Human Genome Sequence Editing the arrangement of a sequence of these letters corrected a genetic mutation in stem cells, a breakthrough combination of stem cell therapy and genetic modification. Wikimedia Commons, October 13, 2011, by By Rebecca Boyle  —   For the first time, scientists using a combination of gene-editing technologies have corrected mutations in a patient’s own induced stem cells. The breakthrough could pave the way toward reprogramming a person’s own cells to cure genetic diseases, rather than using transplanted organs and drug therapies.

Researchers led by two institutions in the UK corrected a mutation in cells derived from a patient with a metabolic liver disease.

Stem cells — embryonic ones and induced pluripotent ones — can turn into any type of cell, so they hold promise for treating a host of disorders. They can come with unwanted mutations, however. For one thing, induced pluripotent stem cells (iPS cells) would contain the same genetic defects as the rest of a patient’s body, so you’d have to remove those defects before you could treat a person with his or her own cells. But this removal can be imprecise; current editing methods can cause misplaced alleles or residual genetic sequences, which can lead to formation of cancer or other unwanted side effects. And recent breakthroughs in gene editing methods have not involved stem cells.

To work on stem cells, you would need a very careful editing method to snip out incorrect gene sequences in the stem cells and replace them with the correct kind. And that’s what these researchers have done.

Scientists at the Wellcome Trust Sanger Institute and the University of Cambridge worked with a mutation in a gene responsible for coding a specific protein in the liver. It’s a common mutation, found in about 1 of every 2,000 people of European descent, and it’s also a fairly simple mutation, with just one transposition of letters.

The team took skin cells from a patient and turned them into iPS cells. Then they used genetic scissors, zinc-finger nucleases, to snip the genetic sequence at the site of the mutation. They also used a piggyBAC transposon, which cuts and pastes genetic information. In this way, they were able to correct both alleles involved in the mutation of this liver gene.

Once the stem cells were corrected, the team induced them to become liver cells. These were transplanted in mice with the liver disorder. The cells restored the liver’s proper function, and were still working properly after six weeks, the researchers said.

This was an incredibly difficult maneuver, and it’s the first time anyone has been able to pull it off, the researchers say. Researcher David Lomas told the BBC it was “ridiculously hard.”

But it’s proof, at least in principle, that well-edited genetic sequences in induced human stem cells can provide new cells for a variety of clinical treatments. The paper was published in today’s issue of Nature.

Peyton Manning Manning on the sidelines, where he’s likely to remain for the 2011 season. Wikimedia Commons, Sept/Oct 2011, by Rebecca Boyle  —  As fantasy teams across the nation crumbled in Week 1, football’s greatest current quarterback (yeah, that’s right) sat down for the first time in his NFL career, following a third procedure on his neck that may have ended his season. But before that, Peyton Manning apparently flew to Europe for an experimental stem cell treatment, according to Fox Sports.


Manning had surgery in May to correct a bulging disc in his neck, but it didn’t solve his problems. Prior to a third surgery in September, he took a private plane to an unknown hospital in an unnamed country, Fox’s Jay Glazer reported in a pregame show Sunday (clip below). The procedure is not available in the United States.

In an email, a Colts spokesman said the team’s only comment was head coach Jim Caldwell’s “no comment” during a Monday press conference. Caldwell said the team would not discuss Manning’s medical issues.

That means details are scarce, but Fox’s report said this was not a procedure involving embryonic stem cells. It’s likely Manning underwent a procedure involving induced pluripotent stem cells, or iPS cells, which can be reprogrammed to become any type of cell. Glazer said he was informed that doctors cultured some of Manning’s own fat cells and injected them into his neck, where they would ideally help regenerate damaged tissue. Researchers showed back in 2009 that fat cells could easily be turned into iPS cells, and do so much more quickly than the other common iPS cell progenitor, skin cells.

In any event, the fat-stem-cell procedure was insufficient, leading to Manning’s third neck surgery Sept. 8. The anterior fusion of two neck vertebrae was a success, but the future Hall-of-Famer will be sidelined for two to three months, likely missing the entire regular season.

Sports analysts said the stem cell procedure was evidence that Manning really wants to get back on the field this year (he’s missed, like, one snap in his career prior to last week). It could also mean that his injury was more serious than some people thought. But let us pose another theory: It’s also evidence American stem cell therapy is still lagging. Researchers at Stanford started working with fat-derived iPS cells in 2009 — so why is a marquee NFL quarterback flying overseas for this therapy?

So-called stem cell tourism is nothing new, of course; risk-takers of wealthy and/or desperate stripes have been doing it for some time. And the reasons for American reluctance regarding stem cell procedures are many and varied. But when high-profile people like Peyton Manning start leaving this country for treatment that could be done at home, it’s a good opportunity to ask bigger questions than the quarterback’s personal motivations.

<a href=';mkt=en-us&#038;from=sp^foxsports_en-us_videocentral&#038;src=FLPl:embed::uuids' target='_new' title='Glazer&#39;s Edge: Peyton&#39;s Stem Cells' >Video: Glazer&#39;s Edge: Peyton&#39;s Stem Cells</a>

Human Embryonic Stem Cells PLoS Biology via Wikimedia Commons  —  In 2010, doctors drilled a hole into a Scottish truck driver’s head and injected his brain with 2 million stem cells, in the first-ever regulated human trial for stem cell stroke treatment.

Doctors at Glasgow’s Southern General Hospital will conduct periodic MRI scans to look for repairs or changes in areas of the patient’s brain damaged by stroke. The trial, called Pilot Investigation of Stem Cells in Stroke (PISCES), is designed to check the procedure’s safety, but any signs of physical improvement would be a major leap in neural medicine.

Before the surgery, researchers at UK company ReNeuron grew the stem cells into neural stem cells. British media said the company obtained the cells from a donated 12-week-old human fetus from the U.S. (An embryo becomes a fetus about eight weeks after fertilization.)

The procedure was initially approved last year.

Keith Muir of the University of Glasgow, the lead researcher on the trial, said some of the injected neural stem cells would grow into neurons. But they could prove even more versatile — earlier studies in rats showed that the stem cells triggered a wide variety of cell development, including new brain blood vessels.

During the next year, as many as 12 other patients will get progressively higher doses of stem cell injections, reaching as many as 20 million cells, according to Muir.

Stem cells are valuable because they can become any type of cell in the body, but are controversial because embryonic stem cells require the destruction of human embryos. The National Institutes of Health is embroiled in a legal wrangle over whether its federally funded researchers can study human embryonic stem cells; for now, research is progressing, but the future is in the hands of the courts.

Doctors in Russia, China and other nations offer stem cell therapy to patients with a host of maladies, but the treatments are often poorly regulated or not at all.

Despite the controversies, the Scottish trial is the second notable embryonic stem cell implantation procedure in as many months. After years of delays, the first American embryonic stem cell therapy for spinal injuries started last month, when a spinal patient received a stem cell injection into the spinal cord. Again, the study’s goal is to prove the treatment is safe, but doctors hope the paralyzed patient will see some physical improvement. As many as nine other patients may join that study.

In August, an Iraq war veteran became the first recipient of an adult stem cell implant, also to treat a spinal injury.

Stem Cell Exerts Pressure On Microscopic Posts, Reveals Its Own Future Jianping Fu, by Lana Birbrair — Like a child, a stem cell can grow up to be just about anything. Eventually it picks a job, however, during a process called differentiation. Scientists can influence, if not always control, the outcome by applying compounds called growth factors. Now Jianping Fu, a biomedical and mechanical engineer at the University of Michigan, and his colleagues have discovered that the force exerted by a stem cell onto a surface is an important part of in both predicting and altering what type of cell it will develop into.

Fu placed a stem cell on a scaffold of 13-micron-long silicone posts and found that the amount of force the cell exerted on those posts indicated it would eventually become a fat cell. But he also found that when he stiffened the surface by shortening the posts, it caused the same line of stem cells to turn into bone. Knowing how to predict and manipulate the fate of stem cells will make therapies based on them—for spinal-injury repair, bone grafts, skin transplants—easier to develop.

ORNL’s Petascale Jaguar Supercomputer The petascale Jaguar is the world’s fastest computer, but DARPA wants to take computing to the next level.



The new souped-up supercomputer will be renamed Titan, October 13, 2011, by Clay Dillow  —  Back in June when the latest edition of TOP500 dropped (TOP500 lists the world’s top supercomputers), Japan’s K Computer leapt ahead of China’s Tianhe-1A supercomputer to become the biggest, baddest computing platform on the planet. But after more than a year of slipping down the ranks as its competitors across the Pacific surged ahead, Oak Ridge National Labs Jaguar supercomputer is poised to become the fastest computer in the world once more.

Cray Inc., maker of the XT5-HE supercomputer at the heart of Jaguar, says it has inked a deal with ORNL to overhaul the Department of Energy computer with thousand of graphics processors from NVIDIA as well as chips from Advanced Micro Devices. The tune-up will push the peak performance beyond K Computer’s current capacity, putting ORNL’s computer at the top of the TOP500 for the first time since last year. After the overhaul, Jaguar will be renamed Titan.

Graphics processing units, or GPUs, are chips that specialize in screen graphics (they’re what manage all the individual screen pixels when you play your Xbox, for instance). As such, they are also quite good at processing a range of different tasks simultaneously, a process known as parallel computing. Parallel computing speeds up complex computing processes, and the NVIDIA Tesla GPUs are critical to enabling the 20-petaflop peak performance envisioned for Titan. The powerful AMD chips will process data in sequence, more like the average PC.

Even as recently as last June when Jaguar topped the TOP500, it had already technically been dethroned, as its peak performance had already been theoretically surpassed by a Chinese system called Nebulae. Titan should in short order re-establish ORNL’s supercomputing lab as the world’s elite–for the time being, at least.