Archive for the ‘Gene Therapy Research’ Category

ScienceDaily (July 9, 2012) In research funded by the National Institutes of Health and the American Heart Association and published in EMBO Molecular Medicine, Saint Louis University investigator ngel Baldn, Ph.D., found that the microRNA miR-33 plays a key role in regulating bile metabolism. Further, the research suggests that, in an animal model, the manipulation of this microRNA can improve the liver toxicity that can be caused by statins.

"As we learn more about the way cholesterol is moved and metabolized through the body, we have more tools at our disposal to try to limit potential side effects of cholesterol-managing drugs like statins," said Baldn, who is assistant professor of biochemistry and molecular biology at Saint Louis University.

This study continues Baldn's exploration of the microRNA miR-33, which is expressed from within SREBP-2, an important gene in the body that previously had been shown to regulate cholesterol metabolism. In earlier research, the Baldn laboratory found that miR-33 plays a key role in regulating cholesterol. In particular, his team found that decreasing the levels of the microRNA (which is a piece of genetic coding) helped to raise HDL, or "good cholesterol," in an animal model. Five laboratories, including Baldan's, simultaneously reported these results in 2010.

Now, as Baldn continues to study the role of miR-33, he has examined two particular bile transporters, ABCB11 and ATP8B1, and found that miR-33 directly regulates these transporters. The research team found that when they silenced miR-33, turning off the microRNA's signal, they caused increases in bile secretion from the liver, so more bile was recovered in the gallbladder.

Further confirming the suspicion that this pathway was responsible for regulating the flow of bile, researchers treated two groups of mice with an anti-miR-33 drug and tracked radioactively labeled cholesterol as it moved through and was eliminated by these animals.

"We hypothesized we should see changes in the amount of radioactivity in the cholesterol that was eliminated in the mice's feces, depending on whether they were given placebo or anti-miR-33," Baldn said. "That is in fact what we found. When the microRNA is silenced, the pathway is enhanced and more cholesterol is passed through."

Bile is produced by the liver to help the body digest dietary lipids. Bile is itself made up, in part, of cholesterol and cholesterol-derived bile acids, and it also serves a key function in controlling the body's balance of cholesterol.

When the body doesn't secrete and transport bile well, due to an obstruction like a gallstone, or, as examined in this study, because of a genetic variation or medication side effect, bile cannot flow from the liver to the small intestine. The resulting blockage causes cholestasis, a kind of liver damage.

In the final segment of the study, researchers took note of a genetic condition, called progressive familial intrahepatic cholestasis (PFIC), an inherited disease that causes cholestasis and can lead to liver failure. PFIC is caused by defects in the biliary transporters, such as ABCB11 and ATP8B1, the very genes that are regulated by miR-33. Interestingly, the same group of symptoms can occur in a less severe form, called benign recurrent intrahepatic cholestasis (BRIC) in some people with less severe genetic mutations.

"Intriguingly, a very small number of patients who take statins develop a syndrome identical to BRIC, a milder version of the same illness experienced by people who have the genetic disease PFIC," Baldn said. "In this case, though, statins caused the condition pharmacologically.

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Turning off key piece of genetic coding eliminates toxic effect of statins, study suggests

23andMe has named Andy Page to its board of directors. He currently serves as president of Gilt Group. Previously he served as chief operating and financial officer at PlayPhone; chief financial officer and senior vice president of business strategy at StubHub; and has held senior executive positions at Panasas, ONI Systems, and Robertson Stephens & Company.

Sera Prognostics has named Sherree Frazier to be VP of sales and marketing. Frazier will head the company's commercial activities, including development and launch of the ProNid diagnostic test to predict preterm birth risk. Frazier formerly was senior director of molecular diagnostics and head of North American clinical sales at Qiagen, and prior to that she was women's health manager at Adeza Biomedical, before it was acquired by Cytyc.

Douglas Kell has been reappointed as chief executive and deputy chair of the UK Biotechnology and Biological Sciences Research Council, the Minister for Universities and Science David Willetts said this week.

Kell has held the top post at BBSRC since 2008, and before that he was director of the Manchester Centre for Integrative Systems Biology. He also has served as director of research at the Institute of Biological Sciences at the University of Aberystwyth, and he was a founding director of Aber Instruments. His research has included a range of topics including systems biology, analytical chemistry, and biochemical and data modeling.

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Population Genetics, Autism Research Centre to Study Asperger Variants

ScienceDaily (July 9, 2012) The challenge of treating patients with genetic disorders in which a single mutated gene is simply too large to be replaced using traditional gene therapy techniques may soon be a thing of the past. A Nationwide Children's Hospital study describes a new gene therapy approach capable of delivering full-length versions of large genes and improving skeletal muscle function. The strategy may hold new hope for treating dysferlinopathies and other muscular dystrophies.

A group of untreatable muscle disorders known as dysferlinopathies are caused by mutations in the dysferlin gene. Patients with these disorders, including limb girdle muscular dystrophy type 2B, are typically diagnosed in their early twenties. Approximately one-third will become wheelchair dependent by their mid-30s.

Gene therapy using adeno-associated virus (AAV) to deliver genes to cells has been pursued as an option for some patients with muscular dystrophy. However, AAV's packaging limitations have served as obstacles in using gene therapy to deliver large genes like dysferlin. Scientists in the past have attempted to work around AAV's packaging limitations by inserting a small version of large genes into the viral vector to induce gene expression. Some have also used more than one viral vector at a time to deliver a large gene. However, micro and mini versions of large genes don't always have the power of full-length gene expression and an increased viral load can lead to negative side effects.

"We have had success in the clinic using AAV gene therapy with limb girdle muscular dystrophy type 2D, which is caused by mutations in the alpha-sarcoglycan gene," said Louise Rodino-Klapac, PhD, principal investigator in the Center for Gene Therapy at The Research Institute of Nationwide Children's Hospital. "However, the dysferlin gene is very large, about six times larger than the alpha-sarcoglycan gene and can't fit into a traditional AAV vector."

A 2008 study identified AAV5, an AAV serotype that could package large transcripts. "This made us wonder whether it could be used for gene replacement requiring inserts as large as the dysferlin gene," said Dr. Rodino-Klapac.

In their 2012 study appearing in PLoS ONE, Dr. Rodino-Klapac's team used AAV5 to package a full-length, intact dysferlin gene and directly deliver it to the diaphragm of dysferlin-deficient mice. They also injected the leg muscles of dysferlin-deficient mice using both intramuscular and vascular approaches to further evaluate whether the gene delivery could improve skeletal muscle function.

They found that both the intravascular and intramuscular delivery approaches led to full-length, intact dysferlin gene expression in the leg and diaphragm muscle cells of the mice. More importantly, they saw that the newly-restored dysferlin repaired membrane deficits previously seen in the dysferlin-deficient mice.

"Our findings demonstrate highly favorable results with full restoration of dysferlin without compromise in function," said Dr. Rodino-Klapac. "With regard to neuromuscular diseases, these studies provide new perspective for conditions caused by mutations of large genes. Duchenne muscular dystrophy is the most common severe childhood muscular dystrophy and would seem to benefit from expression of the larger transcripts than mini- and micro-dystrophins that only partially restore physiologic function in mouse models of the disease."

Dr. Rodino-Klapac and her team are currently defining a path for a dysferlin clinical gene therapy trial. "We have shown that AAV5-dysferlin delivery is a very promising therapeutic approach that could restore functional deficits in dysferlinopathy patients," she says.

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New gene transfer strategy shows promise for limb girdle and other muscular dystrophies

DUBLIN--(BUSINESS WIRE)--

Research and Markets (http://www.researchandmarkets.com/research/kxltqj/gene_therapy_marke) has announced the addition of the "Gene Therapy Market to 2018 - Product Development Slowed by Clinical Failures, Close Regulatory Surveillance and High Compliance Standards" report to their offering.

Gene Therapy: the Next Big Step in Cancer Treatments.

The fight against cancer is leading a new movement in gene therapy, as the failure of conventional cancer therapies is fuelling demand for new treatments, according to a new report by healthcare experts GBI Research.

The new report* states that gene therapy technology is still in its nascent stage, and high levels of regulatory surveillance in clinical development is affecting progress. However, the increasing potential of upcoming treatments and shortcomings in traditional therapies is gradually leading to broader acceptance of gene therapy in medicine.

Therapies such as chemotherapy and hormone therapy control the progression of diseases, but are often associated with severe side effects, such as nausea, hair loss and abnormal blood cell counts. Once administered, the drugs induce systemic action throughout the body, and patients often die due to the side effects of treatment rather than the cancer itself. The inability of these conventional therapies to cure diseases has created a significant unmet need in the treatment of cancer, as well as Human Immunodeficiency Virus (HIV), autoimmune diseases, and viral infections.

Targeted therapies such as monoclonal antibodies, stem cell therapies, Ribonucliec Acid (RNA) therapies and gene therapies have initially shown better efficacy and safety profiles compared to chemotherapies.

Gene therapy has several promising drug candidates, which are likely to drive the growth of the gene therapy market if clinical trials are successful. Collategene by AnGes MG, Cardium Therapeutics' Generx, and Vical Incorporation's Allovectin-7 are in development for a wide range of cancer indications, and are expected to compete in the oncology therapeutics market as the market acceptance of gene therapy improves over time.

Companies Mentioned

- ReGenX Biosciences

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Research and Markets: Gene Therapy Market to 2018 - Product Development Slowed by Clinical Failures, Close Regulatory ...

ScienceDaily (July 8, 2012) Comparing the DNA from patients at the best and worst extremes of a health condition can reveal genes for resistance and susceptibility. This approach discovered rare variations in the DCTN4 gene among cystic fibrosis patients most prone to early, chronic airway infections.

The DCTN4 gene codes for dynactin 4. This protein is a component of a molecular motor that moves trouble-making microbes along a cellular conveyer belt into miniscule chemical vats, called lysosomes, for annihilation.

This study, led by the University of Washington, is part of the National Heart Lung and Blood Institute GO Exome Sequencing Project and its Lung GO, both major National Institutes of Health chronic disease research efforts.

Similar "testing the extremes" strategies may have important applications in uncovering genetic factors behind other more common, traits, such as healthy and unhealthy hearts.

The results of the cystic fibrosis infection susceptibility study appear on July 8, in Nature Genetics. The infection in question was Pseudomonas aeruginosa, an opportunistic soil bacterium that commonly infects the lungs of people with cystic fibrosis and other airway-clogging disorders. The bacteria can unite into a slithery, hard-to-treat biofilm that hampers breathing and harms lung tissue. Chronic infections are linked to poor lung function and shorter lives among cystic fibrosis patients. These bacteria rarely attack people with normal lungs and well-functioning immune systems.

In the study, these rare variations in DCTN4 did not appear in any of the cystic fibrosis patients who were the most resistant to Pseudomonas infection. The study subjects most susceptible to early, chronic infection had at least one DCTN4 missense variant. A missense variant produces a protein that likely can't function properly.

The lead author of the report published July 8 in Nature Genetics is Dr. Mary J. Emond, research associate professor of biostatistics at the University of Washington School of Public Health in Seattle. The senior author is medical geneticist Dr. Michael Bamshad, UW professor of pediatrics in the Division of Genetic Medicine.

To the extent of their knowledge, the researchers think that this might be the first time that genetic variants underlying complex trait were discovered by sequencing all the protein-coding portions of the genomes of people at each extreme of a disease spectrum.

"We did not have a candidate gene in mind when we did this study," said Emond. Statistical analysis of the DNA of 91 patients led the research team to this particular gene. Of the initial study group, 43 children had their first onset of chronic lung infection with Pseudomonas as when they were very young, and the 48 oldest individuals had not yet reached a state of chronic infection. The patients selected for sequencing were from the Early Pseudomonas Infection Control (EPIC) Observational Study, a project at the Seattle Children's Research Institute, and the North American Cystic Fibrosis Genetic Modifiers Study. Exome sequencing was done by UW researchers in the laboratory of Deborah Nickerson, UW professor of genome sciences.

Comparisons of the protein coding portions of the study subjects' DNA called the researchers attention to missense variations of the DCTN4 gene. The researchers went on to screen a selected group of 1,322 other EPIC participants to check their findings.

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Exome sequencing of health condition extremes can reveal susceptibility genes

ISTANBUL, Turkey, July 7 (UPI) -- Cigarette smoke reduces the production of a Fallopian tube gene, which helps explain the link between smoking and ectopic pregnancy, Scottish researchers say.

Drs. Andrew Horne and Colin Duncan of the Medical Research Council Centre for Reproductive Health in Edinburgh, Scotland, said ectopic pregnancy -- when the embryo implants in the Fallopian tube -- is the most common cause of maternal death in early pregnancy.

Ectopic pregnancy occurs in up to 2 percent of all pregnancies.There is no way to prevent the condition, which must be treated by abdominal surgery or, if the ectopic is small and stable, by injection of a drug called methotrexate.

Horne and colleagues exposed cells from the Fallopian tube to a breakdown product of nicotine -- cotinine. They then showed that cotinine had a negative effect on genes known to be associated with cell death, or apoptosis, and in particular with a particular gene.

In a further study the researchers showed that the gene's reduced production in the Fallopian tube of women who were smokers.

"The research is exciting because it provides new scientific evidence to help understand why women who smoke are more likely to have ectopic pregnancies," Horne said. "It appears that smoking reduces the production of genes, which are involved in the control of cell death and promote an environment in the Fallopian tube which is attractive to the developing embryo."

The findings were presented at the European Society of Human Reproduction and Embryology in Istanbul, Turkey.

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Smoking linked to ectopic pregnancy

The Republican primary race in Kansas House District 91 features the incumbent from the previous District 105, Gene Suellentrop, and small business owner Robin Clements.

Clements says her business, Public Solutions, consults with small businesses on public policy.

Suellentrop, also a business owner, was chosen by the Sedgwick County Republican Party in late 2009 to fill a vacant seat in the Legislature. He won re-election in 2010.

Both candidates live in northwest Wichita.

The district covers parts of north and west Wichita as well as Park City. It includes about 25 percent of the residents of the old District 105, about 20 percent of the old District 91, 21 percent of the old District 90, 27 percent of the old District 89 and 7 percent of the old District 85.

The winner in the Aug. 7 primary election will face Democrat Katelyn Delvaux in November.

Turn to Page 3B for information on the candidates and their stances on tax cuts, education and more.

Robin Clements

Age: 61

Occupation: CEO of Public Solutions LLC

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Gene Suellentrop and Robin Clements are GOP candidates in House District 91

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

BETHESDA, MD July 6, 2012 Yona Goldshmit, Ph.D., is a former physical therapist who worked in rehabilitation centers with spinal cord injury patients for many years before deciding to switch her focus to the underlying science.

"After a few years in the clinic, I realized that we don't really know what's going on," she said.

Now a scientist working with Peter Currie, Ph.D., at Monash University in Australia, Dr. Goldshmit is studying the mechanisms of spinal cord repair in zebrafish, which, unlike humans and other mammals, can regenerate their spinal cord following injury. On June 23 at the 2012 International Zebrafish Development and Genetics Conference in Madison, Wisconsin, she described a protein that may be a key difference between regeneration in fish and mammals.

One of the major barriers to spinal regeneration in mammals is a natural protective mechanism, which incongruously results in an unfortunate side effect. After a spinal injury, nervous system cells called glia are activated and flood the area to seal the wound to protect the brain and spinal cord. In doing so, however, the glia create scar tissue that acts as a physical and chemical barrier, which prevents new nerves from growing through the injury site.

One striking difference between the glial cells in mammals and fish is the resulting shape: mammalian glia take on highly branched, star-like arrangements that appear to intertwine into dense tissue. Fish glia cells, by contrast, adopt a simple elongated shape called bipolar morphology that bridges the injury site and appears to help new nerve cells grow through the damaged area to heal the spinal cord.

"Zebrafish don't have so much inflammation and the injury is not so severe as in mammals, so we can actually see the pro-regenerative effects that can happen," Dr. Goldshmit explained.

Studies in mice have found that mammalian glia can take up the same elongated shape, but in response to the environment around the injury they instead mature into scar tissue that does not allow nerve regrowth.

Dr. Goldshmit and her colleagues have focused on a family of molecules called fibroblast growth factors (Fgf), which have shown some evidence of improving recovery in mice and humans with spinal cord damage. The Monash University group found that Fgf activity around the damage site promotes the bipolar glial shape and encourages nerve regeneration in zebrafish.

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Zebrafish reveal promising mechanism for healing spinal cord injury

MANILA, Philippines Stem cell therapy, aside from being a potential cure for a wide range of illnesses, can also make a patient look and feel younger, a stem cell therapist said.

Dr. Ricardo Quiones, a cosmetic surgeon and dermatologist, has trained to conduct stem cell therapy, which he describes as the future of medicine.

Quiones said stem cell therapy has become popular for its ability to regenerate and heal properties of adult stem cells.

As we grow old, our stem cells dramatically decline. When we were children, we had 80 million stem cells. As we reach the age of 40, our stem cells decline to 35 million, he told Mornings@ANC on Friday.

Quiones explained that the procedure is similar to turning back the clock because it can increase a persons stem cells to 100 million.

Ive done two patients from Zamboanga City. I called them up after the procedure and they told me they look younger. They have the stamina, the vigor and they have felt an increase in short-term memory, powers of attention and concentration, he said.

Quiones also said the procedure has the potential to cure diabetes, heart damage, brain damage such as Parkinsons and Alzheimers, osteoarthritis, stroke, baldness and even sports injuries.

3-hour procedure

Quiones said any patient, except those diagnosed with cancer, can undergo the procedure, which he said will only last for about 3 to 4 hours.

After receiving clearance from a physician and passing medical and laboratory tests, anesthesia will be administered to a patient before stem cells are harvested.

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Stem cell therapy 'turns back clock'

BLOOMFIELD HILLS, MI--(Marketwire -07/06/12)- Arizona-based algae producer Health Enhancement Products (HEPI) was notified by Dr. Smiti Gupta, associate professor in the Department of Nutrition and Food Science at Wayne State University that research conducted by her team has been recently published in the Journal of Nutrition & Dietary Supplements, an international, peer-reviewed journal focused on academic research.

The article "ProAlgaZyme and its sub-fractions increase plasma HDL-cholesterol via up regulation of ApoA1, ABCA1 and SRB1 and inhibition of CETP in hypercholesterolemic hamsters," is authored by the team of Andreea Geamanu, Nadia Saadat, Arvind Goja, Monika Wadehra, Xiangming Ji and Smiti V. Gupta, all researchers associated with the Smiti Gupta laboratory at Wayne State University.

"Our data suggests that dietary intake of the algal extract ProAlgaZyme and its specific sub-fractions result in an improved cholesterol distribution in hamsters, primarily via its effects on multiple gene targets in the HDL (or reverse cholesterol transport) pathway," states Dr. Gupta. "Thus the potential for ProAlgaZyme to raise HDL, i.e. the 'good' cholesterol, warrants further investigation."

The article covers more than two years of work conducted by Dr. Gupta and her team. She continues to consult and work with the Company as various compounds and candidate molecules are isolated and analyzed, and is a member of an interdisciplinary working group of scientists and researchers.

Incoming Chief Science Officer Dr. Scott Freeman had this to say: "We congratulate Dr. Gupta and her team for achieving this milestone. These preliminary results commit us to further investigation in order to maximize future market potential."

About Wayne State University

Wayne State University is one of the nation's pre-eminent public research universities in an urban setting, ranking in the top 50 in R & D expenditures of all public universities by the National Science Foundation. Through its multidisciplinary approach to research and education, and its ongoing collaboration with government, industry and other institutions, the university seeks to enhance economic growth and improve the quality of life in the city of Detroit, state of Michigan and throughout the world. For more information about research at Wayne State University, visit http://www.research.wayne.edu.

About Health Enhancement Products, Inc.

Health Enhancement Products, Inc. (HEPI) is a health & wellness company engaged in the development of natural products derived from algae cultures for use as dietary supplements and food ingredients. These natural products are extracted from living algae grown in purified water.

Safe Harbor Statement

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Research Funded by Health Enhancement Products Published in Peer-Reviewed Journal of Nutrition & Dietary Supplements

Researchers have developed a moisturizing lotion with the potential to treat deadly skins cancers, psoriasis and promote diabetic wound healing.

The cream's unique molecular properties were engineered using nanotechnology, allowing its active ingredients to penetrate deeply into the skin to switch off disease-causing genes.

Nanotechnology involves the manipulation of miniscule particles, a thousand times smaller than the diameter of a human hair. The nanoparticles in the new lotion consist of nucleic acids of messenger RNA, a chain of genetic material designed to target the disease-causing genes in the skin cells.

Because these nucleic acids cannot get into cells in their normal linear form, researchers rearranged them into spherical structures. Then they organized the RNA spheres into a kind of densely-packed shell surrounding a gold nanoparticle.

The result was an object that could easily penetrate the deepest layers of skin cells and disable disease-causing genes responsible for conditions such as melanoma, a deadly skin cancer, and help the healing of diabetic foot wounds.

The gold in the structures allowed scientists to easily follow the movement of the nanoparticles. Chad Mirkin, director of the International Institute for Nanotechnology at Northwestern University in Illinois, developed the disease-fighting skin cream and says the nanoparticle-containing moisturizer opens up the possibility of what he calls topical gene regulation.

And what that means is we can take disease cells and flip genetic switches that correct the disease or, in the case of even things like cancer, cause cancer cells to selectively die," Mirkin says. "And thats really, really exciting because theres no other game in town in that regard.

Mirkin notes that at the same time they target disease-promoting genes, the so-called interfering RNA leaves the genes in the nuclei of healthy cells alone.

The nanoparticles were mixed in a thick, soothing cream called Aquaphor that can be applied directly to the skin. There, the cream enhances the skin's natural protective barrier to keep in bodily fluids, such as water, and keep out disease-causing molecules.

Amy Paller, head of Northwestern University's department of dermatology and its skin disease research center, worked with Mirkin on the cream. She says lab experiments in mice and with human skin samples showed the nanoparticles switched off a protein on skin-cell surfaces thats important to how they function. Shockingly, there was almost no change at all in any genes we were trying to suppress," Paller says. "We did not activate the immune system. So, we think we have a unique system that not only gets through the barrier and is taken up beautifully by cells and, to date doesnt seem to have any problem.

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Gene-Altering Lotion Could Fight Cancer, Skin Disease

SAN DIEGO, July 6, 2012 /PRNewswire/ -- Sequenom, Inc. (SQNM), a life sciences company providing innovative genetic analysis solutions, today confirmed that the United States District Court for the Northern District of California has denied a motion by Sequenom for a preliminary injunction against Ariosa Diagnostics.

"While obviously a disappointing preliminary outcome in our efforts to enforce an issued US patent against infringement, yesterday's decision is by no means a final ruling on the infringement or the validity of the '540 patent, but is one step in a long process to enforce our patent and protect our proprietary rights in the non-invasive prenatal testing marketplace," said Harry F. Hixson, Jr., Ph.D., Chairman and CEO, Sequenom, Inc.

Earlier this year, Sequenom filed the motion against Ariosa (formerly Aria) Diagnostics to stop the company from making, using, selling or offering for sale, importing or exporting, infringing tests for detecting fetal chromosomal aneuploidy, such as Ariosa's Harmony Prenatal Test, pending the ultimate resolution of the litigation.

Sequenom's request for preliminary injunctive relief followed its counterclaims filed in the lawsuit in the United States District Court for the Northern District of California, which allege that Ariosa is infringing U.S. Patent No. 6,258,540 ("'540 patent"). The case is ongoing and Sequenom will be seeking a full trial on the merits of its claims and all relief, including damages and a permanent injunction against Ariosa.

Using circulating cell free fetal nucleic acid as the analyte, as covered in the issued '540 patent, Sequenom Center for Molecular Medicine (Sequenom CMM) was the first fully certified clinical diagnostic laboratory to develop, fully validate in a major clinical study and bring to market a high precision non-invasive prenatal diagnostics laboratory developed test (LDT) for chromosomal aneuploidy. Sequenom CMM's MaterniT21 PLUS LDT detects a genetic chromosomal anomaly known as Trisomy 21, the most common cause of Down syndrome, as well as trisomies 18 and 13, in single, twin or triplet pregnancies. MaterniT21 PLUS is available as a testing service to physicians through Sequenom CMM.

About SequenomSequenom, Inc. (SQNM) is a life sciences company committed to improving healthcare through revolutionary genetic analysis solutions. Sequenom develops innovative technology, products and diagnostic tests that target and serve discovery and clinical research, and molecular diagnostics markets. The company was founded in 1994 and is headquartered in San Diego, California. Sequenom maintains a Web site at http://www.sequenom.com to which Sequenom regularly posts copies of its press releases as well as additional information about Sequenom. Interested persons can subscribe on the Sequenom Web site to email alerts or RSS feeds that are sent automatically when Sequenom issues press releases, files its reports with the Securities and Exchange Commission or posts certain other information to the Web site.

About Sequenom Center for Molecular MedicineSequenom Center for Molecular Medicine (Sequenom CMM) has two CAP accredited and CLIA-certified molecular diagnostics reference laboratories dedicated to the development and commercialization of laboratory developed tests for prenatal and eye conditions and diseases. Utilizing innovative proprietary technologies, Sequenom CMM provides test results that can be used as tools by clinicians in managing patient care. Testing services are available only upon request to physicians. Sequenom CMM works closely with key opinion leaders and experts in obstetrics, retinal care and genetics. Sequenom CMM scientists use a variety of sophisticated and cutting-edge methodologies in the development and validation of tests. Sequenom CMM is changing the landscape in genetic diagnostics. Visit http://www.scmmlab.com for more information on laboratory testing services.

Forward-Looking StatementsExcept for the historical information contained herein, the matters set forth in this press release, including statements regarding the process to enforce Sequenom's patent and protect its rights, that Sequenom will be seeking a full trial on the merits of its claims and all relief, including damages and a permanent injunction, the ultimate resolution of the litigation, Sequenom's commitment to improving healthcare through revolutionary genetic analysis solutions, and Sequenom CMM's dedication to the development and commercialization of laboratory developed tests and changing the landscape in genetic diagnostics, are forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995. These forward-looking statements are subject to risks and uncertainties that may cause actual results to differ materially, including the risks and uncertainties associated with ongoing patent litigation, Sequenom's ability to develop and commercialize new technologies and products, particularly new technologies such as prenatal and other diagnostics and laboratory developed tests, Sequenom's ability to manage its existing cash resources or raise additional cash resources, competition, intellectual property protection and intellectual property rights of others, government regulation particularly with respect to diagnostic products and laboratory developed tests, obtaining or maintaining regulatory approvals, ongoing litigation, and other risks detailed from time to time in Sequenom, Inc.'s most recent Annual Report on Securities and Exchange Commission Form 10-K and other documents subsequently filed with or furnished to the Securities and Exchange Commission. These forward-looking statements are based on current information that may change and you are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date of this press release. All forward-looking statements are qualified in their entirety by this cautionary statement, and Sequenom, Inc. undertakes no obligation to revise or update any forward-looking statement to reflect events or circumstances after the issuance of this press release.

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Sequenom Addresses Preliminary Injunction Decision, Reaffirms Defense of Patent

ScienceDaily (July 6, 2012) Dr Changfa Guo, Professor Chunsheng Wang and their co-investigators from Zhongshan hospital Fudan University, Shanghai, China have established a novel hyperbranched poly(amidoamine) (hPAMAM) nanoparticle based hypoxia regulated vascular endothelial growth factor (HRE-VEGF) gene therapy strategy which is an excellent substitute for the current expensive and uncontrollable VEGF gene delivery system.

This discovery, reported in the June 2012 issue of Experimental Biology and Medicine, provides an economical, feasible and biocompatible gene therapy strategy for cardiac repair.

Transplantation of VEGF gene manipulated mesenchymal stem cells (MSCs) has been proposed as a promising therapeutic method for cardiac repair after myocardium infarction. However, the gene delivery system, including the VEGF gene and delivery vehicle, needs to be optimized. On one hand, long-term and uncontrollable VEGF over-expression in vivo has been observed to lead to hemangioma formation instead of functional vessels in animal models. On the other hand, though non-viral gene vector can circumvent the limitations of virus, drawbacks of the current non-viral vectors, such as complex synthesis procedure, limited transfection efficiency and high cytotoxicity, still needs to be overcome.

Co-investigators, Drs. Kai Zhu and Hao Lai, said "Hypoxia response elements were inserted into the promoter region of VEGF gene to form HRE-VEGF, which provided a safer alternative to the conventionally available VEGF gene." "The HRE-VEGF up-regulates gene expression under hypoxic conditions caused by ischemic myocardium and turns it off under normoxia condition when the regional oxygen supply is adequate."

The hPAMAM nanoparticles, which exhibit high gene transfection efficiency and low cytotoxicity during the gene delivery process, can be synthesized by a simpler and more economical one-step/pot polymerization technique. Drs. Zhu and Lai, said "Using the hPAMAM based gene delivery approach, our published and unpublished results explicitly demonstrated that it was an economical, effective and biocompatible gene delivery vehicle."

Dr Guo concluded that "Treatment with hPAMAM-HRE-VEGF transfected MSCs after myocardium infarction improved the myocardial VEGF level, which improved graft MSC survival, increased neovascularization and ultimately improved heart function. And this novel VEGF gene delivery system may have clinical relevance for tissue repair in other ischemic diseases."

Dr. Steve Goodman, Editor-in-Chief of Experimental Biology and Medicine said "Guo and colleagues have provided an exciting new nanoparticle based gene therapy for cardiac repair. This novel approach has great promise for repair of the heart after myocardial infarction."

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An economical, effective and biocompatible gene therapy strategy promotes cardiac repair

05-07-2012 03:11 Facts about ACTL by a Polish Doctor escorting her patient from Poland to China, and the very truth experiences presented by patient's relative.

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Facts about ACTL targeted cancer cell therapy.wmv - Video

Chinese scientists identify yield-boosting rice gene

Li Jiao

5 July 2012 | EN |

Scientists say the new gene could improve both rice yields and quality, in China and across the world

Flickr/randomwire

[BEIJING] Researchers in China have identified a rice gene that could improve both the quality and yield of the staple crop.

Xiangdong Fu, a geneticist at the Institute of Genetics and Development Biology of the Chinese Academy of Sciences in Beijing, China, and colleagues first discovered the gene known as GW8 while studying basmati rice in Pakistan.

Basmati rice is well known for its good grain quality. The researchers found that this feature is influenced by the presence of a gene known as GW8, which can also improve the appearance and flavour of rice.

Fu and his colleagues hypothesised that high-quality Chinese rice varieties might also have the GW8 gene.

Following a series of field studies in Beijing, Guangzhou and Hainan in 2009, the researchers found that a variant of the GW8 gene does exist in certain varieties of high-yielding rice in China.

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Chinese scientists identify yield-boosting rice gene

NEW YORK - The days of pregnant women having a 3-inch-long (8-centimetre-long) hollow needle jabbed into their abdomens may be numbered.

For the second time in a month, scientists have announced that a simple blood test, rather than more invasive tests such as amniocentesis, can determine a fetus's genetic make-up, identifying mutations causing any of about 3,000 inherited disorders that arise from a glitch in a single gene, such as cystic fibrosis.

Unlike a procedure unveiled last month, the one announced Wednesday in the journal Nature can be done without knowing who the father is, much less obtaining a sample of his DNA. Since paternity is unknown or incorrect in an estimated 3 to 10% of births in the United States, the father-free method promises to make fetal DNA sequencing possible in every pregnancy, if hurdles including cost and accuracy are overcome.

"We're really on the verge of an enormous increase in our ability to understand what an infant will be like," said Dr Michael Katz, a senior adviser to the March of Dimes, a foundation that supports research on pregnancy and birth defects. Katz was not involved in the study. "You'll be able to detect any kind of abnormality early, quickly, without distress and safely. This is the way of the future."

Determining a fetus's genome might give women more reasons to end a pregnancy. But it would also let physicians identify conditions that can be treated before birth or immediately after, said Stephen Quake of Stanford University in California, who led the new study: "The way it's done now, parents wait until a newborn gets sick and suffers in the first weeks of life, and only then does the doctor start figuring out the baby has a metabolic or immune disorder."

With prenatal genetic testing, in contrast, the parents would know by the end of the first trimester (12 to 13 weeks) if the fetus has a genetic or chromosomal defect. That way, they can be ready if the baby has special needs, which can be as simple as a certain diet.

Knowing so early that something has gone wrong might also allow physicians to treat a fetus. Prenatal surgery, introduced 30 years ago, is currently performed at a few specialized hospitals to correct just a few heart, bladder or other defects.

"Now we can challenge our colleagues in surgery and pharmacology," said Quake. "We'll soon be able to diagnose all these genetic disorders; what are you going to do about them?"

ABORTING UNWANTED TRAITS?

Knowing every detail of a fetus's genome could open the door to more controversial steps, however. In China and India, parents use ultrasound for sex selection, aborting 1.3 million to 1.6 million female fetuses every year, according to estimates in a 2011 study in the journal Lancet and a 2009 study in the World Bank Economic Review.

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Editor's Choice Main Category: Alzheimer's / Dementia Also Included In: Genetics Article Date: 05 Jul 2012 - 10:00 PDT

Current ratings for: Researchers Working Hard To Unlock Alzheimer's Genetic Secrets

The study, which will be conducted by researchers at Indiana University School of Medicine and colleagues across the country, could significantly affect the development of treatments for Alzheimer's.

The researchers will sequence and examine genomes of more than 800 adults taking part in the Alzheimer's Disease Neuroimaging Initiative (ADNI). The study, supported by the Brin Wojcicki Foundation and the Alzheimer's Association, will provide a listing of all 3 billion segments (base pairs) of genetic code in each participant's DNA.

Andrew Saykin, Psy.D., director of the Indiana University Center for Neuroimaging and lead researcher for the genetics core of the Alzheimer's Disease Neuroimaging Initiative, explained: "This is the equivalent of going from a good quality map of the United States to having the detailed blueprints for everything within our borders."

The DNA samples gathered from participants are stored at the National Cell Repository for Alzheimer's Disease at the IU School of Medicine. The repository, directed by Tatiana Foroud, Ph.D., P. Michael Conneally Professor of Medical and Molecular Genetics, prepared the DNA samples that will be used in the study.

Dr. Foroud and Li Shen are co-lead researchers for the genetics core. Shen, Ph.D., is a computer scientist and assistant professor of radiology and imaging sciences. Illumina, a biotechnology equipment and services company will conduct the sequencing.

Martin Farlow, M.D., professor of neurology and associate director of the Indiana Alzheimer's Disease Center, explained:

At present, researchers around the world use data produced by the ADNI, but the new study will provide next-generation sequencing data on a significantly larger and refined scale.

Dr. Saykin, Raymond C. Beeler Professor of Radiology and Imaging Sciences. said:

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Researchers Working Hard To Unlock Alzheimer's Genetic Secrets

NEW YORK, July 5, 2012 /PRNewswire/ --Reportlinker.com announces that a new market research report is available in its catalogue:

Therapy Trends: Alzheimer`s Disease - Breaking new ground in disease modificationhttp://www.reportlinker.com/p0924465/Therapy-Trends-Alzheimer`s-Disease---Breaking-new-ground-in-disease-modification.html#utm_source=prnewswire&utm_medium=pr&utm_campaign=Therapy

An incisive, dynamic analytical report that uses insight from the most influential key opinion leaders (KOLs) in Alzheimer's disease (AD) to map the current treatment landscape and identify future trends.Alzheimer's Disease: Breaking new ground in disease modification

By the end of 2012, the Alzheimer's disease market will reach a pivotal stage with the expected release of Phase III results of disease-modifying therapies, and new research into biomarkers to aid early AD diagnosis. If positive, these results will represent a ground-breaking milestone in AD treatment. Recent trends in the AD market also represent a clear shift in researchers' approaches to detecting, treating and preventing AD.

'Therapy Trends: Alzheimer's Disease' is compiled from exclusive, in-depth interviews with the world's leading KOLs in AD. It identifies and analyses the major factors, advances and trends currently influencing the AD treatment landscape. The report focuses on late-stage pipeline products, and how these could modify future AD management.Drive your strategic decision-making with inside intelligence'Therapy Trends: Alzheimer's Disease' disseminates the critical opinions of KOLs, giving you greater insight to the latest AD market advances. This information includes:

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Therapy Trends: Alzheimer`s Disease - Breaking new ground in disease modification

NOVATO, Calif., July 5, 2012 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc., a biotechnology company focused on developing treatments for rare and ultra-rare genetic disorders, today announced the dosing of the first two patients in a Phase 2 study of UX001 for hereditary inclusion body myopathy (HIBM). HIBM is a rare, severe, neuromuscular disease caused by sialic acid deficiency. UX001 is an extended-release oral tablet formulation of sialic acid (SA-ER) intended as a substrate replacement therapy for HIBM.

The Phase 2 clinical trial is an international, multi-center, randomized, double-blind, placebo-controlled, parallel group study of UX001 in HIBM patients. The study plans to enroll up to 45 patients between 18 and 65 years of age with a previously demonstrated mutation in the GNE gene causing HIBM. The subjects will receive either of two dose levels of SA-ER or placebo over 24 weeks, with all patients continuing on active treatment after 24 weeks. The study's primary objectives are evaluating safety, and improvements in sialylation biochemistry of muscle (pharmacodynamic endpoint). Clinical and patient-reported outcomes will also be evaluated, though the study is not powered for these endpoints. Study sites are located in the US and Israel. The total duration of the Phase 2 study is up to 48 weeks, with data expected in 2013.

Emil D. Kakkis, MD, PhD, Chief Executive Officer of Ultragenyx commented: "The initiation of this Phase 2 study is a critical milestone for our team in developing a therapeutic for HIBM patients who currently lack treatment options for this devastating disease. It follows quickly upon the positive results from our Phase 1 trial. This Phase 2 study should help us determine if UX001 is improving the biochemistry of the muscle in these patients and help us learn more about the disease. We look forward to seeing top line results next year."

About HIBM

HIBM is also known as GNE myopathy, Quadriceps Sparing Myopathy (QSM), Inclusion Body Myopathy type 2, Distal Myopathy with Rimmed Vacuoles (DMRV) and Nonaka myopathy. HIBM is a severe, adult-onset, progressive, genetic neuromuscular disease caused by a deficiency of an enzyme in the first step of sialic acid biosynthesis needed for the modification of proteins and fats. Patients with HIBM typically begin to have weakness and abnormal walking at 18 to 30 years of age. Over the ensuing 10 to 20 years, many patients progressively lose significant functional ability and become wheelchair-bound. There are no current treatments for this disease.

About Ultragenyx

Ultragenyx is a privately held, developmental stage biotechnology company committed to bringing life-enhancing therapeutics for patients with rare and ultra-rare genetic diseases, also known as orphan and ultra-orphan diseases, to market. The company focuses on rare metabolic diseases that affect small numbers of patients, but for which the unmet medical need is high and there are no effective treatments. Ultragenyx intends to build a sustainable pipeline of safe and effective therapies to address these underserved diseases. Ultragenyx' lead program, UX001, is being evaluated as a potential treatment for hereditary inclusion body myopathy (HIBM), also known as GNE myopathy. The UX001 program has been granted orphan drug designation in the US and the EU.

The company is led by an experienced management team in rare disease therapeutics. Ultragenyx is striving toward an improved model for successful rare disease drug development, which has the potential to increase efficiency while maintaining appropriate safety and efficacy standards. The company believes that it can deliver significant value to patients by building a high-quality pipeline of rare disease therapeutics and efficiently transforming good science into great medicine.

For more information on Ultragenyx, please visit the company's website at http://www.ultragenyx.com.

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Ultragenyx Initiates Phase 2 Study of UX001 in Hereditary Inclusion Body Myopathy, a Rare Neuromuscular Disease

Researchers said Wednesday they were able to sequence the entire genome of a fetus using only a blood sample from the mother, an advance in the effort to find noninvasive ways for expectant parents to determine if their babies will be born with genetic conditions.

The findings, from researchers at Stanford University School of Medicine, reflect intense interest in finding fast, relatively inexpensive and accurate ways to predict genetic conditions without the risks associated with currently available tests. Amniocentesis, for instance, which many couples rely on to obtain some genetic information about the fetus, requires insertion of a needle through the walls of a pregnant woman's abdomen and uterus, and has a small risk of miscarriage. The study was funded by the National Institutes of Health and the Howard Hughes Medical Institute.

The Stanford technique, researchers said, doesn't require DNA from the father, an advantage given that a child's paternity may not be known in as estimated 3% to 10% of births in the U.S., according to sources cited in the Nature paper. "It is not that practical to assume you can get DNA from dad or you even know who dad is,'' said Stephen R. Quake, a professor of applied physics and bioengineering at Stanford and senior author of the study. The findings were published in the July 4 issue of the journal Nature.

The study grew out of a discovery made in the late 1990s that a fetus releases DNA into the mother's blood during pregnancy. A number of companies, including two co-founded by Dr. Quake, are already using molecular counting techniques to measure the presence of elevated amounts of chromosome 21 relative to other chromosomes in a mother's blood to determine if a fetus has Down syndrome, a genetic condition that causes cognitive disabilities. The current study takes the method "and applies it to the whole genome,'' said Dr. Quake.

It will probably take a few more years before such noninvasive whole genome tests are ready for use in the clinic, said Jay A. Shendure, associate professor of genome sciences at the University of Washington, who wasn't involved in the latest research. He was principal investigator in a study published last month in Science Translational Medicine that sequenced the fetal genome using a blood sample from the mother and saliva from the father.

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Mother's blood shows birth defects in fetal DNA

July 5, 2012

redOrbit Staff & Wire Reports Your Universe Online

Scientists say they have put together a more complete string of genetic letters that may control how well parrots learn to imitate their owners and other noises.

Researchers unraveled the certain regions of the parrots genome using a new technology, single molecule sequencing, and fixing its flaws with data from older DNA-decoding devices. Researchers also decoded hard-to-sequence genetic material from corn and bacteria as proof of their new sequencing approach. The results of the study appeared online recently in the journal Nature Biotechnology.

Single molecule sequencing got a lot of hype last year because it generates long sequencing reads, supposedly making it easier to assemble complex parts of the genome, said Duke University neurobiologist Erich Jarvis, a co-author of the study.

Jarvis is interested in the sequences that regulate parrots imitation abilities because they could give neuroscientists information about the gene regions that control speaking development in humans.

Jarvis began his project with others by trying to piece together the genome regions with what are known as next-generation sequencers, which read chunks of 100 to 400 DNA base pairs at a time and then take a few days to put them together into a draft genome. After doing the sequencing, the scientists noticed that the read lengths were not long enough to assemble the regulatory regions of some of the genes that control brain circuits for vocal learning.

University of Maryland computational biologists Adam Phillippy and Sergey Koren experts at assembling genomes heard about Jarviss sequencing struggles at a conference and approached him with a possible solution of adjusting the algorithms that order the DNA base pairs. But the fix was still not sufficient.

Last year, 1000 base-pair reads by Roch 454 became available, as did the single molecule sequencer by Pacific Biosciences. The Pacbio technology generates strands of 2,250 to 23,000 base pairs at a time and can draft an entire genome in about a day.

Jarvis and others assumed the new technologies would solve the genome-sequencing challenges. Through a competition, called the Assemblathon, the scientists discovered that the Pacbio machine had trouble accurately decoding complex regions of the parrot, Melopsittacus undulates, genome. The machine had a high error rate, generating the wrong genetic letter at every fifth or sixth spot in a string of DNA. The errors made it nearly impossible to create a genome assembly with the very long reads, Jarvis said.

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Genetics Could Explain Parrots Ability To Parrot

(07-04) 12:00 PDT Stanford -- In a discovery that widens a lens into lives not yet lived, Stanford University School of Medicine researchers have for the first time determined an unborn child's genome with nothing but a blood sample from the mother.

This new approach to genetic testing, the scientists say, could expand families' ability to screen for potential disorders in fetuses without the risk of miscarriage that comes with conventional tests. In a few years, the testing could be part of a routine trip to the doctor.

But outside experts argue it raises an ethical question that physicians and parents are not prepared to answer: Who deserves to be born?

"Many families would dread having a child with Down syndrome," said Marcy Darnovsky, associate executive director of the Center for Genetics and Society, a public interest group in Berkeley. "And, absolutely no questions asked, that would be a reason for them to terminate."

The latest development in genome testing, outlined Wednesday in the science journal Nature, falls in the same vein as a June study from the University of Washington. Scientists there sequenced a fetus's DNA using both a blood sample from the pregnant woman and a saliva sample from the father.

The Stanford team accomplished the same feat with Dad out of the picture. Their method is advantageous, they say, when a child's paternity is unknown, as it is for an estimated 3 to 10 percent of births in the United States.

"Oftentimes, the person who thinks he's a father is not the biological father," said the study's senior author, Stephen Quake, a professor of bioengineering and applied physics.

With his approach, which can be used any time during the pregnancy, parents could screen for a range of medical conditions, allowing them to prepare for proper care, Quake said.

"Things like metabolic disorders and immune disorders you could find out ahead of time, so when the baby's born, you know exactly what to feed them or, more importantly, not to feed them so they don't get sick; or what environment they need to be in to protect them from germs," Quake said.

But one ethicist pointed to a potential dark side.

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Stanford researchers move fetal genome testing ahead

Toxic chemicals wreak havoc on cells, damaging DNA and other critical molecules. A new study from researchers at MIT and the University at Albany reveals how a molecular emergency-response system shifts the cell into damage-control mode and helps it survive such attacks by rapidly producing proteins that counteract the harm.

Peter Dedon, a professor of biological engineering at MIT, and colleagues had previously shown that cells treated with poisons such as arsenic alter their chemical modification of molecules known as transfer RNA (tRNA), which deliver protein building blocks within a cell. In their new paper, appearing in the July 3 issue of Nature Communications, the research team delved into how these modifications help cells survive.

The researchers found that toxic stresses reprogram the tRNA modifications to turn on a system that diverts the cell's protein-building machinery away from its routine activities to emergency action. "In the end, a stepwise mechanism leads to selective expression of proteins that you need to survive," says Dedon, senior author of the Nature Communications paper.

The findings offer insight into not only cells' response to toxins, but also their reactions to all kinds of stimuli, such as nutrients or hormones, Dedon says. "We're proposing that any time there's a stimulus, you're going to have a reprogramming [of tRNA] that causes selective translation of proteins you need for the next step in whatever you're going to do," he says.

Lead author of the paper is recent MIT PhD recipient Clement Chan. Other MIT authors are postdocs Yan Ling Joy Pang and Wenjun Deng and research scientist Ramesh Indrakanti. Authors from the University at Albany are Thomas Begley, an associate professor of nanobioscience, and research scientist Madhu Dyavaiah.

A new role for RNA

Transfer RNA is made of 70 to 90 ribonucleotide building blocks. After synthesis, the ribonucleotides usually undergo dozens of chemical modifications that alter their structure and function. The primary job of tRNA is to bring amino acids to the ribosomes, which string them together to make proteins.

In a 2010 paper, Dedon and colleagues exposed yeast cells to different toxic chemicals, including hydrogen peroxide, bleach and arsenic. In each case, the cells responded by uniquely reprogramming the location and amount of each tRNA modification. If the cells lost the ability to reprogram the modifications, they were much less likely to survive the toxic attack.

In the new study, the researchers focused on a particular tRNA modification, known as m5C, which occurs when cells encounter hydrogen peroxide, a chemical produced by white blood cells.

They first discovered that this modification occurs predominantly in one of the tRNAs that carry the amino acid leucine. Every amino acid is encoded by three-letter sequences in the genome called codons. Each tRNA corresponds to one amino acid, but most amino acids can be coded by several tRNA sequences. For example, leucine can be coded by six different genome sequences: TTA, TTG, CTT, CTC, CTA and CTG.

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Genetic 911: Study examines how cells exploit gene sequences to cope with toxic stress

02-07-2012 14:16 This is the VOA Special English Agriculture Report, from | http Scientists have made a genetic map of the tomato. Tomatoes are second only to potatoes as the world's most valuable vegetable crop. Eight years of work went into making the map, or genome. Three hundred scientists around the world took part in the project to sequence the tomato's DNA code. Giovanni Giuliano, a researcher in Italy, is part of the Tomato Genome Consortium. He says they started as ten countries and now have fourteen. Having the tomato's genetic map will help growers who are always trying to produce a better tomato. Mr. Giuliano says they now know not only what genes are there, but their order. Researchers published the genome of a tomato used by Heinz, the American food company famous for its tomato ketchup. Ketchup is a thick sauce used on hamburgers, hot dogs and other foods. Heinz's research manager, Rich Ozminkowski, says the company knows what it wants in a tomato. "Traits like sugars and, for Heinz, viscosity, or the juice thickness, and the redness of the tomatoes are all very critical traits for us," he says. Those are all controlled by a lot of different genes within the tomato. Mr. Ozminkowski says genome sequencing takes away much of the guesswork for breeders of tomatoes or other crops that have been mapped. In his words, "By having the genome information, we can pick out those tomato plants that have more of those genes." Until the late nineteen sixties, the ...

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Tomato's Genetic Secrets Are Peeled Away - Video

SHENZHEN, China, July 3, 2012 /PRNewswire-Asia/-- A study conducted by Beike Biotechnology Company (http://www.beikebiotech.com) in conjunction with physicians and researchers at two Chinese hospitals, documents the effectiveness of cord blood-derived stem cells in treating primary biliary cirrhosis (PBC). The study, which was published in the April 2012 issue of the Stem Cell Discovery, was the first of its kind. Researchers noted that additional clinical trials would be required before stem cells can become an accepted therapy for liver cirrhosis.

Prof. Jin-hui Yang, Director of the Department of Hepatology in the 2nd Affiliated Hospital of Kunming Medical College stated, "Given the severity of liver cirrhosis and its related conditions, and the limited number of options available to treat those who suffer from it, this finding represents an important, potentially significant breakthrough."

PBC is a chronic, progressive liver disease that leads eventually to fibrosis and cirrhosis of the liver. It affects 1 in 1,000 women over the age of 40.Approximately one-third of those who suffer from PBC and its related conditions do not respond well to Ursodeoxycholic acid (UDCA) treatment, which is the only currently FDA-approved standard medical treatment for the condition. Many of those patients ultimately require liver transplantation.

Beike Chairman, Dr. Sean Hu, commented, "With a growing body of research that demonstrates the effectiveness of cord blood-derived stem cell therapies in treating a broad range of chronic conditions, this latest study is a milestone in the continuing effort to gain broad acceptance and recognition of regenerative medicine as a mainstream treatment option.We look forward to conducting more comprehensive clinical trials to attempt to validate the positive outcomes we have already observed."

The case study reported in the Stem Cell Discovery involved a 58 year old woman suffering from PBC who developed an incarcerated hernia and uncontrolled hydrothorax after undergoing UDCA treatment.One week after completing two stem cell transplantations with no observed adverse effects, the patient showed improvement in both liver function and in her general condition. She was released from the hospital but continued to receive twice-daily UDCA treatments. Six months after her discharge, doctors observed continued improvements in her liver function and overall condition.

To review the full text of the published study, please visit: http://www.scirp.org/journal/PaperInformation.aspx?paperID=18710. Study authors included physicians and researchers from the 2nd Affiliated Hospital of Kunming Medical College, Beike Biotechnology Company, and the Yunnan Provincial 1st People's Hospital in Kunming, China.

About Beike Biotechnology Company

Shenzhen Beike Biotechnology Co., Ltd. is China's leading biotechnology company focusing on the production of adult stem cells for use in medical therapies. Headquartered in Shenzhen (near Hong Kong) with a flagship regenerative medicine facility at the China Medical City in Jiangsu province, Beike produces a full line of stem cell products derived from umbilical cord, cord blood and autologous bone marrow.

For any questions regarding this release, please call:

Contact Person: T. Gutmann Phone Number: +86-532-6677-6659

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Stem Cell Therapy Shown to be Effective in Treating Liver Cirrhosis