PHILADELPHIA Douglas C. Wallace, PhD, professor of Pathology and Laboratory Medicine, at the Perelman School of Medicine, University of Pennsylvania, is the recipient of the 2012 Genetics Prize of the Gruber Foundation. Wallace is a pioneering genetics researcher who founded the field of mitochondrial genetics in humans. He is also the director of the Center for Mitochondrial and Epigenomic Medicine at The Children's Hospital of Philadelphia.

Wallace is being honored with this prestigious international award for his groundbreaking achievements in understanding the role of mitochondriathe "power plants" of cellsin the development of disease and as markers for human evolution. He is also being honored for training and inspiring numerous pre- and postdoctoral students who have gone on to have distinguished careers of their own.

Wallace will receive the award on November 9 at the annual meeting of the American Society of Human Genetics in San Francisco. The Gruber Foundation, now based at Yale University, announced the Genetics Prize on June 28. The Foundation's Genetics Prize annually honors leading scientists for groundbreaking contributions to genetics research. The Peter and Patricia Gruber Foundation's International Prize Program honors contemporary individuals in the fields of Cosmology, Genetics, Neuroscience, Justice and Women's Rights, whose groundbreaking work provides new models that inspire and enable fundamental shifts in knowledge and culture. The Gruber Foundation's Genetics Prize, a gold medal and an unrestricted $500,000 cash award for fundamental insights in the field of genetics, was established in 2001.

"Douglas Wallace's contributions to our understanding of mitochondrial genetics have changed the way human and medical geneticists think about the role of mitochondria in human health and disease." said Elizabeth Blackburn, chair of the Selection Advisory Board to the Prize. Blackburn is the 2006 Gruber Genetics Prize laureate and shared the 2009 Nobel Prize in Physiology and Medicine.

Wallace began his research on mitochondrial biology 40 years ago, at a time when few people thought the study of mitochondria and its DNA (mtDNA) would have any significant applications for clinical medicine. In the late 1970s, Wallace demonstrated that human mtDNA is inherited solely through the mother. Using maternal inheritance as a guide, Wallace identified the first inherited mtDNA disease -Leber's hereditary optic neuropathy - and subsequently linked mtDNA mutations to a wide range of clinical symptoms, including deafness, neuropsychiatric disorders, cardiac and muscle problems, and metabolic diseases such as diabetes. Wallace also showed that mtDNA mutations accumulate in human tissue with age, and thus may play a role in age-related diseases, such as heart disease and cancer. In addition, he found that the levels of these age-related mtDNA mutations are higher in the brains of people with certain neurodegenerative diseases, including Alzheimer disease, Parkinson disease, and Huntington disease.

Wallace's research has also made a major contribution to the field of molecular anthropology. Using mtDNA variation, he has reconstructed the origins and ancient migrations of women, tracing all mtDNA lineages back some 200,000 years to a single African origin the so-called mitochondrial Eve.

Wallace holds the Michael and Charles Barnett Endowed Chair in Pediatric Mitochondrial Medicine at Children's Hospital. He is a member of the National Academy of Sciences, as well as the Academy's Institute of Medicine, and is also a member of the American Academy of Arts and Sciences. Wallace joined the Penn Department of Pathology and Laboratory Medicine in 2010 and previously held academic positions at Stanford University, at Emory University, where he chaired the Department of Genetics and Molecular Medicine, and most recently at the University of California Irvine, where he was Director of the Center for Molecular and Mitochondrial Medicine and Genetics.

For more information, read the Gruber Foundation news release.

The Perelman School of Medicine is currently ranked #2 in U.S. News & World Report's survey of research-oriented medical schools. The School is consistently among the nation's top recipients of funding from the National Institutes of Health, with $479.3 million awarded in the 2011 fiscal year.

The University of Pennsylvania Health System's patient care facilities include: The Hospital of the University of Pennsylvania -- recognized as one of the nation's top 10 hospitals by U.S. News & World Report; Penn Presbyterian Medical Center; and Pennsylvania Hospital the nation's first hospital, founded in 1751. Penn Medicine also includes additional patient care facilities and services throughout the Philadelphia region.

Continued here:
Douglas Wallace to Receive Gruber Foundation 2012 Genetics Prize

Recommendation and review posted by Bethany Smith

When last we met, the subject was athletic performance enhancement. I spoke of modalities for raising one's game, including surgery, lucky genetics and, of course, eau de Canseco, also known as anabolic steroids. That column contended that many world-class athletes are freaksof nature, yes, but freaks nonetheless. In effect, they make use of performance-enhancing substances that happen to be produced by their own bodies rather than by a friend of a friend who knows a really good pharmaceutical chemist.

I'll continue to pull on that thread briefly here because within days of that column going to press, news broke that is directly related to the topic. After being lobbied by the union representing its players, the National Football League has agreed to do a study. The investigation will try to determine if football players, who represent the last remnants of a once thriving pre-Clovis North American population of megafauna, naturally have crazy high amounts of compounds that can make one large.

As the New York Times put it on April 21, the union has said that football players, because of their size, might have a higher level of naturally occurring human growth hormone [HGH] and could be at risk of having false positives. At which point, league officials would presumably stand on a chair to raise the level of HGH that counts as a positive test result in pigskin land.

All of which brings me back to the question I asked last time: If users of performance-enhancing drugs are disqualified, should holders of performance-enhancing mutations be barred, too? In other wordsand I do not know the right answer to this questionwhy is it okay for a guy to have a body that makes a lot of hormone but not a buddy who makes a lot of hormone to inject?

Speaking of hormones and injections, have you seen Museum of Copulatory Organs? Part of the 18th Sydney Biennale in Australia, this collection of 3-D models of insect genitalia was the Ph.D. project of Colombian-born artist Maria Fernanda Cardoso.

Her previous claim to fame was a recreation of a 19th-century-style flea circus, which is paradoxically no small task. A blog post at the Australian Broadcasting Corporation (ABC) Web site quotes Cardoso as saying, It's one of the hardest things in life to train fleas, it took six years and it requires a lot of patience, no one knew how to train fleas anymore. Actually the New York City subway system still trains fleas on a daily basis, judging by the number of passengers carrying tiny dogs around with them for some reason probably related to the effect of Paris Hilton on our culture.

According to the ABC article, Cardoso was inspired to pursue the copulatory organ project when she found within the flea literature this quote about the insects' penises: It's not size that matters, it is shape. Indeed, some insect penises come equipped with hooks that enable the ensconced male to grab a previous suitor's sperm packet and remove it from the female. I suggest that these hooks be called cuckholders.

Speaking of shaft-shaped devices used to convey information, have you visited the Cumberland Pencil Museum in England lately? It bills itself as a great all weather attraction for the whole family, although I would submit that a pencil museum is best appreciated when rain necessitates the cancellation of outdoor festivities. Fortunately for pencil aficionados, this is England.

The museum's Web site speculates that Cumberland locals first struck graphite some five centuries ago, when a violent storm uprooted trees and unearthed vast stores of the carbon allotrope. Shepherds soon used the material to mark their sheep. Meanwhile aspiring scribes wrapped sticks of graphite in sheep hides to make rudimentary pencils. This animal-implement relationship was clearly the source of the old adage He was as write as a sheep.

Pencils reached their pinnacle in the U.S. in the second half of the 20th century, when millions of high school students clutched No. 2 versions in their clammy hands to mark the answers on their SATs. Some who may not have done well still managed to earn sheepskins by carrying pigskins.

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Genetics and Graphite Provide Scribe Fodder

Recommendation and review posted by Bethany Smith

ScienceDaily (July 2, 2012) Getting under your skin takes on a brave new meaning thanks to Northwestern University research that could transform gene regulation.

A team led by a physician-scientist and a chemist -- from the fields of dermatology and nanotechnology -- is the first to demonstrate the use of commercial moisturizers to deliver gene regulation technology that has great potential for life-saving therapies for skin cancers.

The topical delivery of gene regulation technology to cells deep in the skin is extremely difficult because of the formidable defenses skin provides for the body. The Northwestern approach takes advantage of drugs consisting of novel spherical arrangements of nucleic acids. These structures, each about 1,000 times smaller than the diameter of a human hair, have the unique ability to recruit and bind to natural proteins that allow them to traverse the skin and enter cells.

Applied directly to the skin, the drug penetrates all of the skins layers and can selectively target disease-causing genes while sparing normal genes. Once in cells, the drug simply flips the switch of the troublesome genes to off.

A detailed study of a method that could dramatically redefine the field of gene regulation will be published online during the week of July 2 by the Proceedings of the National Academy of Sciences (PNAS).

Early targets of the novel treatment are melanoma and squamous cell carcinoma (two of the most common types of skin cancer), the common inflammatory skin disorder psoriasis, diabetic wound healing and a rare genetic skin disorder that has no effective treatment (epidermolytic ichthyosis). Other targets could even include wrinkles that come with aging skin.

The technology developed by my collaborator Chad Mirkin and his lab is incredibly exciting because it can break through the skin barrier, said co-senior author Amy S. Paller, M.D., the Walter J. Hamlin Professor, chair of dermatology and professor of pediatrics at Northwestern University Feinberg School of Medicine. She also is director of Northwesterns Skin Disease Research Center.

This allows us to treat a skin problem precisely where it is manifesting -- on the skin, she said. We can target our therapy to the drivers of disease, at a level so minute that it can distinguish mutant genes from normal genes. Risks are minimized, and side effects have not been seen to date in our human skin and mouse models.

A co-senior author of the paper, Mirkin is the George B. Rathmann Professor of Chemistry in the Weinberg College of Arts and Sciences and professor of medicine, chemical and biological engineering, biomedical engineering and materials science and engineering. He also is the director of Northwesterns International Institute for Nanotechnology.

Mirkin first developed the nanostructure platform used in this study in 1996 at Northwestern, and the FDA-cleared technology now is the basis of powerful commercialized medical diagnostic tools. This, however, is the first realization that the nanostructures naturally enter skin and that they can deliver a large payload of therapeutics.

Read this article:
Breaking the skin barrier: Drugs topically deliver gene therapy via commercial moisturizers for skin disease treatment

Recommendation and review posted by Bethany Smith

NOTCH1 Signaling Promotes T-Cell Acute Lymphoblastic Leukemia-Initiating Cell Regeneration

Newswise Research suggests that patients with leukemia sometimes relapse because standard chemotherapy fails to kill the self-renewing leukemia initiating cells, often referred to as cancer stem cells. In such cancers, the cells lie dormant for a time, only to later begin cloning, resulting in a return and metastasis of the disease.

One such type of cancer is called pediatric T cell acute lymphoblastic leukemia, or T-ALL, often found in children, who have few treatment options beyond chemotherapy.

A team of researchers led by Catriona H. M. Jamieson, MD, PhD, associate professor of medicine at the University of California, San Diego School of Medicine and Director of Stem Cell Research at UC San Diego Moores Cancer Center studied these cells in mouse models that had been transplanted with human leukemia cells. They discovered that the leukemia initiating cells which clone, or replicate, themselves most robustly activate the NOTCH1 pathway, usually in the context of a mutation.

Earlier studies showed that as many as half of patients with T-ALL have mutations in the NOTCH1 pathway an evolutionarily conserved developmental pathway used during differentiation of many cell and tissue types. The new study shows that when NOTCH1 activation was inhibited in animal models using a monoclonal antibody, the leukemia initiating cells did not survive. In addition, the antibody treatment significantly reduced a subset of these cancer stem cells (identified by the presence of specific markers, CD2 and CD7, on the cell surface.)

We were able to substantially reduce the potential of these cancer stem cells to self-renew, said Jamieson. So were not just getting rid of cancerous cells: were getting to the root of their resistance to treatment leukemic stem cells that lie dormant.

The study results suggest that such therapy would also be effective in other types of cancer stem cells, such as those that cause breast cancer, that also rely on NOTCH1 for self-renewal.

Therapies based on monoclonal antibodies that inhibit NOTCH 1 are much more selective than using gamma-secretase inhibitors, which also block other essential cellular functions in addition to the NOTCH1 signaling pathway, said contributor A. Thomas Look, MD of Dana-Farber/Children Hospital Cancer Center in Boston. We are excited about the promise of NOTCH1-specific antibodies to counter resistance to therapy in T-ALL and possibly additional types of cancer.

In investigating the role of NOTCH1 activation in cancer cell cloning, the researchers showed that leukemia initiating cells possess enhanced survival and self-renewal potential in specific blood-cell, or hematopoietic, niches: the microenvironment of the body in which the cells live and self-renew.

The scientists studied the molecular characterization of CD34+ cells a protein that shows expression in early hematopoietic cells and that facilitates cell migration from a dozen T-ALL patient samples.

Read this article:
Researchers Block Pathway to Cancer Cell Replication

Recommendation and review posted by simmons

02-07-2012 09:43 State of the Nation is a nightly newscast anchored by award-winning broadcast journalist, Jessica Soho. It airs Mondays to Fridays at 9:00 PM (PHL Time) on GMA News TV Channel 11. For more videos from State of the Nation, visit fthenation.

Continued here:
SONA: Stem cell therapy, kaya raw makapagpabata ng pangangatawan - Video

Recommendation and review posted by simmons

Ed Reschke / Getty Images

A color-enhanced photograph of spongy (Cancellous) bone red bone marrow fills the space.

How much would it take for you to consider selling your bone marrow? A U.S. appeals court puts the price at about $3,000 in a ruling that now makes it legal to pay donors for their bone marrow tissue.

The courts decision may well help thousands of sick patients who need bone marrow transplants to survive, but it also begs the question, what other body parts might next be up for sale?

The ruling came about at the end of 2011, in a decision to an October 2009 lawsuit brought by a group of cancer patients, parents and bone marrow donation advocates against the government over the federal law banning the buying and selling of bodily organs. The plaintiffs were led by Doreen Flynn, who has three daughters who suffer from Fanconi anemia, a blood disorder that requires bone marrow transplants to treat. Flynn and the other plaintiffs said that too many such patients die waiting for transplants and argued that we should be allowed to pay people to donate their marrow as a way of ensuring a more reliable supply. The U.S. Court of Appeals for the Ninth Circuit agreed.

(MORE: Facebook Now Lets Organ Donors Tell Their Friends)

At the core of the plaintiffs argument was the National Organ Transplantation Act (NOTA), which since 1984 has forbid the buying and selling of human organs, including bone marrow. But new developments in bone marrow extraction have made marrow donation not much different from donating blood: traditionally, bone marrow donation required anesthesia and long needles to extract the marrow from the hipbones of donors. Now, a technique called peripheral apheresis allows doctors to extract blood stem cells directly from the blood, instead of the bone patients first take a drug that pulls stem cells from the bone and into the blood meaning that the marrow cells should be considered a fluid like blood, rather than an organ, the plaintiffs argued. NOTA doesnt prohibit payments for blood or other fluids, such as plasma or semen.

U.S. Attorney General Eric Holder decided not to ask the U.S. Supreme Court to review the appellate courts decision, which would have been the next step in overturning it. That means the ruling stands and that people can now be paid up to $3,000 for their marrow, as long as it is collected by apheresis. In a concession to the spirit of NOTA, however, the compensation cant be in cash; it needs to be in the form of a voucher that can be applied to things such as scholarships, education, housing or a donation to a charity.

While the decision applies only to the nine states covered by the Ninth Circuit court, and only to bone marrow obtained through apheresis, it does raise bigger questions about how we will look at organ donation in the future. With about 114,000 people waiting for organs in the U.S. alone on any given day, and only 3,300 donors, the urgent medical need runs up against moral standards of the value human life. Once we start paying for the parts we need, though, how far do we go? We dont allow people to buy and sell human beings, thats slavery, says Dr. Robert Klitzman, director of the bioethics program at Columbia University. Should we allow people to buy and sell human body parts?

(MORE: Where Do (Some) Babies Come From? In Washington, a New Law Bans Anonymous Sperm and Egg Donors)

See the article here:
Paying for Bone Marrow: Should We Be Able to Sell Our Parts?

Recommendation and review posted by sam

BOSTON--(BUSINESS WIRE)--

Inform Genomics, Inc., a private company focused on developing novel platforms of genomic based personalized medicine products for cancer supportive care and inflammatory diseases, today announced preliminary results from its initial study for its lead product, OnPART, which is designed to determine an individuals risk of side-effects associated with chemotherapy regimens based on his or her individual genomic profile. The study utilized advanced Bayesian network technology to identify (single-nucleotide polymorphism) SNP networks associated with common side-effects of chemotherapy regimens. In the plenary session Molecular Predictors in Supportive Care, Dr. Stephen T. Sonis, D.M.D., D.M.Sc. who is a co-founder of Inform Genomics and also a Clinical Professor of Oral Medicine at the Harvard School of Dental Medicine, reported that for patients receiving dose-dense doxorubicin, cyclophosphamide, plus paclitaxel (AC+T) a preliminary SNP network correctly identified patients at risk for chemotherapy-induced diarrhea with an accuracy of 96.7% and an area under the receiver operating characteristic (ROC) curve of 0.994.

We are very encouraged by these early results and they are in line with what we previously demonstrated with a high-degree of precision using our Bayesian network technology to predict oral mucositis with our transplant product, said Ed Rubenstein, President & CEO of Inform Genomics. We are excited to see further data demonstrating OnPARTs ability to predict serious toxicities from chemotherapy regimens based on his or her individual genomic profile which may lead to actions to reduce the burden of potentially curable interventions for patients with cancer.

About OnPART

OnPART, Oncology Preferences And Risk of Toxicity, is Inform Genomics lead platform personalized medicine product for treatment decisions in patients who will receive chemotherapy for colorectal, breast, lung, or ovarian cancer. Based upon response rates and survival, more than one chemotherapy regimen may be considered appropriate care for patients with these common solid tumors, yet the regimens vary widely in their side-effect profiles. OnPART is being developed to assess genomic risk for common and often debilitating therapy-related side-effects, including fatigue, nausea and vomiting, diarrhea, oral mucositis, cognitive dysfunction and peripheral neuropathy. The product includes a differentiating factor in personalized medicine, quantifying patient concerns for side-effects, using a copyrighted patient questionnaire (Preference Assessment Inventory). OnPART is expected to provide valuable information for patients and medical oncologists to help clarify critical clinical choices and be commercially available in 2014.

About Cancer Supportive Care

Most patients with cancer receive supportive care as part of their multimodal anti-cancer therapy, regardless of cancer diagnosis, stage of disease, or treatment modality. Common symptoms associated with cancer or its treatments include fatigue, nausea and vomiting, diarrhea, oral mucositis, cognitive dysfunction, and peripheral neuropathy. Some of these conditions are manageable with commercially available medications, while others are the focus of drug development programs. The development of these side-effects may interfere with ongoing anti-cancer treatment, impair patient functioning, negatively impact the patients quality of life, and may increase the risk of mortality. Treatment of these side-effects also results in significant costs for payers and providers.

About Inform Genomics

Inform Genomics, Inc. is a private company focused on developing novel platforms of genomic based personalized medicine products for cancer supportive care and inflammatory diseases, including its lead platform product, OnPART, designed to determine an individuals risk of side-effects associated with chemotherapy regimens based on his or her individual genomic profile. The companys business model leverages existing technology in conjunction with proprietary analytic methods for conducting genome-wide association studies. Product development programs will lead to commercial, single source laboratory tests consisting of single-nucleotide polymorphism (SNP) networks that determine the likelihood of individual patient clinical outcomes to drug therapies. The U.S. market opportunity for these differentiated products exceeds $2 billion annually. Inform Genomics is headquartered in Boston, Massachusetts. For more information, please visit http://www.informgenomics.com.

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Inform Genomics Announces Preliminary Results of OnPART™ Personalized Medicine Product: SNP Network Identifies ...

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

Advanced Cell Technology, Inc. (ACT; OTCBB: ACTC), a leader in the field of regenerative medicine, today announced treatment of the second patient in its Phase 1/2 clinical trial for Stargardts macular dystrophy (SMD) using retinal pigment epithelial (RPE) cells derived from human embryonic stem cells (hESCs). The surgery was performed on Friday, June 29 at Moorfields Eye Hospital in London, the same site as the first patient treatment in January, by a team of surgeons led by Professor James Bainbridge, consultant surgeon at Moorfields and Chair of Retinal Studies at University College London. The procedure was successfully performed without any complications. ACT and Moorfields Eye Hospital recently received clearance from the Data and Safety Monitoring Board (DSMB) to treat the final two patients in the first cohort of this clinical trial.

We are very pleased to continue our forward momentum with both our U.S. trials and our European trial, commented Gary Rabin, chairman and CEO. It was less than a month ago that we received DSMB approval to treat the second and third patients in our E.U. trial, and it is very gratifying to have already completed dosing of the second. It is a pleasure to be working with Professor Bainbridge and the rest of his team at Moorfields Eye Hospital, and we continue to be encouraged by the steady progress of the trial thus far.

The Phase 1/2 trial is designed to determine the safety and tolerability of hESC-derived RPE cells following sub-retinal transplantation in patients with SMD at 12 months, the studys primary endpoint. It will involve a total of 12 patients, with cohorts of three patients each in an ascending dosage format. It is similar in design to the U.S. trial for SMD that was initiated in July 2011.

The European Medicines Agency's (EMA) Committee for Orphan Medicinal Products (COMP) has officially designated ACT's human embryonic stem cell (hESC)-derived retinal pigment epithelial (RPE) cells as an orphan medicinal product for the treatment of Stargardt's Macular Dystrophy (SMD).

More information on the status of the companys clinical trials will be posted today on Mr. Rabins Chairmans blog.

About Stargardts Disease Stargardts disease or Stargardts Macular Dystrophy is a genetic disease that causes progressive vision loss, usually starting in children between 10 to 20 years of age. Eventually, blindness results from photoreceptor loss associated with degeneration in the pigmented layer of the retina, called the retinal pigment epithelium, which is the site of damage that the company believes the hESC-derived RPE may be able to target for repair after administration.

About Advanced Cell Technology, Inc. Advanced Cell Technology, Inc. is a biotechnology company applying cellular technology in the field of regenerative medicine. For more information, visit http://www.advancedcell.com.

Forward-Looking Statements Statements in this news release regarding future financial and operating results, future growth in research and development programs, potential applications of our technology, opportunities for the company and any other statements about the future expectations, beliefs, goals, plans, or prospects expressed by management constitute forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Any statements that are not statements of historical fact (including statements containing the words will, believes, plans, anticipates, expects, estimates, and similar expressions) should also be considered to be forward-looking statements. There are a number of important factors that could cause actual results or events to differ materially from those indicated by such forward-looking statements, including: limited operating history, need for future capital, risks inherent in the development and commercialization of potential products, protection of our intellectual property, and economic conditions generally. Additional information on potential factors that could affect our results and other risks and uncertainties are detailed from time to time in the companys periodic reports, including the report on Form 10-K for the year ended December 31, 2011. Forward-looking statements are based on the beliefs, opinions, and expectations of the companys management at the time they are made, and the company does not assume any obligation to update its forward-looking statements if those beliefs, opinions, expectations, or other circumstances should change. Forward-looking statements are based on the beliefs, opinions, and expectations of the companys management at the time they are made, and the company does not assume any obligation to update its forward-looking statements if those beliefs, opinions, expectations, or other circumstances should change. There can be no assurance that the Companys clinical trials will be successful.

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ACT Announces Second Patient with Stargardt’s Disease Treated in EU Clinical Trial

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SAN DIEGO, CA--(Marketwire -07/02/12)- DPR Construction (DPR) has been awarded a multi-million dollar contract to manage the construction of a regenerative medicine campus on behalf of Advanced BioHealing and Shire plc, the global specialty biopharmaceutical company, with real estate partner BioMed Realty Trust, Inc. and designers Ferguson Pape Baldwin Architects.

The new campus in Sorrento Mesa will expand the operational footprint of Shire's regenerative medicine business, Advanced BioHealing, Inc., and create the increased capacity it needs to meet future demand for its lead product, DERMAGRAFT, while offering additional space and infrastructure to manufacture new regenerative medicine products.

"This project will generate hundreds of jobs, including much needed construction jobs, during the build out over the new two years, and we are honored to be a part of it," said Jay Leopold, Regional Manager of DPR's San Diego office. "We look forward to working with the project partners to implement our High Performance Team strategies and create a model facility for attracting more biomedical business here in San Diego."

Phase I of the site development will be in excess of 150,000 square feet and will house the company's manufacturing and associated support operations, commercial operations, corporate, and administrative functions. Preconstruction and design is underway with construction scheduled to begin in 2013. Initial occupancy is targeted for 2014.

About DPR ConstructionDPR Construction, named by FORTUNE as #13 of the "100 Best Companies To Work For," is a forward-thinking national general contractor and construction manager specializing in life science, healthcare, corporate office, advanced technology and higher education projects. A privately held, employee-owned company, DPR has grown to more than $1 billion in annual revenue, making it one of the largest general contractors in the nation. Locally, DPR is a perennial Top 5 general contractor with annual revenue averaging well over $200 million in projects ranging from $5k to $600M. DPR's San Diego office is located at 5010 Shoreham Place, San Diego, CA 92122. For more information about DPR, visit: http://www.dpr.com.

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DPR Construction Awarded Multi-Million Dollar Contract to Manage Build Out of Shire Advance BioHealing Campus in ...

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Ed Reschke / Getty Images

A color-enhanced photograph of spongy (Cancellous) bone red bone marrow fills the space.

How much would it take for you to consider selling your bone marrow? A U.S. appeals court puts the price at about $3,000 in a ruling that now makes it legal to pay donors for their bone marrow tissue.

The courts decision may well help thousands of sick patients who need bone marrow transplants to survive, but it also begs the question, what other body parts might next be up for sale?

The ruling came about at the end of 2011, in a decision to an October 2009 lawsuit brought by a group of cancer patients, parents and bone marrow donation advocates against the government over the federal law banning the buying and selling of bodily organs. The plaintiffs were led by Doreen Flynn, who has three daughters who suffer from Fanconi anemia, a blood disorder that requires bone marrow transplants to treat. Flynn and the other plaintiffs said that too many such patients die waiting for transplants and argued that we should be allowed to pay people to donate their marrow as a way of ensuring a more reliable supply. The U.S. Court of Appeals for the Ninth Circuit agreed.

(MORE: Facebook Now Lets Organ Donors Tell Their Friends)

At the core of the plaintiffs argument was the National Organ Transplantation Act (NOTA), which since 1984 has forbid the buying and selling of human organs, including bone marrow. But new developments in bone marrow extraction have made marrow donation not much different from donating blood: traditionally, bone marrow donation required anesthesia and long needles to extract the marrow from the hipbones of donors. Now, a technique called peripheral apheresis allows doctors to extract blood stem cells directly from the blood, instead of the bone patients first take a drug that pulls stem cells from the bone and into the blood meaning that the marrow cells should be considered a fluid like blood, rather than an organ, the plaintiffs argued. NOTA doesnt prohibit payments for blood or other fluids, such as plasma or semen.

U.S. Attorney General Eric Holder decided not to ask the U.S. Supreme Court to review the appellate courts decision, which would have been the next step in overturning it. That means the ruling stands and that people can now be paid up to $3,000 for their marrow, as long as it is collected by apheresis. In a concession to the spirit of NOTA, however, the compensation cant be in cash; it needs to be in the form of a voucher that can be applied to things such as scholarships, education, housing or a donation to a charity.

While the decision applies only to the nine states covered by the Ninth Circuit court, and only to bone marrow obtained through apheresis, it does raise bigger questions about how we will look at organ donation in the future. With about 114,000 people waiting for organs in the U.S. alone on any given day, and only 3,300 donors, the urgent medical need runs up against moral standards of the value human life. Once we start paying for the parts we need, though, how far do we go? We dont allow people to buy and sell human beings, thats slavery, says Dr. Robert Klitzman, director of the bioethics program at Columbia University. Should we allow people to buy and sell human body parts?

(MORE: Where Do (Some) Babies Come From? In Washington, a New Law Bans Anonymous Sperm and Egg Donors)

Continue reading here:
A Court Allows Payment for Bone Marrow. Should People Be Able to Sell Their Parts?

Recommendation and review posted by Bethany Smith

Ed Reschke / Getty Images

A color-enhanced photograph of spongy (Cancellous) bone red bone marrow fills the space.

How much would it take for you to consider selling your bone marrow? A U.S. appeals court puts the price at about $3,000 in a ruling that now makes it legal to pay donors for their bone marrow tissue.

The courts decision may well help thousands of sick patients who need bone marrow transplants to survive, but it also begs the question, what other body parts might next be up for sale?

The ruling came about at the end of 2011, in a decision to an October 2009 lawsuit brought by a group of cancer patients, parents and bone marrow donation advocates against the government over the federal law banning the buying and selling of bodily organs. The plaintiffs were led by Doreen Flynn, who has three daughters who suffer from Fanconi anemia, a blood disorder that requires bone marrow transplants to treat. Flynn and the other plaintiffs said that too many such patients die waiting for transplants and argued that we should be allowed to pay people to donate their marrow as a way of ensuring a more reliable supply. The U.S. Court of Appeals for the Ninth Circuit agreed.

(MORE: Facebook Now Lets Organ Donors Tell Their Friends)

At the core of the plaintiffs argument was the National Organ Transplantation Act (NOTA), which since 1984 has forbid the buying and selling of human organs, including bone marrow. But new developments in bone marrow extraction have made marrow donation not much different from donating blood: traditionally, bone marrow donation required anesthesia and long needles to extract the marrow from the hipbones of donors. Now, a technique called peripheral apheresis allows doctors to extract blood stem cells directly from the blood, instead of the bone patients first take a drug that pulls stem cells from the bone and into the blood meaning that the marrow cells should be considered a fluid like blood, rather than an organ, the plaintiffs argued. NOTA doesnt prohibit payments for blood or other fluids, such as plasma or semen.

U.S. Attorney General Eric Holder decided not to ask the U.S. Supreme Court to review the appellate courts decision, which would have been the next step in overturning it. That means the ruling stands and that people can now be paid up to $3,000 for their marrow, as long as it is collected by apheresis. In a concession to the spirit of NOTA, however, the compensation cant be in cash; it needs to be in the form of a voucher that can be applied to things such as scholarships, education, housing or a donation to a charity.

While the decision applies only to the nine states covered by the Ninth Circuit court, and only to bone marrow obtained through apheresis, it does raise bigger questions about how we will look at organ donation in the future. With about 114,000 people waiting for organs in the U.S. alone on any given day, and only 3,300 donors, the urgent medical need runs up against moral standards of the value human life. Once we start paying for the parts we need, though, how far do we go? We dont allow people to buy and sell human beings, thats slavery, says Dr. Robert Klitzman, director of the bioethics program at Columbia University. Should we allow people to buy and sell human body parts?

(MORE: Where Do (Some) Babies Come From? In Washington, a New Law Bans Anonymous Sperm and Egg Donors)

Read more:
Paying for Bone Marrow: Should We Be Able to Sell Our Body Parts?

Recommendation and review posted by Bethany Smith

02-07-2012 09:43 State of the Nation is a nightly newscast anchored by award-winning broadcast journalist, Jessica Soho. It airs Mondays to Fridays at 9:00 PM (PHL Time) on GMA News TV Channel 11. For more videos from State of the Nation, visit fthenation.

Read more from the original source:
SONA: Stem cell therapy, kaya raw makapagpabata ng pangangatawan - Video

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Public release date: 2-Jul-2012 [ | E-mail | Share ]

Contact: Sarah Jackson press_releases@the-jci.org Journal of Clinical Investigation

In Parkinson's disease, the loss of dopamine-producing cells in the midbrain causes well-characterized motor symptoms. Though embryonic stem cells could potentially be used to replace dopaminergic (DA) neurons in Parkinson's disease patients, such cell therapy options must still overcome technical obstacles before the approach is ready for the clinic. Embryonic stem cell-based transplantation regimens carry a risk of introducing inappropriate cells or even cancer-prone cells. To develop cell purification strategies to minimize these risks, Dr. Lorenza Studer and colleagues at Memorial Sloan Kettering Cancer Center in New York developed three different mouse lines to fluorescently label dopaminergic neurons at early, mid, and late stages of differentiation. Their data suggest that mouse embryonic stem cells induced to the mid stage of neuronal differentiation are best suited for transplantation to replace dopaminergic neurons. Further, their work identified new genes associated with each stage of neuronal differentiation. Their results in the mouse model system help define the differentiation stage and specific attributes of embryonic stem cell-derived, dopamine-generating cells that hold promise for cell therapy applications.

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TITLE:

Identification of embryonic stem cellderived midbrain dopaminergic neurons for engraftment

AUTHOR CONTACT:

Lorenz Studer

Memorial Sloan Kettering Cancer Center, New York, NY, USA

Phone: 212.639.6126; E-mail: studerl@mskcc.org

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Generating dopamine via cell therapy for Parkinson's disease

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01-07-2012 19:52 Does identifying athletic genes matter to you? Professor Kathryn North's research led to the discovery of the 'gene for speed'. Learn more and have your say at

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Identifying athletic genes matters to Kathryn North - Video

Recommendation and review posted by Bethany Smith

Public release date: 2-Jul-2012 [ | E-mail | Share ]

Contact: Marla Paul marla-paul@northwestern.edu 312-503-8928 Northwestern University

EVANSTON, Ill. --- Getting under your skin takes on a brave new meaning thanks to Northwestern University research that could transform gene regulation.

A team led by a physician-scientist and a chemist -- from the fields of dermatology and nanotechnology -- is the first to demonstrate the use of commercial moisturizers to deliver gene regulation technology that has great potential for life-saving therapies for skin cancers.

The topical delivery of gene regulation technology to cells deep in the skin is extremely difficult because of the formidable defenses skin provides for the body. The Northwestern approach takes advantage of drugs consisting of novel spherical arrangements of nucleic acids. These structures, each about 1,000 times smaller than the diameter of a human hair, have the unique ability to recruit and bind to natural proteins that allow them to traverse the skin and enter cells.

Applied directly to the skin, the drug penetrates all of the skins layers and can selectively target disease-causing genes while sparing normal genes. Once in cells, the drug simply flips the switch of the troublesome genes to off.

A detailed study of a method that could dramatically redefine the field of gene regulation will be published online during the week of July 2 by the Proceedings of the National Academy of Sciences (PNAS).

Early targets of the novel treatment are melanoma and squamous cell carcinoma (two of the most common types of skin cancer), the common inflammatory skin disorder psoriasis, diabetic wound healing and a rare genetic skin disorder that has no effective treatment (epidermolytic ichthyosis). Other targets could even include wrinkles that come with aging skin.

The technology developed by my collaborator Chad Mirkin and his lab is incredibly exciting because it can break through the skin barrier, said co-senior author Amy S. Paller, M.D., the Walter J. Hamlin Professor, chair of dermatology and professor of pediatrics at Northwestern University Feinberg School of Medicine. She also is director of Northwesterns Skin Disease Research Center.

This allows us to treat a skin problem precisely where it is manifesting -- on the skin, she said. We can target our therapy to the drivers of disease, at a level so minute that it can distinguish mutant genes from normal genes. Risks are minimized, and side effects have not been seen to date in our human skin and mouse models.

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Breaking the skin barrier

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By Brian Alexander

Most people who buy cosmetic lotions and potions know that while the people working behind the department store makeup counters may wear white lab coats, the stuff they sell is more about packaging than science.

But a Northwestern University team is bucking that image, reporting today that theyve created a way to regulate genes affecting the skin -- merely by applying moisturizer.

Not only could their technology pave the way for cosmetics that actually work, but it also might also prove to be a valuable weapon in fighting melanoma, the deadliest form of skin cancer, or diseases like psoriasis, and wounds like the intractable sores that often plague diabetics.

This is a blockbuster in the ways we will treat diseases of the skin, saidChad Mirkin, director of the International Institute for Nanotechnology and the George B. Rathmann Professor of Chemistry at Northwestern said. Were talking about ailments, scarring, wound healing, ways of regulating them or retarding them.

In a research paper published today in the Proceedings of the National Academy of Sciences, Mirkin and his colleagues describe not a drug, exactly, but a way of delivering small sections of nucleic acids (DNA and RNA are nucleic acids) called short interfering RNA, or siRNA, to cells. The cells take up the siRNA, which then alters the way a gene inside each cell can be read by the protein-making system.

The team used gold particles with a diameter of 13 nanometers. (One nanometer is 1-billionth of a meter. A typical strand of human hair is roughly 60,000 nanometers wide.) They coated the particles with siRNA to create what they call spherical nucleic acid nanoparticleconjugates, or SNAs. Millions of SNAs were then added to a commercially available petroleum-based skin moisturizer and the mixture was applied to mice and to lab-grown human skin.

In their key experiment in mice, they used their new system to tamp down the activity of a gene called epidermal growth factor receptor, or EGFR, thats involved in the growth of melanoma. As its name implies, EGFR receives messages from the epidermal growth factor protein. So toning down EGFR will interrupt the message; growth will be reduced or stop.

After mice were treated with the mixture three times per week for three weeks, the expression of the EGFR gene was reduced by 65 percent.

'Impressive' resultsSteve Dowdy, professor of cellular and molecular medicine at the University of California San Diego, and a Howard Hughes Medical Institute investigator specializing in RNA inhibition and ways to deliver siRNAs, called that result impressive.

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Gene healing in a lotion? Researchers are close

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In February last year, The New York Times published an interview with my University of Chicago colleague Janet Rowley. Janet is deservedly famous for finding a repeated chromosome rearrangement in certain types of leukemia. This was one of the earliest indications that genome changes in cancer cells do not occur randomly.

In the interview, Janet explained how she discovered this particular chromosome change, now called the "Philadelphia Chromosome." She was just looking through the microscope, motivated by her curiosity to know more about these tumor cells.

Janet pointed out that she might well not be able to repeat her discovery in today's scientific environment. She was practicing what she called "observationally driven research." Today, she said, granting agencies don't support that kind of work. "That's the kiss of death if you're looking for funding today. We're so fixated now on hypothesis-driven research that if you do what I did, it would be called a 'fishing expedition,' a bad thing."

In other words, you have to know what kind of result to expect before the funding agencies will give you money to look for it. Surprises are not fundable. But "surprise" is just another word for "discovery." As Janet put it, "I keep saying that fishing is good. You're fishing because you want to know what's there."

Let's look at how we would "fish" for complex genomic novelty through natural genetic engineering. I can think of two approaches. There will definitely turn out to be more.

One approach was included in my book. The idea was to do interspecific hybridization with a well-characterized organism, like the mustard weed Arabidopsis, and follow what happens with the genetically unstable hybrid progeny.

We know that interspecific hybridization and genome duplication lead to high levels of genomic and phenotypic variation. DNA sequencing has found evidence of genome duplication at many critical points of evolutionary divergence, especially in plants. There is a fine Scientific American article by the famous 20th-century evolutionist G. Ledyard Stebbins entitled "Cataclysmic Evolution," which describes how hybridization between two wild grasses can recreate the origin of flour wheat.

The hybrid progeny can be followed, and those plants that develop significant new traits, such as flower patterns, can then be analyzed. Sequencing the whole Arabidopsis genome in a short time is now feasible. The sequence data will let the Arabidopsis genome speak for itself in telling us how the new traits evolved.

We can then look for multiple changes that show signs of coordination in the underlying natural genetic engineering events. Such coordinated events might be insertions of the same or related mobile elements at distinct locations in the genome or the addition of the same domains to more than one protein in the network responsible for development of the novel trait.

The second "fishing" approach to asking how a novel feature can evolve would use a microbe, as suggested in the previous blog on experimental evolution. In this case, however, the changes would not be pre-targeted to a number of different sites in the genome.

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James A. Shapiro: Experimental Evolution II: More Ways to Watch Natural Genetic Engineering in Real Time

Recommendation and review posted by Bethany Smith

July 2, 2012

Lawrence LeBlond for redOrbit.com Your Universe Online

At least three genetic mutations found in the human brain have been linked to enlarged brain size (megalencephaly) and a number of disorders, including cancer, epilepsy and autism, according to new research led by Seattle Childrens Research Institute.

The mutations were found in the genes AKT3, PIK3R2 and PIK3CA. The mutations were also linked to vascular disorders and skin growth disorders, said the researchers. The study, published in the online edition of the journal Nature Genetics on June 24, offers important implications for the future of medicine through the research findings.

Study leaders, geneticist William Dobyns, MD, and Jean-Baptiste Rivire, PhD, discovered through their research additional proof that the genetic makeup of a person is not completely determined at the moment of conception. The new evidence ties in with previous research that recognized that genetic changes can occur after conception, although considered quite rare.

The researchers also discovered the genetic causes of these human diseases, including developmental disorders, may also directly lead to new possibilities for treatment.

AKT3, PIK3R2 and PIK3CA are found in all humans, but only when they are mutated do they lead to the diseases and disorders. PIK3CA is known as a cancer-related gene, and appears to make cancer more aggressive. Boston Childrens Hospital researchers recently found a common link between the PIK3CA gene and a rare condition known as CLOVES syndrome.

James Olson, MD, PhD, a pediatric cancer expert at Seattle Childrens and Fred Hutchinson Cancer Research Center acknowledged the two decades-worth of work that led to the findings.

This study represents ideal integration of clinical medicine and cutting-edge genomics, said Olson, who was not involved in the latest research. I hope and believe that the research will establish a foundation for successfully using drugs that were originally developed to treat cancer in a way that helps normalize intellectual and physical development of affected children.

He noted that the team did an excellent job by deep sequencing exceptionally rare familial cases and unrelated cases to identify the culprit pathway. He further noted that the three genes all encode core components of the phosphatidylinositol-3-kinase/AKT pathway, the culprit pathway, as referenced by his work.

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Gene Mutations Associated With Enlarged Brain Size, Disorders

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Newswise Researchers at Moffitt Cancer Center working with colleagues at three other institutions have validated a link between a rare genetic variant and the risk of glioma, the most common and lethal type of brain tumor. The validation study also uncovered an association between the same rare genetic variant and improved rates of survival for patients with glioma.

The study, the first to confirm a rare susceptibility variant in glioma, appeared in a recent issue of the Journal of Medical Genetics, a journal published by the British Medical Association.

"Glioma is a poorly understood cancer with high morbidity and devastating outcomes," said study lead author Kathleen M. Egan, Sc.D., interim program leader of Cancer Epidemiology and vice chair of the Department of Cancer Epidemiology. "However, the discovery of the association of the TP53 genetic variant rs78378222 with glioma provides new insights into these tumors and offers better prospects for identifying people at risk."

According to the authors, their study "genotyped' the single nucleotide polymorphism (SNP, or "snip") rs78378222 in TP53, an important tumor suppressor gene. The researchers said the SNP disrupts the TP53 signal and, because of its activity, has been linked to a variety of cancers. This study linked the presence of the rare form of rs78378222 to deadly glioma.

The researchers conducted a large, clinic-based, case-control study of individuals age 18 and older with a recent glioma diagnosis. A total of 566 glioma cases and 603 controls were genotyped for the rs78378222 variant.

Study results reveal that the odds of developing glioma were increased 3.5 times among the rare variant allele carriers. However, when researchers examined the impact of rs78378222 on survival, they found an approximately 50 percent reduction in death rates for those who were variant allele carriers.

"That the variant increased survival chances was an unexpected finding," Egan said. "It is tempting to speculate that the presence of the risk allele could direct tumor development into a less aggressive path."

The researchers concluded that their study results "may shed light on the etiology and progression of these tumors."

In addition to researchers from Moffitt, researchers from The University of Alabama at Birmingham, Emory School of Medicine and Vanderbilt University participated in the study and co-authored the paper.

The study was supported by funding from Public Health Service Grants R01CA11674 from the National Cancer Institute and the U.S. Department of Health and Human Services, as well as institutional funding from Moffitt and the Vanderbilt-Ingram Cancer Center.

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Moffitt Cancer Center Study Validates Activity of Rare Genetic Variant in Glioma

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ScienceDaily (July 2, 2012) Researchers at Moffitt Cancer Center working with colleagues at three other institutions have validated a link between a rare genetic variant and the risk of glioma, the most common and lethal type of brain tumor. The validation study also uncovered an association between the same rare genetic variant and improved rates of survival for patients with glioma.

The study, the first to confirm a rare susceptibility variant in glioma, appeared in a recent issue of the Journal of Medical Genetics, a journal published by the British Medical Association.

"Glioma is a poorly understood cancer with high morbidity and devastating outcomes," said study lead author Kathleen M. Egan, Sc.D., interim program leader of Cancer Epidemiology and vice chair of the Department of Cancer Epidemiology. "However, the discovery of the association of the TP53 genetic variant rs78378222 with glioma provides new insights into these tumors and offers better prospects for identifying people at risk."

According to the authors, their study "genotyped' the single nucleotide polymorphism (SNP, or "snip") rs78378222 in TP53, an important tumor suppressor gene. The researchers said the SNP disrupts the TP53 signal and, because of its activity, has been linked to a variety of cancers. This study linked the presence of the rare form of rs78378222 to deadly glioma.

The researchers conducted a large, clinic-based, case-control study of individuals age 18 and older with a recent glioma diagnosis. A total of 566 glioma cases and 603 controls were genotyped for the rs78378222 variant.

Study results reveal that the odds of developing glioma were increased 3.5 times among the rare variant allele carriers. However, when researchers examined the impact of rs78378222 on survival, they found an approximately 50 percent reduction in death rates for those who were variant allele carriers.

"That the variant increased survival chances was an unexpected finding," Egan said. "It is tempting to speculate that the presence of the risk allele could direct tumor development into a less aggressive path."

The researchers concluded that their study results "may shed light on the etiology and progression of these tumors."

In addition to researchers from Moffitt, researchers from The University of Alabama at Birmingham, Emory School of Medicine and Vanderbilt University participated in the study and co-authored the paper.

The study was supported by funding from Public Health Service Grants R01CA11674 from the National Cancer Institute and the U.S. Department of Health and Human Services, as well as institutional funding from Moffitt and the Vanderbilt-Ingram Cancer Center.

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Activity of rare genetic variant in glioma validated

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The Irish Times - Monday, July 2, 2012

LORNA SIGGINS

WORLD leaders in stem-cell technology are due to exchange knowledge of potential treatments at a conference opening in NUI Galway today.

Researchers from NUIG, University College Cork and NUI Maynooth will participate in the event, which has been billed as the first major conference on stem-cell therapy in Ireland.

Prof Anthony Hollander of the University of Bristol, England who was one of a team which successful created and then transplanted the first tissue-engineered trachea or windpipe is among a number of international speakers presenting findings.

The gathering will focus on the realities of stem-cell treatment, Prof Frank Barry, director of NUIGs National Centre for Biomedical Engineering Science has said.

The therapy is complex and controversial, and sometimes exaggerated claims are made, he said.

The researchers are specialists in Mesenchymal, or adult, stem cells, and will be concentrating on what is likely in the future, he added.

The list of conditions which could be treated successfully by stem cells is small, but growing, Prof Barry said.

Leukaemia and other diseases of the blood appear to respond best.

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Stem-cell research leaders to meet in NUIG

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RANCHO CORDOVA, Calif., July 2, 2012 /PRNewswire/ -- ThermoGenesis Corp. (KOOL), a leading supplier of enabling technologies for the processing, storage and administration of cell therapies, said today that Asahi Kasei Medical Co., Ltd., has exercised its option to purchase the Company's CryoSeal Fibrin Sealant System wound care product line for $2 million in cash, effective June 30, 2012. Payment is due during the Company's first fiscal quarter of 2013.

This transaction is the culmination of the Company's previously announced strategy and option agreement to divest the CryoSeal product line, according to Matthew Plavan, Chief Executive Officer of ThermoGenesis. "We are very pleased to consummate the sale of the CryoSeal product line to our long-time strategic partner, Asahi. We look forward to their successful commercialization of CryoSeal in the wound care market," Plavan said.

"This divestiture is an important milestone in our long-term strategy to focus on the development of enabling technologies for the stem cell regenerative medicine market. It significantly strengthens our balance sheet and the proceeds from this transaction will be used to fund our market expansion efforts for our cord blood and bone marrow stem cell processing and storage offerings, particularly in markets outside North America. In addition, it frees up management and corporate resources to address these more strategic market opportunities, and furthers our initiative to reduce operating costs," Plavan added.

About ThermoGenesis Corp.ThermoGenesis Corp. (www.thermogenesis.com)is a leader in developing and manufacturing automated blood processing systems and disposable products that enable the manufacture, preservation and delivery of cell and tissue therapy products. These include:

This press release contains forward-looking statements. These statements involve risks and uncertainties that could cause actual outcomes to differ materially from those contemplated by the forward-looking statements. Several factors including timing of FDA and foreign regulatory approvals, changes in customer forecasts, our failure to meet customers' purchase order and quality requirements, supply shortages, production delays, changes in the markets for customers' products, introduction timing and acceptance of our new products scheduled for fiscal years 2012 and 2013, and introduction of competitive products and other factors beyond our control could result in a materially different revenue outcome and/or in our failure to achieve the revenue levels we expect for fiscal 2012 and 2013. A more complete description of these and other risks that could cause actual events to differ from the outcomes predicted by our forward-looking statements is set forth under the caption "Risk Factors" in our annual report on Form 10-K and other reports we file with the Securities and Exchange Commission from time to time, and you should consider each of those factors when evaluating the forward-looking statements.

ThermoGenesis Corp.Web site: http://www.thermogenesis.comContact: Investor Relations+1-916-858-5107, orir@thermogenesis.com

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ThermoGenesis Announces Sale Of CryoSeal® Product Line To Asahi

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

Advanced Cell Technology, Inc. (ACT; OTCBB: ACTC), a leader in the field of regenerative medicine, today announced treatment of the second patient in its Phase 1/2 clinical trial for Stargardts macular dystrophy (SMD) using retinal pigment epithelial (RPE) cells derived from human embryonic stem cells (hESCs). The surgery was performed on Friday, June 29 at Moorfields Eye Hospital in London, the same site as the first patient treatment in January, by a team of surgeons led by Professor James Bainbridge, consultant surgeon at Moorfields and Chair of Retinal Studies at University College London. The procedure was successfully performed without any complications. ACT and Moorfields Eye Hospital recently received clearance from the Data and Safety Monitoring Board (DSMB) to treat the final two patients in the first cohort of this clinical trial.

We are very pleased to continue our forward momentum with both our U.S. trials and our European trial, commented Gary Rabin, chairman and CEO. It was less than a month ago that we received DSMB approval to treat the second and third patients in our E.U. trial, and it is very gratifying to have already completed dosing of the second. It is a pleasure to be working with Professor Bainbridge and the rest of his team at Moorfields Eye Hospital, and we continue to be encouraged by the steady progress of the trial thus far.

The Phase 1/2 trial is designed to determine the safety and tolerability of hESC-derived RPE cells following sub-retinal transplantation in patients with SMD at 12 months, the studys primary endpoint. It will involve a total of 12 patients, with cohorts of three patients each in an ascending dosage format. It is similar in design to the U.S. trial for SMD that was initiated in July 2011.

The European Medicines Agency's (EMA) Committee for Orphan Medicinal Products (COMP) has officially designated ACT's human embryonic stem cell (hESC)-derived retinal pigment epithelial (RPE) cells as an orphan medicinal product for the treatment of Stargardt's Macular Dystrophy (SMD).

More information on the status of the companys clinical trials will be posted today on Mr. Rabins Chairmans blog.

About Stargardts Disease Stargardts disease or Stargardts Macular Dystrophy is a genetic disease that causes progressive vision loss, usually starting in children between 10 to 20 years of age. Eventually, blindness results from photoreceptor loss associated with degeneration in the pigmented layer of the retina, called the retinal pigment epithelium, which is the site of damage that the company believes the hESC-derived RPE may be able to target for repair after administration.

About Advanced Cell Technology, Inc. Advanced Cell Technology, Inc. is a biotechnology company applying cellular technology in the field of regenerative medicine. For more information, visit http://www.advancedcell.com.

Forward-Looking Statements Statements in this news release regarding future financial and operating results, future growth in research and development programs, potential applications of our technology, opportunities for the company and any other statements about the future expectations, beliefs, goals, plans, or prospects expressed by management constitute forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Any statements that are not statements of historical fact (including statements containing the words will, believes, plans, anticipates, expects, estimates, and similar expressions) should also be considered to be forward-looking statements. There are a number of important factors that could cause actual results or events to differ materially from those indicated by such forward-looking statements, including: limited operating history, need for future capital, risks inherent in the development and commercialization of potential products, protection of our intellectual property, and economic conditions generally. Additional information on potential factors that could affect our results and other risks and uncertainties are detailed from time to time in the companys periodic reports, including the report on Form 10-K for the year ended December 31, 2011. Forward-looking statements are based on the beliefs, opinions, and expectations of the companys management at the time they are made, and the company does not assume any obligation to update its forward-looking statements if those beliefs, opinions, expectations, or other circumstances should change. Forward-looking statements are based on the beliefs, opinions, and expectations of the companys management at the time they are made, and the company does not assume any obligation to update its forward-looking statements if those beliefs, opinions, expectations, or other circumstances should change. There can be no assurance that the Companys clinical trials will be successful.

Original post:
ACT Announces Second Patient with Stargardt’s Disease Treated in EU Clinical Trial

Recommendation and review posted by simmons

COLUMBIA, Md.--(BUSINESS WIRE)--

Osiris Therapeutics, Inc. (OSIR), announced today interim one-year results from its groundbreaking clinical trial evaluating Prochymal (remestemcel-L) for the treatment of patients experiencing first-time acute myocardial infarction. The trial is the largest study of allogeneic or "off-the-shelf" stem cells ever conducted in heart attack patients. A total of 220 patients were given a single infusion of either Prochymal or placebo through a standard intravenous line within seven days of an acute heart attack.

Cardiac MRI assessments were conducted for six months following infarct to evaluate cardiac remodeling. Patients receiving Prochymal had significantly less cardiac hypertrophy, as measured by cardiac MRI, compared to patients receiving placebo (p<0.05). Patients treated with Prochymal also experienced significantly less stress-induced ventricular arrhythmia (p<0.05). Cardiac hypertrophy and ventricular arrhythmia are indicators of pathological remodeling following heart injury and provide insight into the mechanism by which mesenchymal stem cells attenuate heart injury following a myocardial infarction.

The mechanistic data is complemented by clinical data showing treatment with Prochymal resulted in a statistically significant reduction in heart failure. In the study, seven patients who were treated with placebo have progressed to heart failure requiring treatment with intravenous diuretics, compared to none of the Prochymal patients (p=0.01). Furthermore, patients receiving placebo tended to require re-hospitalization for cardiac issues sooner than the patients receiving Prochymal (median 27.5 days vs. 85.5 days).

This study is the largest of its kind and provides key insights into the mechanism of action of mesenchymal stem cells in the setting of acute myocardial infarction, said Lode Debrabandere, Ph.D., Senior Vice President of Therapeutics at Osiris. These important mechanistic observations are consistent with data obtained from our preclinical models and from the first placebo-controlled human trial with Prochymal published in the Journal of the American College of Cardiology. Given the quality of the data and highly encouraging results observed thus far, we are extending the trial's duration to capture a better understanding of the long-term clinical benefits of MSCs."

The trial also demonstrated that treatment with Prochymal was safe. There were no infusional toxicities observed in patients receiving Prochymal. Serious adverse events occurred with equal frequency in both treatment groups (31.8%). To date, there have been 5 deaths in the trial, 2 in the Prochymal group and 3 in the placebo group.

For interventional cardiologists, keeping our myocardial infarction patients from progressing to heart failure is central to our mission, said Mark Vesely, M.D., Principal Investigator on the Study and Assistant Professor of Medicine (Interventional Cardiology) at the University of Maryland School of Medicine. It is remarkable and very encouraging to see significant changes in clinically meaningful parameters this early in the study. We look forward to the additional data that will be gathered as the study progresses, which will help us to better understand both the magnitude and durability of the benefit to treatment.

Prochymal, the worlds first and only stem cell drug approved by an internationally recognized regulatory authority, is used for the treatment of graft vs. host disease (GvHD). GvHD is a devastating complication of bone marrow transplantation that kills up to 80 percent of children affected. Prochymal is now approved in Canada and New Zealand, and is currently available in seven other countries including the United States under an Expanded Access Program (EAP).

About the Trial

This Phase 2, multi-center, randomized, double-blind, placebo-controlled study is evaluating the safety and efficacy of Prochymal (ex-vivo cultured adult human mesenchymal stem cells) intravenous infusion following acute myocardial infarction. A total of 220 patients were randomized (1:1) at 33 centers in the United States and Canada and received a single intravenous infusion of Prochymal or placebo within 7 days following first acute myocardial infarction. In addition to screening and baseline visits prior to the infusion, initially follow-up evaluations were scheduled to be conducted through 2 years. Given the encouraging results observed at the one year time-point, the trial is being extended to include 5 years of follow-up. Both male and female subjects between 21 and 85 years of age were enrolled. Patients had to have a left ventricular ejection fraction (LVEF) between 20% and 45% as determined by quantitative echocardiography or cardiac MRI at least 24 hours after successful reperfusion of the culprit vessel. In addition, troponin levels must have been greater than 4 times the upper limit of normal during the first 72 hours of hospitalization for the MI.

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Prochymal Significantly Reduces Hypertrophy, Arrhythmia and Progression to Heart Failure in Patients Suffering a Heart ...

Recommendation and review posted by Bethany Smith

Public release date: 2-Jul-2012 [ | E-mail | Share ]

Contact: Kim Newman sciencenews@einstein.yu.edu 718-430-3101 Albert Einstein College of Medicine

July 2, 2012 -- (Bronx, NY) -- Researchers at Albert Einstein College of Medicine of Yeshiva University have found that abnormal bone marrow stem cells drive the development of myelodysplastic syndromes (MDS), serious blood diseases that are common among the elderly and that can progress to acute leukemia. The findings could lead to targeted therapies against MDS and prevent MDS-related cancers. The study is published today in the online edition of the journal Blood.

"Researchers have suspected that MDS is a 'stem cell disease,' and now we finally have proof," said co-senior author Amit Verma, M.B.B.S., associate professor of medicine and of developmental and molecular biology at Einstein and attending physician in oncology at Montefiore Einstein Center for Cancer Care. "Equally important, we found that even after MDS standard treatment, abnormal stem cells persist in the bone marrow. So, although the patient may be in remission, those stem cells don't die and the disease will inevitably return. Based on our findings, it's clear that we need to wipe out the abnormal stem cells in order to improve cure rates."

MDS are a diverse group of incurable diseases that affect the bone marrow and lead to low numbers of blood cells. While some forms of MDS are mild and easily managed, some 25 to 30 percent of cases develop into an aggressive disease called acute myeloid leukemia. Each year, about 10,000 to 15,000 people in the U.S. are diagnosed with MDS, according to the National Marrow Donor Program.

Most cases of MDS occur in people over age 60, but the disease can affect people of any age and is more common in men than women. Symptoms vary widely, ranging from anemia to infections, fever and bleeding. Treatment usually involves chemotherapy to destroy abnormal blood cells plus supportive care such as blood transfusions.

In the current study, lead author Britta Will, Ph.D., research associate in the department of cell biology, and her colleagues analyzed bone marrow stem cells and progenitor cells (i.e., cells formed by stem cells) from 16 patients with various types of MDS and 17 healthy controls. The stem and progenitor cells were isolated from bone marrow using novel cell-sorting methods developed in the laboratory of co-senior author Ulrich Steidl, M.D., Ph.D., assistant professor of cell biology and of medicine and the Diane and Arthur B. Belfer Faculty Scholar in Cancer Research at Einstein.

Genome-wide analysis revealed widespread genetic and epigenetic alterations in stem and progenitor cells taken from MDS patients, in comparison to cells taken from healthy controls. The abnormalities were more pronounced in patients with types of MDS likely to prove fatal than in patients with lower-risk types.

"Our study offers new hope that MDS can be more effectively treated, with therapies that specifically target genes that are deregulated in early stem and progenitor cells," said Dr. Steidl. "In addition, our findings could help to detect minimal residual disease in patients in remission, allowing for more individualized treatment strategies that permanently eradicate the disease."

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Myelodysplastic syndromes (MDS) linked to abnormal stem cells

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