Archive for the ‘Gene Therapy Research’ Category

Gene therapy for back pain
Nine News, Melbourne, 6pm, 30/1/2014.

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6th Borough - Cell Therapy
Another Track That was in the Vault, Figure I Release it Since It #39;s Throwback Thursday They Say... Shouts Out to The Goodie Mob.

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Purtier Placenta Stem Cell Therapy Presented By Dr. Chen
Presentasi Purtier Placenta oleh Dr. Chen Nikmati hidup bebas rasa sakit dan selalu awet muda bersama Purtier Placenta: http://www.stemcellworld.net.

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Adipose Derived Cell Therapy

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Newswise NEW YORK (January 30, 2014) Steven Libutti, M.D., F.A.C.S., professor and vice chairman in the Department of Surgery at Montefiore Medical Center and Albert Einstein College of Medicine at Yeshiva University, director at the Montefiore Einstein Center for Cancer Care and professor in the Department of Genetics and associate director of clinical services at Albert Einstein College of Medicine has been named Editor-in-Chief of Cancer Gene Therapy, a journal for cancer researchers and clinicians.

Cancer Gene Therapy serves as a respected resource for scientists and clinicians on the latest gene and cellular therapies for cancer. Topics will range from DNA synthesis and repair to the latest original laboratory research and case reports on translational research and tumor immunotherapies.

The progression of personalized cancer treatments and evolution of new technologies for monitoring genetic changes holds new promise for those impacted by cancer, said Dr. Libutti. Cancer Gene Therapy will cover innovative scientific developments and prompt clinical questions that I hope spark new perspectives and industry dialogue about whats next.

As a pioneer in tumor-targeted gene therapy, Dr. Libutti is developing novel cancer therapies through his study of the complex interactions that occur within a tumors microenvironment. Since 2009, he has spearheaded multidisciplinary efforts in research and treatment of cancer at the Montefiore Einstein Center for Cancer Care.

We feel honored to be working in conjunction with an expert of Dr. Libuttis caliber, said Andrea Macaluso, publishing manager, Nature Publishing Group. His vast experience discovering new and effective ways to treat cancer combined with his record of excellent basic research and clinical care made him a natural choice to lead our journal and further strengthen Nature Publishing Groups library of scientific and medical publications.

Dr. Libutti is an internationally recognized expert in surgical oncology and endocrine surgery and has published more than 250 peer-reviewed journal articles and 16 oncology book chapters. He has received the NIH Directors Award and the National Cancer Institutes Directors Gold Star and Intramural Innovation Awards. Dr. Libutti also has appeared on Castle Connollys list of Top Doctors in America and on New York Magazines list of Top Doctors in New York. In 2009, he was named the Marvin L. Gliedman, M.D., Distinguished Surgeon at Montefiore Medical Center. Prior to being appointed director of the Montefiore Einstein Center for Cancer Care, Dr. Libutti was a researcher, surgeon and Section Head at the National Cancer Institute.

About Montefiore Medical Center As the University Hospital for Albert Einstein College of Medicine, Montefiore is a premier academic medical center nationally renowned for its clinical excellence, scientific discovery and commitment to its community. Recognized among the top hospitals nationally and regionally by U.S. News & World Report, Montefiore provides compassionate, patient- and family-centered care and educates the healthcare professionals of tomorrow. The Children's Hospital at Montefiore is consistently named in U.S. News' "America's Best Children's Hospitals." With four hospitals, 1,491 beds and 90,000 annual admissions, Montefiore is an integrated health system seamlessly linked by advanced technology. State-of-the-art primary and specialty care is provided through a network of more than 130 locations across the region, including the largest school health program in the nation and a home health program. Montefiore's partnership with Einstein advances clinical and translational research to accelerate the pace at which new discoveries become the treatments and therapies that benefit patients. The medical center derives its inspiration for excellence from its patients and community, and continues to be on the frontlines of developing innovative approaches to care. For more information please visit http://www.montefiore.org and http://www.montekids.org. Follow us on Twitter; like us on Facebook; view us on YouTube.

About Albert Einstein College of Medicine of Yeshiva University Albert Einstein College of Medicine of Yeshiva University is one of the nations premier centers for research, medical education and clinical investigation. During the 2013-2014 academic year, Einstein is home to 734 M.D. students, 236 Ph.D. students, 106 students in the combined M.D./Ph.D. program, and 353 postdoctoral research fellows. The College of Medicine has more than 2,000 full-time faculty members located on the main campus and at its clinical affiliates. In 2013, Einstein received more than $155 million in awards from the NIH. This includes the funding of major research centers at Einstein in diabetes, cancer, liver disease, and AIDS. Other areas where the College of Medicine is concentrating its efforts include developmental brain research, neuroscience, cardiac disease, and initiatives to reduce and eliminate ethnic and racial health disparities. Its partnership with Montefiore Medical Center, the University Hospital and academic medical center for Einstein, advances clinical and translational research to accelerate the pace at which new discoveries become the treatments and therapies that benefit patients. Through its extensive affiliation network involving Montefiore, Jacobi Medical Center Einsteins founding hospital, and five other hospital systems in the Bronx, Manhattan, Long Island and Brooklyn, Einstein runs one of the largest residency and fellowship training programs in the medical and dental professions in the United States. For more information, please visit http://www.einstein.yu.edu, read our blog, follow us on Twitter, like us on Facebook, and view us on YouTube.

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Montefiore Einstein Center for Cancer Care Director Named Editor-in-Chief of Cancer Gene Therapy

Jan 31, 2014 by Fred Love Amy Toth is one of several ISU faculty members working to understand the decline of pollinating insects. Credit: Bob Elbert.

Last spring, when Mary Harris started looking for particular pesticides in the pollen carried by honey bees in northwest Iowa, she didn't find any. But that changed the week tractors hit the fields to plant crops.

That week, every pollen sample she took tested positive for the presence of neonicotinoids, pesticides often used to coat seeds before they're planted.

Harris, an Iowa State University adjunct assistant professor of natural resource ecology and management, was part of a research team formed by the nonprofit Pollinator Partnership to monitor the level of neonicotinoid pesticides found in plant pollen collected by honey bees. The research, released on Thursday, indicates that the pesticides also contaminate nearby plants that are visited by a range of helpful pollinating insects.

Harris's effort to study pesticides is one thread in a patchwork of research at Iowa State to identify the factors that have led to steep declines in the populations of pollinating insects in Iowa and across the globe.

ISU faculty members have found several causes that likely lie at the heart of the problem, each one compounding the others.

"People want a single issue to blame it on, and that would be great because we could fix it," said Amy Toth, an assistant professor of ecology, evolution and organismal biology. "But it's not that simple."

Over the last five years, average annual winter losses among U.S. beekeepers have totaled about 30 percent, said Andrew Joseph, the state apiarist for the Iowa Department of Agriculture. That means that American beekeepers are losing almost a third of their bees each winter. Iowa beekeepers have seen even higher mortality, with average annual losses reaching 54.1 percent during that time period, Joseph said.

It's a growing crisis that could eventually drive up costs at grocery stores for a range of foods that can't reach store shelves without the help of pollinators.

Neonics on the wind

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Researchers are piecing together causes of decline in honey bees

Jan 31, 2014 by Jennie Matthew Swans are pictured on a lake in Slovakia on December 29, 2010

In Britain, wild swans may be prized for their beauty and protected by the Queen, but the US state of New York has declared war on them, branding them a violent menace.

Draft proposals to kill or resettle the state's 2,200 wild mute swans by 2025 may be supported by some conservationists but have sparked uproar among animal rights activists.

Mute swans were brought to North America by European settlers to adorn their estates in the late 1800s but the authorities no longer consider them a beauty worthy of roaming free.

The New York state department of environmental conservation says swans attack people, destroy vegetation, pose a threat to jetliners and damage water because their feces contain e coli.

Ever since US Airways flight 1549 collided with a flock of geese in 2009 and landed on the Hudson river, the US Department of Agriculture has set about annually culling Canada geese.

Now the New York state conservation department wants to expand the offensive and eliminate free-ranging mute swans by 2025, killing them or allowing "responsible ownership" of the birds in captivity.

"Lethal control methods will include shooting of free-ranging swans and live capture and euthanasia in accordance with established guidelines for wildlife," said the draft proposal.

Nests would also be destroyed, and eggs oiled, punctured or sterilized to prevent hatching, it added.

Pressure group Goose Watch NYC, which was set up to protest against the geese culls, demanded the plan be scrapped.

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New York declares war on swans

Jan 31, 2014 by Bob Yirka Mirabilis jalapa (the four o'clock flower). This plant is the host of Phytophthora mirabilis, the sister species of the Irish potato famine pathogen Phytophthora infestans. Credit: Sophien Kamoun, The Sainsbury Laboratory (Norwich, UK)

(Phys.org) A research team with members from the U.K., Germany and the U.S. has identified an amino acid sequence in effector proteins in two pathogens that helps explain how a firmly established pathogen can leap from one plant to another. In their paper published in the journal Science, the team describes genomic research they conducted on two types of well-known plant pathogens and what it revealed regarding effector proteins and their role in helping the pathogen jump from one plant species to another.

Understanding how plant pathogens jump from one plant to another can help prevent severe outbreaks such as the famous potato famine in Ireland back in the 19th century. Scientists suspect it as to do with evolutionary developments in effector proteinsthose proteins produced by a pathogen that disable pathogen fighting proteins in the host. In this new effort, the researchers looked at two particular types of pathogens, Phytophthora infestans and Phytophthora mirabilisthey are believed to have diverged from a single parent approximately 1,200 years agorecently enough to have left behind evidence of what caused them to go their separate ways.

P. infestans is the pathogen responsible for the potato famine. P. mirabilis on the other hand, is only able to infect a plant known as the four o'clock flower. To uncover what made the divergence possible, the researchers picked up where another team had left off back in 2010that team had found evidence of adaptive evolution in 82 effector genesthose responsible for the creation of effector proteins. The new team looked at just one of those genes and found that the proteins that were produced by each plantprotease inhibitorsbound more strongly to proteases from their host plants than to those of the other host plant, suggesting a genetic predisposition. That explained their ability to infect one host but not another.

In taking a closer look at the effector proteins, the researchers found just one single amino acid difference in the two pathogens. Thus it appears that the divergence of the two pathogens came down to a simple evolutionary development in a single amino acid. More striking perhaps was the realization that such a small difference between pathogens can mean the difference between which types of plants can be infectedjumping to another plant generally renders the pathogen unable to infect its original host.

Explore further: How an aggressive fungal pathogen causes mold in fruits and vegetables

More information: Effector Specialization in a Lineage of the Irish Potato Famine Pathogen, Science 31 January 2014: Vol. 343 no. 6170 pp. 552-555. DOI: 10.1126/science.1246300

Abstract Accelerated gene evolution is a hallmark of pathogen adaptation following a host jump. Here, we describe the biochemical basis of adaptation and specialization of a plant pathogen effector after its colonization of a new host. Orthologous protease inhibitor effectors from the Irish potato famine pathogen, Phytophthora infestans, and its sister species, Phytophthora mirabilis, which is responsible for infection of Mirabilis jalapa, are adapted to protease targets unique to their respective host plants. Amino acid polymorphisms in both the inhibitors and their target proteases underpin this biochemical specialization. Our results link effector specialization to diversification and speciation of this plant pathogen.

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Journal reference: Science

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Researchers identify amino acid change that allows pathogens to jump from one plant to another

Genetic research is focused on which specific genes are involved in the inflammatory process, immune response, and breakdown of cartilage. Understanding the genetic connection may lead to a cure or better treatment options for arthritis and related conditions.

Is Arthritis Hereditary? If a family member has arthritis, should you worry? Is arthritis hereditary?

HLA-B27 and HLA-DR4 - Arthritis and the Genetic Factor Genetics has become the focal point of new and ongoing arthritis research.

Senior Mobility Gene JAMA reports that the presence of a particular geotype that interacts with exercise to enhance mobility in seniors.

Clinical Trials: Gene Therapy Clinical trials for gene therapy, from ClinicalTrials.gov.

Genomics & Health Weekly Update This weekly update provides information about the impact of human genetic research on disease prevention and public health, from CDC.

The Human Genome Project (1990-2003) Completed in 2003, the Human Genome Project (HGP) was a 13-year project. Explore this site for information about the Human Genome Project (1990-2003), from ORNL.gov.

The New Genetics: National Institute of General Medical Sciences The New Genetics explains how genes affect your health. The New Genetics describes the basics of how DNA and RNA work. It also explains how studies of evolution drive medical research, how genes influence health and disease, and how computer science is advancing genetics in the 21st century.

NHGRI: The National Human Genome Research Institute The National Human Genome Research Institute led the Human Genome Project for the National Institutes of Health, which culminated in the completion of the full human genome sequence in April 2003. Now, NHGRI moves forward into the genomic era with research aimed at improving human health and fighting disease.

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31-Jan-2014

Contact: Julie O'Connor julie.oconnor@wayne.edu 313-577-8845 Wayne State University - Office of the Vice President for Research

DETROIT A research team led by Wayne State University, in collaboration with Michigan State University, has identified a single gene in honeybees that separates the queens from the workers.

The scientists unraveled the gene's inner workings and published the results in the current issue of Biology Letters. The gene, which is responsible for leg and wing development, plays a crucial role in the evolution of bees' ability to carry pollen.

"The gene Ultrabithorax, or Ubx is responsible for making hind legs different from fore legs so they can carry pollen" said Aleksandar Popadic, associate professor of biological sciences in Wayne State University's College of Liberal Arts and Science and principal investigator of the study. "In some groups, like crickets, Ubx is responsible for creating a 'jumping' hind leg. In others, such as bees, it makes a pollen basket a 'naked,' bristle-free leg region that creates a space for packing pollen."

"Other studies have shed some light on this gene's role in this realm, but our team examined in great detail how the modifications take place," added Zachary Huang, MSU entomologist.

Ubx represses the development of bristles on bees' hind legs, creating a smooth surface that can be used for packing pollen. This important discovery can be used as a foray into more commercial studies focused on providing means to enhance a bee's pollination ability the bigger the pollen basket, the more pollen that can be packed in it and transported back to the hive.

While workers have these distinct features, queens do not. The team confirmed this by isolating and silencing Ubx. This made the pollen baskets completely disappear, altered the growth of the pollen comb and reduced the size of the pollen press. Interestingly, Ubx is also expressed in the same region of the hind legs in bumble bees, which are in the same family as honey bees. This finding suggests that the evolution of the pollen-gathering apparatus in all corbiculate bees may have a shared origin and could be traced to the acquisition of novel functions by Ubx.

In another interesting finding, researchers identified that bees living in more complex social structures have an advantage over isolated populations in developing these important functions.

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Research led by Wayne State discovers single gene in bees separating queens from workers

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Newswise DETROIT A research team led by Wayne State University, in collaboration with Michigan State University, has identified a single gene in honeybees that separates the queens from the workers.

The scientists unraveled the genes inner workings and published the results in the current issue of Biology Letters. The gene, which is responsible for leg and wing development, plays a crucial role in the evolution of bees ability to carry pollen.

The gene Ultrabithorax, or Ubx is responsible for making hind legs different from fore legs so they can carry pollen said Aleksandar Popadic, associate professor of biological sciences in Wayne State Universitys College of Liberal Arts and Science and principal investigator of the study. In some groups, like crickets, Ubx is responsible for creating a jumping hind leg. In others, such as bees, it makes a pollen basket a naked, bristle-free leg region that creates a space for packing pollen.

Other studies have shed some light on this genes role in this realm, but our team examined in great detail how the modifications take place, added Zachary Huang, MSU entomologist.

Ubx represses the development of bristles on bees hind legs, creating a smooth surface that can be used for packing pollen. This important discovery can be used as a foray into more commercial studies focused on providing means to enhance a bees pollination ability the bigger the pollen basket, the more pollen that can be packed in it and transported back to the hive.

While workers have these distinct features, queens do not. The team confirmed this by isolating and silencing Ubx. This made the pollen baskets completely disappear, altered the growth of the pollen comb and reduced the size of the pollen press. Interestingly, Ubx is also expressed in the same region of the hind legs in bumble bees, which are in the same family as honey bees. This finding suggests that the evolution of the pollen-gathering apparatus in all corbiculate bees may have a shared origin and could be traced to the acquisition of novel functions by Ubx.

In another interesting finding, researchers identified that bees living in more complex social structures have an advantage over isolated populations in developing these important functions.

The pollen baskets are much less elaborate or completely absent in bees that are less socially complex, Huang said. We conclude that the evolution of pollen baskets is a major innovation among social insects and is tied directly to more complex social behaviors.

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Research Team Discovers Single Gene in Bees Separating Queens From Workers

PUBLIC RELEASE DATE:

31-Jan-2014

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

New Rochelle, NY, January 31, 2014In recognition of his seminal work on adenoviral vectors, which accelerated the translation of gene therapy from the research laboratory to the clinic, Ronald G. Crystal, MD (Weill Cornell Medical College, Cornell University, New York City), has received a Pioneer Award from Human Gene Therapy, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers. Human Gene Therapy is commemorating its 25th anniversary by bestowing this honor on the leading 12 Pioneers in the field of cell and gene therapy selected by a blue ribbon committee* and publishing a Pioneer Perspective by each of the award recipients. The article by Dr. Crystal is available on the Human Gene Therapy website.

Currently it is standard practice to use a modified virus as a transport vehicle to deliver therapeutic genes to patients. But this concept was new, innovative, and technically challenging when Dr. Crystal began developing the molecular tools and methods in the late 1980s. In the Pioneer Perspective "Adenovirus: The First Effective In Vivo Gene Delivery Vector," Dr. Crystal provides historical insights on the many years of research and testing needed to design, optimize, manufacture, and evaluate the performance of adenoviral vectors. He describes the first in vivo studies, the first human studies, and the many current applications of this useful gene delivery system.

"Ron led the way in the clinical translation of adenoviral vectors in the very early days of gene therapy," says James M. Wilson, MD, PhD, Editor-in-Chief of Human Gene Therapy, and Director of the Gene Therapy Program, Department of Pathology and Laboratory Medicine, University of Pennsylvania Perelman School of Medicine, Philadelphia.

###

*The blue ribbon panel of leaders in cell and gene therapy, led by Chair Mary Collins, PhD, MRC Centre for Medical Molecular Virology, University College London, selected the Pioneer Award recipients. The Award Selection Committee selected scientists that had devoted much of their careers to cell and gene therapy research and had made a seminal contribution to the field--defined as a basic science or clinical advance that greatly influenced progress in translational research.

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 journals, Human Gene Therapy Methods, published bimonthly, focuses on the application of gene therapy to product testing and development, and Human Gene Therapy Clinical Development, published quarterly, features data relevant to the regulatory review and commercial development of cell and gene therapy products. Tables of content for all three publications and a free sample issue may be viewed on the Human Gene Therapy website.

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Ronald Crystal, M.D., receives Pioneer Award

MineCraft Modded Survival Cac Tech #4 Advanced Genetics

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Cancer Genetics, Inc. (NASDAQCM: CGIX): RedChip Emerging Growth Showcase (January 22-23, 2014)
Virtual Conference Presentation and Investor Q A with Panna Sharma, CEO of Cancer Genetics, Inc. (NASDAQCM: CGIX), an emerging leader in the field of persona...

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Cancer Genetics, Inc. (NASDAQCM: CGIX): RedChip Emerging Growth Showcase (January 22-23, 2014) - Video

The Biology of Genetics
Learn what goes in to making you, you.

By: Bill Nye

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The Biology of Genetics - Video

Stretch Goal Cast - Episode #4: Jurassic Genetics, Divinity: Original Sin The Castle Doctrine
Your SGC podcasting crew shrinks to three this week with Jared, Justin and Clinton presiding over the main topics. Jared kicks things off with some genuine, ...

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Stem Cell Therapy: Non-Surgical Treatment for Neck Pain Whiplash
An informative guide to how Platelet Rich Plasma can heal the tough minority of whiplash cases where traditional treatments do not offer significant relief. For more information, visit http://www.stemcell...

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Stem Cell Therapy: Non-Surgical Treatment for Neck Pain & Whiplash - Video

Stress before conception can cause changes to a woman's eggs These changes can lead to genetic differences in her future children Even stress during the woman's teens can affect her future offspring

By Emma Innes

PUBLISHED: 10:56 EST, 31 January 2014 | UPDATED: 10:58 EST, 31 January 2014

A woman's levels of stress even before conception can influence her child's ability to deal with stressful situations

All women know that their lifestyle during pregnancy can have important effects on their childs future health.

Now research suggests that a womans levels of stress even before conception can influence her childs ability to deal with stressful situations.

Research has shown that stress before conception can cause genetic changes to children because it can cause changes in the mother-to-bes eggs.

The research, conducted by the University of Haifa, in Israel, was carried out on rats but scientists believe the conclusions can be applied to humans.

Researcher Hiba Zaidan said: The systemic similarity in many instances between us and rats raises questions about the transgenerational influences in humans as well.

If until now we saw evidence only of behavioural effects, now weve found proof of effects at the genetic level.

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A joint research team led by an NUI Galwayscientist has found that changes in a little-known gene called ULK4 were observed in individuals with schizophrenia.

A rare risk factor which is associated with mental illnesses like schizophrenia has been identified by a joint research team led by an NUI Galway (NUIG) scientist.

The research team has found that changes in a little-known gene called ULK4 were observed in individuals with schizophrenia.

The findings are published today in the Journal of Cell Science.

Prof Sanbing Shen of NUIGs Regenerative Medicine Institute, who led the research, says that this could contribute to more effective treatment of the condition in time.

The multi-institutional study examined a database of up to 7,000 people, half of whom had schizophrenia and half of whom did not.

Many genetic risk factors have been associated with schizophrenia and other mental illnesses such as bipolar disorder and depression, but Prof Shen and his team were able to characterise how the ULK4 gene functions in the brain.

He and his colleagues found that when levels of ULK4 were decreased, through mutation or deletion, the neuronal (brain) cells tend to function less well.

This leads to reduced synaptic function and other changes that are also known as risk factors of schizophrenia.

Prof Shen said ULK4 is essential for the formation of the nerve fibres which connect the two sides of the brain.

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Risk factor linked to schizophrenia identified by NUI Galway scientist

Macaques in China are the first primates born with genomes engineered by precision gene-targeting methods.

Prime cuts: The faint ladder-rung patterns in an image of a DNA gel show that genome editing successfully modified a gene in two macaque infants (central columns), but not in an untreated animal (right column). The left column shows a size standard.

Researchers at Nanjing Medical University and Yunnan Key Laboratory of Primate Biomedical Research in Kunming, China, have created genetically modified monkeys using a new method of DNA engineering known as Crispr. The infant macaques show that targeted genome editing is feasible in primatesa potential boon for scientists studying complex diseases, including neurological ones, and an advance that suggests that the method could one day work in humans. The work was reported in the journal Cell on Thursday.

Scientists have previously used the new genome-editing technique to delete, insert, and modify DNA in human cells and other animal cells grown in petri dishes. The method has also been used to create gene modifications in whole animals such as mice, rats, and zebrafish. The new study shows for the first time that Crispr can create viable primates with genomes modified at specific targeted genes.

The Chinese researchers injected single-cell macaque embryos with RNAs to guide the genome-editing process. The team modified three genes in the monkeys: one that regulates metabolism, another that regulates immune cell development and a third that regulates stem cells and sex determination, says study coauthor Wezhi Ji, a researcher at the Yunnan Key Laboratory of Primate Biomedical Research. The researchers found that the genome-editing tools created multiple changes in their target genes at different stages of embryonic development. The infant monkeys are too young for the team to yet determine if the genetic changes have an effect on physiology or behavior, says Ji. But, he adds, data from this species should be very useful for curing human disease and improving human health.

Researchers have previously created a handful of transgenic monkeys, such as a rhesus macaque that produces the disease-causing version of theHuntingtons gene. Researchers at Emory University in Atlanta created this avatar of human disease by injecting a virus into macaque eggs. The virus delivered a disease-version of the human Huntingtons gene into a random location in the monkeys genome.

Primate pioneers: Twin infant macaques whose genomes were modified within three different genes.

Crispr, on the other hand, can be used to insert, delete, or rewrite a DNA sequence at a specific location within a genome. Like the random viral insertion used by the Emory team, the Crispr method employed by Ji and colIeagues can create genetically modified animals in a single generation, an important consideration for researchers working with animals that can take three years to reach sexual maturity and are expensive and difficult to rear.

Others say they are anxious to use Crispr to create their own monkeys. Robert Desimone, director of MITs McGovern Brain Institute for Brain Research, says he and colleagues are planning on using genome editing to create modified monkeys. He says its possible the success of the Chinese researchers will encourage other groups to use primates in their work. Although mice are giving us tremendous insight into basic brain biology and the biology of the disease, theres still a big gap in between the mouse brain and the monkey brain, says Desimone.

For example, he says, lots of drugs that work in mice to treat disease dont work in humans. Desimone says hes hoping that some success in monkeys will interest drug companies in neurosciencealluding to a recent trend of large drug companies abandoning research on brain diseases because the work often proved unsuccessful. The hope is that disease and drug research in monkeys will more likely lead to therapies in humans because the primates share complex behaviors and social structures. We are cautiously optimistic, says Desimone.

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Monkeys Modified with Genome Editing

Niu et al., Cell

Twin cynomolgus monkeys born in China are the first with mutations in specific target genes.

The ultimate potential of precision gene-editing techniques is beginning to be realised. Today, researchers in China report the first monkeys engineered with targeted mutations1, an achievement that could be a stepping stone to making more realistic research models of human diseases.

Xingxu Huang, a geneticist at the Model Animal Research Center of Nanjing University in China, and his colleagues successfully engineered twin cynomolgus monkeys (Macaca fascicularis) with two targeted mutations using the CRISPR/Cas9 system a technology that has taken the field of genetic engineering by storm in the past year. Researchers have leveraged the technique to disrupt genes in mice and rats2, 3, but until now none had succeeded in primates.

"This is an important step," says Feng Zhang, a synthetic biologist who was not involved in the study, but who has helped to develop CRISPR technology at the Massachusetts Institute of Technology in Cambridge. "It shows that the system is working."

Transgenic mice have long dominated as models for human diseases, in part because scientists have honed a gene-editing method for the animals that uses homologous recombination rare, spontaneous DNA-swapping events to introduce mutations. The strategy works because mice reproduce quickly and in large numbers, but the low rates of homologous recombination make such a method unfeasible in creatures such as monkeys, which reproduce slowly.

"We need some non-human primate models," says Hideyuki Okano, a stem-cell biologist at Keio University in Tokyo. Human neuropsychiatric disorders can be particularly difficult to replicate in the simple nervous systems of mice, he says.

Previous attempts to genetically modify primates have relied on viral methods4, 5, which create mutations efficiently, but at unpredictable locations and in uncontrolled numbers. Prospects for primates brightened with the emergence of the CRISPR/Cas9 gene-editing system, which uses customizable snippets of RNA to guide the DNA-cutting enzyme Cas9 to the desired mutation site.

Huang and his team first tested the technology in a monkey cell line, disrupting each of three genes with 1025% success. Encouraged, the scientists subsequently targeted the three genes simultaneously in more than 180 single-celled monkey embryos. Ten pregnancies resulted from 83 embryos that were implanted, one of which led to the birth of a pair with mutations in two genes: Ppar-, which helps to regulate metabolism, and Rag1, which is involved in healthy immune function.

Stem-cell researcher Rudolf Jaenisch of the Whitehead Institute for Biomedical Research in Cambridge, Massachusetts, calls the result an interesting demonstration, but says that it offers little scientific insight. "The next step is to see if we can learn anything from it," says Jaenisch, who pioneered the use of transgenic mice in the 1970s.

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First monkeys with customized mutations born

PARIS, Jan. 31 (UPI) -- Female development in the fetus is activated by the FOXL2 gene, blocking male gene expression, meaning the default of sex of a fetus is not female.

Researchers at the INRA, in Paris, France, have identified the FOXL2 gene as responsible for female differentiation.

Females were long believed to be the "default" sex of a fetus, with a gene on the Y chromosome leading to male differentiation. But in some cases an XX fetus, programmed to be female, fails to develop ovaries and instead is born with male characteristics.

On analyzing the genes of such fetuses, researches identified the FOXL2 gene, which acts like a "defender of the ovary," silencing the male genes as the ovary develops and well into adulthood. These findings have been published in the journal Current Biology.

Using goat embryos, researchers silenced the FOXL2 gene and witnessed that XX fetuses developed testes instead of ovaries, explaining the development of male characteristics in XX fetuses.

The FOXL2 gene had previously been linked to premature menopause in young women. Techniques are being developed based on the goat model to treat certain types of infertility.

[INRA] [Current Biology]

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Female differentiation in the fetus is not default, has to be activated

A central question has been answered regarding a protein that plays an essential role in the bacterial immune system and is fast becoming a valuable tool for genetic engineering. A team of researchers with the Lawrence Berkeley National Laboratory (Berkeley Lab) and the University of California (UC) Berkeley have determined how the bacterial enzyme known as Cas9, guided by RNA, is able to identify and degrade foreign DNA during viral infections, as well as induce site-specific genetic changes in animal and plant cells. Through a combination of single-molecule imaging and bulk biochemical experiments, the research team has shown that the genome-editing ability of Cas9 is made possible by the presence of short DNA sequences known as "PAM," for protospacer adjacent motif.

"Our results reveal two major functions of the PAM that explain why it is so critical to the ability of Cas9 to target and cleave DNA sequences matching the guide RNA," says Jennifer Doudna, the biochemist who led this study. "The presence of the PAM adjacent to target sites in foreign DNA and its absence from those targets in the host genome enables Cas9 to precisely discriminate between non-self DNA that must be degraded and self DNA that may be almost identical. The presence of the PAM is also required to activate the Cas9 enzyme."

With genetically engineered microorganisms, such as bacteria and fungi, playing an increasing role in the green chemistry production of valuable chemical products including therapeutic drugs, advanced biofuels and biodegradable plastics from renewables, Cas9 is emerging as an important genome-editing tool for practitioners of synthetic biology.

"Understanding how Cas9 is able to locate specific 20-base-pair target sequences within genomes that are millions to billions of base pairs long may enable improvements to gene targeting and genome editing efforts in bacteria and other types of cells," says Doudna who holds joint appointments with Berkeley Lab's Physical Biosciences Division and UC Berkeley's Department of Molecular and Cell Biology and Department of Chemistry, and is also an investigator with the Howard Hughes Medical Institute (HHMI).

Doudna is one of two corresponding authors of a paper describing this research in the journal Nature. The paper is titled "DNA interrogation by the CRISPR RNA-guided endonuclease Cas9." The other corresponding author is Eric Greene of Columbia University. Co-authoring this paper were Samuel Sternberg, Sy Redding and Martin Jinek.

Bacterial microbes face a never-ending onslaught from viruses and invasive snippets of nucleic acid known as plasmids. To survive, the microbes deploy an adaptive nucleic acid-based immune system that revolves around a genetic element known as CRISPR, which stands for Clustered Regularly Interspaced Short Palindromic Repeats. Through the combination of CRISPRs and RNA-guided endonucleases, such as Cas9, ("Cas" stands for CRISPR-associated), bacteria are able to utilize small customized crRNA molecules (for CRISPR RNA) to guide the targeting and degradation of matching DNA sequences in invading viruses and plasmids to prevent them from replicating. There are three distinct types of CRISPR-Cas immunity systems. Doudna and her research group have focused on the Type II system which relies exclusively upon RNA-programmed Cas9 to cleave double-stranded DNA at target sites.

"What has been a major puzzle in the CRISPR-Cas field is how Cas9 and similar RNA-guided complexes locate and recognize matching DNA targets in the context of an entire genome, the classic needle in a haystack problem," says Samuel Sternberg, lead author of the Nature paper and a member of Doudna's research group. "All of the scientists who are developing RNA-programmable Cas9 for genome engineering are relying on its ability to target unique 20-base-pair long sequences inside the cell. However, if Cas9 were to just blindly bind DNA at random sites across a genome until colliding with its target, the process would be incredibly time-consuming and probably too inefficient to be effective for bacterial immunity, or as a tool for genome engineers. Our study shows that Cas9 confines its search by first looking for PAM sequences. This accelerates the rate at which the target can be located, and minimizes the time spent interrogating non-target DNA sites."

Doudna, Sternberg and their colleagues used a unique DNA curtains assay and total internal reflection fluorescence microscopy (TIRFM) to image single molecules of Cas9 in real time as they bound to and interrogated DNA. The DNA curtains technology provided unprecedented insights into the mechanism of the Cas9 target search process. Imaging results were verified using traditional bulk biochemical assays.

"We found that Cas9 interrogates DNA for a matching sequence using RNA-DNA base-pairing only after recognition of the PAM, which avoids accidentally targeting matching sites within the bacterium's own genome," Sternberg says. "However, even if Cas9 somehow mistakenly binds to a matching sequence on its own genome, the catalytic nuclease activity is not triggered without a PAM being present. With this mechanism of DNA interrogation, the PAM provides two redundant checkpoints that ensure that Cas9 can't mistakenly destroy its own genomic DNA."

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Puzzling question in bacterial immune system answered

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Short DNA sequences known as PAM (shown in yellow) enable the bacterial enzyme Cas9 to identify and degrade foreign DNA, as well as induce site-specific genetic changes in animal and plant cells. The presence of PAM is also required to activate the Cas9 enzyme. (Illustration by KC Roeyer.)

A central question has been answered regarding a protein that plays an essential role in the bacterial immune system and is fast becoming a valuable tool for genetic engineering. A team of researchers with the Lawrence Berkeley National Laboratory (Berkeley Lab) and the University of California (UC) Berkeley have determined how the bacterial enzyme known as Cas9, guided by RNA, is able to identify and degrade foreign DNA during viral infections, as well as induce site-specific genetic changes in animal and plant cells. Through a combination of single-molecule imaging and bulk biochemical experiments, the research team has shown that the genome-editing ability of Cas9 is made possible by the presence of short DNA sequences known as PAM, for protospacer adjacent motif.

Our results reveal two major functions of the PAM that explain why it is so critical to the ability of Cas9 to target and cleave DNA sequences matching the guide RNA, says Jennifer Doudna, the biochemist who led this study. The presence of the PAM adjacent to target sites in foreign DNA and its absence from those targets in the host genome enables Cas9 to precisely discriminate between non-self DNA that must be degraded and self DNA that may be almost identical. The presence of the PAM is also required to activate the Cas9 enzyme.

With genetically engineered microorganisms, such as bacteria and fungi, playing an increasing role in the green chemistry production of valuable chemical products including therapeutic drugs, advanced biofuels and biodegradable plastics from renewables, Cas9 is emerging as an important genome-editing tool for practitioners of synthetic biology.

Understanding how Cas9 is able to locate specific 20-base-pair target sequences within genomes that are millions to billions of base pairs long may enable improvements to gene targeting and genome editing efforts in bacteria and other types of cells, says Doudna who holds joint appointments with Berkeley Labs Physical Biosciences Division and UC Berkeleys Department of Molecular and Cell Biology and Department of Chemistry, and is also an investigator with the Howard Hughes Medical Institute (HHMI).

Jennifer Doudna and Samuel Sternberg used a combination of single-molecule imaging and bulk biochemical experiments to show how the RNA-guided Cas9 enzyme is able to locate specific 20-base-pair target sequences within genomes that are millions to billions of base pairs long. (Photo by Roy Kaltschmdit)

Doudna is one of two corresponding authors of a paper describing this research in the journal Nature. The paper is titled DNA interrogation by the CRISPR RNA-guided endonuclease Cas9. The other corresponding author is Eric Greene of Columbia University. Co-authoring this paper were Samuel Sternberg, Sy Redding and Martin Jinek.

Bacterial microbes face a never-ending onslaught from viruses and invasive snippets of nucleic acid known as plasmids. To survive, the microbes deploy an adaptive nucleic acid-based immune system that revolves around a genetic element known as CRISPR, which stands for Clustered Regularly Interspaced Short Palindromic Repeats. Through the combination of CRISPRs and RNA-guided endonucleases, such as Cas9, (Cas stands for CRISPR-associated), bacteria are able to utilize small customized crRNA molecules (for CRISPR RNA) to guide the targeting and degradation of matching DNA sequences in invading viruses and plasmids to prevent them from replicating. There are three distinct types of CRISPRCas immunity systems. Doudna and her research group have focused on the Type II system which relies exclusively upon RNA-programmed Cas9 to cleave double-stranded DNA at target sites.

What has been a major puzzle in the CRISPRCas field is how Cas9 and similar RNA-guided complexes locate and recognize matching DNA targets in the context of an entire genome, the classic needle in a haystack problem, says Samuel Sternberg, lead author of the Nature paper and a member of Doudnas research group. All of the scientists who are developing RNA-programmable Cas9 for genome engineering are relying on its ability to target unique 20-base-pair long sequences inside the cell. However, if Cas9 were to just blindly bind DNA at random sites across a genome until colliding with its target, the process would be incredibly time-consuming and probably too inefficient to be effective for bacterial immunity, or as a tool for genome engineers. Our study shows that Cas9 confines its search by first looking for PAM sequences. This accelerates the rate at which the target can be located, and minimizes the time spent interrogating non-target DNA sites.

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Running with genetic scissors: how a breakthrough technology works

A new gene-editing technique could lead to more useful animal models of disease, and perhaps one day more effective gene therapy for humans

Genetically modified long-tailed macaques. Credit: Cell, Niu et al.

Like many babies, the wide-eyed twins are cute. The fact that they are macaque monkeys is almost beside the point. What is not beside the point, however, is their genetic heritage. These baby macaques are, as reported inCell, the first primates to have been genetically modified using an extremely precise gene-editing tool based on the so-called CRISPR/Cas system.

Conducted by researchers in China, the new study is significant because it paves the way for the custom development of laboratory monkeys with genetic profiles that are similar to those found in humans with certain medical disorders. Although mice and rats have long been the animals of choice when creating living models of human disease, they have not been very helpful for studying neurological conditions such as autism and Alzheimers disease; the differences between rodent and human brains are just too great.

To be sure, a few other genetically modified monkeys have been born over the past decade and a half, but the methods used to alter their DNA were not as efficient or as easy to use as the CRISPR/Cas technology. The amount of genome engineering in monkeys is pretty small, says George Church, a professor of genetics at Harvard Medical School.So yes, this [paper] is a pretty big deal.

CRISPR stands for clustered regularly interspaced short palindromic repeats and refers to what at first glance appear to be meaningless variations and repeats in the sequence of molecular letters (A, T, C and G) that make up DNA. These CRISPR patterns are found in many bacteria and most archaea (an ancient group of bacteria that is now considered to be different enough from other one-celled organisms to merit is own taxonomic kingdom, along with bacteria, protists, fungi, plants and animals).

First identified in bacteria in 1987, CRISPR elements started being widely used to create genetic engineering tools only in 2013. It took that long to figure out that the patterns actually served a purpose, determine out what that purpose washelping archaea and bacteria to recognize and defend themselves against virusesand then adapt that original function to a new goal.

Basically, biologists learned that certain proteins associated with the CRISPR system (dubbed, straightforwardly enough, CRISPR-associated, or Cas, proteins) act like scissors that cut any strands of DNA they come across. These cutting proteins, in turn, are guided to specific strands of DNA by complementary pieces of RNA (a sister molecule to DNA). The bacteria generate specific guide strands of RNA whenever they encounter a virus that is starting to hijack their cellular machinery. The guide-RNA complements the viral DNA, which is how the Cas proteins know where to cut. The bacteria then keep a copy of the viral DNA in their own genetic sequence between two CRISPR elements for future reference in case a similar virus tries to cause trouble later on.

In the past couple of years researchers have learned how to trick the Cas proteins into targeting and slicing through a sequence of DNA of their own choosing. By developing strands of RNA that precisely complement the part of the DNA molecule that they want to change, investigators can steer the Cas proteins to a predesignated spot and cut out enough genetic material to permanently disrupt the usual expression of the DNA molecule at that location.

In essence, scientists have turned a bacterial self-defense mechanism into an incredibly precise gene-editing tool. By some accounts CRISPR technology has been successfully tried out on 20 different kinds of higher organisms (meaning higher than bacteria) in just the past year or so.

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New Biotech Makes It Much Easier to Genetically Modify Monkeys