UCLA Stem Cell Researcher Pioneers Gene Therapy Cure for Children with "Bubble Baby" Disease

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Newswise UCLA stem cell researchers have pioneered a stem cell gene therapy cure for children born with adenosine deaminase (ADA)-deficient severe combined immunodeficiency (SCID), often called Bubble Baby disease, a life-threatening condition that if left untreated can be fatal within the first year of life.

The groundbreaking treatment was developed by renowned stem cell researcher and UCLA Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research member Dr. Donald Kohn, whose breakthrough was developed over three decades of research to create a gene therapy that safely restores immune systems in children with ADA-deficient SCID using the patients own cells with no side effects.

To date, 18 children with SCID have been cured of the disease after receiving the stem cell gene therapy in clinical trials at UCLA and the National Institutes of Health.

All of the children with SCID that I have treated in these stem cell clinical trials would have died in a year or less without this gene therapy, instead they are all thriving with fully functioning immune systems said Kohn, a professor of pediatrics and of microbiology, immunology and molecular genetics in Life Sciences.

To protect children born with SCID they are kept in isolation, in controlled environments because without an immune system they are extremely vulnerable to illness and infection that could be lethal.

Other current options for treating ADA-deficient SCID are not always optimal or feasible for many children, said Kohn. We can now, for the first time, offer these children and their families a cure, and the chance to live a full healthy life.

Defeating ADA-Deficient SCID: A Game-Changing Approach

Children born with SCID, an inherited immunodeficiency, are generally diagnosed at about six months. They are extremely vulnerable to infectious diseases, and in a child with ADA-deficient SCID even the common cold can prove fatal. The disease causes cells to not create an enzyme called ADA, which is critical for production of the healthy white blood cells that drive a normal, fully-functioning immune system. About 15 percent of all SCID patients are ADA-deficient.

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UCLA Stem Cell Researcher Pioneers Gene Therapy Cure for Children with "Bubble Baby" Disease

Bubble Baby: Gene Therapy Cured 2-year-old Girl Of Deadly SCID At UCLA

The gene therapy that has seemingly cured a 2-year-old girl could be used to treat other diseases, like Sickle Cell Anemia.

UCLA DR. DONALD KHON has perfected the treatment that has now affected the lives of over a dozen families, including the parents of Evangelina, who was born with the deadly SCID. The immune system disease is known by its common name, Bubble Baby disease, because the children have to live in bubbles, since their immune systems do not function. Most die within the first year of life, unless they can get and survive a bone marrow transplant.

Evangelina's twin sister, Annabella, was not a bone marrow transplant. So Dr Kohn included the sick twin in his study, transfering the patient's own bone marrow to the child. At two, her immune system is working well.

It's a difficult and time consuming process, but it seems to work. The same procedure could work with other blood diseases that are curable with bone marrow transfusions. That includes sickle cell anemia, which will be the focus of the next trial.

The implications are huge, as there are 30 or 40 diseases where the treatment could work.

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Bubble Baby: Gene Therapy Cured 2-year-old Girl Of Deadly SCID At UCLA

UCLA Doctors Hail Potential Cure For Bubble Baby Syndrome

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WESTWOOD (CBSLA.com) Doctors say a groundbreaking stem cell therapy treatment out of UCLA may have cured Bubble Baby syndrome once and for all.

KNX 1070s Brian Ping reports Dr. Donald Kohn has perfected a gene therapy that has now cured 18 children born without an immune system, known as ADA-deficient severe combined immunodeficiency (SCID).

Only weeks after giving birth to fraternal twins in 2012, Alysia and Christian Padilla-Vaccaro found out their daughter Evangelinas immune system was so deficient that she could have no exposure to the outside world.

After enrolling their daughter in Dr. Donald Kohns revolutionary stem cell gene therapy treatment which was nearly three decades in the making doctors extracted stem cells from the bone marrow in Evangelinas hip, then used a modified mouse virus to correct her faulty gene before replacing the marrow.

You hear the words mouse virus and you want to run the other way, said mom Alysia. But they modify it so that its teaching it to do something that they want it to do, which is put something in there that was missing.

Evangelinas new immune system developed without side effects and she is now living a healthy normal life.

Her mother Alysia said while the process was difficult for any mom to go through, it was all worth it.

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UCLA Doctors Hail Potential Cure For Bubble Baby Syndrome

China Once Again Boasts The World's Fastest Supercomputer

Every six months, the Top500 Organization ranks the five hundred fastest supercomputers in the world. And for the fourth consecutive list, Chinas Tianhe-2 is on top, performing at 33.86 petaflop/s according to the organizations benchmarks. Thats nearly twice as fast as the number two computer, Crays Titan at the Oak Ridge National Laboratory.

The Tianhe-2 resides at Chinas National University of Defense Technology. It cost approximately $390 million to build and is comprised of thousands of Intel Intel Xeon E5-2692v2 12C 2.2GHz processors. It runs a version of Linux that was developed by the NUDT.

The rest of the top ten was the same as last years list, with one exception a Cray supercomputer thats installed at an undisclosed U.S. government site. Interestingly enough, this is the second list in a row where the only newcomer to the top 10 list was a Cray supercomputer installed at an undisclosed U.S. government site.

Cray has actually seen a nice bump on this top 500 list. It has 62 total systems on the list, 11 more than it did in June. HP has the most supercomputers on the list, with 179. IBM has the second-most with 153 systems. However, both HP and IBM have fewer systems on this list than they did on the last one.

When it comes to processors, though, Intel dominates. 85.8% of the supercomputers on the list use Intel processors and 25 use Intels Xeon Phi co-processors, including the Tianhe-2.

You can see the full list of the 500 fastest supercomputers here.

Follow me onTwitterorFacebook. Read my Forbes bloghere.

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China Once Again Boasts The World's Fastest Supercomputer

HBO Remaking Westworld, The Best Killer Theme Park Robot Movie Yet

Well, it's official. The 1973 sci-fi movie Westworld is becoming an HBO series. "What's Westworld?" everybody under the age of 50 is probably asking themselves right about now. Only the greatest movie about killer theme park robots ever made.

The original Westworld was both written and directed by Michael Crichton of Jurassic Park fame (and tiny dick rule infamy). Crichton passed away in 2008. The film centered around a common theme from 1970s futurism: That one day we'd all be able to enact our wildest fantasies in a completely immersive, simulated environment.

The original film featured three environments: West World, Medieval World, and Roman World. But they all turn out to be a little too immersive, because the parks soon become ground zero for the robot uprising. The gun-slinging robots of West World go off-script and start hunting our mustachioed protagonist through the various lands. It's all fun and games in the Wild West simulation until the men in white lab coats lose control of their human-like creations.

The new series has some huge names attached, including a starring role for Anthony Hopkins, with J.J. Abrams and Bryan Burk producing through their company Bad Robot Productions. According to the Hollywood Reporter the robots will be played by James Marsden, Evan Rachel Wood and Thandie Newton.

Jonathan Nolan, the younger brother of Christopher Nolan (who directed Interstellar, Inception, and the latest Batman trilogy), has directed the pilot. The series was reportedly shot this past July and August. Actors Ed Harris, Miranda Otto and Jeffrey Wright are also featured in the series. Here's the Vine HBO used to announce it, because that is how we announce things these days apparently?

It's too early to tell, but the coolest thing HBO could do (at least in this humble blogger's opinion) would be to set the "modern day" scenes in the 1970s. The series doesn't have an air date yet. All we know is that we should look for it sometime in 2015.

Image: Screenshot from the original Westworld (1973)

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HBO Remaking Westworld, The Best Killer Theme Park Robot Movie Yet

Twenty-Five Years of Freedom Through the Eyes of Czech and American Envoys – Video


Twenty-Five Years of Freedom Through the Eyes of Czech and American Envoys
Welcome Remarks The Hon. Paula Dobriansky, Senior Fellow, JFK Belfer Center for Science and International Affairs, Harvard University Keynote Address H.E. Jan Hamek, Speaker of the Chamber.

By: AtlanticCouncil

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Twenty-Five Years of Freedom Through the Eyes of Czech and American Envoys - Video

Freedom to Marry launches statewide gay marriage campaign in Texas

AUSTIN Freedom to Marry, one of the most prominent same-sex marriage advocacy organizations in the nation, is launching a campaign in Texas to amplify its message ahead of an important appeals court hearing next year.

The organization will announce the "Texas for Marriage" campaign and unveil its accompanying website on Tuesday. It plans to spend roughly $200,000 on the effort, and will enlist Ward Curtin, three-time deputy campaign manager to Houston Mayor Annise Parker, and former George W. Bush adviser Mark McKinnon in its efforts.

"We want to make sure that the court hears that America is ready for the freedom to marry, and that includes Texas," Freedom to Marry President and CEO Evan Wolfson, considered by many to be the father of the modern gay marriage movement, told the Chronicle on Monday. "Obviously I would love to be everywhere, but we have to look where we can have a real impact."

The campaign will feature statewide TV ads, town hall meetings and efforts to bring together young conservatives ahead of the January hearing in the 5th Circuit Court challenging Texas' gay marriage ban. In February, San Antonio-based U.S. District Judge Orlando Garcia ruled the ban unconstitutional because it violated gay couples' 14th Amendment rights to due process and equal protection.

Attorney General Greg Abbott, now the governor-elect, appealed the ruling to the New Orleans-based 5th Circuit Court. While the court initially indicated it would expedite a hearing, oral arguments were eventually set for the week of Jan. 5, 2015.

When asked how the enhanced effort would work in Texas, a strongly red state that just elected a full slate of new Republican leaders, Wolfson said candidates chose not to focus heavily on gay marriage this election cycle because opposition to the movement no longer sells like it used to.

"Whatever this election was about, it wasn't about the freedom to marry and it wasn't about gay people," said Wolfson. McKinnon, already the Texas chair for Freedom to Marry, said the issue was "a blip" on the election scene "because Texans from all walks of life, from big cities to small towns, believe strongly in freedom and family. Supporting gay couples marrying is squarely in line with these Texas values."

Freedom to Marry has launched similar efforts in about two dozen other states before similarly important court hearings there. Gay marriage is now legal in 33 states and the District of Columbia. While a recent circuit court decision upholding same-sex marriage bans in four states means the U.S. Supreme Court will likely review the issue, the Texas case will continue undeterred during that time.

Lauren McGaughy is a reporter in the Houston Chronicle's Austin bureau. She can be reached at lauren.mcgaughy@chron.com or on Twitter @lmcgaughy.

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Freedom to Marry launches statewide gay marriage campaign in Texas

Freedom heads home from regions, Potomac takes another step

Tue., Nov. 18 - Although Prince William County is largely made up of Group 6A high schools, the two Group 5A schools on the eastern end actually benefit to a certain extend from playing the bigger schools in varsity football. If nothing else, that experience surely didn't hurt Potomac.

By Jason S. Rufner and Dan Roem

Gainesville Times

Times Photo/Marny Malin Potomac sophomore running back Mike Hawkins takes the handoff and contributes a long gain. The Panthers defeated visiting Patrick Henry (Ashland) 31-28 on Friday night in Dumfries during the first round of the Group 5A North Region tournament. The Panthers head to Fredericksburg this Friday for Round 2 against another group of Panthers in undefeated host Massaponax.

If nothing else, that experience surely didn't hurt Potomac.

The Panthers advanced to the second region of the Group 5A North Region tournament on Friday with a close 31-28 win at home in Dumfries over Patrick Henry (Ashland) as a last-second field goal by the visiting team went awry, securing the victory for Potomac.

Potomac senior running back Jaren Johnson pounded in a trio of touchdowns and one more came from sophomore leading rusher Mike Hawkins to help the Panthers secure a trip to Fredericksburg this week to take on undefeated Massaponax in the second round.

Meanwhile in Ashburn on Nov. 14, Freedom faced a much more difficult task.

Freedom When they were down 35 points, playing on the road and in the cold versus a team with a better record and a more impressive pedigree, the players of the Freedom High School football program showed something to their supporters and to themselves.

That's when the Eagles (3-8) ran off 16 straight points and 22 of the last 28, refusing to go away quietly as they battled third-seeded Stone Bridge (9-2) to a 41-22 loss in the 5A North Region's first round.

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Freedom heads home from regions, Potomac takes another step

Bill Limiting NSA Surveillance Practices Fails In Senate

The USA Freedom Act had the support of not only the White House and Democratic Sen. Patrick Leahy but also that of Republican Sen. Ted Cruz, but the bid to reform the NSA failed late Tuesday after it didn't receive enough votes to cut off debate.

After a 58-42 vote, the measure had the support of the majority but it didn't get the 60 votes necessary to break a Republican filibuster. It was something of an odd end for a bill that had been approved by the Republican-controlled House back in May.

The USA Freedom Act sought to amend the Foreign Intelligence Surveillance Act of 1978, to "rein in the dragnet collection of data by the National Security Agency (NSA) and other government agencies, increase transparency of the Foreign Intelligence Surveillance Court," as its chief House sponsor, Rep. Jim Sensenbrenner, R-Wis., says in a summary on his website.

The Hill reports:

"The defeat of the legislation to stop the government's bulk data collection program will put off legislation responding to Edward Snowden's leaks about the controversial programs until next year."

Leahy, the Judiciary Committee chairman, said that in blocking the bill he had sponsored, his Republican colleagues "failed to answer the call of the American people who elected them, and all of us, to stand up and to work across the aisle. Once again, they reverted to scare tactics rather than to working productively to protect Americans' basic privacy rights and our national security."

Earlier Tuesday, Senate Minority Leader Mitch McConnell, R-Ky., took a stand against the bill. Citing the U.S. fight against extremist terrorist group ISIS, he said, "At a minimum, we shouldn't be doing anything to make the situation worse. Yet, that's just what this bill would do."

Another "No" vote came from Sen. Rand Paul, who said that he voted against the bill because "it currently extends key provisions of the Patriot Act until 2017."

Digital rights group the Electronic Frontier Foundation said of the measure's failure, "We are disappointed that the Senate has failed to advance the USA Freedom Act, a good start for bipartisan surveillance reform that should have passed the Senate."

Leahy vowed to keep working to pass the bill, which sprang out of what the Two-Way called "an unusual alliance involving a prominent House Republican and a veteran Senate Democrat" when it was first introduced in October of 2013.

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Bill Limiting NSA Surveillance Practices Fails In Senate

FTA win for Freedom Foods

CHINESE giant New Hope will invest up to $500 million in Australian dairy farms and processing plants as part of a deal with ASX-listed Freedom Foods in what could be the first of many deals fuelled by the historic China-Australia free-trade agreement.

On Tuesday, Freedom Foods and the Perich Group said they had signed a memorandum of understanding with New Hope, China's biggest private agricultural company.

"With increasing demand from markets in China and south-east Asia for high-quality, value-added product, Australia, through companies such as Freedom Foods and Perich Group, is well placed to play an important role in the development of this supply chain," Freedom Foods managing director Rory Macleod said.

To secure milk supply, New Hope has established an investment fund of up to $500 million to invest in dairy farms and dairy processing.

Freedom Foods said it has built on its supply arrangements with New Hope for UHT [long-life] milk and it is considering collaboration in other strategic branded dairy operations in Australia and overseas to take Freedom Foods products to China.

Perich Group, New Hope and other investors will contribute most of the equity for farm investment, which will commence in 2015.

News of the deal comes on the back of Monday's free-trade agreement with China that gave Australia unprecedented access to the Chinese market.

Dairy producers fared particularly well, with all tariffs (up to 20 per cent) on Australian dairy eliminated in four to 11 years, depending on the product.

"We couldn't wish for a better deal. The agreement will do for us what it did for New Zealand. New Zealand [dairy] trade with China exploded after they signed their deal [in 2008]," Murray Goulburn managing director Gary Helou said.

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FTA win for Freedom Foods

This freedom fighter's struggle for his right is now a movie

It took Gour Hari Das three decades to wrangle out a certificate recognising his work as a freedom fighter. His struggle is now the subject of a film

In the dead of night during the mid-1940s, Gour Hari Das would customarily set out of his house and sprint alongside passing trains, relying on their beacons to guide his 14-year-old feet.

He was joined in this exercise by others roughly his age, all part of a group called the vanar sena that nimbly ferried covert messages and publications of the freedom movement.

Many years later, the same pair of legs tirelessly carried him up and down the stairs of government offices in Mumbai. The communication he sought this time was a certificate recognising his work as a freedom fighter - it took three full decades to arrive.

The prolonged personal tryst for acknowledgment got him more attention than even his participation in the freedom struggle. His travails form the subject of an upcoming film Gour Hari Dastaan - The Freedom File.

After reading a newspaper report, director Ananth Mahadevan was struck by the irony in Das' experience.

"Here was a man fighting for identity in a country that he helped to free," he observes. Mahadevan traced Das to his current home in suburban Mumbai and convinced him to share his story. The film, with screenplay by journalist and poet C P Surendran, has been shortlisted for screenings at three Indian film festivals so far.

In his first meeting, Surendran says he did not find Das terribly impressive but noted an understated grit about him. Mahadevan too was nonplussed during an initial interaction.

"Leave alone three decades, this man did not look like he could have fought for three days."

His largely self-effacing persona led the film's makers to play on silences.

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This freedom fighter's struggle for his right is now a movie