Archive for the ‘Gene Therapy Research’ Category

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.

Originally posted here:
Research points to potential window for treating CMV and preventing mother-to-child transmission

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...

By: oneshotgrow

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

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 ...

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.

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

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.

###

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?

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

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'

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

By: ToddGrows420

Excerpt from:
A big thank you to MILEHIGH GENETICS. - Video

SALT LAKE CITY, Oct. 29, 2013 (GLOBE NEWSWIRE) -- Myriad Genetics, Inc. (MYGN) today announced that it has launched a new prognostic test, Myriad myPlan Lung Cancer, for patients diagnosed with early-stage lung adenocarcinoma. myPlan Lung Cancer is an RNA expression panel of 31 cell-cycle-progression genes, in combination with tumor staging information, to determine a patient's risk of dying from lung cancer within five years.

The myPlan Lung Cancer test has been extensively studied in more than 1,500 patients. In these studies, myPlan Lung Cancer was shown to be significantly predictive of five-year lung cancer mortality and considerably more predictive than tumor staging information alone. In a large validation study presented at the IASLC annual meeting, patients with a high-risk myPlan Lung Cancer score had nearly twice the rate of lung cancer deaths (35 percent) than patients with a low-risk score (18 percent). These findings build on data published in Clinical Cancer Research that showed the myPlan Lung Cancer test is the strongest independent predictor of survival in patients with early-stage lung cancer as compared to the conventional clinical variables of disease progression including age, stage of disease, gender, smoking status and tumor size.

"Post-surgical treatment decisions for early-stage lung cancer patients are largely guided by tumor staging. However, the prognostic power of tumor stage alone is limited and there remain unacceptably high mortality rates for this patient population," said Mark C. Capone, president of Myriad Genetics Laboratories. "Myriad myPlan Lung Cancer is a powerful new prognostic test that stratifies patients based on their risk of lung cancer-related, five-year mortality. Our test will empower physicians to know with confidence which patients should receive surgery alone versus those who may need more aggressive therapy."

Myriad myPlan Lung Cancer is being launched in a phased approach beginning with an early-access, clinical-experience program to medical and scientific thought leaders, followed by a full commercial launch in calendar year 2014. myPlan Lung Cancer will be sold through Myriad's own specialty sales force to assist physicians in providing their lung cancer patients with the most appropriate healthcare management. myPlan Lung Cancer has a list price of $3,400.

Myriad myPlan Lung Cancer is the second molecular diagnostic test launched by Myriad this fiscal year. In September, the Company launched Myriad myRisk(TM) Hereditary Cancer, a new multi-gene diagnostic test for eight major hereditary cancers including breast, colorectal, ovarian, endometrial, pancreatic, prostate, gastric and melanoma.

About Myriad Genetics

Myriad Genetics is a leading molecular diagnostic company dedicated to making a difference in patients' lives through the discovery and commercialization of transformative tests to assess a person's risk of developing disease, guide treatment decisions and assess risk of disease progression and recurrence. Myriad's molecular diagnostic tests are based on an understanding of the role genes play in human disease and were developed with a commitment to improving an individual's decision making process for monitoring and treating disease. Myriad is focused on strategic directives to introduce new products, including companion diagnostics, as well as expanding internationally. For more information on how Myriad is making a difference, please visit the Company's website: http://www.myriad.com.

Myriad, the Myriad logo and Myriad myPlan Lung Cancer, Myriad myRisk Hereditary Cancer, are trademarks or registered trademarks of Myriad Genetics, Inc. in the United States and foreign countries. MYGN-F, MYGN-G.

Safe Harbor Statement

This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, including statements relating to Myriad myPlan Lung Cancer being launched in a phased approach beginning with an early-access, clinical-experience program to medical and scientific thought leaders, followed by a full commercial launch in calendar year 2014; Myriad myPlan Lung Cancer being sold through Myriad's own specialty sales force to assist physicians in providing their lung cancer patients with the most appropriate healthcare management; Myriad myPlan Lung Cancer being an important new molecular diagnostic tool that will help physicians in predicting the aggressiveness of early-stage lung adenocarcinoma in conjunction with conventional clinical parameters; and the Company's strategic directives under the caption "About Myriad Genetics". These "forward-looking statements" are management's present expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those described in the forward-looking statements. These risks include, but are not limited to: the risk that sales and profit margins of our existing molecular diagnostic tests and companion diagnostic services may decline or will not continue to increase at historical rates; risks related to changes in the governmental or private insurers reimbursement levels for our tests; the risk that we may be unable to develop or achieve commercial success for additional molecular diagnostic tests and companion diagnostic services in a timely manner, or at all; the risk that we may not successfully develop new markets for our molecular diagnostic tests and companion diagnostic services, including our ability to successfully generate revenue outside the United States; the risk that licenses to the technology underlying our molecular diagnostic tests and companion diagnostic services tests and any future tests are terminated or cannot be maintained on satisfactory terms; risks related to delays or other problems with operating our laboratory testing facilities; risks related to public concern over our genetic testing in general or our tests in particular; risks related to regulatory requirements or enforcement in the United States and foreign countries and changes in the structure of the healthcare system or healthcare payment systems; risks related to our ability to obtain new corporate collaborations or licenses and acquire new technologies or businesses on satisfactory terms, if at all; risks related to our ability to successfully integrate and derive benefits from any technologies or businesses that we license or acquire; risks related to increased competition and the development of new competing tests and services; the risk that we or our licensors may be unable to protect or that third parties will infringe the proprietary technologies underlying our tests; the risk of patent-infringement claims or challenges to the validity of our patents; risks related to changes in intellectual property laws covering our molecular diagnostic tests and companion diagnostic services and patents or enforcement in the United States and foreign countries, such as the Supreme Court decision in the lawsuit brought against us by the Association for Molecular Pathology et al; risks of new, changing and competitive technologies and regulations in the United States and internationally; and other factors discussed under the heading "Risk Factors" contained in Item 1A of our most recent Annual Report on Form 10-K filed with the Securities and Exchange Commission, as well as any updates to those risk factors filed from time to time in our Quarterly Reports on Form 10-Q or Current Reports on Form 8-K. All information in this press release is as of the date of the release, and Myriad undertakes no duty to update this information unless required by law.

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Myriad myPlan(TM) Lung Cancer Test Launched to Leading Oncologists

Google Exec Ray Kurzweil Says Robots Will Keep Us Alive | The Rubin Report
Google engineering director and futurist Ray Kurzweil believes we are close to realizing everlasting life and is making it his mission to get us there. This ...

By: Rubin Report

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Google Exec Ray Kurzweil Says Robots Will Keep Us Alive | The Rubin Report - Video

Bolton, UK (PRWEB UK) 29 October 2013

According to the BBC, the University Hospital in Birmingham are trialing a new gene therapy hospital treatment, aiding in the fight against prostate cancer (1). The pioneering treatment could help prevent the incidence of surgical errors, increasing the success rate associated with treating the disease.

It is believed, that if the treatment works, surgery for prostate cancer will become a thing of the past, eliminating the surgical errors associated with the procedure.

According to the BBC, the University Hospital in Birmingham is among the first to trial the new treatment, stimulating the bodys own immune system to attack the tumor (1). With studies on mice showing complete eradication of the cancer, hopes are high for the imminent human trials.

A new form of gene therapy, the treatment requires a virus to be injected directly into the prostate cancer tumour; a gene attached to the virus (GM-CSF,) is then released, activating the body's own immune system, attracting white blood cells to attack the cancer (1). As reported by the BBC, the virus also carries an enzyme - nitroreductase - which sits inside the cancer cell.

Two days after the injection, the patient in question will be put on a drip, which contains a cancer drug (CB1954), which is initially inactive. When the drug comes into contact with the enzyme, it reacts and starts killing the cancer cells. The inactive drug, CB1954, does not harm cells which do not have the enzyme inside, protecting the healthy tissue surrounding the tumour (1).

Urology Specialist, Prashant Patel, is hopeful that gene therapy could provide real hope for patients who are running out of treatment options (1).

"I have to stress that this is only a phase one safety trial to test that there are no side effects. However, we are excited by this."

"If this works, 15 to 20 years from now, we could be using the patient's own immune system in this way to fight early onset prostate cancer so that patients won't need painful treatments or even surgery."

Considering the procedure, Asons Executive, Laura Williams

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New Gene Therapy Treatment Helps Fight Prostate Cancer, Asons Solicitors Comment

Newswise Scientists in The Research Institute at Nationwide Childrens Hospital have found a way to overcome one of the biggest obstacles to using viruses to deliver therapeutic genes: how to keep the immune system from neutralizing the virus before it can deliver its genetic payload. In a study published recently in Molecular Therapy, researchers found that giving subjects a treatment to temporarily rid the body of antibodies provides the virus safe passage to targeted cells, allowing it to release a corrective or replacement gene to treat disease.

Gene therapy is among the most promising treatment options for such genetic disorders as muscular dystrophy, congenital blindness and hemophilia. Scientists also are investigating gene therapy as a cure for some cancers, neurodegenerative diseases, viral infections and other acquired illnesses. To get the therapeutic gene into cells, researchers have turned to viruses, which deliver their genetic material into cells as part of their normal replication process. Time and time again, these efforts have been thwarted by the bodys own immune system, which attacks the viral vector. The therapeutic genes arent delivered and disease rages on.

Now, a team led by Louis G. Chicoine, MD, Louise Rodino-Klapac, PhD, and Jerry R. Mendell, MD, principal investigators in the Center for Gene Therapy at Nationwide Childrens, has shown for the first time that using a process called plasmapheresis just before delivering a virus-packed gene therapy protects the virus long enough for it to enter the cell and deliver the gene.

Plasmapheresis, widely used to treat patients with autoimmune disorders, removes blood from the body, separates the plasma and cells, filters out antibodies, and returns the blood to the patient. The antibody loss is temporary; the body begins producing new antibodies within a few hours of the procedure.

In a study of a gene therapy designed to treat Duchenne muscular dystrophy (DMD), Drs. Chicoine and Rodino-Klapac used plasmapheresis in a large animal model, then injected a virus packed with a micro-dystrophin gene. When they examined the levels of micro-dystrophin gene expression in the animals, they found a 500 percent percent increase over gene expression in animals that did not receive plasmapheresis. Dr. Mendell, director of the Center for Gene Therapy, helped conceive of this treatment for DMD patients based on experience with autoimmune diseases such as myasthenia gravis and inflammatory nerve diseases.

Right now, gene therapy seems to work best in patients who have no antibodies for the virus being used to deliver the gene, Dr. Mendell says. That limits the number of patients who can benefit from gene therapy.

Using plasmapheresis would increase the potential for gene therapy, Dr. Chicoine adds, by eliminating one obstacle of immune reaction.

As gene therapy becomes more prevalent, patients may need to receive more than one treatment, Dr. Rodino-Klapac says. The problem is that when they get the first treatment, their body will develop antibodies to the virus used to deliver the gene. Using plasmapheresis on someone who previously received gene therapy could allow them to be treated again.

Full citation: L. G. Chicoine, C. L. Montgomery, W. G. Bremer, K. M. Shontz, D. A. Griffin, K. N. Heller, S. Lewis, V. Malik, W.E. Grose, C. J. Shilling, K. J. Campbell, T. J. Preston, B. D. Coley, P. T. Martin, C. M. Walker, K. R. Clark, Z. Sahenk, J. R. Mendell, and L. R. Rodino-Klapac, Plasmapheresis eliminates the negative impact of AAV antibodies on micro-dystrophin gene expression following vascular delivery. Molecular Therapy. Epub 2013 Oct 23. doi:10.1038/mt.2013.244.

Funding: This research was supported by grants from the Childrens Hospital Foundation; National Institutes of Health; Senator Paul D. Wellstone Muscular Dystrophy Cooperative Research Center Grant at Nationwide Childrens Hospital; Muscular Dystrophy Association; and Jesses Journey Foundation for Gene and Cell Therapy.

Original post:
Researchers Identify Way to Increase Gene Therapy Success

THURSDAY, Oct. 24 (HealthDay News) -- Obesity is on the rise among children, and a particular genetic mutation might play a role for some kids, a new study suggests.

Researchers in Britain conducted genetic analyses of more than 2,100 severely obese youngsters. They found that those with mutations in the KSR2 gene had larger appetites and slower metabolism than those with a normal copy of the gene, according to the study published in the Oct. 24 issue of the journal Cell.

"Changes in diet and levels of physical activity underlie the recent increase in obesity; however, some people gain weight more easily than others," study author Sadaf Farooqi, of the University of Cambridge, noted in a journal news release. "This variation between people is largely influenced by genetic factors. The discovery of a new obesity gene, KSR2, demonstrates that genes can contribute to obesity by reducing metabolic rate -- how well the body burns calories."

The findings could someday lead to new treatments for obesity and type 2 diabetes, the researchers said.

Farooqi and colleagues had previously found that deleting the KSR2 gene led to obesity in mice, highlighting the gene's role in controlling energy balance and metabolism. These findings confirm KSR2's role in the regulation of weight and metabolism in humans.

"This work adds to a growing body of evidence that genes play a major role in influencing a person's weight and may be useful for developing new ways to treat people who are heavy and develop diabetes," Farooqi said.

More information

The American Academy of Pediatrics has more about childhood obesity.

Copyright 2013 HealthDay. All rights reserved.

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Gene mutation tied to higher obesity risk in kids

A rare childhood disease may hold clues to treating Alzheimer's and Parkinson's, a study has found.

Scientists analysing A-T disease, which leaves youngsters unable to walk by adolescence, have found new ways of understanding the more common neurodegenerative diseases, according to the report in the online journal Nature Neuroscience.

Children born with A-T have mutations in both of their copies of the ATM gene and cannot make normal ATM protein, and experts at Rutgers University in New Jersey in the US hope their research will lead to new therapies for Alzheimer's and Parkinson's.

The rare genetic childhood disorder - which occurs in about one in 40,000 births - leads to problems in movement, co-ordination, equilibrium and muscle control as well as a number of other deficiencies outside the nervous system.

Using mouse and human brain tissue studies, the researchers from Rutgers found that without ATM, the levels of a regulatory protein known as EZH2 go up.

Looking through the characteristics of A-T disease in cells in tissue culture and in brain samples from both humans and mice with ATM mutation, they found that the increase in EZH2 was a major contributing factor to the neuromuscular problems caused by A-T.

Plan a conversation in a familiar, non-threatening environment

Explain why talking is important - youre worried because you care

Use examples to make things clearer

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Gene Discovery Could Hold The Key To Treating Alzheimer's And Parkinson's Disease

PUBLIC RELEASE DATE:

28-Oct-2013

Contact: Holly Evarts holly.evarts@columbia.edu 347-453-7408 Columbia University

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. It's 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 study's co-author, "it doesn't 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 House's 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.

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Using genetic algorithms to discover new nanostructured materials

PALO ALTO, Calif.--(BUSINESS WIRE)--

EMD Serono, a subsidiary of Merck KGaA, Darmstadt, Germany, and Open Monoclonal Technology, Inc. (OMT), a leader in the genetic engineering of animals for development of human therapeutic antibodies, today announced expansion of their collaboration agreement from June 2012. Under the terms of the new agreement, EMD Serono will make an upfront payment to secure unlimited access to the OmniRat platform as well as success-based development milestones and royalties. Further details of the contract are not being disclosed.

The expansion of our collaboration with OMT is reflective of its strong quality and the significant progress we've made together over the last 15 months," said Dr. Annalisa Jenkins, Executive Vice President and head of Global Research & Development for Merck Serono, a biopharmaceutical division of Merck KGaA, Darmstadt, Germany. This is a good example of our focus on exploring targeted partnerships with innovative companies such as OMT, with the goal of generating access to technologies that advance our capabilities in biotech development.

Dr. Roland Buelow, founder and CEO of OMT, said: "EMD Seronos transition to unlimited platform access is evidence of their success with OmniRat on multiple therapeutic targets during the past 15 months. The expansion illustrates how OMT provides its partners with access to complementary platforms for discovery of superior antibodies against challenging targets in a cost-effective fashion.

About Merck KGaA

Merck is a leading pharmaceutical, chemical and life science company with total revenues of 11.2 billion in 2012, a history that began in 1668, and a future shaped by approximately 38,000 employees in 66 countries. Its success is characterized by innovations from entrepreneurial employees. Merck's operating activities come under the umbrella of Merck KGaA, in which the Merck family holds an approximately 70% interest and free shareholders own the remaining approximately 30%. In 1917 the US subsidiary Merck & Co. was expropriated and has been an independent company ever since. For more information, please visit http://www.merckserono.com or http://www.merckgroup.com.

About EMD Serono, Inc.

EMD Serono, Inc., a subsidiary of Merck KGaA, Darmstadt, Germany, is a specialized biopharmaceutical company dedicated to developing therapies with ground breaking potential. The company has strong market positions in neurology, endocrinology and reproductive health. In addition, EMD Serono has an enduring commitment to solve the unsolvable, with state-of-the-art science dedicated to developing new therapies in the core focus areas of neurology, oncology, immune oncology and immunology. With a long-standing history of industry expertise and a dedication to shape the future of healthcare, the companys US footprint continues to grow, with approximately 1,000 employees around the country and fully integrated commercial, clinical and research operations in its home state, Massachusetts. For more information, please visit http://www.emdserono.com.

Open Monoclonal Technology, Inc.

Open Monoclonal Technology, Inc. (OMT) is a leader in genetic engineering of animals for discovery of human therapeutic antibodies naturally optimized human antibodies.

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EMD Serono and Open Monoclonal Technology Expand Collaboration to Include Unlimited Access to OmniRat® Human Antibody ...

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SALT LAKE CITY, Oct. 28, 2013 (GLOBE NEWSWIRE) -- Myriad Genetics, Inc. (MYGN) today announced that validation data for the Myriad myPlan Lung Cancer test showed that it significantly predicted patients' risk of death from early-stage lung adenocarcinoma within five years of being diagnosed. A key finding of the validation study is that patients with a high-risk myPlan prognostic score had approximately twice the number of lung cancer related deaths compared to patients with a low-risk score.

Myriad is presenting these results and another myPlan clinical study this week at the International Association for the Study of Lung Cancer (IASLC) 15th World Conference on Lung Cancer in Sydney, Australia. myPlan Lung Cancer is the only prognostic test on the market today that can assess a patient's risk of lung cancer mortality and thereby guide the physician in determining which patients may benefit from post-surgical chemotherapy.

"The Myriad myPlan Lung Cancer test provides new information that can identify people at high risk of lung cancer recurrence and will enable us to offer these patients additional therapy to improve their survival," said Raphael Bueno, M.D., Associate Chief of Thoracic Surgery at Brigham and Women's Hospital and Harvard Medical School. "Once we know a patient's risk profile, we can align our clinical management strategies to achieve better medical outcomes and save more lives."

The two studies being presented at the IASLC annual meeting are:

Validation of a Proliferation-based Expression Signature as Prognostic Marker in Early Stage Lung Adenocarcinoma. [Bueno et al., Poster: Oct. 28, 2013, 3:30 p.m. -- 4:15 p.m. AEDT]

This study evaluated the association of cell cycle progression (CCP) genes with five-year lung cancer mortality in 650 patients diagnosed with stage 1 or 2 lung adenocarcinoma. In this clinical validation study, patients with a high-risk Myriad myPlan Lung Cancer prognostic score had a significantly higher average five-year mortality rate (35 percent) than patients with a low-risk score (18 percent). The prognostic score (CCP + tumor stage) segregated stage 1A to 2B patients with five-year risk estimates ranging from 11 percent to 68 percent. This study demonstrated that the myPlan Lung Cancer prognostic score was statistically significant in predicting five-year lung cancer mortality, and was significantly more predictive than tumor staging information alone. These data strongly support the use of the Myriad myPlan Lung Cancer test to help stratify patients' risk profiles and determine which patients are candidates for conservative management with surgery alone versus more intensive therapy based on a higher personal risk profile.

Integrated Prognosis in Early-Stage Resectable Lung Adenocarcinoma. [Kim et al., Poster: Oct. 30, 2013, 3:30 p.m. -- 4:15 p.m. AEDT]

This study evaluated a prognostic model that combines a cell cycle progression (CCP) score with tumor stage in order to maximize the prognostic utility of both predictive markers. The primary outcome measure was cancer specific death within five years of surgery. The results for this study show that both tumor stage and CCP score are statistically significant independent predictors of lung cancer death. However, the combination of tumor stage and the CCP expression score was a more powerful predictor of post-surgical risk of cancer-specific death than tumor stage alone. Myriad myPlan Lung Cancer is a prognostic test that combines CCP score and tumor stage and offers clinicians a more precise risk assessment of patients with early-stage lung cancer, which may improve medical management of these patients.

About Myriad Genetics

Myriad Genetics is a leading molecular diagnostic company dedicated to making a difference in patients' lives through the discovery and commercialization of transformative tests to assess a person's risk of developing disease, guide treatment decisions and assess risk of disease progression and recurrence. Myriad's molecular diagnostic tests are based on an understanding of the role genes play in human disease and were developed with a commitment to improving an individual's decision making process for monitoring and treating disease. Myriad is focused on strategic directives to introduce new products, including companion diagnostics, as well as expanding internationally. For more information on how Myriad is making a difference, please visit the Company's website: http://www.myriad.com.

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Myriad myPlan(TM) Lung Cancer Test Meets Primary Endpoint in Validation Study

LOS ANGELES--(BUSINESS WIRE)--

Life Stem Genetics Inc., an emerging innovator in the advancement of Adult Stem Cell therapy, is pleased to announce that Matthew Sullivan, COO of global heath products company Asana International, and Shahab Bakhtyar, an independent medical business consultant with global experience, have joined Life Stems Executive Advisory Board.

Established earlier this year, the Executive Advisory Board has become a cornerstone of Life Stems global stem-cell growth model by actively retaining influential business leaders with broad backgrounds in corporate development and finance in our targeted industries with a focus on business expansion into Canada, Europe, Asia, and the Middle East.

Matthew Sullivan, BA, MBA, is a corporate finance specialist who has worked with numerous early-stage and well-established companies in operations as well as strategic and financial planning. Matthews background is in venture capital and business analysis. His role has ranged from business planning/implementation, M&A, market analysis to operational implementation. He is currently COO of Asana International, a global health products company, CFO of Kat Gold Holdings, a publicly traded gold production and exploration company based in Ghana, and CFO of Travelvu, a business that places smart devices in hotel rooms. Matthew holds a Bachelor of Arts degree from the University of British Columbia, Canada, and an MBA from Dalhousie University, Canada.

Shahab Bakhtyar, MBA, is an independent business consultant with a focus on expansion and financing of small to medium sized businesses. In the last 15 years, Mr. Bakhtyars consultancy has focused mainly on Western Canada where he includes medical/health service providers among his clientele. Prior to establishing his business in Canada, Mr. Bakhtyar worked in Dubai, UAE. Included among his clients were Emirate Air and FIFA to whom he provided marketing and advertising services. Mr. Bakhtyar holds an MBA from Queens University, Canada. He has traveled to over 63 countries and maintains a strong international network of business contacts with a focus throughout the Middle East and Canada.

The addition of Matthew and Shahab to our advisory board aligns with our goal of developing a team of advisors who share our core values and can help us attain our growth initiatives in the rapidly advancing Adult Stem Cell therapy business sector, says Gloria Simov, president and CEO of Life Stem Genetics. Life Stem will greatly benefit from the depth of industry expertise and overall business acumen that both Matthew and Shahab bring, and we look forward to their value-added contributions as we continue to build our base of stem cell clinics throughout the world.

About Life Stem Genetics

Life Stem Genetics (LSG) is a progressive healthcare company focused on Adult Stem Cell (ASC) healing therapies. For decades, stem cells have been utilized in the successful treatment of a variety of ailments. Today, advanced ASC therapies are being offered to patients as an efficient and painless alternative treatment for a wide range of ailments including, but not limited to, orthopedic injuries, neurological disorders such as Parkinsons and Alzheimers, cancer, arthritis, diabetes, multiple sclerosis, as well as age management. Adult Stem Cell therapies and LSGs proprietary techniques are experiencing some of the best results in the industry in helping to repair or reprogram damaged or diseased tissues and organs. Life Stems ASC specialist has performed thousands of stem cell treatments including some of the top names in PGA golf, NFL football, NBA basketball, and Major League Baseball. LSG will offer its proprietary treatments through a series of affiliate doctors and medical clinics with 60 affiliated clinics thus far. LSGs mission is to develop a comprehensive approach to the treatment and maintenance of diseases while breaking free from the medical insurance maze by tapping into an affordable private-pay sector.

Contrarian Press, the publisher, has been engaged by Life Stem Genetics to assist with identification of potential market participants who may be interested in learning more about the company and its securities. Updated disclaimer and disclosure information is available at the publisher's website listed above and at the following link:

http://www.contrarianwealthcoalition.com/guide/LIFS.pdf

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Life Stem Genetics Strengthens Its Executive Advisory Board by Adding MBAs Matthew Sullivan and Shahab Bakhtyar

RUTHERFORD, N.J., Oct. 28, 2013 (GLOBE NEWSWIRE) -- Cancer Genetics, Inc. (CGIX), a diagnostics company focused on developing genomic-based, oncology tests and services, today announced the closing of its previously announced public offering of 3,286,700 shares of its common stock (including 428,700 shares that were offered and sold by Cancer Genetics pursuant to the exercise in full of the underwriters' over-allotment option) at a price to the public of $14.00 per share. The gross proceeds to Cancer Genetics from the offering were $46 million, before underwriting discounts and commissions and other offering expenses payable by Cancer Genetics.

Aegis Capital Corp. acted as sole book-running manager for the offering.

Feltl and Company, Inc., Cantor Fitzgerald & Co. and Dougherty & Company acted as co-managers for the offering.

This offering was made only by means of a prospectus.

A copy of the prospectus relating to this offering may be obtained by contacting Aegis Capital Corp., Prospectus Department, 810 Seventh Avenue, 18th Floor, New York, NY 10019, telephone: 212-813-1010, e-mail: prospectus@aegiscap.com.

A registration statement relating to these securities was declared effective by the Securities and Exchange Commission on October 22, 2013. This press release shall not constitute an offer to sell or a solicitation of an offer to buy, nor shall there be any sale of these securities in any state or jurisdiction in which such an offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.

About Cancer Genetics:

Cancer Genetics, Inc. is an emerging leader in DNA-based cancer diagnostics and services some of the most prestigious medical institutions in the world. Our tests target cancers that are difficult to diagnose and predict treatment outcomes. These cancers include hematological, urogenital and HPV-associated cancers. We also offer a comprehensive range of non-proprietary oncology-focused tests and laboratory services that provide critical genomic information to healthcare professionals as well as biopharma and biotech. Our state-of-the-art reference lab is focused entirely on maintaining clinical excellence and is both CLIA certified and CAP accredited and has licensure from several states including New York State. We have established strong research collaborations with major cancer centers such as Memorial Sloan-Kettering, The Cleveland Clinic, Mayo Clinic and the National Cancer Institute. For further information, please see http://www.cancergenetics.com.

Forward Looking Statements:

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements pertaining to future financial and/or operating results, future growth in research, technology, clinical development and potential opportunities for Cancer Genetics, Inc. products and services, along with other statements about the future expectations, beliefs, goals, plans, or prospects expressed by management constitute forward-looking statements. In addition, the offering is subject to market and other conditions and there can be no assurance as to the estimated proceeds from the offering and the anticipated use of proceeds from the offering. Any statements that are not historical fact (including, but not limited to, statements that contain words such as "will," "believes," "plans," "anticipates," "expects," "estimates") should also be considered to be forward-looking statements. Forward-looking statements involve risks and uncertainties, including, without limitation, risks inherent in the development and/or commercialization of potential products, uncertainty in the results of clinical trials or regulatory approvals, need and ability to obtain future capital, and maintenance of intellectual property rights and other risks discussed in the Company's Form 10-Q for the quarter ended June 30, 2013 and other filings with the Securities and Exchange Commission. These forward-looking statements speak only as of the date hereof. Cancer Genetics disclaims any obligation to update these forward-looking statements.

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Cancer Genetics Announces Closing of Public Offering of $46 Million of Shares of Common Stock

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