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U.S. scientists try 1st gene editing in the body

The Associated Press
CBC.ca

Scientists for the first time have tried editing a gene inside the body in a bold attempt to permanently change a person’s DNA to try to cure a disease.

The experiment was done Monday in California on 44-year-old Brian Madeux. Through intravenous (IV), he received billions of copies of a corrective gene and a genetic tool to cut his DNA in a precise spot.

“It’s kind of humbling” to be the first to test this, said Madeux, who has Hunter syndrome, a metabolic disease. “I’m willing to take that risk. Hopefully it will help me and other people.”

Signs of whether it’s working may come in a month; tests will show for sure in three months.

If it’s successful, it could give a major boost to the fledgling field of gene therapy. Scientists have edited human genes before, altering cells in the lab that are then returned to patients. There also are gene therapies that don’t involve editing DNA.

But these methods can only be used for a few types of diseases. Some give results that may not last. Some others supply a new gene like a spare part, but can’t control where it inserts in the DNA, possibly causing a new problem like cancer.

‘We cut your DNA, open it up, insert a gene, stitch it back up. Invisible mending.’ – Dr. Sandy Macrae, Sangamo Therapeutics

This time, the gene tinkering is happening in a precise way inside the body. It’s like sending a mini-surgeon along to place the new gene in exactly the right location.

“We cut your DNA, open it up, insert a gene, stitch it back up. Invisible mending,” said Dr. Sandy Macrae, president of Sangamo Therapeutics, the California company testing this for two metabolic diseases and hemophilia. “It becomes part of your DNA and is there for the rest of your life.”

That also means there’s no going back — no way to erase any mistakes the editing might cause.

“You’re really toying with Mother Nature” and the risks can’t be fully known, but the studies should move forward because these are incurable diseases, said one independent expert, Dr. Eric Topol of the Scripps Translational Science Institute in San Diego.

Protections are in place to help ensure safety, and animal tests were encouraging, said Dr. Howard Kaufman, a Boston scientist on the National Institutes of Health panel that approved the studies.

He said gene editing’s promise is too great to ignore.

“So far there’s been no evidence that this is going to be dangerous,” he said. “Now is not the time to get scared.”
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DIY Gene Editing: Fast, Cheap—and Worrisome

BY AMY DOCKSER MARCUS
WALL STREET JOURNAL

Kian Sadeghi has postponed homework assignments, sports practice and all the other demands of being a 17-year-old high-school junior for today. On a Saturday afternoon, he is in a lab learning how to use Crispr-Cas9, a gene-editing technique that has electrified scientists around the world—and sparked a widespread debate about its use. Scientific breakthroughs often raise big ethical questions. Moral concerns around the 1996 cloning of Dolly the sheep or the 2000 announcement of a rough draft of the human genome still reverberate today. The public benefits from scientific advances, particularly in improving health. Continue Reading →

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Ethical questions raised in search for Sardinian centenarians’ secrets

There is something like gold flowing through the veins of 100-year-old Maria Tegas, and everyone wants a piece of the treasure. The centenarian, who lives in a poor and remote area of central eastern Sardinia – in one of 14 villages known to geneticists and genealogists as the Blue Zone – has not had an easy life. Orphaned at the age of one, she remembers what it was like to go hungry, when homemade acorn bread was her main sustenance. As a young woman, she often walked 15 miles (24km) a day in steep and rocky terrain to bring food home to her six children. “We lived like birds in the sky,” she says in a tiny whisper of a voice. Continue Reading →

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Genetically Modified Mosquitoes: What Could Possibly Go Wrong?

BY ADRIENNE LAFRANCE
THE ATLANTIC
History is filthy with stories of pest control gone terribly, terribly wrong. Consider, for example, the infamous tale of how the mongoose got to the Hawaiian Islands. The sleek carnivore was imported in the 1880s as part of a plan by the sugar industry to subdue the rats that wouldn’t stop gnawing through stalks of sugar cane. Mongoose do enjoy a tasty rat supper, when the opportunity presents itself, but there was a problem: Rats are active at night, while mongoose are active during the day. So instead of decimating the rat population, the mongoose came to Hawaii and feasted on native birds and their eggs. Continue Reading →

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New study shows cloned sheep are living long lives with few health problems

BY ERIC ABENT
SLASHGEAR

Those of you who survived the roarin’ 1990s will almost certainly remember Dolly the Sheep, who was created from a single adult cell that was combined with an egg cell that had been stripped of its DNA. In other words, Dolly was a clone. Dolly was all over the news when she was born in 1996, but soon after, she started to suffer from health problems, with many people assuming that she was facing these issues because she was a clone. A new study of 13 cloned sheep, including four from the same cell line as Dolly, is showing that may not actually be the case. While Dolly was plagued early on by problems like osteoarthritis and eventually had to be put down in 2003 because of a tumors in her lungs, the clones studied here seem to be aging without many problems.

Though the study, which was led by developmental biologist Kevin Sinclair and a team of scientists from the University in Nottingham in England, says that it observed “no clinical signs of degenerative joint disease apart from mild, or in one case moderate, osteoarthritis in some animals,” it also states that somatic-cell nuclear transfer (the process by which the sheep were cloned) has “no obvious detrimental long-term health effects.”

This is pretty big news, as the health problems Dolly experienced brought into question the overall healthiness of cloned animals. Continue Reading →

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Crispr: Chinese scientists to pioneer gene-editing trial on humans

BY NICKY WOOLF
THE GUARDIAN

A team of Chinese scientists will be the first in the world to apply the revolutionary gene-editing technique known as Crispr on human subjects. Led by Lu You, an oncologist at Sichuan University’s West China hospital in Chengdu, China, the team plan to start testing cells modified with Crispr on patients with lung cancer in August, according to the journal Nature. Crispr is a game-changer in bioscience; a groundbreaking technique which can find, cut out and replace specific parts of DNA using a specially programmed enzyme named Cas9. Its ramifications are next to endless, from changing the color of mouse fur to designing malaria-free mosquitoes and pest-resistant crops to correcting a wide swath of genetic diseases like sickle-cell anaemia in humans. The concept of editing human DNA has often been controversial. Continue Reading →

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Swedish politicians consider opening population’s medical-research DNA database for private insurance companies

BY RICK FALKVINGE
PRIVATE INTERNET ACCESS

Since 1975, Sweden has taken a DNA sample from all newborns for medical research purposes, and asked parents’ consent to do so for this research purpose. This means that over time, Sweden has built the world’s most comprehensive DNA database over everybody under 43 years of age. But now, politicians are considering opening up this research-only DNA database to law enforcement and private insurance companies. It was a treasure to the scientific community, at the same time as it held enormous privacy risks that were not foreseen at the time. Scientists desired to study Phenylketonuria (PKU), a hereditary metabolism deficiency that, among other things, turns the common diet-soda-sweetener aspartame into a lethal poison. Continue Reading →

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Gene therapy partially restores vision in rare blindness disorder

BY IAM SAMPLE
THE GUARDIAN

Two men with progressive blindness have regained some of their vision after taking part in the first clinical trial of a gene therapy for the condition. The men were among six patients to have experimental treatment for a rare, inherited, disorder called choroideremia, which steadily destroys eyesight and leaves people blind in middle age. After therapy to correct a faulty gene, the men could read two to four more lines on an optician’s sight chart, a dramatic improvement that has held since the doctors treated them. One man was treated more than two years ago. The other four patients, who had less advanced disease and good eyesight before the trial, had better night vision after the therapy. Continue Reading →

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Britain gives scientist go-ahead to genetically modify human embryos

BY KATE KELLAND
REUTERS

Scientists in Britain have been give the go-ahead to edit the genes of human embryos for research purposes, using a technique that some say could eventually be used to create “designer babies”. Less than a year after Chinese scientists caused an international furor by saying they had genetically modified human embryos, Kathy Niakan, a stem cell scientist from London’s Francis Crick Institute, was granted a license to carry out similar experiments. “The Human Fertilisation and Embryology Authority (HFEA) has approved a research application from the Francis Crick Institute to use new ‘gene editing’ techniques on human embryos,” Niakan’s lab said on Monday. It said the work carried out “will be for research purposes and will look at the first seven days of a fertilized egg’s development, from a single cell to around 250 cells”. The scientists will not be allowed to develop the modified embryos for clinical purposes or implant them into any women. Continue Reading →

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