Adult Stem Cell Breakthrough Will Transform Medicine …

Dr. Marc Darrow, M.D., J.D.

(CNSNews.com) A scientific breakthrough that enables researchers to create adult stem cells much faster and easier will radically transform the way medicine is practiced, predicts Dr. Marc Darrow,assistant clinical professor of medicine at the University of California/Los Angeles (UCLA) School of Medicine.

It will be the standard of care, said Darrow, who teaches regenerative techniques utilizing platelets and adult stem cells to medical residents at UCLA, and who has been using the same techniques to treat patients with joint, tendon, ligament and muscle injuries in his own LAclinic.

Darrow explained that in the past, creating stem cell lines was a very tedious procedure which required using a pipette to take nuclear material from one cell to put into another.

But an article published January 29th in the peer-reviewed journalNature describes a new technique for creating undifferentiatedadult stem cells by immersing blood cells in an acid bath for half an hour.

BiologistHaruko Obokata, a stem cell researcher from Japans RIKEN Center of Developmental Biology, then injected the acid-stressed, florescently-tagged blood cells into a mouse embryo, where they created entire organs including a beatingheart.

Haruko Obokata (RIKEN Center for Developmental Biology)

Its amazing. I would have never thought external stress could have this effect, said study co-author YoshikiSasai. (See STAP cells.pdf)

The generation of these cells is essentially Mother Natures way of responding to injury, added co-author Charles Vacanti, director of the Laboratory for Tissue Engineering and Regenerative Medicine at the Harvard-affiliated Brigham Womens Hospital.

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Adult Stem Cell Breakthrough Will Transform Medicine ...

Genome editing goes hi-fi: Technique in stem cells to …

13 hours ago Beating-heart cells derived from iPS cells are shown. A single DNA base-pair of the PRKAG2 gene was edited using the method developed by Drs. Miyaoka and Conklin. Credit: Luke Judge/Gladstone Institutes

Sometimes biology is cruel. Sometimes simply a one-letter change in the human genetic code is the difference between health and a deadly disease. But even though doctors and scientists have long studied disorders caused by these tiny changes, replicating them to study in human stem cells has proven challenging. But now, scientists at the Gladstone Institutes have found a way to efficiently edit the human genome one letter at a timenot only boosting researchers' ability to model human disease, but also paving the way for therapies that cure disease by fixing these so-called 'bugs' in a patient's genetic code.

Led by Gladstone Investigator Bruce Conklin, MD, the research team describes in the latest issue of Nature Methods how they have solved one of science and medicine's most pressing problems: how to efficiently and accurately capture rare genetic mutations that cause diseaseas well as how to fix them. This pioneering technique highlights the type of out-of-the-box thinking that is often critical for scientific success.

"Advances in human genetics have led to the discovery of hundreds of genetic changes linked to disease, but until now we've lacked an efficient means of studying them," explained Dr. Conklin. "To meet this challenge, we must have the capability to engineer the human genome, one letter at a time, with tools that are efficient, robust and accurate. And the method that we outline in our study does just that."

One of the major challenges preventing researchers from efficiently generating and studying these genetic diseases is that they can exist at frequencies as low as 1%, making the task of finding and studying them labor-intensive.

"For our method to work, we needed to find a way to efficiently identify a single mutation among hundreds of normal, healthy cells," explained Gladstone Research Scientist Yuichiro Miyaoka, PhD, the paper's lead author. "So we designed a special fluorescent probe that would distinguish the mutated sequence from the original sequences. We were then able to sort through both sets of sequences and detect mutant cellseven when they made up as little one in every thousand cells. This is a level of sensitivity more than one hundred times greater than traditional methods."

The team then applied these new methods to induced pluripotent stem cells, or iPS cells. These cells, derived from the skin cells of human patients, have the same genetic makeupincluding any potential disease-causing mutationsas the patient. In this case, the research team first used a highly advanced gene-editing technique called TALENs to introduce a specific mutation into the genome. Some gene-editing techniques, while effective at modifying the genetic code, involve the use of genetic markers that then leave a 'scar' on the newly edited genome. These scars can then affect subsequent generations of cells, complicating future analysis. Athough TALENs, and other similarly advanced tools, are able to make a clean, scarless single letter edits, these edits are very rare, so that new technique from the Conklin lab is needed.

"Our method provides a novel way to capture and amplify specific mutations that are normally exceedingly rare," said Dr. Conklin. "Our high-efficiency, high-fidelity method could very well be the basis for the next phase of human genetics research."

"Now that powerful gene-editing tools, such as TALENs, are readily available, the next step is to streamline their implementation into stem cell research," said Dirk Hockemeyer, PhD, assistant professor of molecular and cellular biology at the University of California, Berkeley, who was not involved in this study. "This process will be greatly facilitated by the method described by Dr. Conklin and colleagues."

"Some of the most devastating diseases we face are caused by the tiniest of genetic changes," added Dr. Conklin. "But we are hopeful that our technique, by treating the human genome like lines of computer code, could one day be used to reverse these harmful mutations, and essentially repair the damaged code."

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Genome editing goes hi-fi: Technique in stem cells to ...

Genome editing goes hi-fi: Technique in stem cells to boost scientists' ability to study genetic disease

13 hours ago Beating-heart cells derived from iPS cells are shown. A single DNA base-pair of the PRKAG2 gene was edited using the method developed by Drs. Miyaoka and Conklin. Credit: Luke Judge/Gladstone Institutes

Sometimes biology is cruel. Sometimes simply a one-letter change in the human genetic code is the difference between health and a deadly disease. But even though doctors and scientists have long studied disorders caused by these tiny changes, replicating them to study in human stem cells has proven challenging. But now, scientists at the Gladstone Institutes have found a way to efficiently edit the human genome one letter at a timenot only boosting researchers' ability to model human disease, but also paving the way for therapies that cure disease by fixing these so-called 'bugs' in a patient's genetic code.

Led by Gladstone Investigator Bruce Conklin, MD, the research team describes in the latest issue of Nature Methods how they have solved one of science and medicine's most pressing problems: how to efficiently and accurately capture rare genetic mutations that cause diseaseas well as how to fix them. This pioneering technique highlights the type of out-of-the-box thinking that is often critical for scientific success.

"Advances in human genetics have led to the discovery of hundreds of genetic changes linked to disease, but until now we've lacked an efficient means of studying them," explained Dr. Conklin. "To meet this challenge, we must have the capability to engineer the human genome, one letter at a time, with tools that are efficient, robust and accurate. And the method that we outline in our study does just that."

One of the major challenges preventing researchers from efficiently generating and studying these genetic diseases is that they can exist at frequencies as low as 1%, making the task of finding and studying them labor-intensive.

"For our method to work, we needed to find a way to efficiently identify a single mutation among hundreds of normal, healthy cells," explained Gladstone Research Scientist Yuichiro Miyaoka, PhD, the paper's lead author. "So we designed a special fluorescent probe that would distinguish the mutated sequence from the original sequences. We were then able to sort through both sets of sequences and detect mutant cellseven when they made up as little one in every thousand cells. This is a level of sensitivity more than one hundred times greater than traditional methods."

The team then applied these new methods to induced pluripotent stem cells, or iPS cells. These cells, derived from the skin cells of human patients, have the same genetic makeupincluding any potential disease-causing mutationsas the patient. In this case, the research team first used a highly advanced gene-editing technique called TALENs to introduce a specific mutation into the genome. Some gene-editing techniques, while effective at modifying the genetic code, involve the use of genetic markers that then leave a 'scar' on the newly edited genome. These scars can then affect subsequent generations of cells, complicating future analysis. Athough TALENs, and other similarly advanced tools, are able to make a clean, scarless single letter edits, these edits are very rare, so that new technique from the Conklin lab is needed.

"Our method provides a novel way to capture and amplify specific mutations that are normally exceedingly rare," said Dr. Conklin. "Our high-efficiency, high-fidelity method could very well be the basis for the next phase of human genetics research."

"Now that powerful gene-editing tools, such as TALENs, are readily available, the next step is to streamline their implementation into stem cell research," said Dirk Hockemeyer, PhD, assistant professor of molecular and cellular biology at the University of California, Berkeley, who was not involved in this study. "This process will be greatly facilitated by the method described by Dr. Conklin and colleagues."

"Some of the most devastating diseases we face are caused by the tiniest of genetic changes," added Dr. Conklin. "But we are hopeful that our technique, by treating the human genome like lines of computer code, could one day be used to reverse these harmful mutations, and essentially repair the damaged code."

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Genome editing goes hi-fi: Technique in stem cells to boost scientists' ability to study genetic disease

Local mom bounces back after life-threatening illness

Published: Sunday, February 9, 2014 at 4:30 a.m. Last Modified: Saturday, February 8, 2014 at 10:56 p.m.

An avid runner, tennis player and rollerblader, Massagee's journey began nearly 10 years ago, when she began to notice her body slowly change, and included an endless parade of doctors, chemptherapy and a risky stem cell transplant.

Over time, Massagee's muscles grew larger. That wasn't necessarily a bad thing for an athletic woman in her early 50s, but as people age they tend to lose, not gain, muscle mass. Then her muscles began to harden and tighten, causing her significant pain.

Her husband, Buddy, a Hendersonville attorney, declared one day that something just wasn't right. Massagee had become so muscular that some in her social circle silently wondered if she was taking steroids to bulk up. She looked more like a professional body builder than an active mother of five.

As her muscles continued to harden and tighten, physical activity became more and more difficult. Massagee sought medical treatment from at least a dozen doctors, including the best of the best at Duke University Medical Center in Durham. After batteries of tests over several years, not a single doctor could tell Massagee what was wrong. Some doctors later confided to her that they thought I was secretly taking steroids and lying about it.

When a brain scan showed that the muscles behind her eyes were much larger than they should have been, doctors realized she wasn't on steroids, but they still weren't any closer to figuring out what was wrong. Bouncing from doctor to doctor, test to test, began to take its toll on Massagee physically and emotionally.

What broke my heart the most was looking at the pain it was causing Buddy and the children, she recalled. The couple's children, Sarah, 32, Rachel, 28, Kelly, 26, Lucy, 24 and Ty, 22, weren't very open about it we didn't talk about it a lot, Massagee said. But I knew it was very, very difficult for them.

Eventually, she found it impossible to undertake the most rudimentary physical activity, let alone work as a CPA.

I couldn't stand to make dinner, she said. I'd stand to chop something and then I'd need to sit down on a stool. I couldn't walk two blocks without having to stop.

One day, Massagee found it impossible to lift her arms enough to put on a pair of earrings.

Continued here:
Local mom bounces back after life-threatening illness