On a brilliant day in April, tens of thousands of baseball fans stream past Jonathan Thomas's office towards AT&T Park for the first home game of the San Francisco Giants 2014 season. Thomas's standing desk faces away from the window, but the cheering throngs are never far from his mind.

Thomas chairs the board of the California Institute for Regenerative Medicine (CIRM), the US$3-billion agency hailed by scientists around the world for setting a benchmark for stem-cell research funding. But scientists will not be the ones who decide what becomes of CIRM when the cash runs out in 2017. Instead, it will be the orange-and-black-clad masses walking past Thomas's window. And to win their support, Thomas knows that the agency needs to prove that their collective investment has been worthwhile. We need to drive as many projects to the patient as soon as possible, he says.

Californians voted CIRM into existence in 2004, making it the largest funder of stem-cell work in the world. The money the proceeds of bond sales that must be repaid with $3 billion in interest by taxpayers helped to bring 130 scientists to the state, and created several thousand jobs there. It has funded research that led to the publication of more than 1,700 papers, and it has contributed to five early clinical trials.

The institute has navigated a difficult path, however. CIRM had to revamp its structure and practices in response to complaints about inefficiency and potential conflicts of interest. It has also had to adapt its mission to seismic shifts in stem-cell science.

Now, ten years after taking off, the agency is fighting for its future. It has a new president, businessman Randal Mills, who replaces biologist Alan Trounson. Its backers have begun to chart a course for once again reaching out to voters, this time for $5 billion (with another $5 billion in interest) in 2016. And it is under intense pressure to produce results that truly matter to the public.

Whether or not CIRM succeeds, it will serve as a test bed for innovative approaches to funding. It could be a model for moving technologies to patients when conventional funding sources are not interested.

Much of what is celebrated and lamented about CIRM can be traced back to the Palo Alto real-estate developer who conceived of it: Robert Klein. Although officially retired from CIRM he chaired the board from 2004 to 2011 (see 'State of funding') Klein's office is adorned with mementos of the agency: a commemorative shovel from the groundbreaking of a CIRM-funded stem-cell research centre, and a photo of him with former governor Arnold Schwarzenegger at the ribbon-cutting ceremony.

Liz Hafalia/San Francisco Chronicle/Polaris/eyevine

Patient advocates and parents at a 2012 meeting in which US$100 million in CIRM grants were approved.

It was Klein's idea to ask voters to support stem-cell research in 2004, through a ballot measure called Proposition 71. When he succeeded, CIRM instilled a kind of euphoria in stem-cell scientists, who were at the time still reeling from a 2001 decree by then-President George W. Bush that severely limited federal funding for embryonic-stem-cell research. California's commitment removed this roadblock and revealed that many in the state and the country supported the research.

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Stem cells: Hope on the line

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Salk Institute scientist Joseph Ecker holds a flow cell slide used in a genome sequencing machine. Ecker and colleagues compared the genomes of two kinds of artificial embryonic stem cells for a study comparing their quality.

In a setback for hopes of therapy with a promising kind of artificial embryonic stem cells, a study published in the journal Nature has found that these "induced pluripotent stem cells" have serious quality issues.

However, scientists who performed the study, including researchers from the Salk Institute and UC San Diego, say it should be possible to improve the quality of these IPS cells. They say lessons can be learned from studying a newer technique of making human embryonic stem cells through nuclear transfer, the same technology used to create Dolly the cloned sheep.

In addition, the study does not prove that the quality problems will affect therapy with the cells, said scientists who examined the study. That remains to be tested.

The IPS cells are made from skin cells treated with "reprogramming" factors that turn back the clock, so they very closely resemble embryonic stem cells. The hope is that these IPS cells could be differentiated into cells that can repair injuries or relieve diseases. Because they can be made from a patient's own cells, the cells are genetically matched, reducing worries of immune rejection.

In San Diego, scientists led by Jeanne Loring at The Scripps Research Institute have created IPS cells from the skin cells of Parkinson's disease patients, and turned the IPS cells into neurons that produce dopamine. They hope to get approval next year to implant these cells into the patients, relieving symptoms for many years. The project is online under the name Summit4StemCell.org.

A major concern is that IPS cells display abnormal patterns of gene activation and repression. This is controlled by a process called methylation. This process adds chemicals called methyl groups to DNA, but these "epigenetic" changes do not change the underlying DNA sequence. Methylation represses gene function; removing the methyl groups, or demethylation, activates them.

The Nature study was led by Shoukhrat Mitalipov of Oregon Health & Scence University. Mitalipov made headlines last year for applying nuclear transfer to derive human embryonic stem cells, the first time this has been achieved in human cells. These cells can be made to be a near-perfect genetic match to the patient, and their quality closely resembles those of true embryonic stem cells.

"We know that the embryonic stem cells are the gold standard, and we've been always trying to make patient-matched cells that would match the gold standard," Mitalipov said. "And at this point it looks like the NT (nuclear transfer) cells produce exactly those cells that would be best."

Nuclear transfer involves placing a nucleus from a skin cell into an egg cell that has had its nucleus removed. The cell is then stimulated, and starts dividing in the same way a fertilized egg cell divides to form an embryo.

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Artificial embryonic stem cells have quality problems: study

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Dallas, TX (PRWEB) July 02, 2014

Cell Culture Market by Equipment (Bioreactor, Incubator, Centrifuge), by Reagent (Media, Sera, Growth Factors, Serum Free Media), by Application (Cancer Research, Gene Therapy, Drug Development, Vaccine Production, Toxicity Testing) Global Forecast to 2018 research report segments the cell culture market into two distinct market segments, namely, the cell culture equipment market and the cell culture media, sera, and reagents market. The segments of the cell culture equipment market included in this report are bio-safety cabinets, consumables, lab equipment, sterilization equipment, and storage equipment. The lab equipment segment consists of four subsegments, namely, cell counters, centrifuges, fermentors and bioreactors, and incubators. The storage equipment segment consists of two subsegments, namely, cryogenic storage and refrigerators and freezers.

The segments of the cell culture media, sera, and reagents market included in this report (http://www.lifescienceindustryresearch.com/cell-culture-market-by-equipment-bioreactor-incubator-centrifuge-by-reagent-media-sera-growth-factors-serum-free-media-by-application-cancer-research-gene-therapy-drug-development-vacci.html ) are contamination detection kits, cryoprotective agents, lab reagents, media, serum, and other reagents. The lab reagents segment consists of four subsegments, namely, balanced salt solutions, buffers and chemicals, cell dissociation reagents, and supplements and growth factors. The media segment consists of three subsegments, namely, basal media, reduced serum media, and serum-free media.

Cell culture has its applications in a large number of fields. On the basis of application, the report is segmented into biopharmaceutical production, cancer research, drug screening and development, gene therapy, tissue culture and engineering, toxicity testing, vaccine production, and other applications. The geographic segments included in this report are North America, Europe, Asia-Pacific, and Rest of the World.

The key drivers for this market are rapid increase in the demand for biopharmaceuticals and increased adoption of single-use technology. According to IMS Health, biopharmaceutical is expected to be the fastest growing pharmaceutical product segment by2017. Biopharmaceuticals are pharmaceutical products isolated from living organisms. Cell culture is one of the most important and widely used techniques for biopharmaceutical production. It was also the largest application segment of the cell culture market in 2013. Growth in the biopharmaceutical market will drive the growth of the cell culture market.

Companies profiled in this cell culture market research report include Becton, Dickinson and Company, Corning, Inc., Eppendorf AG, Lonza Group Ltd., Merck Kgaa, Sartorius AG, Sigma-Aldrich Corporation, Thermo Fisher Scientific, Inc. and Promocell GMBH. Order a copy of this report at http://www.lifescienceindustryresearch.com/purchase?rname=15929 .

Secondary information was used to identify the overall revenues, geographic reach, and product portfolios of the market players. Estimates of their cell culture segment revenues were validated through primary interviews. Primary interviews with key opinion leaders were also used to determine percentage shares of each subsegment and the relative differences in the growth rates.

The report provides qualitative insights about key market shares, growth rates, and market drivers for all important subsegments. It maps the market sizes and growth rates of each subsegment and identifies the segments poised for rapid growth in each of the geographic segments. The report also includes company profiles of the market leaders. These company profiles include financial performances, product portfolios, and market developments for each company. The report also provides a competitive landscape of the cell culture market. The competitive landscape covers the growth strategies adopted by the industry players over the last three years. It also includes analysis of industry developments like mergers and acquisitions, agreements and partnerships, and new product launches.

Reasons to Buy the Report:

The report will enable both established firms and new entrants to gauge the pulse of the market and help them make important strategic growth decisions. The report provides insights on the following:

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10.71% CAGR for Cell Culture Market Forecast to 2018 in a New Global Research Report Available at ...

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NEW YORKTibetans living on the roof of the world can thank an extinct human relative for providing a gene that helps them adapt to the high altitude, a study suggests.

Past research has concluded that a particular gene helps people live in the thin air of the Tibetan plateau. Now scientists report the Tibetan version of that gene is found in DNA from Denisovans, a poorly understood human relative more closely related to Neanderthals than modern people.

Denisovans are known only from fossils in a Siberian cave that are dated to about 50,000 years ago. Some of their DNA has also been found in other modern populations, indicating they interbred with ancient members of todays human race long ago.

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But the version of the high-altitude gene shared by Denisovans and Tibetans is found in virtually no other population today, researchers report in an article released Wednesday by the journal Nature.

That suggests that Denisovans or close relatives of theirs introduced the gene variant into the modern human species, but that it remained rare until some people started moving into the Tibetan plateau, said the studys main author, Rasmus Nielsen of the University of California, Berkeley.

At that point, it conferred a survival advantage and so spread through the Tibetan population, he said in an email. Its not clear whether the Denisovans were also adapted to high altitudes, he said.

The results show that as early members of todays human species expanded outside of Africa and encountered new environments, they could call on their genetic legacies from other species, he said. Thats easier than waiting for a helpful genetic mutation to arise, he said.

The Tibetan plateau rises above 4,000 metres in elevation. The genetic variant helps survival there by affecting the amount of oxygen the blood can carry when a person is in thin air. Apart from Tibetans, it is found very rarely in Han Chinese and also exists in Mongolians and Sherpas, Nielsen said. The researchers found no trace of it outside East Asia.

David Reich, an expert on ancient DNA at Harvard Medical School, called the paper important and exciting in showing the gene came from an ancient human relative. But he said that relative could have been Neanderthals, who are also known to have contributed DNA to modern people.

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Tibetans inherited high-altitude gene from extinct human relatives: study

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Mutation is similar to a 'super athlete' gene believed to give improved athletic performance Gene came from Denisovans, a human relative that became extinct 40,000-50,000 years ago Ethnic Tibetans split off from the Chinese Han less than 3,000 years ago

By Mark Prigg

Published: 16:16 EST, 2 July 2014 | Updated: 17:22 EST, 2 July 2014

Tibetans were able to adapt to high altitudes thanks to a gene picked up when their ancestors mated with a now extinct early species of human.

It is the first time a gene from another species of human has been shown unequivocally to have helped modern humans adapt to their environment, researchers said.

They found the gene came from Denisovans a human relative that became extinct 40,000-50,000 years ago, around the same time as the more well-known Neanderthals.

Tibetan nomads play billiards on an open grassland near Namtso Lake: Researchers have revealed the population evolved to thrive at high altitudes in just 3,000 years.

The gene has been referred to as the 'superathlete' gene because at low elevations, some variants of it help athletes quickly boost hemoglobin and thus the oxygen-carrying capacity of their blood, upping endurance.

At high altitudes, however, the common variants of the gene boost hemoglobin and its carrier, red blood cells, too much, increasing the thickness of the blood and leading to hypertension and heart attacks as well as low birth weight babies and increased infant mortality.

The variant, or allele, found in Tibetans raises hemoglobin and red blood cell levels only slightly at high elevations, avoiding the side effects seen in most people who relocate to elevations above 13,000 feet.

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'Super athlete' gene in Tibetans evolved in just 3,000 years

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This undated photo provided by BGI shows daily life in Tibet, an autonomous region of China. Tibetans can thank an extinct human relative for providing a gene that helps them adapt to the high altitude, according to a study released on Wednesday, July 2, 2014. (AP Photo/BGI)

NEW YORK -- Tibetans living on the "roof of the world" can thank an extinct human relative for providing a gene that helps them adapt to the high altitude, a study suggests.

Past research has concluded that a particular gene helps people live in the thin air of the Tibetan plateau. Now scientists report that the Tibetan version of that gene is found in DNA from Denisovans, a poorly understood human relative more closely related to Neanderthals than modern people.

Denisovans (de-NEE'-soh-vens) are known only from fossils in a Siberian cave that are dated to at least about 50,000 years ago. Some of their DNA has also been found in other modern populations, indicating they interbred with ancient members of today's human race long ago.

But the version of the high-altitude gene shared by Denisovans and Tibetans is found in virtually no other population today, researchers report in an article released Wednesday by the journal Nature.

That suggests that Denisovans or close relatives of theirs introduced the gene variant into the modern human species, but that it remained rare until some people started moving into the Tibetan plateau, said study main author Rasmus Nielsen of the University of California, Berkeley.

At that point, it conferred a survival advantage and so spread through the Tibetan population, he said in an email. It's not clear whether the Denisovans were also adapted to high altitudes, he said.

The results show that as early members of today's human species expanded outside of Africa and encountered new environments, they could call on their genetic legacies from other species, he said. That's easier than waiting for a helpful genetic mutation to arise, he said.

The Tibetan plateau rises above 13,000 feet in elevation. The genetic variant helps survival there by affecting the amount of oxygen the blood can carry when a person is in thin air.

FILE - In this June 11, 2008 file photo, Chinese Tibetan ethnic herdsmen try to catch a yak for sale in Dengsheng of Aba, China's southwest Sichuan province. Tibetans can thank an extinct human relative for providing a gene that helps them adapt to the high altitude, according to a study released on Wednesday, July 2, 2014. (AP Photo/Alexander F. Yuan, File)

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Ancient gene helps Tibetans cope with high altitude

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Tibetans, who thrive in an oxygen-scarce, high-altitude environment that leaves most gasping for breath, may have inherited their ability to adapt from an ancient human ancestor discovered just four years ago.

Earlier studies identified a version of a gene called EPAS1 as being responsible to Tibetans ability to adjust to their environment 4,000 meters (13,000 feet) above sea level. An analysis of the variant, which isnt found in other peoples, showed it in the remains of a Denisovan, the name for a species of human that lived about 41,000 years ago.

The findings, reported today in the journal Nature, suggest that interbreeding between modern humans and Denisovans provided the right genetic ingredients to enable their Tibetan descendants to thrive on the worlds highest plateau. In a broader sense, they also point to interbreeding between humans and other hominins as a key to survival in new environments.

Perhaps that process of adaptive introgression, of getting genes from other species, might be more important in evolution than previously thought, said Rasmus Nielsen, a professor at the University of California at Berkeley and one of the studys authors.

The Denisovans, a human species distinct from Neanderthals and modern humans, could have interbred with the ancestors of Tibetans before they migrated upwards, making the adaptations an essential advantage, Nielsen said.

The study focused on the EPAS1 gene because it regulates levels of oxygen-carrying hemoglobin. In most people, the gene causes the body to produce more hemoglobin when less oxygen is available. At high altitudes, it can go too far. Thickened blood can cause heart problems, hypertension and higher infant mortality.

The variation carried by Tibetans only raises hemoglobin production slightly, leading to a distinct survival advantage. The same high-altitude friendly variation was present in the genome sequenced from a single finger bone found in the Denisova Cave in southern Siberia. DNA sequencing in 2010 found that bone to be distinct from both Neanderthals and humans, which also inhabited the cave at different times. The full Denisovan genome was sequenced in 2012.

Studies have found humans probably interbred with Neanderthals and Denisovans before they became extinct. About 2 to 4 percent of human DNA today is made up of Neanderthal genes, according to research published earlier this year. Research has suggested the interbreeding may have conferred advantages enabling humans to survive in non-African environments.

The Tibetan adaptation strengthens the argument for adaptive introgression, in which advantageous genes are absorbed from existing populations, said Anna Di Rienzo, a professor of human genetics at the University of Chicago who has also researched the origin of the Tibetans genetic variation.

Its one of the most clear-cut cases of adaptive introgression with archaic humans, Di Rienzo said in an interview. Its clearly a very good case where we understand a lot about whats going on with this adaptation.

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Tibetans Got High-Altitude Gene From Archaic Humans

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Stephen Cass: Hello, Im Stephen Cass for IEEE Spectrums Techwise Conversations.

Nancy Kress is a celebrated author of science fiction and fantasy novels and short stories. First published in 1976, her work often focuses on the implications of genetic engineering and other biomedical technologies. Among other awards, she has won five Nebulas from the Science Fiction & Fantasy Writers of America.

As part of its 50th anniversary celebrations this August, Spectrum will be publishing Coming Soon Enough, an anthology of six original science fiction stories, including one by Kress titled Someone to Watch Over Me. But you dont have to wait to read it: The story is available now as part of Spectrums June special issue about the long-term future of technology.

Without giving anything away, Someone to Watch Over Me is a dark tale about the unintended consequences of advanced biomedical implant technology. To talk about the story and the broader themes that run throughout her work, Kress joins us now by phone from her home in Seattle. Nancy, welcome to the podcast.

Nancy Kress: Thank you. Im glad to be here.

Stephen Cass: So what was the inspiration for Someone to Watch Over Me?

Nancy Kress: I often write about children. The next generation of any society is, of course, what carries it forward. But in addition, we are faced right now with so many interesting possibilities, with genetic engineering and in other technologies as well, that the generation being born now is growing up far different than, say, the way I did. Texting, to me, would have seemed unimaginable. To them, its just normal ho-hum everyday kind of things. So when I was thinking about this story, I was thinking about cameraswhich, of course, are a far next generation of Google Glassthat actually fit in the eye and are not noticeable to anybody else or even possibly to the wearer, except in that they are recording. I naturally turn to the idea of a child wearing one, and I asked myself, What child? Under what circumstances? And from there the story grew. I frequently do start with a character.

Stephen Cass: So how closely do you then follow real-world technological developments? You mentioned Google Glass, which is an emerging technology

Nancy Kress: Im not trained as a scientist, which I deeply regret. When I was in high school, I didnt have chemistry because it conflicted with French 4, and now I cant do chemistry or speak French. It was a great tragedy. However, I try to keep up with the journals that are written for laymen, and when something captures my attention, Ill try to get the latest books on it. And I also collect microbiologists the way some people collect butterflies.

And I have a list of people that I can ask questions of when something captures my attention. So its kind of hit and miss whether some emergent technology comes to my attention. But if it does, and if it also succeeds in capturing that attention, then I try to find out about it in anyplace thats available to me.

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Nancy Kress: How Science Fiction Helps Us Rehearse for the Future

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Everyone loves a medical mystery, except the afflicted patient and his or her family who shuffle from doctor to doctor in search of an explanation for a disorder whose name, origin, prognosis and cure are all unknown. Now, the National Institutes of Health have underwritten a nationwide "whodunnit" campaign, with a $43 million-initiative to fund the diagnosis and exploration of undiagnosed, unrecognized and misunderstood diseases.

Think of it as a bid to clear a backlog of medical cold cases, using dazzling new tools now at the disposal of medical sleuths. Think of it also as an opportunity for scientists to glean new insights into the human genome and its role in causing -- and perhaps curing -- disease.

This week, the NIH announced the expansion of its Undiagnosed Disease Network, which by the summer of 2017 is expected to enroll at least 300 new patients with mysterious and intractable conditions per year, nationwide.

The diseases that afflict these patients are conditions that have stumped even skilled physicians. Doctors may fail to recognize the conditions because they are rarely seen, have never been described in the medical literature or are rare forms of more common diseases. Some may be caused outright by infection or environmental exposure. But many will have their origins in a patient's genes but have been unexpressed or gone undetected earlier in the patient's family tree.

Prospective patients interested in participating in the Undiagnosed Diseases Network may learn more here.

The initiative builds on a pilot program run for six years from NIH's Bethesda, Md., clinical center. There, some 600 patients whose symptoms have stumped their doctors have been brought in for extensive diagnostic work-ups in a bid to identify and treat their diseases.

Using genomic analysis and a full toolbox of standard diagnostic tests, the multidisciplinary clinical and research team has diagnosed approximately 100 patients. In the process, the NIH team discovered two unknown diseases and identified 15 genes not previously associated with any other human disease.

Now, the experts at NIH will be joined in the hunt by physician/scientists at six institutions across the country -- Baylor College of Medicine in Houston; Boston Children's Hospital, Brigham and Women's Hospital, and Massachusetts General Hospital in Boston; Duke University in Durham, N.C.; Stanford University; UCLA; and Vanderbilt University Medical Center in Nashville, Tenn.

The expansion of the mystery diseases network comes at a time that scientists could only dream about when the Human Genome Project was launched in 1990. Today, genomic analysis -- faster, cheaper, more comprehensive and more reliable than ever before -- is finding its way into clinical practices.

The genetic analysis of solid tumors is widely used to guide the choice of targeted treatments for cancer. The microbiota of everything from human guts to combat wounds is being genetically characterized by the new techniques. Scientists are using them to uncover the fingerprints of ancient plagues and plot the course of prehistoric human migration. And a new generation of massively parallel computer technology has made it possible to sequence a human being's 3 billion base pairs of DNA in a day -- at a cost of less than $5,000.

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Medical sleuths seek patients with mystery diseases, offer new tools

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Lets Play The Sims 3 Perfect Genetics Part 6: Baby #1
Watch as the first child is born. Will it be the Perfect Genetic Heir? My Sims 3 Page: http://mypage.thesims3.com/mypage/becky050890 My Husband #39;s Sims 3 Page...

By: GBabyChallenger

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Lets Play The Sims 3 Perfect Genetics Part 6: Baby #1 - Video

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The Sims 3 | Perfect Genetics Challenge Part 10: Heir?
In this part, you #39;ll have to find out for yourself 😉 dont hurt me ily Backstory: "Once upon a time, the Mighty Player sent a Sim to live in the world where all its creations were living...

By: simplyapril

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The Sims 3 | Perfect Genetics Challenge Part 10: Heir? - Video

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Amy on faith, genetics, and opportunities for discussion
Amy talks about the sometimes complicated intersection between genetics and faith and the value of discussion about genetics and the sticky ethical questions...

By: Personal Genetics Education Project - pgEd.org

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Amy on faith, genetics, and opportunities for discussion - Video

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The Most Tough German Shepherd Puppy/ Puppy Test/ Top Genetics/ Top Breeding
In this video you see one of my puppies that is 6 weeks old getting tested for the first time. What I am looking for when I am testing a puppy is the top genetic traits. Let me explain to you..I...

By: DDRPROGSD1

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The Most Tough German Shepherd Puppy/ Puppy Test/ Top Genetics/ Top Breeding - Video

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ACGT president and co-founder, Barbara Netter, at left, with Savio L.C. Woo, founding chair, ACGT Scientific Advisory Council and professor at the Mt. Sinai School of Medicine.

Barbara Netter and her late husband Edward Netter have been awarded the American Society of Gene and Cell Therapy (ASGCT) Distinguished Service Award for their contributions to the field of genetic and cellar therapy.

As the founders of Alliance for Cancer Gene Therapy (ACGT), the Netters have helped grant almost $25 million in funding to cancer researchers and physicians worldwide. The award was presented during the 17th annual meeting of the ASGCT May 22, in front of some 1,700 scientists and cancer research advocates.

Its an honor to receive this accolade from an organization at the center of our medical community, Ms. Netter said in a statement. Through the work at ACGT and in the cell and gene therapy realm, I have the rare opportunity to see new and optimistic methods of treating cancer unfold. With fantastic breakthroughs in cancer cell and gene therapy occurring more frequently, the future is bright.

Founded in 1998, the ASGCT is a non-profit medical organization dedicated to boosting awareness for gene and cell therapies. These therapies have been shown to be effective against cardiovascular, genetic, infectious and degenerative disorders in addition to cancer. ASGCT publishes the medical journal Molecular Therapy to report on the latest advancements in the field.

The ASGCT Advisory Council selected Barbara and Edward Netter (posthumously) for this award based on the founding of ACGT and the enormous contributions made to advancing cancer gene therapy research, ASGCT advisory council chair Terence R. Flotte said.

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Philanthropists honored for service

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Medicine Based on Your Genetic Makeup
Personalized medicine is upending hierarchies with consumer products like Scanadu, designed to track physiological signals, and 23andMe.com, which provides raw genetic data. Meanwhile, our...

By: The Aspen Institute

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Medicine Based on Your Genetic Makeup - Video

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From Horizontal to Vertical Thinking: Interpreting a Diagnosis
Personalized medicine is upending hierarchies with consumer products like Scanadu, designed to track physiological signals, and 23andMe.com, which provides raw genetic data. Meanwhile, our...

By: The Aspen Institute

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From Horizontal to Vertical Thinking: Interpreting a Diagnosis - Video

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How Same-Day Test Results Could Revolutionize the Health Industry
Personalized medicine is upending hierarchies with consumer products like Scanadu, designed to track physiological signals, and 23andMe.com, which provides raw genetic data. Meanwhile, our...

By: The Aspen Institute

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How Same-Day Test Results Could Revolutionize the Health Industry - Video

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GoldLab Symposium 2010 - Daniel Kracov
Daniel Kracov, J.D., Partner, Arnold and Porter spoke at GLS2010. His presentation: "Personalized Medicine: Facilitating a Partnership with the FDA"

By: GOLDLABCOLORADO

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GoldLab Symposium 2010 - Daniel Kracov - Video

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Bubba sings with Max Renshaw at Lew Reed Spinal Cord Injury Fund Benefit 6-28-14
Thanks to all of Bubba #39;s friends, Sunny Jo Loudin, Rich Edwards Puyallup Eagles volunteers who helped raise funds for victims of spinal cord injuries.

By: Marty Solis

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Bubba sings with Max Renshaw at Lew Reed Spinal Cord Injury Fund Benefit 6-28-14 - Video

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The STAP stem cell saga has reached its bitter conclusion for now.

The authors of two papers published by the journal Nature, which claimed to have produced embryonic-like stem cells from adult cells, have retracted them.

The papers said that almost any adult cell could be coaxed into becoming a stem cell just by dipping them in a bath of acid for 30 minutes. The method held great promise for regenerative medicine because it could be used to create any cell without needing to reprogram genes, or destroy an embryo. The team, led by researchers at the Riken Institute in Kobe, Japan, called this technique stimulustriggered acquisition of pluripotency, or STAP.

But in the months after publication, no independent team was able to replicate the experiments. Instead, the researchers around the world scrutinising the papers exposed many flaws in the papers including manipulated pictures of protein gel panels and mislabelled images. A public flogging of many high profile researchers ensued (see ""How the STAP cell story unfolded", below) and Nature's review process was thrust into the spotlight.

The journal published two statements today from the authors saying they were retracting both papers. The statements include an apology from the authors, in which they admit that multiple errors impair the credibility of the study. They concede that they are unable to say without doubt whether the STAP cell phenomenon is real.

An accompanying Nature editorial says that in practice, it may be impossible for journals to police gel panels routinely "without disproportionate editorial effort". The journal says it is now reviewing its screening practices to increase such checks.

The editorial goes on to say that Nature believes that its editors and referees could not have detected the fatal faults in this work. However, it emerged during the investigation that the papers were first submitted for publication in Science. According to a Nature News blog, Science rejected them after spotting the manipulated images and warning the lead author of the papers, Haruko Obokata, that such composite images need to be marked. Soon after the papers were published, independent bloggers started finding discrepancies in the work.

The Nature editorial states that the episode has highlighted flaws in Nature's procedures. The journal says that it needs to put quality assurance even higher on its agenda to make sure that people's trust in science is not betrayed.

Charles Vacanti at Harvard Medical School, one of the authors on the papers, has said that he is deeply saddened by the whole episode, although he continues to believe that none of the issues cast doubt on the existence of STAP cells themselves. He says he is encouraged that Riken president Ryoji Noyori and other independent labs will now allow sufficient time to try to replicate the experiments.

29 January Two high profile papers are published in Nature claiming that adult cells could be coaxed into becoming stem cells by dipping them in a bath of acid for 30 minutes. The team call these new cells stimulustriggered acquisition of stem cells, or STAP cells.

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Acid-bath stem cell papers are finally retracted

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Stem cell scientists had what first appeared to be an easy win for regenerative medicine when they discovered mesenchymal stem cells several decades ago. These cells, found in the bone marrow, can give rise to bone, fat, and muscle tissue, and have been used in hundreds of clinical trials for tissue repair. Unfortunately, the results of these trials have been underwhelming. One problem is that these stem cells don't stick around in the body long enough to benefit the patient.

But Harvard Stem Cell Institute (HSCI) scientists at Boston Children's Hospital aren't ready to give up. A research team led by Juan Melero-Martin, PhD, recently found that transplanting mesenchymal stem cells along with blood vessel-forming cells naturally found in circulation improves results. This co-transplantation keeps the mesenchymal stem cells alive longer in mice after engraftment, up to a few weeks compared to hours without co-transplantation. This improved survival gives the mesenchymal stem cells sufficient time to display their full regenerative potential, generating new bone or fat tissue in the recipient mouse body. The finding was published in the Proceedings of the National Academy of Sciences (PNAS).

"We are losing mesenchymal stem cells very rapidly when we transplant them into the body, in part, because we are not giving them what they need," said Melero-Martin, an HSCI affiliated faculty member and an assistant professor of surgery at Boston Children's Hospital, Harvard Medical School.

"In the body, these cells sit very close to the capillaries, constantly receiving signals from them, and even though this communication is broken when we isolate mesenchymal stem cells in a laboratory dish, they seem to be ok because we have learned how to feed them," he said. "But when you put the mesenchymal stem cells back into the body, there is a period of time when they will not have this proximity to capillary cells and they start to die; so including these blood vessel-forming cells from the very beginning of a transplantation made a major difference."

Melero-Martin's research has immediate translational implications, as current mesenchymal clinical trials don't follow a co-transplantation procedure. He is already collaborating with surgical colleagues at Boston Children's Hospital to see if his discovery can help improve fat and bone grafts. However, giving patients two different types of cells, as opposed to just one, would require more time and experiments to determine safety and efficacy. Melero-Martin is seeking to identify the specific signals mesenchymal stem cells receive from the blood vessel-forming cells in order to be able to mimic the signals without the cells themselves.

"Even though mesenchymal stem cells have been around for a while, I think there is still a lack of fundamental knowledge about communication between them and other cells in the body," he said. "My lab is interested in going even beyond what we found to try to understand whether these cell-cell signals are different in each tissue of the body, and to learn how to educate both blood vessel-forming and mesenchymal stem cells to co-ordinate tissue specific regenerative responses."

Other Harvard Stem Cell Institute researchers are studying mesenchymal stem cells as bioengineering tools to deliver therapeutics, which is possible because of the cell type's unique ability to not trigger an immune response. Jeffrey Karp, PhD, at Brigham and Women's Hospital has developed several methods to turn these cells into drug-delivery vehicles, so that after transplantation they can, for example, hone in on swollen tissue and secrete anti-inflammatory compounds. And Khalid Shah, PhD, at Massachusetts General Hospital has designed a gel that holds mesenchymal stem cells in place so that they can expose brain tumors to cancer-killing herpes viruses.

"A lot of these applications have no real direct link with mesenchymal stem cells' supposed progenitor cell function," Melero-Martin said. "In our study, we went back to the collective ambition to use these cells as a way to regenerate tissues and we are not in a position to say how that affects other uses that people are proposing."

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The above story is based on materials provided by Harvard University. Note: Materials may be edited for content and length.

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Research team pursues techniques to improve elusive stem cell therapy

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1-Jul-2014

Contact: Krysten Carrera krysten.carrera@nih.gov 301-435-8112 The JAMA Network Journals

Use of a lower intensity bone marrow transplantation method showed promising results among 30 patients (16-65 years of age) with severe sickle cell disease, according to a study in the July 2 issue of JAMA.

Myeloablative (use of high-dose chemotherapy or radiation) allogeneic hematopoietic stem cell transplantation (HSCT; receipt of hematopoietic stem cells "bone marrow" from another individual) is curative for children with severe sickle cell disease, but associated toxicity has made the procedure prohibitive for adults. The development of nonmyeloablative conditioning regimens (use of lower doses of chemotherapy or radiation to prepare the bone marrow to receive new cells) may facilitate safer application of allogeneic HSCT to eligible adults, according to background information in the article.

Matthew M. Hsieh, M.D., of the National Institute of Diabetes and Digestive and Kidney Diseases, Bethesda, Md., and colleagues explored a nonmyeloablative approach in a pilot group of 10 adults with severe sickle cell disease, using a simplified HSCT regimen (with stem cell donation from a immunologically matched sibling), that had few toxic effects, yet all patients continued taking immunosuppression medication. The researchers have since revised the protocol to include an option to stop immunosuppression after 1 year in selected patients (those with donor CD3 engraftment of greater than 50 percent and normalization of hemoglobin). In this report, the authors describe the outcomes for 20 additional patients with severe sickle cell disease, along with updated results from the first 10 patients. All 30 patients (ages 16-65 years) were enrolled in the study from July 2004 to October 2013.

As of October 25, 2013, 29 patients were alive with a median follow-up of 3.4 years, and 26 patients (87 percent) had long-term stable donor engraftment without acute or chronic graft-vs-host disease. Hemoglobin levels improved after HSCT; at 1 year, 25 patients (83 percent) had full donor-type hemoglobin. Fifteen engrafted patients discontinued immunosuppression medication and had no graft-vs-host disease.

The average annual hospitalization rate was 3.2 the year before HSCT, 0.63 the first year after, 0.19 the second year after, and 0.11 the third year after transplant. Eleven patients were taking narcotics long-term at the time of transplant. During the week they were hospitalized and received their HSCT, the average narcotics use per week was 639 mg of intravenous morphine-equivalent dose. The dosage decreased to 140 mg 6 months after the transplant.

There were 38 serious adverse events including pain, infections, abdominal events, and toxic effects from the medication sirolimus.

"In this article, we extend our previous results and show that this HSCT procedure can be applied to older adults, even those with severe comorbid conditions " the authors write. "These data reinforce the low toxicity of this regimen, especially among patients with significant end-organ dysfunction."

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Bone marrow transplantation shows potential for treating adults with sickle cell disease

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UVA joins National Marrow Donor Program giving greater access to cancer treatments by Ishaan Sachdeva | Jun 25 2014 | 06/25/14 10:11pm | Updated 06/30/14 9:56pm

The Emily Couric Cancer Center of the University of Virginia Health System has expanded its access to bone marrow and hematopoietic stem cell transplant donors. Now designated as a National Marrow Donor Program (NMDP), the Health System will have access to the Be The Match Registry, the worlds largest and most diverse bone marrow registry. Implications of this change are significant for patients afflicted with blood cancers like leukemia who obtain treatment through the Health System.

Bone marrow, the soft, spongy tissue within bones like the sternum or the ilium of the pelvis, forms hematopoietic or blood-forming stem cells. These cells, unlike embryonic stem cells, differentiate only into types of blood cells- red blood cells, white blood cells or clotting platelets. Leukemia causes bone marrow to produce abnormal, leukemic white blood cells that divide uncontrollably, forming tumors that deprive cells of oxygen and reduce infection defense. One treatment method is autologous bone marrow transplant, in which patients receive stem cells from their healthy, non cancerous bone marrow.

The idea [of autologous transplants] is that you extract healthier bone marrow from the patient to have a source of stored, non-cancerous bone marrow. You can then treat the patient with higher doses of treatment than you can normally give because the most common limitation to treatment is that treatment will kill off healthy bone marrow you might have, said Thomas P. Loughran Jr., MD, the Universitys Cancer Center director.

Essentially, a patients healthy bone marrow is safeguarded outside their body while aggressive treatment is administered to kill cancerous marrow. Another form of treatment is allogeneic treatment, in which bone marrow is transplanted from a sibling or an unrelated donor.

In an allogeneic transplant, you are also transplanting in a new immune system. The new immune system comes in and recognizes the body as a foreign tissue and starts attacking that tissue. This causes a beneficial graft vs. leukemia effect where this new immune system attacks any residual leukemia, but may also cause a harmful graft versus host disease where normal tissue is also attacked, Loughran said.

The donor and recipient tissue interaction underscores the genetic component of bone marrow transplants from external donors. Despite the curative potential of a bone marrow transplant, a strong genetic match between donor and recipient is crucial to the utility of a transplant.

The ability of any donor to be successful is based on genetics. Its called HLA [human leukocyte antigen] typing. The HLA system has four genes called A, B, C and D, and it turns out that A, B and D are influential. We have half of our genes each from both parents, so we have six of these: 2 A, 2 B and 2 D. The best case is a six out of six match from a brother or sister, but the chances are only 1 in 4, said Loughran. The consequence of low genetic probabilities is a large pool of unrelated donors, like the Be The Match Registry. Through such services, patients have a greater chance of finding an unrelated donor who may provide a successful genetic match.

The coordinating center would identify the place where the donor is living and tell them they are potentially able to donate. In the past, the donor would have bone marrow directly extracted. Now it is almost always from the PBSCT [peripheral blood stem cell transplantation] procedure. The donor takes a growth factor that stimulates growth of the needed hematopoietic stem cells within their peripheral blood circulation. A catheter collects this blood and the stem cells are separated from the blood by a machine, and the blood is returned back to the donor. The collected stem cells are sent to the lab where they are purified and frozen, Loughran said.

Meanwhile, the patient in preparation for the transplant is given the highest dose of chemotherapy that can be tolerated. The donated stem cells are administered to the patient in a way similar to IV fluid.

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Bone marrow transplants can reverse severe sickle cell disease in adults, a small study by government scientists found, echoing results from a similar technique used in children.

The researchers and others say the findings show that age need not be a barrier and that the technique could change practice for some adult patients when standard treatment fails.

The transplant worked in 26 of 30 adults, and 15 of them were able to stop taking drugs that prevent rejection one year later.

"We're very pleased," said Dr. John Tisdale, the study's senior author and a senior investigator at the National Institutes of Health. "This is what we hoped for."

Sickle cell disease is a genetic condition that damages oxygen-carrying hemoglobin in red blood cells that then form sickle shapes that can block blood flow through veins. It can cause anemia, pain and organ damage. The disease affects about 100,000 Americans and millions worldwide.

The treatment is a modified version of bone marrow transplants that have worked in kids. Donors are a brother or sister whose stem cell-rich bone marrow is a good match for the patient.

Tisdale said doctors have avoided trying standard transplants in adults with severe sickle cell disease because the treatment is so toxic. Children can often tolerate it because the disease typically hasn't taken as big a toll on their bodies, he said.

The disease is debilitating and often life-shortening. Patients die on average in their 40s, Tisdale said. That's one reason why the researchers decided to try the transplants in adults, hoping the technique could extend their lives.

The treatment involves using chemotherapy and radiation to destroy bone marrow before replacing it with healthy donor marrow cells. In children, bone marrow is completely wiped out. In the adult study, the researchers only partially destroyed the bone marrow, requiring less donor marrow. That marrow's healthy blood cells outlast sickle cells and eventually replace them.

Results from the adult study, involving patients aged 29 on average, were published Tuesday in the Journal of the American Medical Association.

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Study finds new treatment for adult sickle cell disease

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The treatment could edge out joint replacement procedures to a large extent.

Hyderabad, June 30:

A team of doctors from a city hospital have harvested stem cells of a person using bone marrow from the pelvis area to replace some dead tissues in the hip. By doing this, they saved the patient from undergoing a hip replacement.

The Apollo Health City team, headed by orthopaedic specialist Paripati Sharat Kumar, diagnosed a 39-year-old women suffering from Avascular Necrosis. Her condition would require undergoing a replacement of hips.

After assessing her condition, the team has decided to go for the autologous stem cell procedure (where donor and the receiver is the same person) to save both the hip joints.

The minimally invasive procedure involved taking bone marrow aspirate from the patients pelvis. Stem cells were harvested from the aspirate through a process that takes about 15 minutes. Stems cells were planted in the area of damage under fluoroscopy control following core decompression, Kumar said in a statement on Monday.

He feels that the autologous stem cell treatment could edge out joint replacement procedures to a large extent in the days to come. The scope of this procedure in orthopaedics and sports medicine is enormous. This could be extended to indications including osteoarthritis of knee, shoulder, hip, elbows, ankle and spine, he said.

(This article was published on June 30, 2014)

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Autologous stem cell treatment could be the road ahead

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