Durham, NC (PRWEB) March 11, 2015

Scientists report in the current issue of STEM CELLS Translational Medicine that they have been able to clone a line of defective stem cells behind a rare, but devastating disease called Fanconi Anemia (FA). Their achievement opens the door to drug screening and the potential for a new, safe treatment for this often fatal disease.

FA is a hereditary blood disorder that leads to bone marrow failure (FA-BMF) and cancer. Patients who suffer from FA have a life expectancy of 33 years. Currently, a bone marrow transplant offers the only possibility for a cure. However, this treatment has many risks associated with it, especially for FA patients due to their extreme sensitivity to radiation and chemotherapy.

Although various consequences in hematopoietic stem cells (the cells that give rise to all the other blood cells) have been attributed to FA-BMF, its cause is still unknown, said Megumu K. Saito, M.D., Ph.D., of Kyoto Universitys Center for iPS Cell and Application, and a lead investigator on the study. His laboratory specializes in studying the kinds of pediatric diseases in which a thorough analysis using mouse models or cultured cell lines is not feasible, so they apply disease-specific induced pluripotent stem cells (iPSCs) instead.

To address the FA issue, he explained, our team (including colleagues from Tokai University School of Medicine) established iPSCs from two FA patients who have the FANCA gene mutation that is typical in FA. We were then able to obtain fetal type immature blood cells from these iPSCs.

When observing the iPSCs, the researchers found that the characteristics of immature blood cells from FA-iPSCs were different from control cells. The FA-iPSCs showed an increased DNA double-strand break rate, as well as a sharp reduction of hematopoietic stem cells compared to the control group of non-FA iPSCs.

These data indicate that the hematopoietic consequences in FA patients originate from the earliest hematopoietic stage and highlight the potential usefulness of iPSC technology for explaining how FA-BMF occurs, said Dr. Saito. Since conducting a comprehensive analysis of patient-derived affected stem cells is not feasible without iPSC technology, the technology provides an unprecedented opportunity to gain further insight into this disease.

This work shows promise for identifying the initial pathological event that causes the disease, which would be a first step in working toward a cure, said Anthony Atala, M.D., Editor-in-Chief of STEM CELLS Translational Medicine and director of the Wake Forest Institute for Regenerative Medicine.

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The full article, Pluripotent cell models of Fanconi anemia identify the early pathological defect in human hemoangiogenic progenitors, can be accessed at http://www.stemcellstm.com.

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March 10, 2015

These are human blood cells grown in the lab from genetically edited stem cells. (Credit: Ying Wang/Johns Hopkins Medicine)

Provided by Shawna Williams, Johns Hopkins Medicine

Researchers at Johns Hopkins have successfully corrected a genetic error in stem cells from patients with sickle cell disease, and then used those cells to grow mature red blood cells, they report. The study represents an important step toward more effectively treating certain patients with sickle cell disease who need frequent blood transfusions and currently have few options.

The results appear in an upcoming issue of the journalStem Cells.

In sickle cell disease, a genetic variant causes patients blood cells to take on a crescent, or sickle, shape, rather than the typical round shape. The crescent-shaped cells are sticky and can block blood flow through vessels, often causing great pain and fatigue. Getting a transplant of blood-making bone marrow can potentially cure the disease. But for patients who either cannot tolerate the transplant procedure, or whose transplants fail, the best option may be to receive regular blood transfusions from healthy donors with matched blood types.

[STORY: New injection helps stem traumatic blood loss]

The problem, says Linzhao Cheng, Ph.D. , the Edythe Harris Lucas and Clara Lucas Lynn Professor of Hematology and a member of the Institute for Cell Engineering, is that over time, patients bodies often begin to mount an immune response against the foreign blood. Their bodies quickly kill off the blood cells, so they have to get transfusions more and more frequently, he says.

A solution, Cheng and his colleagues thought, could be to grow blood cells in the lab that were matched to each patients own genetic material and thus could evade the immune system. His research group had already devised a way to use stem cells to make human blood cells. The problem for patients with sickle cell disease is that lab-grown stem cells with their genetic material would have the sickle cell defect.

To solve that problem, the researchers started with patients blood cells and reprogrammed them into so-called induced pluripotent stem cells, which can make any other cell in the body and grow indefinitely in the laboratory. They then used a relatively new genetic editing technique called CRISPR to snip out the sickle cell gene variant and replace it with the healthy version of the gene. The final step was to coax the stem cells to grow into mature blood cells. The edited stem cells generated blood cells just as efficiently as stem cells that hadnt been subjected to CRISPR, the researchers found.

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Two landmark studies link the 3-D arrangement of the genome to the variability and control of its expression

February 18, 2015, New York, NY - Two international teams of researchers led by Ludwig San Diego's Bing Ren have published in the current issue of Nature two papers that analyze in unprecedented detail the variability and regulation of gene expression across the entire human genome, and their correspondence with the physical structure of chromosomes.

"We expect that our findings, which describe the interplay of chromosomal structure, regulation and gene expression across a broad array of tissues, will inform research in every branch of mammalian biology and provide information of great value to the study of most human diseases, not least cancer," said Ren.

If the human genome is a recipe book, its chapters are 23 distinct chromosomes--each of which is stuffed, in rough duplicate, into the nucleus of almost all the cells of the human body. But how exactly is that single recipe book read appropriately to build the body's diverse constituency of cells? Or, for that matter, to generate a community of humans so variegated in their appearance, internal biochemistry and susceptibility to disease?

The two papers address key elements of these riddles. One captures the extent to which the same genes--known as alleles--inherited from each parent are expressed at different levels across the genome, so that each version of the gene generates different amounts of the protein it encodes. It links that difference in expression to the distribution and sequence of "enhancers" on each copy of each chromosome. Enhancers are stretches of DNA that do not encode proteins but can boost gene expression from great distances along the linear strand of DNA.

"This is the first time that anyone has looked globally at how gene expression differs between each matching pair of chromosomes across a diverse set of cell types, and our findings are striking," said Ren. "Some 30 percent of the gene set we carry is expressed variably across some 20 types of tissues, depending on which parent the alleles came from. Much of that variation appears to come from differences in sequences that regulate the transcription--or reading--of genes."

The other study examines how the three dimensional structure of chromosomes and the distribution of biochemical (or epigenetic) tags that regulate gene expression differ between different types of cells. It also integrates data from the former paper into this analysis to reveal how all of these phenomena interact to control the appropriate expression of the genome. Taken together, these findings add dimension and depth to our understanding of the physical and functional dynamics of the genome, and how its expression is globally regulated to generate the sublime complexity of the human body.

Both studies are invaluable to a deeper understanding of normal biology as well as disease. The data will, for example, help explain precisely why particular parental traits are often so unevenly expressed and why specific deleterious mutations vary in their effects from person to person. They will also serve as a reference that researchers can use to develop a more sophisticated understanding of how gene regulation and chromosomal structure are altered in diseases such as cancer.

Stemming from five years of research, the papers are two of six published this week in Nature that capture the key discoveries of the $300 million Roadmap Epigenomics Program of the US National Institutes of Health. Ren led one of four reference epigenome mapping centers for the program, and his center focused primarily on how DNA and chromatin--the complex of DNA and its protein packaging that makes chromosomes--are chemically tagged at specific places to control the expression of genes across the human genome.

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Deconstructing the dynamic genome

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First ever genome-wide association study on common, incurable skin condition pinpoints 2 genetic variants associated with rosacea

MOUNTAIN VIEW, Calif., March 10, 2015 -- Today marked the publication of the first ever genome-wide association study of rosacea, a common and incurable skin disorder. Led by Dr. Anne Lynn S. Chang of Stanford University's School of Medicine, and co-authored by 23andMe, the study is the first to identify genetic factors for this condition.

Rosacea (pronounced roh-ZAY-sha) is estimated to affect more than 16 million people in the United States alone1. Symptoms typically include redness, visible blood vessels, and pimple-like sores on the skin of the central face, and many experience stinging, burning, or increased sensitivity over the affected skin. Because rosacea affects facial appearance, it can also have a psychological impact on those who suffer from it. In surveys by the National Rosacea Society, more than 76 percent of rosacea patients said their condition had lowered their self-confidence and self-esteem.

To help better understand the genetics of rosacea, researchers at Stanford University and 23andMe studied the data of more than 46,000 23andMe customers* consented for research. The study, published in the Journal of Investigative Dermatology, found two genetic variants strongly associated with the disease among people of European ancestry.

Further, the study uncovered that the genetic variants, or single nucleotide polymorphisms (SNPs), found to be strongly associated with rosacea are in or near the HLA-DRA and BTNL2 genes, which are associated with other diseases, including diabetes and celiac disease.

The genome-wide association study was broken into two parts: discovery and validation. First, data voluntarily submitted by 22,000 23andMe customers was examined. More than 2,600 customers reported having received a rosacea diagnosis from a physician. The remainder of the study participants did not have the condition and were treated as controls. To validate findings from this initial group, 23andMe researchers then tested the identified SNPs with a separate group of 29,000 consented 23andMe customers (3,000 rosacea patients, 26,000 controls). The researchers were able to confirm the same association with rosacea.

"This is another example of how 23andMe can help in researching common yet poorly understood diseases," said Joyce Tung, Ph.D., 23andMe's director of research and a co-author of the paper. "The study also speaks to the power of large data sets in studying and identifying genetic associations."

In addition to the genome-wide association study, the research included obtaining skin biopsies from six individuals with rosacea and showed that both HLA-DRA and BTNL2 proteins can be found in the skin of people with rosacea. This preliminary work hints toward the biological relevance of HLA-DRA and BTNL2 in rosacea.

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Full paper citation and availability: Assessment of the Genetic Basis of Rosacea by Genome-Wide Association Study

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Lipitor and other statin drugs are commonly prescribed to lower cholesterol. Paul J. Richards/AFP/Getty Images hide caption

Lipitor and other statin drugs are commonly prescribed to lower cholesterol.

Millions of people take statins to lower their cholesterol and reduce the risk of cardiovascular disease. But taking statins does slightly up the risk of Type 2 diabetes. Figuring out whether that means "No statins for you" isn't always easy, despite a proliferation of guidelines intended to help.

Here's in interesting wrinkle: If you've got a hereditary form of high cholesterol you're much less likely to get Type 2 diabetes, according to a study published Tuesday in JAMA, the journal of the American Medical Association.

That's good news for those people, who often have high levels of LDL cholesterol starting in childhood and face a high risk of heart disease and stroke. And it offers intriguing hints as to a possible link between cholesterol receptors in the body and Type 2 diabetes.

To find that out, researchers in the Netherlands delved into an amazing database that has tracked people for familial hypercholesterolemia since 1994. The large number of people tested 63,320 made it possible to not only identify people with genetic mutations that caused the high cholesterol, but to show how it runs in families.

The people with familial hypercholesterolemia had a 51 percent lower risk of Type 2 diabetes than their relatives without the disorder. But the diabetes risk for both groups was low: 1.75 percent versus 2.93 percent. It varied based on the particular genetic mutation involved. That difference makes for a nifty demonstration on how genes affect risk, and confirms a link that doctors who treat patients with the disorder have long observed.

And it also may explain why taking statins boosts the risk of Type 2 diabetes in some people.

One theory on how statins work is that they encourage cells to hoover up the bad LDL cholesterol by turning on LDL receptors. That's good for lowering cholesterol levels in the blood, but the study authors said it may also end up damaging the pancreas, which has lots of LDL receptors and controls blood sugar.

"They're speculating that this LDL receptor may be important in some way in determining the risk of diabetes in a statin," says David Preiss, a metabolic physician at the Glasgow Cardiovascular Research Center at the University of Glasgow who wrote an editorial accompanying the JAMA study. "The data they show is quite strongly supportive of that."

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Personalized Medicine-Risk Assessment with Final Revisions for Final Review - TEMP Link

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Scientist: Personalized Medicine -Susan Baxter Career Girls Role Model
Susan Baxter, scientist and executive director of CSU Program for Education and Research in Biotechnology, shares valuable career guidance and life advice with girls. Learn how to become a...

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WALKASSIST FOR A SPINAL CORD INJURY PATIENT
WALKASSIST WITH A PULLEY FOR WEIGHT SUSPENSION OF PATIENTS WITH NEURO AND SPINAL CORD INJURY.

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New Science of Anti Aging and Regenerative Medicine Robert Goldman, MD, PhD, FAASP, DO, FAOASM
Highlights from a presentation given by Robert Goldman, MD, PhD, FAASP, DO, FAOASM at the 2007 Anti-Aging London Conference talking about "New Science of Anti-Aging and Regenerative ...

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IMAGE:These are human blood cells grown in the lab from genetically edited stem cells. view more

Credit: Ying Wang/Johns Hopkins Medicine

Researchers at Johns Hopkins have successfully corrected a genetic error in stem cells from patients with sickle cell disease, and then used those cells to grow mature red blood cells, they report. The study represents an important step toward more effectively treating certain patients with sickle cell disease who need frequent blood transfusions and currently have few options.

The results appear in an upcoming issue of the journal Stem Cells.

In sickle cell disease, a genetic variant causes patients' blood cells to take on a crescent, or sickle, shape, rather than the typical round shape. The crescent-shaped cells are sticky and can block blood flow through vessels, often causing great pain and fatigue. Getting a transplant of blood-making bone marrow can potentially cure the disease. But for patients who either cannot tolerate the transplant procedure, or whose transplants fail, the best option may be to receive regular blood transfusions from healthy donors with matched blood types.

The problem, says Linzhao Cheng, Ph.D. , the Edythe Harris Lucas and Clara Lucas Lynn Professor of Hematology and a member of the Institute for Cell Engineering, is that over time, patients' bodies often begin to mount an immune response against the foreign blood. "Their bodies quickly kill off the blood cells, so they have to get transfusions more and more frequently," he says.

A solution, Cheng and his colleagues thought, could be to grow blood cells in the lab that were matched to each patient's own genetic material and thus could evade the immune system. His research group had already devised a way to use stem cells to make human blood cells. The problem for patients with sickle cell disease is that lab-grown stem cells with their genetic material would have the sickle cell defect.

To solve that problem, the researchers started with patients' blood cells and reprogrammed them into so-called induced pluripotent stem cells, which can make any other cell in the body and grow indefinitely in the laboratory. They then used a relatively new genetic editing technique called CRISPR to snip out the sickle cell gene variant and replace it with the healthy version of the gene. The final step was to coax the stem cells to grow into mature blood cells. The edited stem cells generated blood cells just as efficiently as stem cells that hadn't been subjected to CRISPR, the researchers found.

Cheng notes that to become medically useful, the technique of growing blood cells from stem cells will have to be made even more efficient and scaled up significantly. The lab-grown stem cells would also need to be tested for safety. But, he says, "This study shows it may be possible in the not-too-distant future to provide patients with sickle cell disease with an exciting new treatment option."

This method of generating custom blood cells may also be applicable for other blood disorders, but its potential does not end there, Cheng says. One possibility, which his group hopes to begin studying soon, is that the blood cells of healthy people could be edited to resist malaria and other infectious agents.

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Johns Hopkins researchers engineer custom blood cells

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Orphan Designation Provides 7-Year Post Approval Marketing Exclusivity, Tax Credits and Elimination of FDA Prescription Drug User Fees

SAN DIEGO, CA--(Marketwired - February 10, 2015) - Targazyme Inc., a clinical-stage biopharmaceutical company developing enzyme technologies and products to improve efficacy outcomes for stem cell transplantation, immunotherapy, gene therapy and regenerative medicine, announced today that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug designation to TZ101 to prevent and reduce the severity and incidence of graft vs. host disease (GVHD) in patients eligible for hematologic stem cell transplant.

GVHD is a serious, life-threating complication of stem cell transplantation.Orphan drug status confirms the importance of Targazyme's novel treatment approach to prevent and reduce the incidence and severity of GVHD in patients with blood cancers where stem cell transplant is prescribed.TZ101 could potentially transform hematopoietic stem cell transplantation by reducing patient morbidity and mortality from GVHD, which occurs in a large percentage of these patients and is very difficult to manage clinically.

"Our work with TZ101 demonstrates impressive increases in the persistence and activity of regulatory T cells in preclinical models of GVHD," said Dr. Elizabeth J. Shpall, Deputy Chair of the Department of Stem Cell Transplantation and Cellular Therapy at The University of Texas MD Anderson Cancer Center."We are looking forward to beginning clinical trials on this promising modality for preventing GVHD in our patients undergoing stem cell transplantation."

Orphan Drug Designation by FDA confers financial benefits and incentives, such as potential Orphan Drug grant funding to defray the cost of clinical testing, tax credits for the cost of clinical research, a 7 year period of exclusive marketing after Approval and a Waiver of Prescription Drug User Fee Act (PDUFA) filing fees which are now greater than $2 million.

"The granting of Orphan Drug status for TZ101 for prevention of GVHD in stem cell transplant patients, as well as our previous Orphan Drug designation of TZ101 for cord blood transplantation, provides additional validation of our innovative platform technologies," said Lynnet Koh, Chairman & Chief Executive Officer of Targazyme."TZ101 and our second product, TZ102 are enabling technologies for improving efficacy outcomes for multiple cell-based therapeutic approaches used to prevent and treat a variety of different diseases for which there is a high unmet medical need.In addition to initiating our registration trial with TZ101 in hematopoietic stem cell transplantation, we plan to embark on our cancer immunotherapy trial later this year."

About Targazyme, Inc.

Targazyme Inc. is a San Diego-based, clinical-stage biopharmaceutical company developing novel enzyme-based platform technologies and products to improve clinical efficacy outcomes for stem cell medicine, auto-immunotherapy, gene therapy and regenerative medicine.

The company's clinical-grade fucosyltransferase enzymes and small molecule products (TZ101 and TZ102) are off-the-shelf products used at the point-of-care to treat therapeutic cells immediately before infusion into the patient using a simple procedure that is easily incorporated into existing medical practice.The company has received a number of world-wide patents, multiple FDA orphan drug designations and major medical/scientific awards and grants.

Targazyme has partnerships and collaborations with Kyowa Hakko Kirin and Florida Biologix, as well as various medical research institutions including The University of Texas MD Anderson Cancer Center, Oklahoma Medical Research Foundation, Texas Transplant Institute, Case Western/University Hospitals, Scripps Hospitals, Fred Hutchinson Cancer Research Center, UCLA Medical Center, Stanford University Medical Center, University of Minnesota Medical Center, University of California San Diego, Sanford-Burnham Medical Research Institute, Indiana University, Memorial Sloan Kettering Cancer Center, and New York Blood Center.For more information please go to http://www.targazyme.com.

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Cardiac Stem Cells: Making a Difference in Duchenne
Dr Eduardo Marban, Director of the Cedars-Sinai Heart Institute, discusses a possible Cardiac Stem Cell breakthrough for Duchenne muscular dystrophy. Coaliti...

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Coronary heart disease is the countrys leading cause of death A new treatment was designed to treat damaged heart muscle The capsule contains stem cells derived from the patients bone marrow

By Roger Dobson for the Daily Mail

Published: 17:33 EST, 9 March 2015 | Updated: 05:45 EST, 10 March 2015

A new treatment using a tiny grow-bag has been designed to treat damaged heart muscle

A tiny grow-bag could be a new way to mend hearts damaged by disease or heart attack.

The capsule, which is pea-sized, contains stem cells that trigger the growth of new cells.

An estimated 2.3 million people in Britain have coronary heart disease the countrys leading cause of death.

It occurs when the arteries supplying the heart become blocked by fatty substances, reducing the flow of blood.

If a bit of this fatty substance breaks off, it can trigger a blood clot, which in turn cuts off the blood supply to heart muscle, causing it to die off. This is what triggers a heart attack.

Heart disease and heart attacks can also lead to heart failure, where the heart becomes too weak to pump blood around the body properly.

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The tiny grow-bag that could mend a heart damaged by disease

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Researchers have created a "heart on a chip" using actual cardiac muscles to help test the effects of heart medication.

Anurag Mathur/Healy Lab

A new device could help make drug testing safer, faster, cheaper -- and eliminate the need for animal testing. It's just an inch long, but inside its silicone body is housed a small piece of cardiac muscle that responds to cardiovascular medications in exactly the same way heart muscle does inside a living human body.

"Ultimately, these chips could replace the use of animals to screen drugs for safety and efficacy," explained Kevin Healy, UC Berkeley professor of engineering, who led the research team that designed the device.

The problems with using animals to test human heart medication aren't merely ethical -- such concerns about lab animals rarely enter scientific discussions. Rather, there are some serious physiological problems -- namely, that drugs designed for humans will not have the same effect on a species that is biologically different from a human.

"These differences often result in inefficient and costly experiments that do not provide accurate answers about the toxicity of a drug in humans," Healy explained.

"It takes about $5 billion on average to develop a drug, and 60 percent of that figure comes from upfront costs in the research and development phase. Using a well-designed model of a human organ could significantly cut the cost and time of bringing a new drug to market."

The chips were created using heart muscle grown in a lab from adult human induced pluripotent stem cells -- stem cells that can be coaxed to grow into many other types of cell. The team then carefully designed the structure to be similar to the geometry and spacing of connective tissue fibre in a living human heart.

Microfluidic channels carved into the silicone on either side of the cell matrix act the same way as blood vessels, mimicking the exchange of nutrients and drugs with human tissue as it would happen in the body.

The cells start beating on their own within 24 hours of being loaded into the chamber at a healthy resting rate of 55 to 80 beats per minute. In order to test the system, the team then administered four well-known cardiovascular drugs -- isoproterenol, E-4031, verapamil and metoprolol. By monitoring the beat rate, the team was able to observe -- and accurately predict -- the chip's response to the drugs. Isoproterenol, for example -- a drug used to treat slow heart rate -- caused the muscle's beat rate to increase from 55 beats per minute to 124 beats per minute half an hour after being administered.

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IMAGE:Environmental Engineering Science, the official journal of the Association of Environmental Engineering and Science Professors (AEESP), is an authoritative peer-reviewed journal published monthly online with Open Access options. Publishing state-of-the-art... view more

Credit: Mary Ann Liebert, Inc., publishers

New Rochelle, NY, March 10, 2015--The increased use of engineered nanoparticles (ENMs) in commercial and industrial applications is raising concern over the environmental and health effects of nanoparticles released into the water supply. A timely study that analyzes the ability of typical water pretreatment methods to remove titanium dioxide, the most commonly used ENM, is published in Environmental Engineering Science, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers. The article is available free on the Environmental Engineering Science website until April 10, 2015.

Nichola Kinsinger, Ryan Honda, Valerie Keene, and Sharon Walker, University of California, Riverside, suggest that current methods of water prefiltration treatment cannot adequately remove titanium dioxide ENMs. They describe the results of scaled-down tests to evaluate the effectiveness of three traditional methods--coagulation, flocculation, and sedimentation--in the article "Titanium Dioxide Nanoparticle Removal in Primary Prefiltration Stages of Water Treatment: Role of Coating, Natural Organic Matter, Source Water, and Solution Chemistry".

"As nanoscience and engineering allow us to develop new exciting products, we must be ever mindful of associated consequences of these advances," says Domenico Grasso, PhD, PE, DEE, Editor-in-Chief of Environmental Engineering Science and Provost, University of Delaware. "Professor Walker and her team have presented an excellent report raising concerns that some engineered nanomaterials may find their ways into our water supplies."

"While further optimization of such treatment processes may allow for improved removal efficiencies, this study illustrates the challenges that we must be prepared to face with the emergence of new engineered nanomaterials," says Sharon Walker, PhD, Professor of Chemical and Environmental Engineering, University of California, Riverside.

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About the Journal

Environmental Engineering Science, the official journal of theThe Association of Environmental Engineering & Science Professors (AEESP) , is an authoritative peer-reviewed journal published monthly online with Open Access options. Publishing state-of-the-art studies of innovative solutions to problems in air, water, and land contamination and waste disposal, the Journal features applications of environmental engineering and scientific discoveries, policy issues, environmental economics, and sustainable development including climate change, complex and adaptive systems, contaminant fate and transport, environmental risk assessment and management, green technologies, industrial ecology, environmental policy, and energy and the environment. Complete tables of content and a sample issue may be viewed on the Environmental Engineering Science website.

About the Association

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Are current water treatment methods sufficient to remove harmful engineered nanoparticle?

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Let #39;s Play The Sims 3 - Perfect Genetics Challenge - Episode 60
Make sure to leave baby names in the comments!. #VampireClan #VampireClan4Life.

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The genes that increase the risk of Type 1 diabetes have lost their hiding place.

A research group that includes a University of Florida genetics expert has located and narrowed down the number of genes that play a role in the disease, according to a study published in the journal Nature Genetics. Knowing the identities and location of causative genes is a crucial development: Other researchers can use this information to better predict who might develop Type 1 diabetes and how to prevent it.

"It's a game-changer for Type 1 diabetes," said Patrick Concannon, director of the University of Florida Genetics Institute.

Researchers gathered information about the genetic makeup of 27,000 people, including those who had Type 1 diabetes and others who did not. They then began looking for individual differences in DNA that raise the risk of Type 1 diabetes. Starting with 200,000 possible locations in the genome, researchers used a technique known as fine mapping to pinpoint DNA sequence variations that can lead to diabetes. In some genomic regions, they narrowed the number of disease-causing DNA variations -- known as single nucleotide polymorphisms or SNPs -- from the thousands down to five or less.

That will make diabetes researchers' work more effective and efficient by giving them the most detailed directions yet about where to look for the genetic variations that cause Type 1 diabetes and perhaps other autoimmune diseases such as arthritis, Concannon said. Now that the group of geneticists has identified the important genes and SNPs, diabetes researchers will reap the benefits, according to Concannon.

"We've taken this genetic data which was interesting but hard to work with, and we've condensed it down into something that people can actually use to begin to explore the mechanism of the disease. It moves it out of the realm of genetics to being broadly applicable to Type 1 diabetes research," he said.

Type 1 diabetes occurs when the body's immune system kills off insulin-producing cells in the pancreas. Some 3 million people in the United States have the disease, according to the JDRF, a group that funds Type 1 diabetes research and education. Experts don't know exactly what causes the disease but suspect that genetics and environmental factors may play a role.

The researchers' findings are the most comprehensive yet in the effort to locate and identify the genetic risk variants for Type 1 diabetes and other autoimmune diseases, said Todd Brusko, a member of the UF Diabetes Institute and an assistant professor in the UF College of Medicine's department of pathology, immunology and laboratory medicine, part of UF Health.

Researchers can now shift away from trying to determine which genes heighten the risk for diseases like Type 1 diabetes, Brusko said. Instead, researchers can focus on how genetic changes alter immune cell activity. That, he said, could eventually lead to new treatments that prevent or stop Type 1 diabetes and other automimmune diseases.

"Ultimately, this information will allow researchers and clinicians to tailor treatments to correct underlying defects in the immune system that allow for autoimmune disease development," Brusko said.

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Genetics breakthrough will boost diabetes research

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FRESNO, Calif. (KFSN) --

Six-year-old Andy Moorhead is learning how to read. But instead of using his eyes, he's using his fingers. Andy told ABC30, "Well, I read the letters with my fingers."

Andy is blind. Andy's Mother, Heather Ingram-Moorhead explained, "He was around nine months, and we started to notice his eyes were twitching."

Andy has leber congenital amaurosis, or LCA. It's the most common type of childhood blindness and is caused by genetic mutations.

"It is just very hard. It's taken us a while to really understand the condition and do everything to help Andy," Heather told ABC30.

Andy's whole family is hands-on. Even his sister Valerie gives him guidance. But despite their efforts, his mom says gene therapy is their only hope.

University of Florida scientist Shannon E. Boye, PhD, is using a $900,000 grant to perfect a gene therapy that could restore vision.

"It's not an attempt just to slow the progression of the disease. It's actually an attempt to halt the progression and make the patient better by delivering them the gene they don't have," Boye told ABC30.

Boye says the therapy has worked in animals. "We're able to show, via what's called an electra retinal gram, that the retinal function has been restored to the mice," she explained.

Gene therapy is still an investigational treatment with risks and only available for those in a clinical trial. Right now there are hundreds of studies underway to treat conditions like LCA, cancer and HIV. It's hope that one day Andy could put down his cane and see his family for the first time.

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Gene therapy: Hope for the blind?

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When University of California, Berkeley, bioengineers say they are holding their hearts in the palms of their hands, they are not talking about emotional vulnerability.

Instead, the research team led by bioengineering professor Kevin Healy is presenting a network of pulsating cardiac muscle cells housed in an inch-long silicone device that effectively models human heart tissue, and they have demonstrated the viability of this system as a drug-screening tool by testing it with cardiovascular medications.

This organ-on-a-chip, reported in a study to be published Monday, March 9, in the journal Scientific Reports, represents a major step forward in the development of accurate, faster methods of testing for drug toxicity. The project is funded through the Tissue Chip for Drug Screening Initiative, an interagency collaboration launched by the National Institutes of Health to develop 3-D human tissue chips that model the structure and function of human organs.

"Ultimately, these chips could replace the use of animals to screen drugs for safety and efficacy," said Healy.

The study authors noted a high failure rate associated with the use of nonhuman animal models to predict human reactions to new drugs. Much of this is due to fundamental differences in biology between species, the researchers explained. For instance, the ion channels through which heart cells conduct electrical currents can vary in both number and type between humans and other animals.

"Many cardiovascular drugs target those channels, so these differences often result in inefficient and costly experiments that do not provide accurate answers about the toxicity of a drug in humans," said Healy. "It takes about $5 billion on average to develop a drug, and 60 percent of that figure comes from upfront costs in the research and development phase. Using a well-designed model of a human organ could significantly cut the cost and time of bringing a new drug to market."

The heart cells were derived from human-induced pluripotent stem cells, the adult stem cells that can be coaxed to become many different types of tissue.

The researchers designed their cardiac microphysiological system, or heart-on-a-chip, so that its 3-D structure would be comparable to the geometry and spacing of connective tissue fiber in a human heart. They added the differentiated human heart cells into the loading area, a process that Healy likened to passengers boarding a subway train at rush hour. The system's confined geometry helps align the cells in multiple layers and in a single direction.

Microfluidic channels on either side of the cell area serve as models for blood vessels, mimicking the exchange by diffusion of nutrients and drugs with human tissue. In the future, this setup could also allow researchers to monitor the removal of metabolic waste products from the cells.

"This system is not a simple cell culture where tissue is being bathed in a static bath of liquid," said study lead author Anurag Mathur, a postdoctoral scholar in Healy's lab and a California Institute for Regenerative Medicine fellow. "We designed this system so that it is dynamic; it replicates how tissue in our bodies actually gets exposed to nutrients and drugs."

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Bioengineers put human hearts on a chip to aid drug screening

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Newswise JUPITER, FL March 9, 2015 A research team from The Scripps Research Institute (TSRI), Mayo Clinic and other institutions has identified a new class of drugs that in animal models dramatically slows the aging processalleviating symptoms of frailty, improving cardiac function and extending a healthy lifespan.

The new research was published March 9 online ahead of print by the journal Aging Cell.

The scientists coined the term senolytics for the new class of drugs.

We view this study as a big, first step toward developing treatments that can be given safely to patients to extend healthspan or to treat age-related diseases and disorders, said TSRI Professor Paul Robbins, PhD, who with Associate Professor Laura Niedernhofer, MD, PhD, led the research efforts for the paper at Scripps Florida. When senolytic agents, like the combination we identified, are used clinically, the results could be transformative.

The prototypes of these senolytic agents have more than proven their ability to alleviate multiple characteristics associated with aging, said Mayo Clinic Professor James Kirkland, MD, PhD, senior author of the new study. It may eventually become feasible to delay, prevent, alleviate or even reverse multiple chronic diseases and disabilities as a group, instead of just one at a time.

Finding the Target

Senescent cellscells that have stopped dividingaccumulate with age and accelerate the aging process. Since the healthspan (time free of disease) in mice is enhanced by killing off these cells, the scientists reasoned that finding treatments that accomplish this in humans could have tremendous potential.

The scientists were faced with the question, though, of how to identify and target senescent cells without damaging other cells.

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Scripps Research, Mayo Clinic Scientists Find New Class of Drugs that Dramatically Increases Healthy Lifespan

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IMAGE:Professor Paul Robbins and Associate Professor Laura Niedernhofer led research efforts for the new study at Scripps Florida. view more

Credit: Photo courtesy of The Scripps Research Institute.

JUPITER, FL - March 9, 2015 - A research team from The Scripps Research Institute (TSRI), Mayo Clinic and other institutions has identified a new class of drugs that in animal models dramatically slows the aging process--alleviating symptoms of frailty, improving cardiac function and extending a healthy lifespan.

The new research was published March 9 online ahead of print by the journal Aging Cell.

The scientists coined the term "senolytics" for the new class of drugs.

"We view this study as a big, first step toward developing treatments that can be given safely to patients to extend healthspan or to treat age-related diseases and disorders," said TSRI Professor Paul Robbins, PhD, who with Associate Professor Laura Niedernhofer, MD, PhD, led the research efforts for the paper at Scripps Florida. "When senolytic agents, like the combination we identified, are used clinically, the results could be transformative."

"The prototypes of these senolytic agents have more than proven their ability to alleviate multiple characteristics associated with aging," said Mayo Clinic Professor James Kirkland, MD, PhD, senior author of the new study. "It may eventually become feasible to delay, prevent, alleviate or even reverse multiple chronic diseases and disabilities as a group, instead of just one at a time."

Finding the Target

Senescent cells--cells that have stopped dividing--accumulate with age and accelerate the aging process. Since the "healthspan" (time free of disease) in mice is enhanced by killing off these cells, the scientists reasoned that finding treatments that accomplish this in humans could have tremendous potential.

The scientists were faced with the question, though, of how to identify and target senescent cells without damaging other cells.

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Scripps Research, Mayo Clinic scientists find class of drugs that boosts healthy lifespan

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Though it may not look at all like the muscle in your chest, this heart-on-a-chip can beat like the real thing. A blend of microfluidics and biological cells, the device will be used as a more efficient means of testing for drug toxicity.

Developed by a team of bioengineers form University of California, Berkeley, the device is designed to mimic the geometry of fibers in a human heart. Pluripotent stem cellsthe cells that can be nudged to become one of the many different types of tissue present in our bodiesare introduced to a channel which is specially designed to encourage cells to grow in multiple layers in one direction, like real cardiac tissue. Here, they grow in to heart cells.

This section is then perfused with blood from microfluidic channels which act as blood vessels. Within 24 hours of lining the structure with heart cells, the structure began to beat at rate of between 55 to 80 beats per minutejust like a real human heart. Anurag Mathur, one of the researchers, explains to PhysOrg:

"This system is not a simple cell culture where tissue is being bathed in a static bath of liquid. We designed this system so that it is dynamic; it replicates how tissue in our bodies actually gets exposed to nutrients and drugs."

The system has already been used to test established cardiovascular drugs such as isoproterenol, E-4031, verapamil and metoprolol. The team observed effects upon the heart-on-a-chip consistent with those brought about in real humanso, drugs intended to speed up heart rate did exactly that to the cells in the device. The findings are published in Scientific Reports.

It's hoped that the device will be used to screen drugs, model human genetic diseasesand perhaps even link up with other organs-on-a-chip to predict whole-body reactions too. [Scientific Reports via PhysOrg]

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This Heart-on-a-Chip Beats Like the Real Thing

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Scientists at Tuft University in Massachusetts grew bone marrow on silk They were able to generate functioning platelet cells that form blood clots The cells could be used to stop bleeding in injured patients in ER rooms It has raised hopes that man-made blood can be created for transfusions However some say it could be up to 15 years before stem cells can be used to create blood that can be safely used for transfusions during surgery

By Richard Gray for MailOnline

Published: 11:46 EST, 19 February 2015 | Updated: 12:50 EST, 23 February 2015

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A major component of blood has been grown in the laboratory by scientists, bringing man-made blood transfusions a step closer.

Biomedical engineers have for the first time produced functional blood platelets - the cells that cause clots to form - from human bone marrow grown in the laboratory.

The achievement raises hopes that it will soon be possible to produce fully functional blood in a similar way.

Scientists have managed to grow fully functioning platelets like the one above surrounded by red blood cells

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Could we soon have man-made blood?

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Severe hypophosphatasia generally fatal during infancy, bone marrow transplant along with mensenchymal stem cell transplants offers hope

Putnam Valley, NY. (Feb 9, 2015) - Recent research carried out by a team of researchers in Japan has investigated the use of bone marrow transplants (BMTs) to treat hypophosphatasia (HPP). In this study, the researchers carried out BMT for two infants with HPP in combination with allogenic (other-donated) mesenchymal stem cell transplants (MSCTs). The allogenic MSC donors were a parent of the infant.

The study will be published in a future issue of Cell Transplantation and is currently freely available on-line as an unedited early e-pub at: http://ingentaconnect.com/content/cog/ct/pre-prints/content-CT-1337_Taketani_et_al

"Hypophosphatasia" (HPP) is a rare and most often fatal genetic bone disease affecting infants that has no current treatment. The disease is caused by mutations in the ALPL gene, which encodes alkaline phosphatase (ALP). Patients with severe HPP develop bone impairment and have extremely low levels of ALP activity, an enzyme necessary for bone mineralization.

Although there are mild and more severe forms, severe hypophosphatasia prevents proper bone mineralization during perinatal development. When the disease develops perinatally, many infants are still-born, with little evidence of bone mineralization. HPP can also appear in later infancy, generally before an infant reaches the age of six months, with the result that most afflicted infants do not live past the age of six months. Milder forms of HPP can present in later youth or in adulthood.

"Mesenchymal stem cells (MSCs) reside in bone marrow and other tissues and have a self-renewal capacity so that after transplantation they can differentiate into various cell lineages, including bone and cartilage," said Dr. Takeshi Taketani of the Division of Blood Transfusion at Shimane University Hospital in Shimane, Japan. "We performed multiple infusions of MSCs for two infant patients with severe HPP who had already undergone BMT. The adverse events from the BMT were managed and there were no adverse events from the MSC infusions."

After each infant had undergone BMT, one infant received four MSCTs and a second infant received nine MSCTs. Previous research had revealed that MSCT without a prior BMT was ineffective.

The researchers reported that the two infants receiving both BMT and MSCTs improved not only in terms of bone mineralization, but also saw improvements in muscle mass, respiratory function and mental development. Both children continue to survive at age three.

"Our data suggest that allogenic MSCT combined with BMT might be one of the safer and more effective remedies for patients with severe HPP, although long-term effectiveness remains unknown and warrants further study," concluded the researchers. "We need to establish curative, MSC-based treatment strategies that can maintain the long-term survival and differentiation capabilities of transplanted allo-MSCs."

"This study highlights the promise of stem cells in presenting a new frontier for regenerative medicine, with an improvement of HPP-associated symptoms and survival following BMT and MSCT." said Dr. David Eve, Cell Transplantation associate editor, and Instructor of neurosurgery and brain repair at the University of South Florida School of Medicine. "In order to elucidate the mechanisms behind recovery and further extrapolate the study to all HPP patients, a larger cohort and more long term follow-up are needed."

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Infants with rare bone disease improve bone formation after cell transplantation

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Since ancient times, humankind has been aware of how important blood is to life. Naturalists speculated for thousands of years on the source of the body's blood supply. For several centuries, the liver was believed to be the site where blood forms. In 1868, however, the German pathologist Ernst Neumann discovered immature precursor cells in bone marrow, which turned out to be the actual site of blood cell formation, also known as hematopoiesis. Blood formation was the first process for which scientists formulated and proved the theory that stem cells are the common origin that gives rise to various types of mature cells.

"However, a problem with almost all research on hematopoiesis in past decades is that it has been restricted to experiments in culture or using transplantation into mice," says Professor Hans-Reimer Rodewald from the German Cancer Research Center (Deutsches Krebsforschungszentrum, DKFZ). "We have now developed the first model where we can observe the development of a stem cell into a mature blood cell in a living organism."

Dr. Katrin Busch from Rodewald's team developed genetically modified mice by introducing a protein into their blood stem cells that sends out a yellow fluorescent signal. This fluorescent marker can be turned on at any time by administering a specific reagent to the animal. Correspondingly, all daughter cells that arise from a cell containing the marker also send out a light signal.

When Busch turned on the marker in adult animals, it became visible that at least one third (approximately 5000 cells) of a mouse's hematopoietic stem cells produce differentiated progenitor cells. "This was the first surprise," says Busch. "Until now, scientists had believed that in the normal state, very few stem cells - only about ten - are actively involved in blood formation."

However, it takes a very long time for the fluorescent marker to spread evenly into peripheral blood cells, an amount of time that even exceeds the lifespan of a mouse. Systems biologist Prof. Thomas Hfer and colleagues (also of the DKFZ) performed mathematical analysis of these experimental data to provide additional insight into blood stem cell dynamics. Their analysis showed that, surprisingly, under normal conditions, the replenishment of blood cells is not accomplished by the stem cells themselves. Instead, they are actually supplied by first progenitor cells that develop during the following differentiation step. These cells are able to regenerate themselves for a long time - though not quite as long as stem cells do. To make sure that the population of this cell type never runs out, blood stem cells must occasionally produce a couple of new first progenitors.

During embryonic development of mice, however, the situation is different: To build up the system, all mature blood and immune cells develop much more rapidly and almost completely from stem cells.

The investigators were also able to accelerate this process in adult animals by artificially depleting their white blood cells. Under these conditions, blood stem cells increase the formation of first progenitor cells, which then immediately start supplying new, mature blood cells. In this process, several hundred times more cells of the so-called myeloid lineage (thrombocytes, erythrocytes, granulocytes, monocytes) form than long-lived lymphocytes (T cells, B cells, natural killer cells) do.

"When we transplanted our labeled blood stem cells from the bone marrow into other mice, only a few stem cells were active in the recipients, and many stem cells were lost," Rodewald explains. "Our new data therefore show that the findings obtained up until now using transplanted stem cells can surely not be reflective of normal hematopoiesis. On the contrary, transplantation is an exception [to the rule]. This shows how important it is that we actually follow hematopoiesis under normal conditions in a living organism."

The scientists in Rodewald's department, working together with Thomas Hfer, now also plan to use the new model to investigate the impact of pathogenic challenges to blood formation: for example, in cancer, cachexia or infection. This method would also enable them to follow potential aging processes that occur in blood stem cells in detail as they occur naturally in a living organism.

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Live assessment of blood formation

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