Archive for the ‘Gene Therapy Research’ Category

September 11, 2013

Brett Smith for redOrbit.com Your Universe Online

According to the National Institutes of Health, almost 3.8 million Americans will suffer from anorexia at some point in their life. Thought to be primarily psychological in nature, anorexia nervosa may have a partial genetic cause according to a new report in Molecular Psychiatry.

These findings point in a direction that probably no one would have considered taking before, said study author Nicholas J. Schork, a professor at The Scripps Research Institute (TSRI).

A condition that predominantly affects women and young girls, anorexia is marked by a severe eating restriction and emaciation. Individuals with anorexia may also see themselves as fat, express perfectionism, exhibit signs of anxiety or depression, and have obsessive tendencies, said Walter Kaye, a co-author on the study and professor at the University of California, San Diego School of Medicine.

Scientists arent entirely sure how anorexia develops in a person, but many suspect cultural, stress, hormonal and social factors.

To explore a potential genetic factor for the condition, TSRI researchers, along with a team of international colleagues, embarked on the largest-ever genetic sequencing study of anorexia. The project was based on genetic data from over 1,200 individuals diagnosed with anorexia and almost 2,000 non-anorexic participants.

In an initial discovery phase of the study that included over 330 subjects, the researchers recorded the genetic variants that had already been associated to feeding behaviors or had been cited in previous anorexia studies. Out of the more than 150 genetic candidates, only a small group demonstrated a significant statistical linkage with anorexia in the study cohort.

One of the strongest initial candidates was the gene EPHX2, which is involved in the production of epoxide hydrolase 2 an enzyme recognized for regulating cholesterol metabolism.

When we saw that, we thought that we might be onto something, because nobody else had reported this gene as having a pronounced role in anorexia, said Schork.

See the original post:
New Study Finds Anorexia Has A Genetic Link, At Least Partially

CAMBRIDGE, Mass.--(BUSINESS WIRE)--

Good Start Genetics, Inc.,an innovative molecular diagnostics company harnessing a powerful, proprietary next-generation DNA sequencing (NGS) capability, today announced the appointment of R. Mark Adams, Ph.D., to chief information officer.

Good Start Genetics is focused on developing commercial applications of our proprietary NGS technology that build on our current market-leading carrier screening test, GoodStart Select, said Don Hardison, president and chief executive officer of Good Start Genetics. Good Start Genetics is an innovator in the healthcare information space, and adding Mark to our team further strengthens our ability to translate human genomics into clinically actionable tests for the reproductive health space and beyond.

Dr. Adams has worked in the field of bioinformatics and data analytics for more than 15 years and brings extensive experience in the strategy, design, and implementation of large-scale informatics organizations and systems in support of key strategic initiatives. He joins Good Start Genetics from Bridgewater Associates, Ltd., where he developed unique data warehousing and machine learning approaches to support the worlds largest hedge fund. Previously, Dr. Adams was a principal with Booz Allen Hamilton, where he developed a leading biomedical informatics consulting practice with a primary focus on informatics strategy for clients in the commercial, government, and non-profit markets. A pioneer in the use, development and deployment of large-scale, open-source software in the national and international bioinformatics community, Dr. Adams is also an expert on building communities comprised of academic, industry, and government partners to fund and address health care issues. Dr. Adams led some of the earliest commercial bioinformatics efforts to explore and use genomic and genetic variation data at Variagenics (Cambridge, MA,) where he was vice president of bioinformatics, and Incyte (Palo Alto, CA) where he worked with Temple Smith, Ph.D., to build the companys first informatics pipeline, supporting high-throughout SNP genomics. Dr. Adams holds a B.A. from Oberlin College and a Ph.D. from Baylor College of Medicine.

It is a privilege to join the team at Good Start Genetics, said Dr. Adams. The companys CAP- and CLIA-certified, high-complexity laboratory is generating unique and voluminous genomic data through its proprietary NGS platform. We are well-positioned to combine that growing data set with public data sources to take advantage of emerging data science approaches and develop unique and highly-valuable intellectual property.

Good Start Genetics is on the leading edge of responsible, clinically relevant NGS-based testing, and I look forward to contributing my expertise to expand our proprietary platform and to deliver valuable information to patients and healthcare practitioners, continued Dr. Adams.

About Good Start Genetics, Inc.

Good Start Genetics is an innovative molecular diagnostics company harnessing a powerful, proprietary next-generation DNA sequencing (NGS) capability combined with other technologies to deliver best-in-class tests for routine genetic screening. Through its GoodStart Select offering, the company provides the most comprehensive and clinically actionable set of tests for known and novel mutations that cause inherited diseases. Good Starts NGS capabilities can be applied to multiple disease areas, including pre-conception carrier screening in the in-vitro fertilization setting. For more information, please visit http://www.goodstartgenetics.com.

Follow this link:
Good Start Genetics Appoints Mark Adams, Ph.D., to Chief Information Officer

Oxford Biomedica Quote more

Price: 2.45

Chg: 0.35

Chg %: 16.67%

Date: 16:57

Gene therapy specialist Oxford Biomedica has been awarded a significant grant to help improve its supply chain.

A UK consortium of companies won 7.7m of government funding in grants and loans, with the FTSE Fledgling company taking 7.1m of this as lead member.

The consortium has been awarded, subject to due diligence and final confirmation, a 2.4m grant, of which Oxford BioMedica will receive 1.8m, and a 5.3m loan to OXB repayable by March 2017.

Chief Executive Officer John Dawson said the award was given to the company in recognition of its potential to become a world-leader in Advanced Therapy Medicinal Product (ATMP) manufacture and supply chain expertise.

He said the funds will help the company, supported by the consortium, in its plans to develop its capability in serum-free, non-adherent manufacturing techniques and expand its proprietary manufacturing facility in Oxford to contain a third production suite and a state-of-the-art fill-and-finish operation.

See the rest here:
Oxford Biomedica wins significant government funding

Scientists have identified a gene on the extra chromosome causing Down syndrome that may be responsible for the early aging and cognitive defects in people with the condition, a finding that could lead to new treatments.

The gene, called Usp16, may hinder the bodys ability to make stem cells needed to maintain tissues and organs, according to research published today in the journal Nature. That includes development of organs such as the brain while in the uterus, said Michael Clarke, a senior study author.

The findings in mice and human cells are the first to help explain why people with Down syndrome show signs of early Alzheimers disease and age faster, the authors said. Further investigation may test whether the gene might slow down these aging effects or fight against other diseases like Alzheimers and cancer, Clarke said.

Like most regulators and stem cell functions, understanding how those things work has potential implications in a wide range of diseases, Clarke, a professor of medicine at Stanford University near Palo Alto, California, said in a telephone interview. Now that we have a pathway, there might be drugs that could be used to improve some of the problems.

People with Down syndrome have three copies of chromosome 21 rather than the standard two. Usp16 is on all three copies. The extra copy of chromosome 21 and the Usp16 gene may speed up the rate that stem cells are used during early development, exhausting the stem cell pools needed to regenerate tissues as adults, Craig Garner, a professor of psychiatry and behavioral sciences at Stanford and a study author, said in a statement. This could make the brains of those with the condition age faster and increase cognitive deficits, he said.

About 400,000 people in the U.S. have Down syndrome and about 6,000 babies are born in the U.S. each year with the disorder, according to the National Down Syndrome Society.

Physical issues that accompany Down syndrome include heart defects, stomach trouble, hearing difficulties and a higher likelihood of childhood leukemia. Alzheimers disease is also common among patients with the disorder. It is estimated that more than 75 percent of those ages 65 and older with Down syndrome have Alzheimers, six times as many as those in the age group without Down syndrome, according to the Alzheimers Association.

In the study, the tests in human cells also showed that an excess of Usp16 in people without Down syndrome caused skin cells to grow more slowly, while reducing Usp16 in skin and nerve cells in people with Down syndrome allowed the cells to have normal growth patterns rather than to regenerate slowly, the authors said.

This gene is clearly regulating processes that are central to aging in mice and humans, Clarke said. Reducing Usp16 expression gives an unambiguous rescue at the stem cell level. The fact that its also involved in this human disorder highlights how critical stems cells are to our well-being.

To contact the reporter on this story: Nicole Ostrow in New York at nostrow1@bloomberg.net

Link:
Gene Tied to Down Syndrome May Suggest Way to New Therapy

Studies of a therapy designed to treat amyotrophic lateral sclerosis (ALS), also called Lou Gehrig's Disease, suggest that the treatment dramatically slows onset and progression of the deadly disorder that is the most common neuromuscular condition in the world. There currently is no cure for ALS. The Centers for Disease Control and Prevention estimates there are about 5,000 new cases in the U.S. each year, mostly in people age 50 to 60.

The researchers, led by teams from The Research Institute at Nationwide Children's Hospital and the Ludwig Institute at the University of California, San Diego, found a survival increase of up to 39 percent in animal models with a one-time treatment, a crucial step toward moving the therapy into human clinical trials.

A release from the hospital reports that the therapy reduces expression of a gene called SOD1, which in some cases of familial ALS has a mutation that weakens and kills nerve cells called motor neurons that control muscle movement. While many drug studies involve only one type of animal model, this effort included analysis in two different models treated before and after disease onset. The in-depth study could vault the drug into human clinical trials, said Brian Kaspar, PhD, a principal investigator in the Center for Gene Therapy at Nationwide Children's and a senior author on the research, which was published online September 6th 2013 in the journal Molecular Therapy.

"We designed these rigorous studies using two different models of the disease with the experimenters blinded to the treatment and in two separate laboratories," said Dr. Kaspar, who collaborated on the study with a team led by Don Cleveland, PhD, at the University of California, San Diego. "We were very pleased with the results, and found that the delivery approach was successful in a larger species, enabling us to initiate a clinical translational plan for this horrible disease." Kevin Foust, PhD, co-first author on the manuscript and an assistant professor in neurosciences at The Ohio State University College of Medicine, said "The extension of survival is fantastic, and the fact that we delayed disease progression in both models when treated at disease onset is what drives our excitement to advance this work to human clinical trials." In addition to the potential therapeutic benefit, the study also offers some interesting insights into the biological underpinnings of ALS. The role of motor neurons in ALS has been well documented, but this study also highlighted another key playerastrocytes, the most abundant cell type in the human brain and supporters of neuronal function. "Recent work from our collaborator Dr. Cleveland has demonstrated that astrocytes and other types of glia are as important if not more important in ALS, as they really drive disease progression," said Dr. Kaspar. "Indeed, in looking at data from mice, more than 50 percent of astrocytes were targeted throughout the spinal cord by this gene-delivery approach."

Ideally, a therapy would hit motor neurons and astrocytes equally hard. The best way to do that is to deliver the drug directly into the cerebrospinal fluid (CSF), which would reduce the amount of SOD1 suppression in cells outside the brain and reduce immune system exposure to AAV9elements that would add weight to an argument for studying the drug in humans. Injections directly into CSF cannot be done easily in mice, so the team took the study a crucial step further by injecting AAV9-SOD1-shRNA into the CSF of healthy nonhuman primates. The results were just as the team hopedthe amount of gene expression dropped by as much as 90 percent in motor neurons and nearly 70 percent in astrocytes and no side effects were reported, laying the groundwork towards moving to human clinical trials. "We have a vast amount of work to do to move this toward a clinical trial, but we're encouraged by the results to date and our team at Nationwide Children's and our outstanding collaborators are fully committed to making a difference in this disease," Dr. Kaspar said. The findings could impact other studies underway in Dr. Kaspar's lab, including research on Spinal Muscular Atrophy, an often fatal genetic disease in infants and children that can cause profoundly weakened muscles in the arms and legs and respiratory failure. "This research provides further proof of targeting motor neurons and glial cells throughout the entire spinal cord for treatment of Spinal Muscular Atrophy and other degenerative diseases of the brain and spinal cord, through a less invasive manner than direct injections," said Dr. Kaspar, who also is an associate professor of pediatrics and neurosciences at The Ohio State University College of Medicine.

Read the rest here:
Therapy Slows ALS Progression

Public release date: 10-Sep-2013 [ | E-mail | Share ]

Contact: Juan J. Gmez juanj.gomez@cnio.es Centro Nacional de Investigaciones Oncologicas (CNIO)

Researchers from the Spanish National Cancer Research Centre (CNIO) have discovered that more than 70% of bladder tumours display somatic mutations in the TERT gene (telomerase reverse transcriptase). The TERT gene is involved in the protection of DNA and in cellular ageing processes and cancer. These results make this gene the most mutated in these tumours.

The study was led by Francisco X. Real, head of the Epithelial Carcinogenesis Group at CNIO, together with Nuria Malats, the head of the Genetic & Molecular Epidemiology Group at CNIO, as well as other European groups, especially Yves Allory, a pathologist at the Mondor Hospital (Crteil, Paris, France), who is on a sabbatical year with Real and Malats's groups at CNIO, and Ellen Zwarthoff's group at the Erasmus Medical Centre in Rotterdam. The results are published in the online version of the journal European Urology.

The conclusions come from an exhaustive genetic and molecular study of more than 450 patients diagnosed with bladder cancer. Among the cases explored are both indolent tumours and more aggressive tumours and, therefore, those most likely to develop localised or spreading metastasis in the organism.

"When we analysed the frequency of TERT mutations in this group of patients, we observed that there was no correlation between the presence of mutations and the aggressiveness of the tumour or the survival or the patients", says Real. The authors' description in the article explains that: "The fact that these mutations are present in any phase of the urothelial tumoural process suggests that they occur in an early phase during carcinogenesis".

The product of the TERT gene is a protein, the reverse transcriptase of the telomerase complex, which increases the length of telomeres, protective structures for genetic material located at the ends of chromosomes and associated with cellular ageing.

"How TERT mutations affect the length of the telomeres and encourage carcinogenesis still needs to be discovered", says Real, adding that: "We believe that they could increase the gene expression, but additional studies are necessary".

The authors think that the clinical implications of their study could be of great relevance, both from a diagnostic point of view and in the follow-up treatment of these patients.

From the diagnostic and treatment-of-patients points of view, the authors propose TERT as a new biomarker for bladder cancer: "We are able to detect gene mutations in urine samples from patients, in such a way that the analysis of these mutations, combined with other markers, could be promising for the detection of the illness".

More here:
3 out of every 4 cases of bladder cancer display mutations in the same gene

Public release date: 10-Sep-2013 [ | E-mail | Share ]

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

New Rochelle, NY, September 10, 2013Use of stimulant medications to treat Attention-Deficit/Hyperactivity Disorder (ADHD) in children and adolescents has increased significantly over the past several years. This trend toward increased use of prescription stimulants extends beyond ADHD to other types of neuropsychiatric disorders in children and teens as well, including Autism Spectrum Disorder (ASD), according to a study published in Journal of Child and Adolescent Psychopharmacology (JCAP), a peer-reviewed journal from Mary Ann Liebert, Inc., publishers. The article is available on the JCAP website.

Sren Dalsgaard, MD, PhD, Helena Skyt Nielsen, PhD, and Marianne Simonsen, PhD, Aarhus University (Denmark), Lundbeck Foundation Initiative for Integrative Psychiatric Research (Denmark), and Hospital of Telemark (Norway), conducted a study of more than 850,000 children born in Denmark between 1990 and 2001. They found that 61% of children with ADHD, 16% of children with ASD, and 3% of those with other psychiatric disorders were treated with one or more medications typically prescribed for ADHDmethylphenidate, dexamphetamine, and atomoxetine. The data indicated significant increases in the prescription rates of these medications during the years 2003 to 2010.

"This study utilizes a population-based national cohort of children and adolescents, and assesses stimulant treatment in children and adolescents with ASD," says Harold S. Koplewicz, MD, Editor-in-Chief of JCAP, and President, Child Mind Institute, New York, NY. "This is the largest and first prospective study to quantify the change in the use of treatment with ADHD medications over time."

###

About the Journal

Journal of Child and Adolescent Psychopharmacology (JCAP) is an authoritative peer-reviewed journal published 10 times a year online with Open Access options and in print. The Journal is dedicated to child and adolescent psychiatry and behavioral pediatrics, covering clinical and biological aspects of child and adolescent psychopharmacology and developmental neurobiology. Complete tables of content and a sample issue may be viewed on the JCAP website.

About the Publisher

Mary Ann Liebert, Inc., publishers is a privately held, fully integrated media company known for establishing authoritative peer-reviewed journals in many promising areas of science and biomedical research, including Cyberpsychology, Behavior, and Social Networking and Games for Health Journal. Its biotechnology trade magazine, Genetic Engineering & Biotechnology News (GEN), was the first in its field and is today the industry's most widely read publication worldwide. A complete list of the firm's over 80 journals, books, and newsmagazines is available on the Mary Ann Liebert, Inc., publishers website.

Excerpt from:
5-fold increase in ADHD medication use in children and adolescents

A more stringent safety regime will be in place for genetic engineering in agriculture or medical sciences once the National Biotechnology Regulatory Authority (NBRA) Bill is cleared by Parliament, a senior official said here today.

"The NBRA bill has been introduced in Parliament and being debated. Once it (bill) is cleared, a more stringent safety regime shall be in place for genetic engineering, be it in the agriculture space or medical sciences," said S R Rao, Scientist, 'G' Department of Biotechnology Government of India.

He was addressing a gathering of farmers at ongoing Vibrant Gujarat Global Agriculture Summit-2013.

The authority is proposed to be an autonomous and statutory agency to regulate the research, transport, import, manufacture and use of organisms and products of modern bio-technology, he said.

India has signed the UN convention on biodiversity and Cartagena Protocol on bio-safety, which calls for a regulatory authority.

Making a strong pitch for adoption of bio-technology in agri sector, Rao said the spurt in Cotton production in India has set an example for adoption of technology.

The cotton production has been growing at a CAGR of 4.38 per cent, Rao said, adding other crops like pulses and others are still in negative territory.

According to experts, after switching over to BT Cotton, the yield in India has shot up from 328 kg per hectare to 491 kg per hectare in 2012, while acreage under the crop has gone up from 78 lakh to 120 lakh hectares.

Experts estimate that around 93 per cent of area in the country is now under BT Cotton even though disease-related issues continue to persist.

Rao said India ranked seventh in the world with 140 million hectares of area under cultivation. "And if the crop productivity has to be increased from 100 per cent to 250 per cent in an acre of land, then 80 per cent of contribution can come through ado."

View original post here:
Stringent Safety Norms in Agriculture after NBRA: Centre

IGM Art Gallery #39;s "Creating A Healthy Life" Forum
For Dr. Howard Murad #39;s book: "Water Secret" please visit: http://www.amazon.com/gp/aw/d/0470554703 For a copy of Dr. Rishi Manchanda #39;s new good "Upstream Doc...

By: IGM Art Gallery

Read the rest here:
IGM Art Gallery's "Creating A Healthy Life" Forum - Video

Newswise Case Western Reserve University is part of a landmark study that has discovered four novel gene variations associated with blood pressure. The 19-site meta-analysis, involving nearly 30,000 African-Americans, also found that the set of genetic mutations are also associated with blood pressure across other populations.

Epidemiology and biostatistics professor Xiaofeng Zhu, PhD, is co-senior author of the paper, which appears in The American Journal of Human Genetics. The Continental Origins and Genetic Epidemiology Network (COGENT) consortium conducted the research, which is the largest genome-wide association study of blood pressure in individuals of African ancestry. Most gene discovery studies to date have been performed using individuals of European ancestry. Previous genome-wide association studies using samples from individuals of African descent failed to detect any replicable genes associated with blood pressure.

In addition to their disproportionate suffering, hypertension occurs earlier in life for African-Americans compared to individuals of other ancestries, Zhu explained. Therefore, it is important to study this population to better understand genetic susceptibility to hypertension.

Zhu and his colleagues also confirmed that previous findings regarding other genes whose presence correlates with increased hypertension risk.

Although it is unknown how the genes regulate blood pressure, Zhu added, our findings contribute to better understanding of blood pressure pathways that can lead to future development of drug target for hypertension and may guide therapy for clinical care.

Experts estimate genetic make-up accounts for roughly 40-50 percent of individuals susceptibility to hypertension. Other factors associated with the disease include lifestyle, diet, and obesity. Compared to Americans of European-ancestry, African-Americans increased hypertension prevalence contributes to a greater risk of stroke, coronary heart disease, and end-stage renal disease.

We anticipated that individuals of African ancestry share similar biology to other populations. However, differences in genomic make-up between African ancestry and other populations have uncovered additional genes affecting blood pressure, in addition to genetic variants that are specific to individuals of African ancestry, said Nora Franceschini, MD, MPH, nephrologist and research assistant professor of epidemiology at the University of North Carolina at Chapel Hill and first author on the paper.

The next phase of study involving the newly discovered gene mutations will investigate their function using human blood samples at the molecular level. Zhu and his colleagues have begun conducting additional research to determine whether the newly identified genes respond to existing hypertension medications. Individuals typically respond differently to a given medication depending on which gene mutation they carry. The more information researchers gather, the greater opportunity clinicians will have prescribed the drug that is most efficacious based on the patients specific mutation.

The research findings do not have immediate implications for treatment, but the hope is that discovering genes associated with disease risks will bring scientists closer to biological pathways and may suggest useful targets for new treatments, said geneticist Brendan J. Keating, DPhil, one of co-senior authors of the paper, of The Center for Applied Genomics at The Childrens Hospital of Philadelphia and faculty at the Department of Pediatrics at the University of Pennsylvania.

###

More here:
African-American Study Identifies Four Common Genetic Variants Associated with Blood Pressure

Public release date: 10-Sep-2013 [ | E-mail | Share ]

Contact: Jessica Studeny Jessica.studeny@case.edu 216-368-4692 Case Western Reserve University

Case Western Reserve University is part of a landmark study that has discovered four novel gene variations which are associated with blood pressure. The 19-site meta-analysis, involving nearly 30,000 African-Americans, also found that the set of genetic mutations are also associated with blood pressure across other populations.

Epidemiology and biostatistics professor Xiaofeng Zhu, PhD, is co-senior author of the paper, which appears in The American Journal of Human Genetics. The Continental Origins and Genetic Epidemiology Network (COGENT) consortium conducted the research, which is the largest genome-wide association study of blood pressure in individuals of African ancestry. Most gene discovery studies to date have been performed using individuals of European ancestry. Previous genome-wide association studies using samples from individuals of African descent failed to detect any replicable genes associated with blood pressure.

"In addition to their disproportionate suffering, hypertension occurs earlier in life for African-Americans compared to individuals of other ancestries," Zhu explained. "Therefore, it is important to study this population to better understand genetic susceptibility to hypertension."

Zhu and his colleagues also confirmed that previous findings regarding other genes whose presence correlates with increased hypertension risk.

"Although it is unknown how the genes regulate blood pressure," Zhu added, "our findings contribute to better understanding of blood pressure pathways that can lead to future development of drug target for hypertension and may guide therapy for clinical care."

Experts estimate genetic make-up accounts for roughly 40-50 percent of individuals' susceptibility to hypertension. Other factors associated with the disease include lifestyle, diet, and obesity. Compared to Americans of European-ancestry, African-Americans' increased hypertension prevalence contributes to a greater risk of stroke, coronary heart disease, and end-stage renal disease.

"We anticipated that individuals of African ancestry share similar biology to other populations. However, differences in genomic make-up between African ancestry and other populations have uncovered additional genes affecting blood pressure, in addition to genetic variants that are specific to individuals of African ancestry," said Nora Franceschini, MD, MPH, nephrologist and research assistant professor of epidemiology at the University of North Carolina at Chapel Hill and first author on the paper.

The next phase of study involving the newly discovered gene mutations will investigate their function using human blood samples at the molecular level. Zhu and his colleagues have begun conducting additional research to determine whether the newly identified genes respond to existing hypertension medications. Individuals typically respond differently to a given medication depending on which gene mutation they carry. The more information researchers gather, the greater opportunity clinicians will have prescribed the drug that is most efficacious based on the patient's specific mutation.

See more here:
African-American study identifies 4 genetic variants associated with blood pressure

SOUTH SAN FRANCISCO, Calif., Sept. 10, 2013 /PRNewswire/ --AvidBiotics today announced a research collaboration with Full Spectrum Genetics for the purpose of optimizing multiple therapeutic protein candidates from AvidBiotics' Micacide protein immuno-oncology and antiviral platform. AvidBiotics also announced that Bob DuBridge, Ph.D., Chief Executive Officer of Full Spectrum Genetics, has joined the AvidBiotics Scientific Advisory Board.

(Logo:http://photos.prnewswire.com/prnh/20130909/NE76577LOGO)

"Micacide proteins represent a new class of molecules that harness the innate cellular immune response to efficiently detect and destroy cancerous or virus-infected cells," said JamesL. Knighton, AvidBiotics' President. "Full Spectrum Genetics' MapEng technology enables the rapid, simultaneous screening of many protein variants, allowing us to quickly identify those molecules with the best binding properties for further development as potential therapeutics."

Under the terms of the agreement, Full Spectrum Genetics will be responsible for generating and analyzing a multitude of variants of up to five specified AvidBiotics' protein molecules using its MapEng platform. AvidBiotics will receive worldwide rights to develop and commercialize product candidates arising from the collaboration. Full Spectrum Genetics will receive an upfront payment and is eligible for additional milestones and royalties on successfully commercialized molecules. Additional terms of the agreement were not disclosed.

"Traditional methods to understand protein-ligand interactions that affect disease outcomes often yield incomplete information despite the considerable time and expense involved," said Dr. DuBridge. "Our MapEng platform is designed to generate these insights significantly faster, less expensively and in more detail than other technologies, thus enabling the engineering of optimized proteins for further development by AvidBiotics."

Bob DuBridge Biography

Bob DuBridge, Ph.D. is the CEO of Full Spectrum Genetics, a company that he founded in 2010. Prior to that, he was employed at PDL BioPharma, Inc. from 2003 to 2010, ultimately serving as Head of New Technologies, and before that at Eos Biotechnology from 1999, which was acquired by PDL in 2003. Dr. DuBridge has also held senior scientific and management positions at such biotechnology firms as Lynx Therapeutics, Cell Genesys, Inc. and Genentech, Inc. He received his Ph.D. in genetics from Stanford University.

About Full Spectrum Genetics

Founded in 2010, Full Spectrum Genetics, Inc. is a privately-held protein engineering platform and product company. The Company's MapEng platform enables ultra-high throughput quantification of the effect on binding of every possible single amino acid substitution within a protein binding site. The MapEng platform provides a comprehensive analysis of protein structure-function relationships, with multiple applications for generating better biotherapeutics and diagnostics. Full Spectrum Genetics' activities cover the spectrum from antibody discovery to generating proteins with improved properties including modulation of binding affinity to one or more targets, de-immunization, humanization, half-life extension and increased stability. For more information on Full Spectrum Genetics and its MapEng platform, visit http://www.fsgene.com.

About AvidBiotics

Continue reading here:
AvidBiotics Announces Research Collaboration with Full Spectrum Genetics

Dr Jamal A Khan holds PC for #39;Dentroitic Cell Therapy #39;

By: Bollywood Royal

Go here to see the original:
Dr Jamal A Khan holds PC for 'Dentroitic Cell Therapy' - Video

Washington, Sept. 10 (ANI): Researchers have developed a therapy that could dramatically slow the onset and progression of the Lou Gehrig's disease.

The researchers, led by teams from The Research Institute at Nationwide Children's Hospital and the Ludwig Institute at the University of California, San Diego, found a survival increase of up to 39 percent in animal models with a one-time treatment, a crucial step toward moving the therapy into human clinical trials.

The therapy reduces expression of a gene called SOD1, which in some cases of familial amyotrophic lateral sclerosis (ALS) has a mutation that weakens and kills nerve cells called motor neurons that control muscle movement.

While many drug studies involve only one type of animal model, this effort included analysis in two different models treated before and after disease onset.

Brian Kaspar, PhD, a principal investigator in the Center for Gene Therapy at Nationwide Children's and a senior author on the research, said that they designed these rigorous studies using two different models of the disease with the experimenters blinded to the treatment and in two separate laboratories.

Ideally, a therapy would hit motor neurons and astrocytes equally hard. The best way to do that is to deliver the drug directly into the cerebrospinal fluid (CSF), which would reduce the amount of SOD1 suppression in cells outside the brain and reduce immune system exposure to AAV9-elements that would add weight to an argument for studying the drug in humans.

Injections directly into CSF cannot be done easily in mice, so the team took the study a crucial step further by injecting AAV9-SOD1-shRNA into the CSF of healthy nonhuman primates.

The results were just as the team hoped-the amount of gene expression dropped by as much as 90 percent in motor neurons and nearly 70 percent in astrocytes and no side effects were reported, laying the groundwork towards moving to human clinical trials.

The study has been published online in Molecular Therapy. (ANI)

Follow this link:
New therapy could help slow down progression of Lou Gehrig's disease

Latest Diet & Weight Management News

FRIDAY, Sept. 6 (HealthDay News) -- Genetic testing for obesity risk does not discourage people from trying to lose weight -- instead, it may help reduce how much they blame themselves for their weight problems, according to a small new study.

Research has shown that genes influence a person's risk of becoming overweight and one gene, called FTO, appears to have the greatest effect. The "A" variant of the gene is associated with a greater risk of weight gain, while the "T" variant of the gene is associated with a lower risk.

One in two people has at least one copy of the A variant. People with two A variants -- one from their mother and one from their father -- are 70 percent more likely to become obese than those with two T variants, according to the study authors at University College London (UCL).

Researchers have access to a genetic test for FTO variants, but the test is not commercially available. Among experts, there's debate about how people would react to getting results of the FTO genetic test. Some believe that such knowledge would help motivate people to manage their weight, while others think it would make people feel there was nothing they could do about their weight.

In this study, researchers assessed how 18 people responded to having the FTO genetic test. The male and female participants, who ranged in weight from underweight to obese, were enthusiastic about getting their genetic test results, according to the study in a recent issue of the Journal of Genetic Counseling.

People who struggled with their weight said the knowledge was helpful because it reduced some of the emotional stress associated with weight control and eased some of the stigma and self-blame. None of the participants had a negative reaction to the genetic test result, nor did they say it made them feel like there was nothing they could do about their weight.

"These results are encouraging. Regardless of gene status or weight, all the volunteers recognized that both genes and behavior are important for weight control. The results indicate that people are unlikely to believe that genes are destiny and stop engaging with weight control once they know their FTO status," study leader Susanne Meisel said in a UCL news release.

"Although they knew that FTO's effect is only small, they found it motivating and informative. We are now doing a larger study to confirm whether more people react in the same way," Meisel added.

Heredity alone doesn't determine obesity, another expert noted.

Read more:
Obesity Gene Tests May Not Hamper Weight-Loss Efforts

HOLLYWOOD, Fla. -

Barbaree King began smoking when she was a teenager. She struggled for years to quit.

"At some time, I had quit for a couple of years and in a moment of weakness, I said, 'I'll just have one' and I did," she said.

King wondered why smoking cessation aids never seemed to help her, but researchers may now have an answer.

The U.S. National Institute on Drug Abuse found several genetic variations that may play a role in whether people will respond well to nicotine replacement therapy and the smoking cessation drug Bupropion. Researchers found 41 genetic variants linked to smokers who were able to quit using nicotine replacement therapy, including patches and gum, and 26 genes that could help people successfully quit with Bupropion.

"Some people will be able to quit without the medication. Some people are going to have a really tough time," said Dr. Mark Block, a lung specialist with Memorial Health Care System.

Block said the research may lead to more tailored treatment approaches.

"You don't want to give medication to people who aren't going to benefit from it and by the same token, you don't want to withhold it from people who are really going to benefit, so it's a nice way to target therapy based on genetics," he said.

View post:
Research may lead to 'smoking gene'

Public release date: 9-Sep-2013 [ | E-mail | Share ]

Contact: Alison Barbuti alison.barbuti@manchester.ac.uk 44-016-127-58383 University of Manchester

Scientists at The University of Manchester and medical institutes in Italy have identified a gene variant that predisposes people to a special type of heart attack.

Their research, published in the International Journal of Cardiology could lead to the development of new drugs to treat the problem.

Dr Paolo Tammaro, who led the team, said: "Heart attacks happen when the blood supply to the heart is reduced by the narrowing or blocking of the coronary artery the vessel that supplies the heart with oxygen and nutrients. Often this is due to fatty deposits which narrow the vessel. However, in some people with perfectly clean arteries, the vessel suddenly constricts shutting off the blood supply. We have discovered that this process, known as vasospasm, can be associated with a rare variant of a particular gene."

Dr Enzo Emanuele, from the University of Pavia, who screened the patients, said: "We knew that this type of heart attack occurs in about 6% of patients and that many of them have a genetic predisposition, but we did not know the gene responsible. Now that it is identified it will be possible to predict who is at risk and to treat them accordingly."

The gene identified by the team encodes a protein termed KATP channel. This protein forms microscopic gated pores that allow potassium ions to move into and out of the cells, in this way giving rise to electrical impulses.

Dr Tammaro and research scientist Keith Smith, both based at the Faculty of Life Sciences at The University of Manchester, added: "These channels are abundant in the cells forming the wall of coronary arteries, and the electrical impulses they generate govern this artery's diameter. Due to the mutation we have identified, the KATP channel in the coronary artery can no longer fulfill this delicate process."

###

The team, whose work was supported by the BBSRC (Biotechnology and Biological Sciences Research Council), now plans to approach pharmaceutical companies with their findings, aiming to design novel drugs that could interact with this new target.

View post:
A tiny channel and a large vessel: A new clue for heart attack

Rensselaer

Notions of the Capital Region as a scientific Smallbany frustrate Douglas Conklin.

In the University at Albany Cancer Research Center a 9-year-old building that the associate professor blithely termed "Joe Bruno pork" Conklin and his team have earned a patent for identifying a gene as a possible target for breast cancer drugs. The gene has been known as the cause of another disease an immune disorder affecting mostly men.

The patent is the result of years of effort casting a broad net to understand how breast cancer invades a woman's body, experimenting, watching and waiting until "the breast cancer cells tell us what's important to them," as Conklin put it.

The work is no different from that going on at research centers with international reputations, he said.

"It cheeses me off to no end, when somebody (local) says, 'We're collecting money for St. Jude's hospital,'" said Conklin, referring to the well-known children's cancer center in Memphis.

Conklin himself came to Albany by way of the Cold Spring Harbor Laboratory on Long Island, known for its groundbreaking work on DNA. (He does a mean impression of James Watson, the co-discoverer of DNA, who directed that lab.)

As blunt as he is in his opinions, Conklin is patient and plainspoken in explaining the complexities of breast cancer research conducted at his lab. His team of 10 scientists are engaged in multiple experiments connected by the common thread of breast cancer.

It was through manipulating the regulation of various genes turning one gene after another "off" to see how cells would function without it that they discovered breast cancer cells die when the Bruton's tyrosene kinase gene is silenced.

BTK is known for its role in X-linked agammaglobulinaemia, a rare immune deficiency disease that affects one in 200,000 newborns, according to the National Institutes of Health. A mutation in the gene destroys the body's immune system response, including the production of antibodies, making even slight infections dangerous. Because the BTK gene is on the X chromosome, women carry it and pass it along to children, but men are almost exclusively the victims.

See the article here:
Quest to solve breast cancer

SAINT PAUL, Minn.--(BUSINESS WIRE)--

The first published research on the groundbreaking use of a modified gene-editing system to produce horn-free dairy cattle was released this week in Proceedings of the National Academy of Sciences (PNAS). PNAS is the official journal of the National Academies of Sciences and is among the world's most-cited multidisciplinary scientific publications.

Drs. Scott Fahrenkrug, Perry Hackett and their team at Recombinetics, a Minnesota-based global innovator in genome editing, authored Efficient nonmeiotic allele introgression in livestock using custom endonucleases, a new paper detailing an important new approach that will not only make hornless dairy cattle a reality, but accelerate the genetic improvement of livestock for food production and the development of regenerative medicines.

For some time now, explains Fahrenkrug, CEO of Recombinetics, farmers have practiced selective breeding for desirable DNA variation with the intention of influencing livestock traits. In this paper, we have shown for the first time that its possible to bypass decades of breeding by introgressing genetic variants directly into livestock genomes using targeted nucleases, without the use of any transgenic DNA. Our team used TALENs and other sequence-specific DNA scissors to custom-engineer and modify several genes of interest in pigs, cows and goats. Recombinetics uses its proprietary genome-editing technologies to accelerate the breeding of natural characteristics in livestock to enhance their health and welfare, and advance worldwide food production and safety.

The group demonstrated that a sequence associated with horns in dairy cattle could be converted to a natural beef cattle variant that is hornless, providing a strategy to improve animal welfare by genetic, instead of physical or chemical dehorning.

Targeted genetic improvement can save numerous generations and decades of selective breeding. The technology promises to rapidly enhance protein production in indigenous livestock breeds and to introduce natural disease resilience traits into breeds and species that dont already have them. The paper also reports the introgression of precise variants from warthogs into the swine genome to protect against the highly-contagious and lethal African Swine Fever Virus; and the copying of a natural sequence into the goat genome to increase the frequency of twinning, helping enhance food security for small farmers in the developing world.

Recombinetics also applies its genome-editing solutions and TALEN technologies to create large animal models for human disease research accelerate the development of therapeutic compounds, medical devices and protocols.

About Recombinetics

A private company founded in 2008, Recombinetics has emerged as a global leader in proprietary gene repair and gene-editing technology. Breakthrough scientific research including the development of TALEN technologies has resulted in global exclusive rights in the biomedical, animal agriculture and livestock vertical markets.

Read the original:
First Published Research Shows Success with Targeted Gene-Editing to Accelerate Genetic Improvement in Dairy Cattle

Home Mail News Sports Finance Weather Games Groups Answers Flickr More omg! Shine Movies Music TV Health Shopping Travel Autos Homes Mobile Yahoo! News Search News Search Web Sign In Mail Help Account Info Help Suggestions Yahoo! Home Video Photos GMA Year in Review Odd Comics Travel Opinion Trending Now Who Knew? Weather The Upbeat U.S. U.S. Video GMA Education Religion Crimes and Trials Local Contributor Network Year In Review World World Video Middle East Europe Latin America Africa Asia Canada Australia/Antarctica Business Video Exclusives Today's Markets Stocks Personal Finance Marketplace Entertainment Video Clinton Concert Celebrity TV Movies Music Fashion Books Arts Theater Dear Abby Comics Odd News Sports Video NFL MLB NBA NCAAF NCAAB Soccer Cycling NHL Tennis Golf Boxing Motor Sports MMA Olympics Tech Gadgets Wireless Apple Social Media Security Open Source Gaming Apps This Could Be Big Upgrade Your Life Politics Remake America The Issues Women and Politics Press Releases Video Science Science Video Weather News Space / Astronomy Pets Dinosaurs / Fossils Biotech Energy Green Health Video Weight Loss Cancer Sexual Health Medications/Drugs Parenting/Kids Seniors/Aging Diseases/Conditions Blogs The Sideshow Katie's Take Power Players This Could Be Big Newsmakers Trending Now The Upbeat Who Knew? Nightline Fix Beyond the Headline Local Popular Search Keyword News Search

Read more:
Rare, inherited mutation leaves children susceptible to acute lymphoblastic leukemia

Public release date: 9-Sep-2013 [ | E-mail | Share ]

Contact: Kathryn Ruehle kruehle@liebertpub.com 914-740-2100 Mary Ann Liebert, Inc./Genetic Engineering News

New Rochelle, NY, September 9, 2013Studies have shown that caffeine users can become dependent on or addicted to caffeine and may have difficulty reducing their consumption, as can occur with other drugs of dependence. A comprehensive review of the current evidence on caffeine dependence is presented in an article in Journal of Caffeine Research, a peer-reviewed publication from Mary Ann Liebert, Inc., publishers. The article is available free on the Journal of Caffeine Research website at http://www.liebertpub.com/jcr.

Steven Meredith and Roland Griffiths, Johns Hopkins University School of Medicine (Baltimore, MD), Laura Juliano, American University (Washington, DC), and John Hughes, University of Vermont (Burlington), reviewed the published research on caffeine dependence. In the article "Caffeine Use Disorder: A Comprehensive Review and Research Agenda" they describe the prevalence of caffeine dependence, clinically relevant indicators of functional impairment among caffeine users, and the criteria for making a diagnosis of caffeine use disorder.

The authors propose an agenda for future research that would include clinical, epidemiologic, and genetic investigations to lead to a better understanding of the clinical signs and the prevalence of caffeine dependence, as well as the risk factors and best approaches for treating caffeine addiction.

"Caffeine-related problems are increasingly being seen as clinically important by addiction professionals," says Jack E. James, PhD, Editor-in-Chief of Journal of Caffeine Research. "The article by Dr. Steven Meredith and colleagues is timely in helping to clarify the dimensions of caffeine dependence problems, while also providing direction for future research in this neglected area."

###

About the Journal

Journal of Caffeine Research: The International Multidisciplinary Journal of Caffeine Science is a quarterly journal published in print and online that covers the effects of caffeine on a wide range of diseases and conditions, including mood disorders, neurological disorders, cognitive performance, cardiovascular disease, and sports performance. The Journal explores all aspects of caffeine science including the biochemistry of caffeine; its actions on the human body; benefits, dangers, and contraindications; and caffeine addiction and withdrawal, across all stages of the human life span from prenatal exposure to end-of-life. Tables of content and a sample issue may be viewed on the Journal of Caffeine Research website at http://www.liebertpub.com/jcr.

About the Publisher

Read the rest here:
More research urgently needed on caffeine

TORONTO, Sept. 9, 2013 /CNW/ - Scientists at the Centre for Addiction and Mental Health (CAMH) and University Health Network (UHN) have found a new link between early-onset Parkinson's disease and a piece of DNA missing from chromosome 22. The findings help shed new light on the molecular changes that lead to Parkinson's disease.

The study appears online today in JAMA Neurology.

Among people aged 35 to 64 who were missing DNA from a specific part of chromosome 22, the research team found a marked increase in the number of cases of Parkinson's disease, compared to expected rates of Parkinson's disease in the general population from the same age group.

The deletion, which occurs when a person is born with about 50 genes missing on one chromosome 22, is associated with 22q11.2 deletion syndrome. People with this condition may have heart or other birth defects, learning or speech difficulties, and some develop schizophrenia. It occurs in an estimated 1 in 2,000 to 4,000 births, but is believed to be under-diagnosed.

"22q11.2 deletion syndrome has been fairly well studied in childhood and adolescence, but less is known about its effects as people age," said Dr. Anne Bassett, Director of CAMH's Clinical Genetics Research Program and Director of the Dalglish Family Hearts and Minds Clinic at UHN, the world's first clinic dedicated to adults with 22q11.2 deletion syndrome. A few cases of patients with the syndrome who had Parkinson's disease symptoms had been previously reported, which suggested that the two conditions might be linked.

Parkinson's disease is one of the most common neurodegenerative disorders worldwide, typically affecting people over the age of 65. Earlier onset of Parkinson's disease, before age 50, is rare and has been associated with several other genetic changes that are not on chromosome 22.

The researchers studied 159 adults with 22q11.2 deletion syndrome to discover how many had been clinically diagnosed with Parkinson's disease. For three individuals with the deletion and Parkinson's disease who were deceased, brain tissue was also examined.

"Through a post-mortem examination, we were able to show that all three patients had a loss of neurons that was typical of that seen in Parkinson's disease. The examination also helped to show that the symptoms of Parkinson's disease were not related to side effects of the medications commonly used to treat schizophrenia," added Dr.Rasmus Kiehl, neuropathologist in UHN's Laboratory Medicine Program, who co-authored the report with CAMH graduate student Nancy Butcher. The team also found that Parkinson's disease in 22q11.2 deletion syndrome is associated with abnormal accumulations of protein called Lewy bodies in the brain in some, but not all cases, just as in another genetic form of Parkinson's disease.

The findings highlight the complexity of clinical care when both Parkinson's disease and 22q11.2 deletion syndrome are present. "Our results may inform best practices in the clinic in these cases," said Dr. Bassett, Senior Scientist in CAMH's Campbell Family Mental Health Research Institute.

Because patients with 22q11.2DS who have schizophrenia are often prescribed anti-psychotic medications, they may experience side-effects such as tremors and muscle stiffness, similar to symptoms of Parkinson's disease.

Link:
Early-onset Parkinson's disease linked to genetic deletion

Let #39;s Play Panzer Dragoon Saga [Part 17] - Ancient Genetics
We #39;re bound to find some interesting facts in this massive place...

By: Zergem

Read the original:
Let's Play Panzer Dragoon Saga [Part 17] - Ancient Genetics - Video

Guido Pontecorvo - Professor of Genetics 1955 to 1968 University of Glasgow.
Guido Pontecorvo (1907-1999), who liked to be known by his nickname, Ponte, was the University #39;s first Professor of Genetics, 1955 to 1968, and has been desc...

By: University of Glasgow

Read more:
Guido Pontecorvo - Professor of Genetics 1955 to 1968 University of Glasgow. - Video

NASHVILLE, Tenn., Sept. 9, 2013 /PRNewswire/ -- NextGxDx Inc., which provides an online genetic testing marketplace for healthcare professionals and hospitals, today announced an agreement with genetic testing laboratory Cancer Genetics, Inc. (CGIX), to offer Cancer Genetics' genomic-based, proprietary oncology tests and services through the NextGxDx platform.

"Genetic testing within oncology is evolving at a tremendous rate," said NextGxDx CEO Mark Harris, PhD, MBA. "Our newest partner, Cancer Genetics, provides our growing marketplace with a robust catalog of more than 100 regulatory approved oncology testing products and solutions for easy search, comparison and ordering."

Cancer Genetics is an emerging leader in DNA-based cancer diagnostics and services for some of the most prestigious medical institutions in the world. The company is focused on transforming diagnosis and disease management for the most complex and difficult-to-treat cancers, including hematologic, urogenital, and gynecological malignancies.

"Our team takes tremendous pride in our focus on personalizing the diagnosis for cancer patients," said Panna Sharma, CEO of Cancer Genetics, Inc. "The NextGxDx platform supports our mission by enhancing our market reach while allowing us to focus on delivering critical genomic information through our clinical trial services and proprietary testing where patients and their physicians need it mostto diagnose, monitor and inform cancer treatment."

About NextGxDxNextGxDx, Inc. provides an online genetic testing marketplace that offers healthcare professionals and hospitals the ability to access up-to-date listings of all genetic tests from CLIA-certified laboratories, view pertinent information about each test, order tests online, and manage results electronically within the HIPAA-compliant portal.By providing clinicians with a one-stop-shop to search, compare and order genetic tests, NextGxDx reduces the time and costs associated with the ordering of genetic tests.For more information, visit http://www.NextGxDx.com.

About Cancer GeneticsCancer Genetics, Inc. is an emerging leader in DNA-based cancer diagnostics that personalizes the diagnosis and prediction of treatment outcomes for difficult to diagnose cancers. These cancers include hematological, urogenital and HPV-associated cancers. The Company's comprehensive range of oncology-focused tests and laboratory services provide critical genomic information to healthcare professionals, cancer centers, and to biopharma companies. Through its CLIA certified and CAP accredited state-of-the-art reference lab, Cancer Genetics servicessome of the most prestigious medical institutions in the world and has 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 seewww.cancergenetics.com.

Media Contact: Erin George 615-946-9914 erin@lovell.com

See the article here:
NextGxDx Adds Cancer Genetics, Inc. to Genetic Testing Platform