bartlett, who also is an assistant professor of pediatrics at The Ohio State University School of Medicine. In this study, we demonstrated a shared mechanism between the two disorders. Language problems and ASD are complicated and have numerous genetic factors, but we think that many genetic factors related to communication could be the same for specific language impairment and language-impaired autism.

The genetic variations appear to be relevant to both disorders and may indicate a greater level of genetic predisposition for impairments in language ability among individuals with and without ASD in those families.

In an earlier study, the researchers found similarities in language deficit type and severity between language-impaired non-ASD and language-impaired ASD individuals in the same family. The behavioral genetics study, published in Biological Psychiatry, found that the same genes active in specific language impairment appear in ASD, but their effect is amplified in ASD. That finding, coupled with this new research, suggests that the two disorders may be on an etiological continuum.

If further research confirms a genetic link between language impairment and ASD, then we may be able to find out why some family members only develop language impairment while others develop autism, says Dr. Bartlett. But most of all, we want to know why there is such a range in communication abilities in children with autismwhy so many children have language difficulties when its not required for the diagnosis.

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Scientist Identify Genetic Link Between Language Impairment and Autism

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California Lightworks 800W Solarstorm - exoticgenetix (Afterlife OG) / DNA Genetics (Tangie) Day 12
I swear this looked WAAAAYYY more white balanced on the cameras LCD. lol 🙁 Make sure to check out my Instagram as well: http://instagram.com/ledoneshot Musi...

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California Lightworks 800W Solarstorm - exoticgenetix (Afterlife OG) / DNA Genetics (Tangie) Day 12 - Video

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Spinal cord injury rehabilitation2
Spinal cord injury rehabilitation is comprehensive program in that various healthcare professionals works together to bring optimal level of independence to ...

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DENVER & CHICAGO--(BUSINESS WIRE)--

The Global Down Syndrome Foundation, the Alzheimers Association, and the Linda Crnic Institute for Down Syndrome have awarded $1.2 million in research grants to five scientists for innovative investigations that explore the development of Alzheimers disease in individuals with Down syndrome. The goal is to eventually translate the findings into improved treatments for all people with Alzheimers.

The organizations are supporting this growing area of study through a new joint grants initiative called Understanding the Development and Devising Treatments for Alzheimers Disease in Individuals with Down Syndrome.

The Alzheimers Association is very interested in understanding why people with Down syndrome are at such high risk for Alzheimers, and how it relates to other variations of the disease, so that we can identify new therapies to treat Alzheimers in both the Down syndrome and typical populations, said Maria Carrillo, Ph.D., Alzheimers Association vice president of Medical and Scientific Relations. Research in this population may also help us develop predictive tools for Alzheimers and design more effective clinical trials.

Investing with the Alzheimers Association has been so rewarding. The science our joint initiative is funding is of the highest caliber, and each grant approaches understanding, treating or preventing Alzheimers in people with Down syndrome from a very different angle. If initial results are promising, we hope that the National Institutes of Health will continue to fund this excellent science, said Michelle Sie Whitten, executive director of the Global Down Syndrome Foundation.

Alzheimers Disease and Down Syndrome

Alzheimer's is a fatal, progressive, degenerative brain disease that causes problems with memory, thinking and behavior. More than 5 million Americans are living with Alzheimers, which is the sixth leading cause of death in the United States.

Down syndrome is a genetic disorder whereby a person has three copies of chromosome 21 instead of two. This chromosome also contains the gene that encodes the amyloid precursor protein (APP). APP is cleaved to form amyloid-beta, which is the primary component of amyloid plaques a lesion found in the brains of people with Alzheimers that many scientists believe is part of the cause of the disease.

According to the U.S. Centers for Disease Control and Prevention, Down syndrome occurs in 1 out of 691 infants in the United States. Due to improved clinical care, people with Down syndrome are now regularly living into their sixth decade of life, causing many to develop dementia due to Alzheimers. Autopsy studies show that by age 40, the brains of almost all individuals with Down syndrome have significant levels of plaques and tangles abnormal protein deposits that are considered Alzheimer's hallmarks. But despite the presence of these brain changes, not everyone with the syndrome develops Alzheimer's symptoms.

One of the many questions researchers hope to answer about Down syndrome is why some people develop dementia symptoms and others don't. Researchers are working to answer a similar key question about those who don't have Down syndrome. This may lead to new opportunities for treatment and prevention of the disease.

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New Research Grants from Alzheimer’s Association and Global Down Syndrome Foundation Explore Links Between Alzheimer’s ...

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PUBLIC RELEASE DATE:

29-Oct-2013

Contact: Vicki Cohn vcohn@liebertpub.com 914-740-2100 Mary Ann Liebert, Inc./Genetic Engineering News

New Rochelle, NY, October 29, 2013Social media such as YouTube videos provide a popular and flexible venue for online activism. How two different social protest movementsOccupy Wall Street and the Proposition 8 same sex marriage initiativeutilized YouTube, and their success in engaging activists are explored in an article in Cyberpsychology, Behavior, and Social Networking, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers. The article is available free on the Cyberpsychology, Behavior, and Social Networking website.

Emily Vraga, PhD and coauthors from George Mason University (Fairfax, VA), Georgetown University (Washington, DC), University of Wisconsin-Madison, and University of Southern California (Los Angeles, CA) emphasize an important advantage of YouTube videos for the purpose of social and political activism: they can be shared easily, quickly, and effectively through a variety of mechanisms, including other forms of social media, email, and print media.

The article "The Rules of Engagement: Comparing Two Social Protest Movements on YouTube" compares how two disparate political movements used YouTube to define and advance their goals. The study shows that social media activism resulted in differing degrees of popularity and engagement, perhaps related to the content of the videos and to the different online environments in which they appear.

"As YouTube matures, and additional social networking tools evolve, it is interesting to note how these tools may be used by individual citizens as well as political activists to advance their goals," says Brenda K. Wiederhold, PhD, MBA, BCIA, Editor-in-Chief of Cyberpsychology, Behavior, and Social Networking, from the Interactive Media Institute, San Diego, CA.

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About the Journal

Cyberpsychology, Behavior, and Social Networking is a peer-reviewed journal published monthly online with Open Access options and in print that explores the psychological and social issues surrounding the Internet and interactive technologies, plus cybertherapy and rehabilitation. Complete tables of content and a sample issue may be viewed on the Cyberpsychology, Behavior, and Social Networking website.

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Is YouTube a driver for social movements like Occupy Wall Street?

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Originally published October 28, 2013 at 8:55 PM | Page modified October 28, 2013 at 11:03 PM

Charles P. Max Moehs is a principal scientist with Arcadia Biosciences in Seattle, where he uses both genetic engineering and conventional breeding to develop low-gluten grains and crops with increased stress tolerance.

Q: Why do you oppose labeling of genetically engineered foods?

A: Genetic engineering is a technology, not a trait. The National Academy of Sciences and other scientific bodies throughout the world have argued that the important thing about crops is what they contain, not how theyre generated.

If a food is unsafe, its not labeled; its taken off the market.

When you make soybean oil from soybeans genetically engineered to be resistant to an insect or herbicide, the oil doesnt contain any genetically engineered components. To add a label that says May contain genetically engineered ingredients is misleading and not really helpful to consumers.

Q: What do you worry might happen if GE foods are labeled?

A: I think it demonizes technology that has potential for a lot of benefits.

I also think it will require producers to segregate their genetically engineered and non-GE products ... and create an additional burden for them. Imagine trying to clean a combine of every last seed. If a farmer has both genetically engineered crops and non-GE crops, they would almost have to run two separate operations.

And it strikes me as an invitation to frivolous lawsuits.

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CON on I-522: GE researcher fears ‘demonization’ of a technology with many potential benefits

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Newswise New York, NYOctober 28, 2013: Researchers at Columbia Engineering, led by Chemical Engineering Professors Venkat Venkatasubramanian and Sanat Kumar, have developed a new approach to designing novel nanostructured materials through an inverse design framework using genetic algorithms. The study, published in the October 28 Early Online edition of Proceedings of the National Academy of Sciences (PNAS), is the first to demonstrate the application of this methodology to the design of self-assembled nanostructures, and shows the potential of machine learning and big data approaches embodied in the new Institute for Data Sciences and Engineering at Columbia.

Our framework can help speed up the materials discovery process, says Venkatasubramanian, Samuel Ruben-Peter G. Viele Professor of Engineering, and co-author of the paper. In a sense, we are leveraging how nature discovers new materialsthe Darwinian model of evolutionby suitably marrying it with computational methods. Its Darwin on steroids!

Using a genetic algorithm they developed, the researchers designed DNA-grafted particles that self-assembled into the crystalline structures they wanted. Theirs was an inverse way of doing research. In conventional research, colloidal particles grafted with single-stranded DNA are allowed to self-assemble, and then the resulting crystal structures are examined. Although this Edisonian approach is useful for a posteriori understanding of the factors that govern assembly, notes Kumar, Chemical Engineering Department Chair and the studys co-author, it doesnt allow us to a priori design these materials into desired structures. Our study addresses this design issue and presents an evolutionary optimization approach that was not only able to reproduce the original phase diagram detailing regions of known crystals, but also to elucidate previously unobserved structures.

The researchers are using big data concepts and techniques to discover and design new nanomaterialsa priority area under the White Houses Materials Genome Initiativeusing a methodology that will revolutionize materials design, impacting a broad range of products that affect our daily lives, from drugs and agricultural chemicals such as pesticides or herbicides to fuel additives, paints and varnishes, and even personal care products such as shampoo.

This inverse design approach demonstrates the potential of machine learning and algorithm engineering approaches to challenging problems in materials science, says Kathleen McKeown, director of the Institute for Data Sciences and Engineering and Henry and Gertrude Rothschild Professor of Computer Science. At the Institute, we are focused on pioneering such advances in a number problems of great practical importance in engineering.

Venkatasubramanian adds, Discovering and designing new advanced materials and formulations with desired properties is an important and challenging problem, encompassing a wide variety of products in industries addressing clean energy, national security, and human welfare. He points out that the traditional Edisonian trial-and-error discovery approach is time-consuming and costlyit can cause major delays in time-to-market as well as miss potential solutions. And the ever-increasing amount of high-throughput experimentation data, while a major modeling and informatics challenge, has also created opportunities for material design and discovery.

The researchers built upon their earlier work to develop what they call an evolutionary framework for the automated discovery of new materials. Venkatasubramanian proposed the design framework and analyzed the results, and Kumar developed the framework in the context of self-assembled nanomaterials. Babji Srinivasan, a postdoc with Venkatasubramanian and Kumar and now an assistant professor at IIT Gandhinagar, and Thi Vo, a PhD candidate at Columbia Engineering, carried out the computational research. The team collaborated with Oleg Gang and Yugang Zhang of Brookhaven National Laboratory, who carried out the supporting experiments.

The team plans to continue exploring the design space of potential ssDNA-grafted colloidal nanostructures, improving its forward models, and bring in more advanced machine learning techniques. We need a new paradigm that increases the idea flow, broadens the search horizon, and archives the knowledge from todays successes to accelerate those of tomorrow, says Venkatasubramanian.

This research has been funded by a $1.4 million three-year grant from the U.S. Department of Energy.

Columbia Engineering Columbia University's Fu Foundation School of Engineering and Applied Science, founded in 1864, offers programs in nine departments to both undergraduate and graduate students. With facilities specifically designed and equipped to meet the laboratory and research needs of faculty and students, Columbia Engineering is home to NSF-NIH funded centers in genomic science, molecular nanostructures, materials science, and energy, as well as one of the worlds leading programs in financial engineering. These interdisciplinary centers are leading the way in their respective fields while individual groups of engineers and scientists collaborate to solve some of modern societys more difficult challenges. http://www.engineering.columbia.edu/

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Using Data Science Tools to Discover New Nanostructured Materials

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Current ratings for: Red and processed meat 'increases colorectal cancer risk'

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People with a common genetic variant who consume red or processed meat may increase their risk of colorectal cancer. This is according to a study presented at the annual American Society of Human Genetics 2013 meeting.

Furthermore, the US researchers say they also found another specific genetic variant that suggests eating more fruit, vegetables and fiber may lower the risk of colorectal cancer.

To reach their findings, the researchers analyzed 9,287 patients suffering from colorectal cancer alongside a control group of 9,117 healthy individuals.

They also analyzed 2.7 million genetic sequences to determine whether there was a link between consumption of red and processed meat and colorectal cancer.

The study shows that individuals with the genetic variant rs4143094 - a variant that affects 1 in 3 people - demonstrate a significantly increased risk of colorectal cancer linked to the consumption of red and processed meat.

The researchers explain that this genetic variant is located on the same chromosome 10 region that has a transcription factor gene called GATA3 - a gene that has previously been linked to many forms of cancer.

The transcription factor encoded by this gene usually plays a part in the immune system, say the researchers.

Speculating on the link with processed meat, the researchers say that when the body digests it, this may trigger an "immunological or inflammatory" response. But if the GATA3 gene region consists of a genetic variant, it is possible it could encode a dysregulated transcription factor, making it hard to overthrow the response.

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Red and processed meat 'increases colorectal cancer risk'

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A big thank you to MILEHIGH GENETICS.
Thank you so much brother .I can #39;t wait to put to good use.

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