Archive for the ‘Gene Therapy Research’ Category

Manila (Philippine Daily Inquirer/ANN) -

Hospitals offering stem cell therapy have until Aug. 31 to seek or renew their accreditation from the Department of Health (DOH), a Philippine official said Friday.

"For the information of the public, the DOH is accrediting hospitals for this kind of treatment, and come Aug. 31, these hospitals should file their accreditation requirements (with DOH) for them to continue to offer this treatment," said deputy presidential spokesperson Abigail Valte.

The government is eyeing stricter regulation of hospitals offering stem cell therapy amid speculations that the recent deaths of three politicians were due to the xenogenic (animal-based stem cell) treatment they had received in Germany last year.

Dr. Leo Olarte, president of the Philippine Medical Association (PMA) and spokesperson of the Philippine Society for Stem Cell Medicine, said the groups were still trying to determine whether the politicians had died due to their illness or due to hypersensitivity reaction from the xenogenic stem cells.

Last week, the PMA also warned of a possible scam involving German doctors coming over to perform stem cell therapy on patients in five-star hotels at around 1 million pesos (US$23,000) per shot.

Valte echoed a similar warning from the DOH against doctors offering the procedure in their clinics, saying that "hospitals, not (individual) doctors, nor stand-alone clinics, are the ones being accredited."

Not a 'cure all'

The Palace official also cautioned the public against claims that stem cell therapy was a "cure all" (for diseases).

"There is no treatment that will cure all of your ills. Much less your love problems," Valte said.

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Philippine gov't to tighten rules on stem cell therapy

By Rima Jessamine Granali Philippine Daily Inquirer

Now she can literally walk a mile.

Lawyer Charito Planas, 83, used to have a hard time getting up. But she can now walk without pain, thanks to stem cell therapy that cost her only five figures.

While hospitals here and abroad charge millions for the treatment, a wellness center in Tagaytay offers the service 10 times cheaper, Planas said.

The cane I used to walk with is just an accessory now, for I walk freely, without pain, without limping, said Planas, a political and human rights activist who sought refuge in the United States during the Marcos martial law administration.

My heavily grey hair has now regained its ebony shade; my skinboth body and facial skinhas regained a youthful, shining glow; and my wrinkles are slowly fading away, she added.

Planas underwent six sessions of stem cell therapy from March to April at Green and Young Health and Wellness Center, owned by Dr. Antonia Carandang Park.

VIP patients

Former President Gloria Macapagal-Arroyo reportedly visited the Filipino physician a day after she was released from detention at Veterans Memorial Medical Center (VMMC) in Quezon City after posting a P1-million bail in July last year. She was taken back to VMMC in October.

Other well-known personalities, such as justices, physicians and a senator, also reportedly frequented the wellness center.

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The prime of Charito Planas, 83, after stem cell therapy on the cheap

By: Jesus G. Dureza Posted: July 1, 2013 8:28 am

Over the last few days, Fresh Stem Cell Therapy (FCT) got into the headlines due to some controversy involving alleged deaths and complaints mentioning prominent names blaming stem cell treatment for their alleged negative effects.Naturally, just like any controversy, the stories unduly put under cloud this innovative and pioneering science of regenerative medicine that is reputed to treat illnesses, bring well being and rejuvenation.

MY FRIENDS The current news reports caught public attention and went viral. Many friends who were following Beths FCT experience and who were planning to also undergo FCT were calling. They were bothered by the reports. I was myself personally disturbed because all of them who were mentioned as having experienced negative results, I knew in person. Congressman ERICO AUMENTADO of Bohol and Camiguin Congressman LOLOY ROMUALDO both were my close friends and whom I worked with when I was with the government. And the controversy all the more struck me because I and my wife Beth saw and experienced for ourselves the positive NOT NEGATIVE results of FCT seven months after treatment at VILLA MEDICA in Germany.

Let me trace back. I knew Congressman Erico well even when he was still Bohol Governor. His successful efforts in clearing his province of armed insurgency gained national attention. When I was Malacanang Presidential Peace Adviser, I used the Bohol experience as example of a success story. So, when initial reports surfaced about FCT having caused his death, I sat up and took special attention. Later, his son came out publicly DENYING the story saying his father died of pneumonia and NOT due to FCT. He said his fathers lungs were already in serious condition but after getting a first infusion of FCT, his condition suddenly and dramatically improved and he was hitting the election campaign trail like superman. In fact they were preparing to again return to Germany for another infusion encouraged by its initial favorable results when death intervened and overtook events.

The other prominent name mentioned with FCT was former Congressman Loloy. We were together as colleagues in Congress. The Romualdo family had dominated politically the Camiguin island province for a long time up to the present. The circumstances of his undergoing stem cell treatment , however, are not yet clear. My cursory check results showed that he did NOT get FCT from Villa Medica in Germany. This will have to be further validated though.

The other prominent name was former Customs official (and now with Dangerous Drugs Board) ANTONIO BEBOT VILLAR. We were together in the Arroyo administration. He said he and his wife had FCT in a 5-star hotel in Metro Manila, by German and Thai doctors but NOT in Germany. His condition allegedly worsened after the treatment. MY OWN STORY I will not venture to further deal on those incidents simply because they are now subject of further close look by the authorities. And I am not privy with the true facts surrounding their cases. But I will deal on some information that are of my own personal knowledge and the personal experience I and my wife BETH can competently attest to.

As I have written previously, I and my wife Beth traveled all the way to EDENKOBEN, GERMANY last November, 2012 some seven (7) months ago for FCT in the VILLA MEDICA (VM) clinic facilities as an alternative remedy to her deteriorating kidney condition. We were NOT assured that there would be positive results by VM representatives but we took the chance after I made extensive research and consultations with doctors and experts. We even traveled to Bangkok, Thailand to make further inquiries. We were encouraged by reports of well-being and regenerative effects on vital organs. And we took efforts to talk to those who themselves underwent the procedure. We gathered that VILLA MEDICA and its FCT, using fresh stem cell from the fetus of clinically controlled and raised sheep had been recognized, authorized, licensed and closely monitored by stringent standards imposed by GERMAN HEALTH AUTHORITIES of the German government. VILLA MEDICA, founded in 1963 had also a long track record of about 50 YEARS. Fresh cell therapy was pioneered by Dr PAUL NIEHANS in 1931. Another doctor in 1980, GUNTER BIOBEL scientifically dissected the technology and was awarded in 1999 the Nobel Prize in Physiology or Medicine.

After some soul-searching and weighing our options, we decided and took the trouble of traveling to Germany at some cost even when we were told that there were stem cell treatments also being done in the Philippines, at more considerable costs, although still in their infancy stage. We had no way of verifying the reliability of local Philippine FCT providers due to their fairly recent track record. When I checked with St. Lukes Hospital Regenerative division at Global City, the division chief told me they were into this procedure: harvesting human stem cells from the patients themselves, usually from the bone marrow, churn them for 2 weeks in the laboratory using German-made machines and injecting them back to the patient over a 12 month period. However she declined to cite or give an example of a success story simply because they had been into the procedure fairly recently. Also, we were informed that to achieve optimum results, fresh stem cells must be injected (in the buttocks) within two hours from its harvesting from the donor fetus. So getting the FCT infusion right there in the clinic in Germany was far better than having the injectables flown from Germany or elsewhere and get the infusion in Manila, travel time obviously reducing the stem cells efficacy or potency. More importantly, seven months ago, there were NO clear Philippine regulations yet governing FCT, due to its infancy, to give us some level of reasonable comfort and assurance. On the other hand, the procedure in Germany we were told adhered to strictest German standards imposed by the German government. These regulations even cover the growing and maintenance of donor sheep and the harvesting of stem cells from 18-week old fetus under clinical control and conditions. And specific success stories were many.

And more importantly, we were in a desperate mode due to the continuous decline of Beths kidney conditions. As preparatory steps, Beth went through a series of tests which were screened and studied by her doctors in Manila and even through German doctors in Villa Medica in Edenkoben who had to make the final decision whether she was qualified to get the treatment. Even her Nephrologist who happens to be also a family friend and a known kidney transplant surgeon at the National Kidney Institute recommended that we try FCT. That sealed our decision.

So, off we went to Germany last November, 2012. Of course at great expense. (Treatment costs EURO 15,000 or about 830,000 PHP ) But nonetheless. AFTER 7 MONTHS Today, seven months after the treatment, and in contrast to the sketchy stories surrounding the cases of my friends ERICO, LOLOY and BEBOT as reported in the media, our own testimonials of the FCT at VILLA MEDICA are a bit different from those horror stories. I also personally met and discussed with others who themselves went to the German facility for FCT and our testimonials coincided.

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FCT Controversy: MY STEM CELL THERAPY STORY

Researchers at UCLA's Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research have successfully established the foundation for using hematopoietic (blood-producing) stem cells from the bone marrow of patients with sickle cell disease to treat the disease. The study was led by Dr. Donald Kohn, professor of pediatrics and of microbiology, immunology and molecular genetics.

Sickle cell disease causes the body to produce red blood cells that are formed like the crescent-shaped blade of a sickle, which hinders blood flow in the blood vessels and deprives the body's organs of oxygen.

Kohn introduced an anti-sickling gene into the hematopoietic stem cells to capitalize on the self-renewing potential of stem cells and create a continual source of healthy red blood cells that do not sickle. The breakthrough gene therapy technique for sickle cell disease is scheduled to begin clinical trials by early 2014. The study was published online today ahead of press in the Journal of Clinical Investigation.

Kohn's gene therapy approach, which uses hematopoietic stem cells from a patient's own blood, is a revolutionary alternative to current sickle cell disease treatments as it creates a self-renewing normal blood cell by inserting a gene that has anti-sickling properties into hematopoietic stem cells. This approach also does not rely on the identification of a matched donor, thus avoiding the risk of rejection of donor cells. The anti-sickling hematopoietic stem cells are transplanted back into the patient's bone marrow and multiply the corrected cells that make red blood cells without sickling.

"The results demonstrate that our technique of lentiviral transduction is capable of efficient transfer and consistent expression of an effective anti-sickling beta-globin gene in human sickle cell disease bone marrow progenitor cells, which improved the physiologic parameters of the resulting red blood cells," Kohn said.

Kohn and colleagues found that in the laboratory the hematopoietic stem cells produced new non-sickled blood cells at a rate sufficient for significant clinical improvement for patients. The new blood cells survive longer than sickled cells, which could also improve treatment outcomes.

Sickle cell disease mostly affects people of Sub-Saharan African descent, and more than 90,000 patients in the U.S. have been diagnosed. It is caused by an inherited mutation in the beta-globin gene that makes red blood cells change from their normal shape, which is round and pliable, into a rigid, sickle-shaped cell. Normal red blood cells are able to pass easily through the tiniest blood vessels, called capillaries, carrying oxygen to organs such as the lungs, liver and kidneys. But due to their rigid structure, sickled blood cells get stuck in the capillaries.

Current treatments include transplanting patients with donor hematopoietic stem cells, which is a potential cure for sickle cell disease, but due to the serious risks of rejection, only a small number of patients have undergone this procedure and it is usually restricted to children with severe symptoms.

This study was supported in part by a Disease Team I Award from the California Institute for Regenerative Medicine, the state's stem cell research agency, which was created by a voter initiative in 2004. The purpose of the disease team program is to support research focused on one particular disease that leads to the filing of an investigational new drug application with the FDA within four years. The program is designed to speed translational research - research that takes scientific discoveries from the laboratory to the patient bedside. This requires new levels of collaboration between basic laboratory scientists, medical clinicians, biotechnology experts and pharmacology experts, to name a few.

Other support came from UCLA's Broad Stem Cell Research Center and Jonsson Comprehensive Cancer Center, and from the Ruth L. Kirschstein National Research Service Award.

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Stem-cell gene therapy for sickle-cell disease advances

July 1, 2013 The idea that worms can be seen as waveforms allowed scientists at Rice University to find new links in gene networks that control movement.

The work led by Rice biochemist Weiwei Zhong, which will appear online this week in the Proceedings of the National Academy of Sciences Early Edition, involved analyzing video records of the movement of thousands of mutant worms of the species Caenorhabditis elegans to identify the neuronal pathways that drive locomotion.

One result was the discovery of 87 genes that, when inactivated, caused movement defects in worms. Fifty of those genes had never been associated with such defects, and 37 have implications in human diseases, the researchers found.

Another discovery was the existence of several network modules among these genes. One module detects environmental conditions. Another resides in all "excitable cells" -- those types that respond to electrical signals -- in the worm's neurons, muscles and digestive tracts. Another coordinates signals in the motor neurons.

The team also uncovered new details about a protein-signaling pathway found in all animals, Zhong said.

Zhong said the study is the first to provide a system-level understanding of how neuronal signaling genes coordinate movement and shows the value of a quantitative approach to genetic studies. She said the approach could be useful in studies of gene-to-drug or drug-to-drug interactions.

What made the research possible is the fact that cameras and computers are able to see variations in movement that are too small for eyes and minds to notice, Zhong said. "The idea is that if a gene is required for maintaining normal movement and we pick a mutant, the computer should be able to detect the defects," she said.

"I'm very observant," she said, "and I thought I could tell the worms with abnormal behaviors. I was surprised to see there were so many things I missed that the computer picked up."

The Rice researchers, with help from associates at the California Institute of Technology and Howard Hughes Medical Institute (HHMI), analyzed 239 mutant C. elegans, a common worm used in studies since the 1970s. Including a set of "wild-type" C. elegans that was used as a baseline, the Rice lab studied more than 4,400 worms. Each type was ordered from the Caenorhabditis Genetics Center and separated by mutation.

The worms were filmed one at a time. Each was placed in a petri dish (seeded with E. coli bacteria for food) on a motorized platform and filmed by a computer-controlled camera/microscope. The computer re-centered the camera on the worms any time they moved near the edge of the camera's field of view.

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Wiggling worms make waves in gene pool

Washington, July 02 (ANI): A new research has found out that RORA, a novel candidate gene for autism, regulates a large number of other genes associated with the condition.

Valerie Hu, Ph.D., professor of biochemistry and molecular medicine at the George Washington University (GW) School of Medicine and Health Sciences (SMHS), who along with his group discovered the gene in 2010, conducted the study.

"We are focusing on this gene, in part, because this gene can act as a master regulator of other genes," Hu said.

"Called nuclear hormone receptors, they are capable of activating or suppressing other genes in the genome. The question was which specific genes are regulated by RORA," he explained.

Hu and co-author, Tewarit Sarachana, Ph.D., a former doctoral student in the molecular medicine doctoral program at SMHS, found that RORA encodes a protein that can regulate the expression of more than 2,500 other genes.

Of these 2,500 genes, many are known to be involved in neuronal development and functions, and 426 of RORA's gene targets are already listed in AutismKB, a database of known autism candidate genes.

To identify genes regulated by RORA, Hu and Sarachana used chromatin immunoprecipitation (ChIP) with an anti-RORA antibody followed by whole-genome promoter array (chip) analysis.

This genome-wide ChIP-on-chip analysis of target genes of RORA, as well as additional methods of validation, confirmed that RORA transcriptionally regulates the genes A2BP1, CYP19A1, HSD17B10, ITPR1, NLGN1, and NTRK2, such that when RORA levels are cut in half, all six genes also go down in their expression.

The expression levels of these six genes are also reduced in RORA-deficient postmortem brain tissues from individuals with autism relative to that of age-matched unaffected controls.

"We see it as a domino effect, where RORA is a particularly shaky domino," said Hu.

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New regulatory autism gene discovered

July 2, 2013 Researchers have pinpointed the role of a gene known as Arl13b in guiding the formation and proper placement of neurons in the early stages of brain development. Mutations in the gene could help explain brain malformations often seen in neurodevelopmental disorders.

The research, led by a team at the University of North Carolina School of Medicine, was published June 30 in the journal Nature Neuroscience.

"We wanted to get a better sense of how the cerebral cortex is constructed," said senior study author Eva Anton, PhD, a professor in the Department of Cell Biology and Physiology and a member of the UNC Neuroscience Center. "The cells we studied -- radial glial cells -- provide a scaffolding for the formation of the brain by making neurons and guiding them to where they have to go. This is the first step in the formation of functional neuronal circuitry in the brain. This study gives us new information about the mechanisms involved in that process."

The researchers became interested in the Arl13b gene because of its expression in a part of the cell called primary cilium and its association with a rare neurological disorder known as Joubert syndrome. The syndrome is characterized by brain malformations and autism like features.

"In addition to helping us understand an important cellular mechanism involved in normal brain development, this study may offer an explanation for some of the malformations seen in Joubert syndrome patients," said Anton. Although there is no immediate clinical application for these patients, the study does help illuminate the factors behind the disease. "It shows what may have gone wrong in some of those patients that led to the malformations," said Anton.

The cerebral cortex, the brain's "gray matter," is responsible for higher-order functions such as memory and consciousness. Like the scaffolding builders use to move people and materials during construction, radial glial cells provide an instructive matrix to create the basic structural features of the cerebral cortex. Mistakes in the formation and development of radial glial cells can translate into structural problems in the brain as it develops, said Anton.

Both mice and humans have the Arl13b gene. The researchers generated a series of mice with mutations on the Arl13b gene at different developmental stages to track the mutations' effects on brain development. They discovered that the gene is crucial to the radial glial cells' ability to sense signals through an appendage called the primary cilium. Without this signaling capability, the radial glia were unable to organize into an instructive scaffold capable of orchestrating the orderly formation of cerebral cortex. "The cilia in these cells play an important role in the initial setup of this scaffolding," said Anton. "Without a functioning Arl13b gene, the cells were not able to determine polarity and formed haphazardly. As a result, they formed a malformed cerebral cortex with ectopic clusters of neurons, instead of the orderly layers of neurons with appropriate connectivity that would be expected, in the developing brain.

Co-authors include Holden Higginbotham, Jiami Guo, Yukako Yokota, Jingjun Li, Nisha Verma, Vladimir Gukkasyan and Joshua Hirt from UNC, and Nicole Umberger, Chen-Ying Su, and Tamara Caspary of Emory University.

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Gene's key role in building the developing brain's scaffolding discovered

Public release date: 1-Jul-2013 [ | E-mail | Share ]

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

New Rochelle, NY, July 1, 2013Among the increasingly valuable roles fungi are playing in the biotechnology industry is their ability to produce enzymes capable of releasing sugars from plants, trees, and other forms of complex biomass, which can then be converted to biofuels and biobased chemicals. Advances in fungal biology and in bioengineering fungal systems industrial applications are explored in a series of articles in Industrial Biotechnology, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers. The articles are available on the Industrial Biotechnology website.

Guest Editors Scott Baker, PhD, Pacific Northwest National Laboratory (PNNL, Richland, WA), Antoine Margeot, PhD, IFP Energies nouvelles (Rueil-Malmaison Cedex, France), and Adrian Tsang, PhD (Concordia University, Montreal, Quebec, Canada), collaborated on the IB IN DEPTHSpecial Section on Fungal Biology in Industrial Biotechnology.

In the Overview "Fungi and Industrial Biotechnology A Special Issue for an Amazing Kingdom," Dr. Baker says, "For more than a century fungi have had an enormous footprint in industrial biotechnology, from the first US biotechnology patent to current research in biofuels and renewable chemicals."

The Special Section includes Review articles by Kevin McCluskey, PhD, Curator of the Fungal Genetics Stock Center at the University of Missouri, Kansas City, entitled "Biological Resource Centers Provide Data and Characterized Living Material for Industrial Biotechnology," and by Justin Powlowski, PhD's group at the Centre for Structural and Functional Genomics, Concordia University, Montreal, Quebec, Canada.

Etienne Jourdier, PhD, and colleagues present a "Comprehensive Study and Modeling of Acetic Acid Effect on Trichoderma reesei Growth." Contributing the research study "In-Stream Itaconic Acid Recovery from Aspergillus terreus Fedbatch Fermentation" is a research team from TNO Microbiology & Systems Biology, Zeist, the Netherlands, let by Professor Peter Punt.

Included in the Fungal Biology Special Section is an IB Interview with Blake Simmons, PhD, Joint BioEnergy Institute (JBEI, Emeryville, CA) and Sandia National Laboratories (Livermore, CA), and Jon Magnuson, PhD, JBEI and PNNL. John Nicksich, Environmental Molecular Sciences Laboratory (PNNL, Richland, WA), describes the cutting-edge technology used to explore and bioengineer fungi in the Catalyzing Innovation article "EMSL Capabilities and Expertise: Pushing the Frontiers of Bioengineering."

"Scientific and technological advances in the life sciences are providing exciting new ways to engage old and familiar microbial friends in a number of novel and innovative industrial biotechnology activities," says Larry Walker, PhD, Co-Editor-in-Chief and Professor, Biological & Environmental Engineering, Cornell University, Ithaca, NY.

###

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Bioengineering fungi for biofuels and chemicals production

Bob Hancox.

Genetic risk assessments could eventually also be used to predict which children with asthma were likely to grow out of the condition, and which would continue having symptoms as they grew older, the research suggested.

The study's lead author is Dr Daniel Belsky, of the Duke University Medical Centre, in North Carolina, in the United States, and one of the Otago authors is Associate Prof Bob Hancox, a respiratory physician in the Otago preventive and social medicine department.

Prof Hancox said the results were ''interesting'' from a scientific viewpoint but, like all good research, also raised many questions.

''There's a lot more to find out.''

It was ''early days'', a great deal more research had to be done, and it was much too early to apply the research to the clinical management of individual patients.

The research again high-lighted the international importance of the long-running Dunedin multidisciplinary study of about 1000 children born in 1972-73, he said.

A team of Otago and Duke University researchers set out to test how genetic discoveries concerning asthma predisposition related to the developmental and biological characteristics of the condition. Their findings were published last month in the online edition of the United Kingdom journal The Lancet Respiratory Medicine.

After analysing data from the Dunedin Multidisciplinary Study, the team discovered that people both with childhood asthma and higher genetic risk scores for being predisposed to it were more than one-third (36%) more likely to develop lifelong asthma than those found to have a lower genetic risk.

Participants with asthma and a higher genetic risk were also more likely to develop atopic (allergic) asthma and impaired lung function (airway hyper-responsiveness and incompletely reversible airflow obstruction), and to miss school or work and to be hospitalised because of asthma.

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Varsity adds to research on asthma

Paleo Primal Diet - Unlocking your Body #39;s Dormant Genetics
http://101how.com/PaleoBurn Paleo Primal Diet. I #39;ll reveal specific foods which trigger your body to release a FLOOD of fat-melting hormones - hormones that ...

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Paleo Primal Diet - Unlocking your Body's Dormant Genetics - Video

Let #39;s Play The Sims 3 - Perfect Genetics Challenge - Episode 20
My Sims 3 Page: http://mypage.thesims3.com/mypage/Llandros2012 My Blog: http://Llandros09.blogspot.com My Facebook: https://www.facebook.com/Llandros09?ref=t...

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Scientific Proof about Genetics and Evolution
What does Observed Population Genetics say about Evolution? Is Evolution a scientific fact or a fairy tale? Liston to what a professor in population genetics...

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Scientific Proof about Genetics and Evolution - Video

Genetics in Cancer Prevention
University of Puerto Rico, Medical Sciences Campus Cancer Genetics Course A 5-day intensive course in the genetics of cancer for upper level undergraduates, ...

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Genetics in Cancer Prevention - Video

Paw Print Genetics New Commerical
Check out Paw Print Genetics new commercial airing this fall.

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Paw Print Genetics New Commerical - Video

Newswise Culminating an extensive national search, Tony Wynshaw-Boris MD, PhD, accomplished researcher and expert in genetics, genomics and neurologic disorders, has been named chair of the Department of Genetics and Genome Sciences at Case Western Reserve University School of Medicine and University Hospitals (UH) Case Medical Center. Wynshaw-Boriss appointment will become official with final approval from the Case Western Reserve Board of Trustees.

Highly accomplished as both a clinician and a scientist, Wynshaw-Boriss research centers on how neurogenetic disorders evolve during early human development. Recent research has concentrated on autism and developing a further understanding of the pathophysiological mechanisms that result in the highly inheritable disorder.

Tony is someone with whom both [UH Case Medical Center President] Fred Rothstein and I have had the pleasure to work with during his initial stints at Case Western Reserve and University Hospitals, said Pamela Davis, MD, PhD, dean of Case Western Reserve University School of Medicine. Our firsthand knowledge of his work makes us all the more excited to have him back in Cleveland, but it is his extraordinary accomplishments since then that represent the greatest reason to recruit him.

Last year, Wynshaw-Boris was elected as a fellow to the American Association for the Advancement of Sciences. He has also been elected to the American Pediatric Society, the Association of American Physicians and the American Society of Clinical Investigation. He was included in Discover magazines Top 100 Science Stories regarding the social behavior of mutant mice, received the National Institutes of Health Directors Award, and won a special achievement award from the National Human Genome Research Institute.

Since 2008, the Cleveland native has served as the Charles J. Epstein Professor of Human Genetics and Pediatrics at the School of Medicine of the University of California at San Francisco as well as Chief of the Division of Genetics in the UCSF Department of Pediatrics. For the preceding eight years, he was at the School of Medicine at the University of California, San Diego and Rady Childrens Hospital San Diego. Dr. Wynshaw-Boris serves as the executive editor of the journal Human Molecular Genetics.

Tony embodies the mission of our academic medical center an innovative researcher, a dedicated educator, and an outstanding physician, says Fred C. Rothstein, MD, President of UH Case Medical Center. He is an internationally known leader in his field and as Chairman he will further enhance our Department of Genetics and Genome Sciences.

The appointment allows Wynshaw-Boris to expand his research beyond his lab. He sees the Department of Genetics and Genome Sciences as an area of study that can advance projects across the university and hospital - ultimately benefitting patients. He also believes scientific and technological advancements make unprecedented breakthroughs possible; the challenge is to engage colleagues across disciplines and skill sets in complementary ways.

I do think genetics and genomics are the organizing principle for all medicine and all of biology, so we should be the center of whats going on in the School of Medicine and in the hospital, Wynshaw-Boris said. Im going to do all that I can to make sure that that happens, building on what we have now.

Wynshaw-Boris earned his medical degree and doctorate in biochemistry from Case Western Reserve. He completed a residency at Rainbow Babies & Children's Hospital and a fellowship at Children's Hospital of Boston and Harvard University. He also completed a prestigious Howard Hughes Medical Institute postdoctoral fellowship, where he studied mouse models of birth defects and developmental disorders.

Among the nations leading academic medical centers, University Hospitals Case Medical Center is the primary affiliate of Case Western Reserve University School of Medicine, a nationally recognized leader in medical research and education.

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Wynshaw-Boris Named Chair of Genetics and Genome Sciences

SEATTLE, WA--(Marketwired - Jul 2, 2013) - Atossa Genetics, Inc. (NASDAQ: ATOS), The Breast Health Company, announced today that Michael H. Kalnoski, M.D., has joined the Company as Medical Director, a newly created position. Dr. Kalnoski's primary focus will be overseeing all operations of the Company's National Reference Laboratory for Breast Health, including quality management from the handling of nipple aspirate fluid specimens coming into the lab, to generating reports for doctors and patients, to developing new assays. He reports to Dr. Steven Quay, Chairman, CEO & President.

Dr. Kalnoski, a board certified pathologist, brings 10 years' experience as a medical director and medical consultant at a number of companies and institutions including the Puget Sound Institute of Pathology in Seattle, the Auburn Regional Medical Center in Auburn, Washington, the Valley General Hospital in Monroe, Washington, the Petersburg Medical Hospital in Petersburg, Alaska, and the Forks Medical Center in Forks, Washington. Earlier, Dr. Kalnoski was also a medical consultant at Hematologics Inc., Seattle, and medical director of Quest Diagnostics Inc., Seattle, and PacLab Inc., Renton, Washington.

"The nationwide marketing efforts we are undertaking for the ForeCYTE Breast Health Test are leading to greater awareness among doctors and an increasing number of samples coming to our lab on a daily basis," said Dr. Quay. "As the number of samples processed in the lab continues to grow, Dr. Kalnoski's extensive experience as a medical director and medical consultant will be invaluable in helping us operate the lab smoothly and efficiently, enabling us to achieve our ambitious growth objectives. I look forward to working with Michael during this exciting time."

"Atossa Genetics is a very innovative company that has the potential to significantly reduce the incidence of breast cancer through superior risk assessment tools and treatments," said Dr. Kalnoski. "I look forward to working with Dr. Quay and the outstanding team at Atossa and the National Reference Laboratory for Breast Health to help drive adoption of the ForeCYTE Breast Health Test through superior services at the laboratory level."

Dr. Kalnoski received his M.D. in 1997 from the Saint Louis University School of Medicine. He completed post graduate medical training at the University of Washington and is Board Certified in Hematopathology and Board Certified in anatomic and clinical pathology (AP/CP). Earlier Dr. Kalnoski attended the University of Washington and earned a bachelor's degree in chemistry. Dr. Kalnoski has co- authored 11 peer-reviewed, published articles.

He is a member of the American Medical Association, Pierce County Medical Society, the College of American Pathologists, the American Society of Clinical Pathology and a member the Washington State Medical Association.

About Atossa Genetics, Inc.

Atossa Genetics, Inc. (NASDAQ: ATOS), The Breast Health Company, based in Seattle, WA, is focused on preventing breast cancer through the commercialization of patented, FDA-designated Class II diagnostic medical devices and patented, laboratory developed tests (LDT) that can detect precursors to breast cancer up to eight years before mammography.

The National Reference Laboratory for Breast Health (NRLBH), a wholly owned subsidiary of Atossa Genetics, Inc., is a CLIA-certified high-complexity molecular diagnostic laboratory located in Seattle, Washington.

For additional information on Atossa and the ForeCYTE test, please visit http://www.atossagenetics.com. For additional information on the National Reference Laboratory for Breast Health, please visit http://www.nrlbh.com.

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Michael H. Kalnoski, M.D., Joins Atossa Genetics as Medical Director

What are the most exciting science developments? - Cheltenham Science Festival 2013 - Head Squeeze
We asked the biggest science brains at Cheltenham Science Festival 2013 what scientific developments they are most excited about! We chatted to Kevin Fong, M...

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Factors Preventing Gene Therapy From Being Effective
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Factors Preventing Gene Therapy From Being Effective - Video

Social and Ethical Considerations of Gene Therapy
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Social and Ethical Considerations of Gene Therapy - Video

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

The Vaccine & Gene Therapy Institute of Florida (VGTI Florida) is pleased to announce the appointment of Richard Jove, Ph.D., as the new director of the Institute. Dr. Jove will take up his new position beginning July 1, 2013.

Dr. Jove brings to VGTI Florida the scientific leadership, research expertise, and a translational philosophy that will bring us to the next level in terms of basic and clinical research and the related improvement of health outcomes, said Jay Nelson, Ph.D., CEO of VGTI Florida.

As an external member of the Scientific Advisory Board for VGTI Florida, Dr. Jove has already assisted the Institute in planning its research strategy for cancer and infectious disease. I am very excited now to become a member of the internal leadership team at VGTI Florida as their mission of `Translating Research into Health' has always been a guiding principle in my own research career, said Dr. Jove. Having the opportunity to contribute what I have learned, and to work closely with the talented researchers and staff at VGTI Florida, will help us bring new scientific discoveries to benefit patients in the clinic.

Richard Jove has most recently served as the director of the Beckman Research Institute at City of Hope and deputy director of its Comprehensive Cancer Center in Los Angeles County, California. His work has been focused on developing more effective and safer therapies for the treatment of cancer. One of his primary roles has been encouragement of collaboration between scientists and clinical investigators for the development of scientific discoveries into novel cancer therapies for early phase clinical trials.

Prior to joining the City of Hope Dr. Jove held various leadership positions at the Moffitt Cancer Center in Tampa, Florida, following his establishing the Molecular Oncology Program at the University of Michigan Cancer Center in Ann Arbor.

Dr. Jove is a welcome addition to the VGTI Florida team and we all look forward to his leadership and scientific background contributing to the growth of the institute. With his global perspective and reach he is ideally suited to expand the depth and breadth on an international basis enhancing VGTI Floridas worldwide translational research healthcare platform, said Mel Rothberg, Chief Operating Officer of VGTI Florida.

Its not just about the science, more importantly its about the patients, said Dr. Jove. VGTI Florida is an Institute that is not only doing the research, but developing the therapies, and I look forward to leading the institute and the recruitment of top researchers that will expand our focus on cancer.

About VGTI Florida:

VGTI Florida is an independent immunological research institute that is on an urgent mission to transform scientific discoveries into novel treatments and cures for existing and emerging infectious diseases, influenza and cancer. VGTI Florida is an independent non-profit 501(c)(3) organization located in the Tradition Center for Innovation in Port St. Lucie, Florida. For more information, please visit http://www.VGTIFL.org. VGTI Florida and Translating Research into Health are Registered Trademarks of the Vaccine & Gene Therapy Institute of Florida.

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Dr. Richard Jove Joins VGTI Florida as the New Institute Director

Newswise Researchers at UCLAs Eli & Edythe Broad Center of Regenerative Medicine & Stem Cell Research have successfully established the foundation for using hematopoietic (blood-producing) stem cells (HSC) from the bone marrow of patients with sickle cell disease (SCD) to treat the disease. The study was led by Dr. Donald Kohn, professor of pediatrics and microbiology, immunology and molecular genetics in the life sciences.

Kohn introduced an anti-sickling gene into the HSC to capitalize on the self-renewing potential of stem cells and create a continual source of healthy red blood cells that do not sickle. The breakthrough gene therapy technique for sickle cell disease is scheduled to begin clinical trials by early 2014. The study was published online ahead of press today in Journal of Clinical Investigation.

Gene Therapy Kohns gene therapy approach using HSC from patients own blood is a revolutionary alternative to current SCD treatments as it creates a self-renewing normal blood cell by inserting a gene that has anti-sickling properties into HSC. This approach also does not rely on the identification of a matched donor, thus avoiding the risk of rejection of donor cells. The anti-sickling HSC will be transplanted back into the patients bone marrow and multiplies the corrected cells that make red blood cells without sickling.

The results demonstrate that our technique of lentiviral transduction is capable of efficient transfer and consistent expression of an effective anti-sickling beta-globin gene in human SCD bone marrow progenitor cells, which improved the physiologic parameters of the resulting red blood cells. Kohn said.

Kohn and colleagues found that in the laboratory the HSC produced new non-sickled blood cells at a rate sufficient for significant clinical improvement for patients. The new blood cells survive longer than sickled cells, which could also improve treatment outcomes. The success of this technique will allow Kohn to begin clinical trials in patients with SCD by early next year.

Sickle Cell Disease Affecting more than 90,000 patients in the US, SCD mostly affects people of Sub-Saharan African descent. It is caused by an inherited mutation in the beta-globin gene that makes red blood cells change from their normal shape, which is round and pliable (like a plastic bag filled with corn oil), into a rigid sickle-shaped cell (like a corn flake). Normal red blood cells are able to pass easily through the tiniest blood vessels, called capillaries, carrying oxygen to organs such as the lungs, liver and kidneys. But due to their rigid structure, sickled blood cells get stuck in the capillaries and deprive the organs of oxygen, which causes organ dysfunction and failure.

Current treatments include transplanting patients with donor HSC, which is a potential cure for SCD, but due to the serious risks of rejection, only a small number of patients have undergone this procedure and it is usually restricted to children with severe symptoms.

CIRM Disease Team Program This study was supported in part by a Disease Team I Award from the California Institute for Regenerative Medicine (CIRM), the states stem cell research agency created by voter initiative in 2004. The purpose of the disease team program is to support research focused on one particular disease that leads to the filing of an investigational new drug application with the FDA within four years. The program is designed to encourage translational research, which means to take scientific discoveries from the laboratory to the patient bedside as quickly as possible. This requires new levels of collaboration between basic laboratory scientists, medical clinicians, biotechnology experts and pharmacology experts, to name a few.

Other support came from the UCLA Broad Stem Cell Research Center and Jonsson Comprehensive Cancer Center and the Ruth L. Kirschstein National Research Service Award.

The stem cell center was launched in 2005 with a UCLA commitment of $20 million over five years. A $20 million gift from the Eli and Edythe Broad Foundation in 2007 resulted in the renaming of the center. With more than 200 members, the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research is committed to a multi-disciplinary, integrated collaboration of scientific, academic and medical disciplines for the purpose of understanding adult and human embryonic stem cells. The center supports innovation, excellence and the highest ethical standards focused on stem cell research with the intent of facilitating basic scientific inquiry directed towards future clinical applications to treat disease. The center is a collaboration of the David Geffen School of Medicine, UCLAs Jonsson Cancer Center, the Henry Samueli School of Engineering and Applied Science and the UCLA College of Letters and Science. To learn more about the center, visit our web site at http://www.stemcell.ucla.edu

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UCLA Stem Cell Gene Therapy for Sickle Cell Disease Advances Toward Clinical Trials

Public release date: 2-Jul-2013 [ | E-mail | Share ]

Contact: Ftima Bosch Fatima.bosch@uab.cat 34-935-814-182 Universitat Autonoma de Barcelona

Sanfilippo Syndrome type A, or Mucopolysaccharidosis type IIIA (MPSIIIA), is a neurodegenerative disease caused by mutations in the gene that encodes the enzyme sulfamidase. Mutations in this gene lead to deficiencies in the production of the enzyme, which is essential for the breakdown of substances known as glycosaminoglicans. If these substances are not broken down, they accumulate in the cells and cause neuroinflammation and organ dysfunction, mainly in the brain, but also in other parts of the body. Children born with this mutation are diagnosed from the age of 4 or 5. They suffer neurodegeneration, causing mental retardation, aggressiveness, hyperactivity, sleep alterations, loss of speech and motor coordination, and they die in adolescence.

A team of researchers headed by the director of the UAB's Centre for Animal Biotechnology and Gene Therapy (CBATEG), Ftima Bosch, has developed a gene therapy treatment that cures this disease in animal models, with pre-clinical studies in mice and dogs. The treatment consists of a single surgical intervention in which an adenoassociated viral vector is injected into the cerebrospinal fluid, the liquid that surrounds the brain and the spinal cord. The virus, which is completely harmless, genetically modifies the cells of the brain and the spinal cord so that they produce sulfamidase, and then spreads to other parts of the body, like the liver, where it continues to induce production of the enzyme.

Once the enzyme's activity is restored, glycosaminoglican levels return to normal for life, their accumulation in cells disappears, along with the neuroinflammation and dysfunctions of the brain and other affected organs, and the animal's behaviour and its life expectancy return to normal. While mice with the disease lived only up to 14 months, those given the treatment survived as long as healthy ones.

This is a joint project between the UAB and the pharmaceutical company Esteve. The study has been published in the online edition of The Journal of Clinical Investigation.

###

AAAS and EurekAlert! are not responsible for the accuracy of news releases posted to EurekAlert! by contributing institutions or for the use of any information through the EurekAlert! system.

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Gene therapy cures a severe paediatric neurodegenerative disease in animal models

July 1, 2013 Researchers at UCLA's Eli & Edythe Broad Center of Regenerative Medicine & Stem Cell Research have successfully established the foundation for using hematopoietic (blood-producing) stem cells (HSC) from the bone marrow of patients with sickle cell disease (SCD) to treat the disease. The study was led by Dr. Donald Kohn, professor of pediatrics and microbiology, immunology and molecular genetics in the life sciences.

Kohn introduced an anti-sickling gene into the HSC to capitalize on the self-renewing potential of stem cells and create a continual source of healthy red blood cells that do not sickle. The breakthrough gene therapy technique for sickle cell disease is scheduled to begin clinical trials by early 2014. The study was published online in the Journal of Clinical Investigation.

Gene Therapy

Kohn's gene therapy approach using HSC from patient's own blood is a revolutionary alternative to current SCD treatments as it creates a self-renewing normal blood cell by inserting a gene that has anti-sickling properties into HSC. This approach also does not rely on the identification of a matched donor, thus avoiding the risk of rejection of donor cells. The anti-sickling HSC will be transplanted back into the patient's bone marrow and multiplies the corrected cells that make red blood cells without sickling.

"The results demonstrate that our technique of lentiviral transduction is capable of efficient transfer and consistent expression of an effective anti-sickling beta-globin gene in human SCD bone marrow progenitor cells, which improved the physiologic parameters of the resulting red blood cells." Kohn said.

Kohn and colleagues found that in the laboratory the HSC produced new non-sickled blood cells at a rate sufficient for significant clinical improvement for patients. The new blood cells survive longer than sickled cells, which could also improve treatment outcomes. The success of this technique will allow Kohn to begin clinical trials in patients with SCD by early next year.

Sickle Cell Disease

Affecting more than 90,000 patients in the US, SCD mostly affects people of Sub-Saharan African descent. It is caused by an inherited mutation in the beta-globin gene that makes red blood cells change from their normal shape, which is round and pliable (like a plastic bag filled with corn oil), into a rigid sickle-shaped cell (like a corn flake). Normal red blood cells are able to pass easily through the tiniest blood vessels, called capillaries, carrying oxygen to organs such as the lungs, liver and kidneys. But due to their rigid structure, sickled blood cells get stuck in the capillaries and deprive the organs of oxygen, which causes organ dysfunction and failure.

Current treatments include transplanting patients with donor HSC, which is a potential cure for SCD, but due to the serious risks of rejection, only a small number of patients have undergone this procedure and it is usually restricted to children with severe symptoms.

CIRM Disease Team Program

See original here:
Stem cell gene therapy for sickle cell disease advances toward clinical trials

Canadian-led study will help scientists identify key genomic regions in canola, other food plants

TORONTO,ON What allows certain plants to survive freezing and thrive in the Canadianclimate, while others are sensitive to the slightest drop in temperature? Thosethat flourish activatespecific genes at just the right time but the waygene activation is controlled remains poorly understood.

Amajor step forward in understanding this process lies in a genomic map producedby an international consortium led by scientists from the University of Toronto(U of T) and McGillUniversity and published online today in the journalNature Genetics.

Themap, which is the first of its kind in plants, will help scientists to localizeregulatory regions in the genomes of crop species such as canola, a major cropin Canada, according to researcherswho worked on the project. The team hassequenced the genomes of several crucifers (a large plant family that includesa number of other food crops) and analyzed them along with previouslypublishedgenomes to map more than 90,000 genomic regions that have been highly conservedbut that do not appear to encode proteins.

Plants are complicated organisms, and they have many types of cells and structures, said Dr. Annabelle Haudry, one of the studys lead authors and former U of T postdoctoral fellow. We found that genes involved in defining how these cells and structures grow as the plant develops from a seed and how it responds to environments stimuli are surrounded by many of these switches.

Amazingly,similar organization of switches was found for the genes that control earlyhuman development from an embryo an example of convergent evolution, saysRobert Williamson, Uof T PhD student and study coauthor. (Convergent evolutionis the scientific term for biological traits that arrive through differentevolutionary lineages.) Work is currently underway toidentify which of thoseregions may be involved in controlling traits of particular importance tofarmers.

Thestudy also weighs in on a major debate among biologists, concerning how much ofan organisms genome has important functions in a cell, and how much is junkDNA, merely along forthe ride, says U of Ts Professor Alan Moses, also involved in the study. While stretches of the genome that code for proteins arerelatively easy to identify, many other noncoding regionsmay be importantfor regulating genes, activating them in the right tissue and under the rightconditions.

Whilehumans and plants have very similar numbers of protein-coding genes, the mappublished inNature Geneticsfurthersuggests that the regulatory sequences controlling plant genes are farsimpler,with a level of complexity between that of fungi and microscopic worms. Plantsseem to have a large fraction of their genome that is junk DNA, says U of TsProfessor StephenWright, another leader of the study. But our analysis allowsfor identification of the tens of thousands of needles in the haystack thatare important for gene regulation.

Fundingfor the research was provided by Genome Canada and Gnome Qubec, along withthe European Regional Development Fund, the Czech Science Foundation, and theNational ScienceFoundation.

-30-

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A genomic atlas of gene switches in plants provides a roadmap for crop research - Canadian-led study will help ...

Public release date: 30-Jun-2013 [ | E-mail | Share ]

Contact: Ueli Schibler ueli.schibler@unige.ch 41-223-796-175 Universit de Genve

Most of our physiological functions fluctuate throughout the day. They are coordinated by a central clock in the brain and by local oscillators, present in virtually every cell. Many molecular gearwheels of this internal clock have been described by Ueli Schibler, professor at the Faculty of Science of the University of Geneva (UNIGE), Switzerland. To study how the central clock synchronizes subordinate oscillators, the researcher's group used a variety of genetic and technological tools developed in collaboration with a team of UNIGE physicians. In this way, the scientists were able to directly observe the bioluminescence emitted by 'clock genes' in mice for several months. This biotechnology is applicable to numerous sectors of biomedical research, which attracted the attention of the editors from the journal "Genes & Development".

In mammals, there are many behaviors and biological functions that are regulated by internal clocks. Most of our cells have one, made from a family of 'clock genes', whose cyclic activity reaches a specific peak in 24 hours. These local oscillators are synchronized by a central 'pacemaker' in the brain which adjusts to light.

The firefly lights the way

The use of genetic engineering techniques enabled the study of molecular mechanisms that activate clock genes directly in cultured mammalian cells: 'We have coupled several of these genes to that of luciferase, the enzyme used by the female firefly for producing green light to attract males,' explained Ueli Schibler, member of the National Research Center Frontiers in Genetics. When a specific clock gene is activated in a cell that was transformed in this way, the light signal emitted can be measured using a highly sensitive bioluminescence detector. However, this device, which is capable of detecting signals on the order of a few photons, cannot be used for studying whole organisms. The contribution of Andr Liani's mechanical workshop, along with Jean-Pierre Wolf's and Luigi Bonacina's teams from UNIGE's Group of Applied Physics, was thus essential. These scientists developed a customized device that can accommodate mice for several months: 'We equipped it with reflective walls to deflect photons toward a highly sensitive photomultiplier tube to capture bioluminescence,' says Andr Liani.

Follow the daily expression of clock genes live

In collaboration with the University of Ulm and the Center for Integrative Genomics (CIG) of Lausanne, the biologists studied how the central clock synchronizes subordinate oscillators in mice. Various clock genes, coupled with the luciferase gene for light emission, were inserted into liver cells using a molecular vector. The time these rodents spent in the bioluminescent device allowed to demonstrate that the central clock generates signals, some of which act directly on the liver oscillators, and others which synchronize them indirectly by controlling the cycles of food intake.

or the effect of a medication in mice

'This technology enables a drastic reduction in the number of mice needed for this type of experiment, and furthermore, it is applicable to many areas of biomedical research,' says Camille Saini, researcher in the Department of Molecular Biology at UNIGE and first author of this article. These complementary genetic and engineering technology tools could be used to directly follow certain biochemical effects of metabolites like cholesterol or glucose, as well as the response to potential treatments of diseases such as hypercholesterolemia or diabetes. Monitoring the response to various hormones, neurotransmitters and other biochemical messengers is also part of this application range.

Originally posted here:
Observing live gene expression in the body