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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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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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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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First Gene Therapy Successful Against Human Aging

Elizabeth Parrish, CEO of Bioviva USA Inc. has become the first human being to be successfully rejuvenated by gene therapy, after her own company’s experimental therapies reversed 20 years of normal telomere shortening. Telomere score is calculated according to telomere length of white blood cells (T-lymphocytes). This result is based on the average T-lymphocyte telomere length compared to the American population at the same age range. The higher the telomere score, the “younger” the cells. In September 2015, then 44 year-old CEO of BioViva USA Inc. Elizabeth Parrish received two of her own company’s experimental gene therapies: one to protect against loss of muscle mass with age, another to battle stem cell depletion responsible for diverse age-related diseases and infirmities. Continue Reading →

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Meet the companies customizing beauty products for your DNA

BY ALEXANDRA BREEN
THE GLOBE AND MAIL

What’s the next frontier in beauty? If you believe in the upswing in science-based brands, it’s your very genetic make up. “Bespoke” skincare brands such as Skin Inc. are popping up in places like Sephora, allowing consumers to create custom blended serums based on their specific needs. Do a quick search online and you’ll find made-to-order facial products by IOMA, TruthArtBeauty and Jennifer Young are just a click away. And then there’s SkinShift and GeneU, two companies making news for their high-tech take on the trend: tailor-made beauty products based on your DNA. Continue Reading →

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Gene-edited ‘micropigs’ to be sold as pets at Chinese institute

BY DAVID CYRANOSKI
NATURE

Cutting-edge gene-editing techniques have produced an unexpected byproduct — tiny pigs that a leading Chinese genomics institute will soon sell as pets.

BGI in Shenzhen, the genomics institute that is famous for a series of high-profile breakthroughs in genomic sequencing, originally created the micropigs as models for human disease, by applying a gene-editing technique to a small breed of pig known as Bama. On 23 September, at the Shenzhen International Biotech Leaders Summit in China, BGI revealed that it would start selling the pigs as pets. The animals weigh about 15 kilograms when mature, or about the same as a medium-sized dog.

At the summit, the institute quoted a price tag of 10,000 yuan (US$1,600) for the micropigs, but that was just to “help us better evaluate the market”, says Yong Li, technical director of BGI’s animal-science platform. In future, customers will be offered pigs with different coat colours and patterns, which BGI says it can also set through gene editing.

With gene editing taking biology by storm, the field’s pioneers say that the application to pets was no big surprise. Some also caution against it. “It’s questionable whether we should impact the life, health and well-being of other animal species on this planet light-heartedly,” says geneticist Jens Boch at the Martin Luther University of Halle-Wittenberg in Germany. Boch helped to develop the gene-editing technique used to create the pigs, which uses enzymes known as TALENs (transcription activator-like effector nucleases) to disable certain genes.

How to regulate the various applications of gene-editing is an open question that scientists are already discussing with agencies across the world. BGI agrees on the need to regulate gene editing in pets as well as in the medical research applications that make up the core of its micropig activities. Any profits from the sale of pets will be invested in this research. “We plan to take orders from customers now and see what the scale of the demand is,” says Li. Continue Reading →

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How DNA barcoding can prevent foodborne illness outbreaks

BY ELIZABETH ARMSTRONG MOORE
GIGAOM
In 2012, when several hundred people fell ill in the U.S. amid a salmonella outbreak, the Food and Drug Administration was quickly able to isolate the exact strain of salmonella that had found its way into the contaminated sushi-grade tuna — and then trace it to the exact processing plant where the fish originated in India. (Not surprisingly, the FDA found 10 sanitation oversights, four of which were considered egregious.)
Then in 2014, the FDA managed to prevent a listeria outbreak from going beyond seven illnesses and one death when it traced the strain of the pathogen to soft cheeses manufactured by Ross Foods, which has since been shut down. Both findings are thanks to DNA sequencing, which is helping not only to identify which species of animals we might be eating, but even which strains of foodborne pathogens might be present in our food. The implications are broad. Knowing at a genetic level what we are eating isn’t just good for our health (think food allergies, high mercury levels, etc.) and for our wallets (how much are we really paying for tilapia?), but also for the animals (some of which are endangered or illegally hunted). Continue Reading →

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Next-generation genomics key to global food and nutritional security

BY ICRISAT
PORK NETWORK

More than 300 delegates representing private and public sectors from 30 countries worldwide convened at the International Crops Research for the Semi-Arid Tropics (ICRISAT) global headquarters to deliberate on future collaborations and ways to integrate next-generation genomics into the future of crop improvement  to contribute to global food and nutrition security. “Making a hunger- and malnutrition-free society is the ultimate goal of every agricultural scientist and stakeholder. Next-generation genomics backed by strong technological advancements will facilitate science-based agricultural innovations such as the development of nutrition-rich crops to eradicate hunger.”

This was according to Dr MS Swaminathan, renowned agricultural scientist and Father of India’s Green Revolution, at today’s successful conclusion of the three-day 5th International Conference on Next Generation Genomics and Integrated Breeding for Crop Improvement (NGGIBCI-V) held on 18-20 February 2015. Genomics – or deciphering the genomic content of crop species using high-throughput and next-generation approaches – allows the scientific community access to ‘good genes’ to speed up breeding for superior crop varieties with agronomically important traits. “It is not so much a question of more food. Continue Reading →

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