Archive for the ‘Gene Therapy Research’ Category

Submission to Governing Authorities? - Chuck Missler
In this segment Chuck Missler discusses submission to governing authorities. This segment comes from the "Timothy" commentary published by Koinonia House. - ...

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Genetic Engineering: Artificial Selection to Designer Babies. What Does It Mean?
SOURCE: http://www.sovereignelementinformation.wordpress.com/2012/12/06/genetic-engineering-artificial-selection-to-designer-babies-what-does-it-mean/ http:/...

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AP Lang -- Opposing Genetic Engineering
AP Lang Opposing Genetic Engineering. angelbratsnats09873 videos. Subscribe Subscribed Unsubscribe 23. 12 views. Like 0 Dislike 0. Like. Sign in to youtube. Sign in with your youtube Accountyoutube Google+ Gmail Orkut Picasa or Chrome to like angelbratsnats0987s video. Sign in. I dislike this. Sign in to youtube. Sign in with your youtube Accountyoutube Google+ Gmail Orkut Picasa or Chrome to dislike angelbratsnats0987s video. Sign in. About Share Add to. Sign in to youtube. Sign in with your ...

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Whole Foods to Provide Labels on Genetically Modified Products
Blinding snow and fierce winds create a travel nightmare across country.

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Genetic Engineering Stop Animation
Genetic Engineering Stop Animation. Uploaded by Lucy Skilling on Mar 12 2013. Skills 2708.

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Public release date: 14-Mar-2013 [ | E-mail | Share ]

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

New Rochelle, NY, March 14, 2013A woman's health status before pregnancy is critical for the health and wellbeing of the fetus and mother-to-be. The U.S. Centers for Disease Control and Prevention (CDC) has set Healthy People 2020 national objectives for women of reproductive age, and young women are making important gains toward achieving some of those health goals, while some trends are less encouraging, as reported in a study published in Journal of Women's Health, a peer-reviewed publication from Mary Ann Liebert, Inc., publishers. The article is available free on the Journal of Women's Health website at http://www.liebertpub.com/jwh.

Pamela Xaverius, PhD and Joanne Salas, MPH, Saint Louis University School of Public Health and School of Medicine, MO, report substantial reductions in smoking and alcohol consumption (including drinking any alcohol and heavy drinking) among women in the U.S. ages 18-44 years. The authors analyzed data on preconception health indicators from over 500,000 women from all 50 states in the U.S. gathered between 2003-2010 from the Behavioral Risk Factor Surveillance System.

In the article "Surveillance of Preconception Health Indicators in Behavioral Risk Factor Surveillance System: Emerging Trends in the 21st Century," they also describe positive preconception health trends related to moderate or vigorous physical activity and a 68% increase in women having an influenza shot within the previous year. Health trends that have worsened and pose a potential threat to maternal and fetal health included binge alcohol drinking and having a chronic medical condition (e.g., diabetes, high blood pressure, asthma, or obesity).

"While the trends in smoking, alcohol use, and influenza prevention have improved, the worsening in binge drinking and chronic medical conditions among reproductive aged women are important concerns," says Susan G. Kornstein, MD, Editor-in-Chief of Journal of Women's Health, Executive Director of the Virginia Commonwealth University Institute for Women's Health, Richmond, VA, and President of the Academy of Women's Health.

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About the Journal

Journal of Women's Health, published monthly, is a core multidisciplinary journal dedicated to the diseases and conditions that hold greater risk for or are more prevalent among women, as well as diseases that present differently in women. The Journal covers the latest advances and clinical applications of new diagnostic procedures and therapeutic protocols for the prevention and management of women's healthcare issues. Complete tables of content and a sample issue may be viewed on the Journal of Women's Health website at http://www.liebertpub.com/jwh. Journal of Women's Health is the Official Journal of the Academy of Women's Health and the Society for Women's Health Research.

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Big improvements in preconception health trends among women of reproductive age reported

Public release date: 14-Mar-2013 [ | E-mail | Share ]

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

New Rochelle, NY, March 14, 2013Severe chronic pain associated with conditions such as bladder pain syndrome/interstitial cystitis often require the use of opioid medication, with the risk of dependency and serious adverse reactions. An alternative treatment strategy increases the levels of a naturally occurring painkiller in and around the nerves that deliver pain signals to the bladder. This new therapeutic approach is described in an article in Human Gene Therapy, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers. The article is available free on the Human Gene Therapy website at http://www.liebertpub.com/hum.

Hitoshi Yokoyama, MD and colleagues from University of Pittsburgh School of Medicine (PA), Shinshu University School of Medicine (Matsumoto, Japan), and Diamyd (Pittsburgh, PA) describe a gene therapy technique in which they inject directly into the bladder wall the gene for enkephalin, an opioid compound produced by the human body. The gene is transported into the target cells via a herpes simplex virus vector that is incapable of replication.

In the article "Effects of Herpes Simplex Virus Vector-Mediated Enkephalin Gene Therapy on Bladder Overactivity and Nociception," the authors demonstrate high levels of enkephalin gene expression in the treated rats and significantly lower measures of pain compared to untreated animals when exposed to stimuli intended to induce bladder irritation. The researchers note that a similar gene therapy delivery vector carrying an enkephalin gene has been used in clinical studies in human patients to treat cancer-related pain, and was shown to be well tolerated and safe and to provide substantial pain relief.

"This is a very innovative application of Herpes Simplex Virus gene therapy in the treatment of a common and painful clinical problem that otherwise requires chronic use of narcotics," says James M. Wilson, MD, PhD, Editor-in-Chief, and Director of the Gene Therapy Program, Department of Pathology and Laboratory Medicine, University of Pennsylvania Perelman School of Medicine, Philadelphia.

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About the Journal

Human Gene Therapy, the Official Journal of the European Society of Gene and Cell Therapy, British Society for Gene and Cell Therapy, French Society of Cell and Gene Therapy, German Society of Gene Therapy, and five other gene therapy societies, is an authoritative peer-reviewed journal published monthly in print and online. Human Gene Therapy presents reports on the transfer and expression of genes in mammals, including humans. Related topics include improvements in vector development, delivery systems, and animal models, particularly in the areas of cancer, heart disease, viral disease, genetic disease, and neurological disease, as well as ethical, legal, and regulatory issues related to the gene transfer in humans. Its sister journal, Human Gene Therapy Methods, published bimonthly, focuses on the application of gene therapy to product testing and development, and Human Gene Therapy Clinical Development, launching in 2013, publishes data relevant to the regulatory review and commercial development of cell and gene therapy products. Tables of content and a sample issue may be viewed on the Human Gene Therapy website at http://www.liebertpub.com/hum.

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Novel treatment approach for bladder pain using a herpes simplex virus vector reported

Home business Two Thais make it to WEF Young Global Leaders list

The Nation March 14, 2013 1:00 am

The young leaders from Myanmar are Thura Ko, founder and managing director of YGA Capital, and Win Win Tint, managing director of City Mart Holdings Co.

Like his father, MR Disnadda Diskul, chief of Mae Fa Luang Foundation, Dispanadda has inherited

the urge to help poor farmers. His project's main objective is to help people in the

farming community and raise their in-

come through the shared ownership of a brand. The project now owns Doi Tung Coffee brand. Although the brand has

not yet spread to global markets, the project has considerable accomplishments domestically.

At an event hosted by Sasin Graduate School, he said success, for a social enterprise, lies in its ability to deliver what the locals need. "We're not giving what they're not ready to take. Many knowledge programmes turn to waste if they don't match the needs [of the people involved]," he said, citing the experience of projects under the Mae Fah Luang Foundation.

According to the Global Young Academy, which is the voice of young scientists around the world, Nitsara obtained a bachelor's degree in chemical engineering from Columbia University in 1999 and master's and doctoral degrees in chemical engineering from Stanford University in 2004. She leads a research team at Biotec in employing microarray technology to study black tiger shrimp, an economically important export for Thailand, and develop antibody arrays for diagnostics.

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Two Thais make it to WEF Young Global Leaders list

BIOFAB researchers have produced high quality standardized public domain DNA sequences for genetic engineering.

(Phys.org) Predictability is often used synonymously with "boring," as in that story or that outcome was soooo predictable. For practitioners of synthetic biology seeking to engineer valuable new microbes, however, predictability is the brass ring that must be captured. Researchers with the multi-institutional partnership known as BIOFAB have become the first to grab at least a portion of this ring by unveiling a package of public domain DNA sequences and statistical models that greatly increase the reliability and precision by which biological systems can be engineered.

The DNA sequences produced by BIOFAB provide precise control of gene expression in Escherichia coli, the rod-shaped bacterium that is one of the principal model organisms for genetic engineering. While these DNA sequences serve as standardized parts specific to E. coli, they also provide a set of rules for how the sequences fit together that should apply to other microbes as well. Controlling the expression of genes is essential for engineering a microbe to produce a specific product or carry out a specific function.

As BIOFAB co-director Adam Arkin, a computational biologist and director of Berkeley Lab's Physical Biosciences Division, has noted, "Fulfilling the great promise of synthetic biology hinges on making the design and construction of biological systems as predictable as the assembly of computer hardware."

Yet even with a microbe as well-characterized and understood as E. coli, the introduction of new genes has in the past been met with as much failure as success.

"You would think after a generation of genetic engineering, expressing genes with precision in an organism as well utilized as E. coli would be pretty straightforward but it's not," says BIOFAB's other co-director Drew Endy, a synthetic biologist at Stanford University.

Arkin and Endy are the corresponding authors of a pair of research papers published simultaneously in the journal Nature Methods, and a third to be published in Nucleic Acids Research, that collectively describe this major BIOFAB breakthrough.

BIOFAB's success is based on two major achievements the design of independent DNA promoter and ribosome binding site sequences for specific E. coli genes, and the development of mathematical models that provide rules for engineering gene expression that should be applicable to nearly all organisms. This work establishes a much-needed technological foundation for the field of synthetic biology, which in turn should facilitate more precise and predictable genetic engineering in the future.

BIOFAB was established in December, 2009 under a grant from the National Science Foundation as "the world's first biological design-build facility." Led by the University of California Berkeley and Stanford University, BIOFAB is operated in partnership with Berkeley Lab, the BioBricks Foundation and the Synthetic Biology Engineering Research Center.

More information: "Quantitative estimation of activity and quality for collections of functional genetic elements." Vivek K Mutalik, Joao C Guimaraes, Guillaume Cambray, Quynh-Anh Mai, Marc Juul Christoffersen, Lance Martin, Ayumi Yu, Colin Lam, Cesar Rodriguez, Gaymon Bennett, Jay D Keasling, Drew Endy & Adam P Arkin, 10 March 2013, Nature Methods. doi:10.1038/nmeth.2403

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Predictability: The brass ring for synthetic biology

PGM Second Lesson, First Swings
PGM Second Lesson, First Swings.

By: Mary Hashagen

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PGM Second Lesson, First Swings - Video

on TreasON
A Bushwackk/Heretic production of Cicero #39;s iconic speech. "A nation can survive its fools, and even the ambitious. But it cannot survive treason from within....

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on TreasON - Video

Published: Thursday, March 14, 2013 at 5:13 p.m. Last Modified: Thursday, March 14, 2013 at 5:13 p.m.

That some people are genetically prone to addictions is nothing new, but some scientists have expressed surprise at the degree of addictive tendencies.

For example, if you have a high-risk genotype for marijuana addiction, and also suffer from neuroticism or anxiety, you have an eight- to nine-fold risk of becoming addicted to marijuana.

What's more, in utero exposure to the drug might induce long-term addictive tendencies -- at least in rats.

Professor Yasmin Hurd, a neuroscientist and professor of psychiatry at the Ichan School of Medicine in Mount Sinai, N.Y., cited these examples Thursday in a talk titled "The Vulnerable Brain: Understanding the Neurobiology of Addiction Risk." Her talk was one of two expert lectures delivered Thursday at the McKnight Brain Institute as part of brain awareness week, a global campaign to raise awareness of the brain.

Early addiction research in neuroscience focused on dopamine dependence -- the "feel-good" chemical released abundantly in the brain during drug usage. The flip side of this immediate surge is a long-term lessening of dopamine actually produced by the brain, which over time, decreases a person's ability to experience pleasure.

But scientists realized addiction in the brain was "much more complicated" than decoding dopamine circuitry, so they began looking at genetic mutations that might play a role.

"Clearly there is not one gene that makes someone a heroin abuser," Hurd said, adding, "Genetics has an important for understanding the vulnerability of heroin abusers."

Hurd noted that marijuana is the most widely abused drug in the U.S., while heroin and cocaine are the most addictive. Heroin overdose has the highest mortality rates of any drug.

One issue that interests Hurd is the effect of marijuana use by pregnant women on their babies. The scientists found that men were more vulnerable to addictions than women. Other studies have shown that people -- especially men -- with certain behavioral traits such as anxiety, when combined with a genetic vulnerability to addiction, or a mother who smoked in utero, were especially at risk.

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Professor: Genetic mutations can amplify drug addictions

Man #39;s DNA Test Rocks Human Genetic Theories
The research means our Y-chromosome tree is much longer than geneticists believed mdash;and that our paternal lineage is more than twice as old as we once thought.

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Man's DNA Test Rocks Human Genetic Theories - Video

MinuteEarth: The Story of Our Planet
Subscribe to MinuteEarth - it #39;s FREE! - http://dft.ba/-minuteearth_sub Agriculture, hula hoops, SARS, and THIS video: how long did they take to get around th...

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Savage - Only You (Harmless Project Remix)
Dar viena puikiai #382;inoma daina, kuri atgaivinta nauju skambesiu. Geriausios kokyb #279;s muzik #261; rasite #269;ia, prenumeruokite ir dalinkit #279;s! Nuotrauka rasite: http:/...

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Savage - Only You (Harmless Project Remix) - Video

How Mendel #39;s pea plants helped us understand genetics - Hortensia Jiménez Díaz
View full lesson: http://ed.ted.com/lessons/how-mendel-s-pea-plants-helped-us-understand-genetics-hortensia-jimenez-diaz Each father and mother pass down tra...

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How Mendel's pea plants helped us understand genetics - Hortensia Jiménez Díaz - Video

HUNTED BY MARILYN MANSON (The Hidden)
Enjoy the video? Subscribe! http://bit.ly/M0mU1V #9669; #9669; #9669; Download Here: http://www.hidden-source.com What is The Hidden? "In the early 1950s human genetics e...

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HUNTED BY MARILYN MANSON (The Hidden) - Video

Newswise BETHESDA, MD March 13, 2013 Genetics and life sciences instructors, who teach undergraduate students about population and evolutionary genetics, have a new teaching resource: the March 2013 Primer in the Genetics Society of Americas journal GENETICS uses current research on transcriptome divergence in two closely related species of field crickets to explain population genetics.

The Primer, Population Genetics and a Study of Speciation Using Next-Generation Sequencing, by Patricia J. Wittkopp, Ph.D., a professor at the University of Michigan, explains how undergraduate instructors can, in their classrooms, use the article, Patterns of Transcriptome Divergence in the Male Accessory Gland of Two Closely Related Species of Field Crickets by Andrs et al., published in the February 2013 issue of GENETICS.

The Primer details background information on the Gryllus firmus and Gryllus pennsylvanicus cricket systems and the use of transcriptome sequence variation to study speciation. Dr. Wittkopp provides cogent explanations of the sequencing technologies used as well as some of the results of the paper, but leaves most of the results for students to interpret on their own. To give students the tools they need to interpret the data, Dr. Wittkopp provides a concise and accessible overview of the necessary genetics concepts on which the research of Andrs et al. is based. For instructors, Dr. Wittkopp provides guidance on how to use the primary literature in the classroom as well as questions for student discussion.

By focusing on contemporary scientific literature, students engage in the learning process and are encouraged to make their own scientific discoveries, said Elizabeth A. De Stasio, Ph.D., a professor at Lawrence University in Appleton, Wisconsin, and editor of the Primer section in the Genetics Society of Americas journal, GENETICS.

Primers are scheduled for the April, May, and June issues of GENETICS. Providing valuable educational resources like this, which enhance the quality of genetics education, teaching and learning, is a mission of GSA, said Mark Johnston, Ph.D., Editor-in-Chief of GENETICS. These articles help educators engage students in critically analyzing current primary research, a vital part of research training.

CITATION: Patricia J. Wittkopp. Population Genetics and a Study of Speciation Using Next Generation Sequencing: An Educational Primer for Use with Patterns of Transciptome Divergence in the Male Accessory Gland of Two Closely Related Species of Field Crickets GENETICS, March 2013, Volume 193, Number 3, 671-675.

ABOUT GENETICS Primers: Primers are designed to bring cutting-edge scientific research into the classroom by making scientific papers accessible to undergraduate students and their instructors. A Primer is intended to be used with the research article, which is published in the same or a recent issue of GENETICS. Primers include topic background, explanation of genetics concepts, suggestions for using the article in the classroom, and questions for classroom discussion. The articles give instructors the opportunity to enliven student interest in genetics by teaching genetics principles in the context of current research.

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

ABOUT GSA: Founded in 1931, the Genetics Society of America (GSA) is the professional membership organization for scientific researchers, educators, bioengineers, bioinformaticians and others interested in the field of genetics. Its nearly 5,000 members work to advance knowledge in the basic mechanisms of inheritance, from the molecular to the population level. The GSA is dedicated to promoting research in genetics and to facilitating communication among geneticists worldwide through its conferences, including the biennial conference on Model Organisms to Human Biology, an interdisciplinary meeting on current and cutting edge topics in genetics research, as well as annual and biennial meetings that focus on the genetics of particular organisms, including C. elegans, Drosophila, fungi, mice, yeast, and zebrafish. GSA publishes GENETICS, a leading journal in the field and an online, open-access journal, G3: Genes|Genomes|Genetics. For more information about GSA, please visit http://www.genetics-gsa.org. Also follow GSA on Facebook at facebook.com/GeneticsGSA and on Twitter @GeneticsGSA.

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Education Resource Teaches Population Genetics Using Current Research

Public release date: 13-Mar-2013 [ | E-mail | Share ]

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

BETHESDA, MD March 13, 2013 Genetics and life sciences instructors, who teach undergraduate students about population and evolutionary genetics, have a new teaching resource: the March 2013 Primer in the Genetics Society of America's journal GENETICS uses current research on transcriptome divergence in two closely related species of field crickets to explain population genetics.

The Primer, "Population Genetics and a Study of Speciation Using Next-Generation Sequencing," by Patricia J. Wittkopp, Ph.D., a professor at the University of Michigan, explains how undergraduate instructors can, in their classrooms, use the article, "Patterns of Transcriptome Divergence in the Male Accessory Gland of Two Closely Related Species of Field Crickets" by Andrs et al., published in the February 2013 issue of GENETICS.

The Primer details background information on the Gryllus firmus and Gryllus pennsylvanicus cricket systems and the use of transcriptome sequence variation to study speciation. Dr. Wittkopp provides cogent explanations of the sequencing technologies used as well as some of the results of the paper, but leaves most of the results for students to interpret on their own. To give students the tools they need to interpret the data, Dr. Wittkopp provides a concise and accessible overview of the necessary genetics concepts on which the research of Andrs et al. is based. For instructors, Dr. Wittkopp provides guidance on how to use the primary literature in the classroom as well as questions for student discussion.

"By focusing on contemporary scientific literature, students engage in the learning process and are encouraged to make their own scientific discoveries," said Elizabeth A. De Stasio, Ph.D., a professor at Lawrence University in Appleton, Wisconsin, and editor of the Primer section in the Genetics Society of America's journal, GENETICS.

Primers are scheduled for the April, May, and June issues of GENETICS. "Providing valuable educational resources like this, which enhance the quality of genetics education, teaching and learning, is a mission of GSA," said Mark Johnston, Ph.D., Editor-in-Chief of GENETICS. "These articles help educators engage students in critically analyzing current primary research, a vital part of research training."

###

CITATION: Patricia J. Wittkopp. Population Genetics and a Study of Speciation Using Next Generation Sequencing: An Educational Primer for Use with "Patterns of Transciptome Divergence in the Male Accessory Gland of Two Closely Related Species of Field Crickets" GENETICS, March 2013, Volume 193, Number 3, 671-675.

ABOUT GENETICS Primers: Primers are designed to bring cutting-edge scientific research into the classroom by making scientific papers accessible to undergraduate students and their instructors. A Primer is intended to be used with the research article, which is published in the same or a recent issue of GENETICS. Primers include topic background, explanation of genetics concepts, suggestions for using the article in the classroom, and questions for classroom discussion. The articles give instructors the opportunity to enliven student interest in genetics by teaching genetics principles in the context of current research.

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Education resource focuses on teaching population genetics using current research

SEATTLE, WA--(Marketwire - Mar 13, 2013) - Atossa Genetics, Inc. ( NASDAQ : ATOS ), The Breast Health Company, has entered into a contractual agreement with FedMed, Inc., one of the largest proprietary Preferred Provider Organization (PPO) networks in the U.S., for diagnostic laboratory testing. FedMed's network is comprised of more than 550,000 providers, including 4,000 hospitals and more than 60,000 ancillary facilities, serving over 40 million Americans.

Atossa's agreement with FedMed will give FedMed's participating providers and its clients' members greater access to Atossa's tests, including the ForeCYTE Breast Health Test and the ArgusCYTE Breast Health Test.

"There is a significant unmet clinical need in the medical community for more effective ways to identify women at high risk of breast cancer," stated Steven C. Quay, M.D., Ph.D., FCAP, Chairman, CEO & President of Atossa Genetics. "Our agreement with FedMed will help ensure that more doctors and their patients have access to the ForeCYTE Breast Health Test, a risk stratification test akin to the cervical Pap smear, and the ArgusCYTE Breast Health Test, a blood test for recurrence targeted at the 3.2 million breast cancer survivors in the U.S."

Dr. Quay added, "At Atossa, our goal is to help physicians manage their patients' breast health by offering a suite of products and services that address the most pressing clinical decisions. Armed with better information, physicians will be able to customize individualized treatment plans to reduce women's risk of breast cancer or cancer recurrence, to improve patient therapeutic compliance and ultimately to decrease the overall cost of care."

About Atossa Genetics, Inc.

Atossa Genetics, Inc. ( NASDAQ : ATOS ), The Breast Health Company, is based in Seattle, WA, and is focused on preventing breast cancer through the commercialization of patented diagnostic medical devices and patented, laboratory developed tests (LDT) that can detect precursors to breast cancer up to eight years before mammography, and through research and development that will permit it to commercialize treatments for pre-cancerous lesions.

The National Reference Laboratory for Breast Health (NRLBH), a wholly owned subsidiary of Atossa Genetics, Inc., is a CLIA-certified high-complexity molecular diagnostic laboratory located in Seattle, WA, that provides the patented ForeCYTE Breast Health Test and the ArgusCYTE Breast Health Test.

About the ForeCYTE Breast Health Test

The ForeCYTE Breast Health Test provides personalized information about the 10-year and lifetime risk of breast cancer for women between ages 18 and 73. It involves collecting a specimen of nipple aspirate fluid, or NAF, using our patented Mammary Aspirate Specimen Cytology Test, or MASCT, System. The NAF specimen is collected by a physician and returned to our CLIA-certified laboratory. We study the patient's NAF specimen and use a proprietary molecular and cellular biomarker test that detects basal or luminal cells to identify the presence of atypical ductal hyperplasia, or ADH, which is considered a precursor to breast cancer.If ADH is detected, steps can be taken to reduce the risk of breast cancer.

About the ArgusCYTE Breast Health Test

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Atossa Genetics Announces National Agreement With Network Provider FedMed

By Randy Dotinga HealthDay Reporter

THURSDAY, March 14 (HealthDay News) -- In a very early sign of medical progress on the osteoarthritis front, scientists report they've used injections of modified genes to reduce the risk that mice will develop the painful, debilitating condition.

There's no way to know if the gene therapy treatment will help humans, and scientists are far from understanding the treatment's side effects and potential cost. But the findings are more than just good news for mice with creaky joints.

"This work identifies an approach that can make a difference," explained study co-author Dr. Brendan Lee, director of the Rolanette and Berdon Lawrence Bone Disease Program of Texas. "There's a great need for treating and preventing osteoarthritis."

The disease, the most common form of arthritis, appears as your joints deteriorate with aging. It often strikes the hands, knees, neck and hips, causing pain, stiffness and difficulty moving.

Seventy percent of Americans aged 55 to 70 struggle with osteoarthritis, for which there is no cure. Doctors try to treat the pain and improve the ability of patients to move, Lee said, and may turn to joint replacement surgeries in advanced cases.

In the new study, researchers examined a protein that diminishes in people with a rare joint disorder. The protein appears to be crucial to the lubrication of joints.

Researchers injected a gene related to the protein into mice and found that the rodent bodies began producing it. The mice appeared to be resistant -- but not immune -- to damage to the cartilage of joints from injury and aging, Lee said.

There are plenty of caveats.

The research is in mice, not humans; the next step is to test the approach in horses, whose joints are similar to those of people. And the gene therapy doesn't seem to do anything for damage that's already occurred.

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Gene therapy helped mice withstand osteoarthritis

ELK GROVE VILLAGE, Ill., March 13, 2013 (GLOBE NEWSWIRE) -- Nationwide Children's Hospital (Columbus, OH) and Families of Spinal Muscular Atrophy (Elk Grove Village, IL) announce the award of a multi-million dollar cooperative agreement from the National Institute of Neurological Disorders and Stroke (NINDS) to advance a gene therapy development program for Spinal Muscular Atrophy (SMA).

This three-year multi-million dollar cooperative agreement to Brian Kaspar, PhD, principal investigator in the Center for Gene Therapy at The Research Institute at Nationwide Children's Hospital in the amount of $3,752,462, funds pre-clinical drug development up to the filing of an Investigational New Drug Application (IND) to the Food and Drug Administration (FDA). This agreement represents an innovative collaboration between Government, Advocacy and Academic groups to advance a promising new therapy for SMA.

In May 2012, Families of SMA (FSMA) announced the award of up to $750,000 to Dr. Kaspar. This ongoing award supports the preclinical development of a Central Nervous System (CNS)-delivered gene therapy for SMA. Direct CNS delivery likely allows for less virus to be used, which significantly increases the likelihood that older and larger SMA patients can be treated with gene therapy. With the funding from FSMA, Dr. Kaspar's team initiated studies to jumpstart the research prior to obtaining government and later commercial involvement. This cooperative award from the NINDS will now support advancing the program to the point of human clinical trials. The program will be evaluated using quantitative go/no-go milestones, determined by Nationwide Children's and NINDS.

SMA is an often-fatal genetic disorder resulting from the loss of both copies of the Survival Motor Neuron (SMN1) gene. This causes a chronic deficiency in the production of the SMN protein, which is essential to the proper functioning of the motor neurons in the spinal cord to the control of muscles in the limbs, neck and chest. SMA is typically marked by the deterioration of the muscles that control crawling, walking, swallowing or breathing. There are no approved therapies for the treatment of SMA. Approximately 1 in 6,000 babies born is affected. One in 40 people, or approximately 8 million in the United States, are genetic carriers of the disease.

Gene therapy is an approach to treating diseases by replacing faulty genes. In the case of SMA, the most direct approach for a gene therapy is to replace the mutated SMN1 gene. In the past, the challenge with gene therapy for SMA has been to find a way to deliver the genetic material efficiently to motor neurons. In recent years, Dr. Kaspar's group was the first to demonstrate Adeno-Associated Virus 9 (AAV9) targeted motor neurons effectively. Administration of AAV9-SMN into one day-old SMA mice resulted in increased SMN protein levels in motor neurons, correction of synaptic function, and a significant extension of life span.

"At Families of SMA we are extremely pleased that our initial investment at an early stage of this program has provided the preliminary data to leverage larger funding from the NIH. We feel this grant award is positive validation of the Families of SMA research funding and partnering strategy, as well as for this approach for gene therapy in SMA," said Jill Jarecki PhD, Research Director at Families of SMA. "The Families of SMA funding strategy for preclinical drug development is to invest seed funds to begin early-stage programs for SMA. As programs advance, we look for funding to transition from non-profit to government and commercial sources."

"My research team at Nationwide Children's Hospital is excited to advance this promising cerebrospinal fluid delivery approach of AAV9-SMN to the clinic for SMA patients and we are extremely grateful to FSMA and NINDS for the support of this important work," said Dr. Kaspar, also a faculty member at The Ohio State University College of Medicine. "We stand committed to bring SMA experimental therapeutics to the clinic in the most rapid and safe manner."

"Development of therapies requires collaboration of academics, advocacy, industry, and government--no single party has the resources to do this alone. The collaboration between Dr. Brian Kaspar, Families of SMA, and the NIH is an exciting model in leveraging resources and expertise in the hope of accelerating therapy development for SMA," said Dr. John Porter, PhD, Program Director at the National Institute of Neurological Disorders and Stroke.

About Families of SMA:

Families of SMA is the world's leader focused on funding SMA research to develop a treatment and cure for the disease. The successful results and progress that the organization has delivered, from basic research to drug discovery to clinical trials, provide real hope for families and patients impacted by the disease. The charity has invested over $55 million in research and has been involved in funding half of all the ongoing novel drug programs for SMA. Families of SMA is a nonprofit 501(c)3 organization, with 31 Chapters and 90,000 members and supporters throughout the United States. The organization's work has produced major discoveries, including identification of the underlying cause and a back-up gene for the disease, which provides a clearly defined target for disease altering therapies. The organization is also dedicated to supporting SMA families through networking, information and services and to improving care for all SMA patients. http://www.curesma.org.

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Families of SMA and Nationwide Children's Announce Multi-Million Dollar Award From NINDS to Advance CNS Gene Therapy ...

In the first paper, researchers from the Fred Hutchinson Cancer Research Institute in Seattle took haematopoeic stem cells (HSCs), bone-marrow cells that are the progenitors of all blood cells, from two pigtail macaque monkeys and transformed them genetically by splicing an inserted gene sequence for mC46 into their CCR5 receptor gene.

They then injected the cells back into the monkeys. One monkey had 20%of its HSCs replaced by the C34-producing cells and the second had over 50% replaced.

A week later, they infected them and two control monkeys with a particularly lethal strain of genetically engineered human/monkey SHIV, which destroys CD4 cells fast and usually develops a steady-state viral load in the order of several million copies/ml.

In the control animals, their CD4 counts declined from around 600 cells/mm3 before infection to between 10 and 50 cells/mm3 within two to three weeks. In the monkeys with mC46, the CD4 cell count dipped to about 100 cells/mm3 within two weeks of infection, but then rose slowly back to pre-infection levels over the next six months.

At the time of highest SHIV viral load and fewest CD4 cells, 90% of the CD4 cells in the mC46 monkeys had the fusion-inhibitor-generating insert in them, which is what one would expect, given that SHIV so decimates non-mutated CD4 cells.

What was unexpected to the scientists, though, was that after the period of peak viral load, the non-mutated CD4 cells made a partial recovery in one monkey to about 60% of all CD4 cells and in the other about 20%. This is promising, as it shows that one would not need to replace all or even most of the CD4 cells in the body with HIV-resistant ones in order to contain an HIV infection, and that increasing numbers of non-resistant cells does not lead to a new burst of virus.

This may also mean that it would not be necessary to take the dangerous step of having to destroy a persons immune system with whole-body radiation, as happened to the 'Berlin patient' Timothy Ray Brown, in order for the new cells to repopulate the immune system.

As we said, SHIV reproduces furiously and peak viral load in all monkeys ten days after infection was one billion copies/ml. After that, viral load in the control monkeys declined to about half a million in one and about ten million in the other. In the mC46 monkeys, it fell to about 100,000 in the monkey with 20% of its cells replaced by mC46 cells and down to a few hundred in the one with more than 50% of its cells replaced.

Viral load was about 320-fold lower (2.5 logs) in the first monkey and about 1400-fold (3.15 logs) lower in the second. This would transform a typical HIV viral load in an untreated human of 70,000 copies/ml to 50 copies/ml before any ARVs were taken.

Although there was a partial recovery of unmutated CD4 cells in the blood, memory cells in the lymph nodes that form the reservoir of proviral HIV DNA remained predominantly the HIV-resistant mC46 cells, which is exactly where one would want them to be.

Original post:
Gene therapy studies show potential for HIV control without drugs

By Lisa Raffensperger | March 13, 2013 1:22 pm

Most people who live to old age will suffer from arthritis. The conditions prevalence is growing alongside a graying world population.

However the only treatments at the moment address the symptoms rather than the causethe loss of cartilage in joints. Joint replacement is a last-ditch solution for some sufferers. Now a gene therapy approach has demonstrated promise in staving off arthritis in mice, opening the door to human testing.

The inspiration for the research came from studying children with a genetic form of arthritis that strikes early. These children are deficient in the gene for a protein called lubricin. Lubricin is thought to act as a lubricant between the bones in a joint.

Since a lack of lubricin caused arthritis, researchers thought perhaps additional lubricin could stave it off.

They tested this hypothesis by creating a strain of mice with an additional lubricin gene in their DNA. When these mice suffered an injury to their knees they didnt develop injury-induced arthritis. Inspection of the mices joints found that their cartilage resembled mice whod never been injured in the first place. Non-modified mice, on the other hand, had symptoms of arthritis just a month after injury.

Whats more, as the mice that made extra lubricin aged, their cartilage stayed youthful. That suggests the protein may protect against both common forms of arthritis: injury-related and age-related.

The treatment also works if the replacement genes are injected right into the joint itself, the researchers report in Science Translational Medicine today. Its delivery to human patients, then, could be similar to the injection of joint lubricants that some arthritis sufferers currently rely on.

However no gene therapies are currently approved by the FDA for human treatment, so this research will likely stay in the lab for some time yet.

More:
Gene Therapy Could Prevent Arthritis

DUBLIN--(BUSINESS WIRE)--

Research and Markets (http://www.researchandmarkets.com/research/2nfn6s/gene_therapy) has announced the addition of Jain PharmaBiotech's new report "Gene Therapy - Technologies, Markets and Companies" to their offering.

Gene therapy can be broadly defined as the transfer of defined genetic material to specific target cells of a patient for the ultimate purpose of preventing or altering a particular disease state. Genes and DNA are now being introduced without the use of vectors and various techniques are being used to modify the function of genes in vivo without gene transfer. If one adds to this the cell therapy particularly with use of genetically modified cells, the scope of gene therapy becomes much broader. Gene therapy can now combined with antisense techniques such as RNA interference (RNAi), further increasing the therapeutic applications. This report takes broad overview of gene therapy and is the most up-to-date presentation from the author on this topic built-up from a series of gene therapy report written by him during the past decade including a textbook of gene therapy and a book on gene therapy companies. This report describes the setbacks of gene therapy and renewed interest in the topic

Gene therapy technologies are described in detail including viral vectors, nonviral vectors and cell therapy with genetically modified vectors. Gene therapy is an excellent method of drug delivery and various routes of administration as well as targeted gene therapy are described. There is an introduction to technologies for gene suppression as well as molecular diagnostics to detect and monitor gene expression.

Clinical applications of gene therapy are extensive and cover most systems and their disorders. Full chapters are devoted to genetic syndromes, cancer, cardiovascular diseases, neurological disorders and viral infections with emphasis on AIDS. Applications of gene therapy in veterinary medicine, particularly for treating cats and dogs, are included.

Research and development is in progress in both the academic and the industrial sectors. The National Institutes of Health (NIH) of the US is playing an important part. As of 2012, over 2030 clinical trials have been completed, are ongoing or have been approved worldwide.A breakdown of these trials is shown according to the areas of application.

Since the death of Jesse Gelsinger in the US following a gene therapy treatment, the FDA has further tightened the regulatory control on gene therapy. A further setback was the reports of leukemia following use of retroviral vectors in successful gene therapy for adenosine deaminase deficiency. Several clinical trials were put on hold and many have resumed now. The report also discusses the adverse effects of various vectors, safety regulations and ethical aspects of gene therapy including germline gene therapy.

The markets for gene therapy are difficult to estimate as there is only one approved gene therapy product and it is marketed in China since 2004. Gene therapy markets are estimated for the years 2012-2022. The estimates are based on epidemiology of diseases to be treated with gene therapy, the portion of those who will be eligible for these treatments, competing technologies and the technical developments anticipated in the next decades. In spite of some setbacks, the future for gene therapy is bright.The markets for DNA vaccines are calculated separately as only genetically modified vaccines and those using viral vectors are included in the gene therapy markets

Profiles of 179 companies involved in developing gene therapy are presented along with 203 collaborations. There were only 44 companies involved in this area in 1995. In spite of some failures and mergers, the number of companies has increased more than 4-fold within a decade. These companies have been followed up since they were the topic of a book on gene therapy companies by the author of this report. John Wiley & Sons published the book in 2000 and from 2001 to 2003, updated versions of these companies (approximately 160 at mid-2003) were available on Wiley's web site. Since that free service was discontinued and the rights reverted to the author, this report remains the only authorized continuously updated version on gene therapy companies.

Benefits of this report

Originally posted here:
Research and Markets: Gene Therapy : Technologies, Markets and Companies 2013 Update with Added Company Profiles