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FDA approves first gene therapy for an inherited disease

BY LAURIE MCGINLEY
WASHINGTON POST

In a historic move, the Food and Drug Administration on Tuesday approved a pioneering gene therapy for a rare form of childhood blindness, the first such treatment cleared in the United States for an inherited disease.

The approval signals a new era for gene therapy, a field that struggled for decades to overcome devastating setbacks but now is pushing forward in an effort to develop treatments for hemophilia, sickle-cell anemia and an array of other genetic diseases. Yet the products, should they reach patients, are likely to carry stratospheric prices — a prospect already worrying consumer advocates and economists.

Philadelphia-based Spark Therapeutics, which makes the childhood-blindness treatment, said it will not announce the price until January. Analysts speculate it could be as much as $1 million for both eyes. Continue Reading →

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It’s time to talk about who can access your digital genomic data

BY CURTIS AND HEREWARD
THE CONVERSATION

We are approaching a time when you might be too scared to have your genome sequenced.

Only last week, a US senator called for an investigation into the privacy policies of direct-to-consumer DNA companies. But this is only one piece of a puzzle that is about to get much more connected.

As with any kind of personal data there are a number of concerns regarding collection, transmission, storage and use. But unlike most other data, your genome reveals intimate information about not only you, but also the people to whom you are related.

It’s time to talk about who can access that data, how, when and why. Continue Reading →

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‘I want to help humans genetically modify themselves’

JOSIAH ZAYNER

BY TOM IRELAND
THE GUARDIAN

Josiah Zayner, 36, recently made headlines by becoming the first person to use the revolutionary gene-editing tool Crispr to try to change their own genes. Part way through a talk on genetic engineering, Zayner pulled out a syringe apparently containing DNA and other chemicals designed to trigger a genetic change in his cells associated with dramatically increased muscle mass. He injected the DIY gene therapy into his left arm, live-streaming the procedure on the internet.

The former Nasa biochemist, based in California, has become a leading figure in the growing “biohacker” movement, which involves loose collectives of scientists, engineers, artists, designers, and activists experimenting with biotechnology outside of conventional institutions and laboratories.

Despite warnings from the US Food and Drug Administration (FDA) that selling gene therapy products without regulatory approval is illegal, Zayner sells kits that allow anyone to get started with basic genetic engineering techniques, and has published a free guide for others who want to take it further and experiment on themselves.

Was administering a dose of Crispr on yourself an experiment, or a stunt to show what amateur scientists/biohackers can do?
Both. The technical feasibility of what I did is not under question – researchers have done this many times, in all sorts of animals. But there’s a barrier – people are afraid of it, and just talk about the possibilities in humans. I wanted to break that down, to say “Hey look, the tools are inexpensive, and somebody with a bit of knowledge can actually go through with these experiments”.

I chose to start with the gene for myostatin [a protein that regulates muscle growth], because it has been extensively studied, and it produces an obvious change if it has worked.

So, how is your arm looking?
In similar experiments with animals, you only start to see results after four to six months of treatment. I would expect that the DNA in some of the cells of my arm has changed, but I am still working on developing assays [tests] to try and detect that. As to whether the actual size of the muscle changes, I’m more sceptical.

Changing the way one gene behaves can have a huge number of knock-on effects on the way other genes are regulated or expressed. Do you really know what you’re doing?
It’s a good question. These things are complicated, and obviously with things like this there are lots of unknowns. I look at what the possible negative outcomes are and ask: “Are those risks insignificant enough that I’m willing to undertake this experiment?” Based on the data I read, for a local injection the answer was yes. A treatment that blocks myostatin throughout the whole body? That would be much more hazardous – you would be messing with the muscles of your heart.

You support the idea of people attempting gene therapy and other experimental procedures on themselves. What’s wrong with the existing system, where treatments are thoroughly tested by professionals before being approved for use?
If we’re going to do these experiments you have to balance two things: how many people can possibly die from testing their own products or making them available prematurely, versus how many people have genetic disorders and are just dying because they don’t have access to them. I think there’s a huge imbalance, where we’re overprotective of hurting people instead of offering a chance to millions of people who are dying right now.

As human beings we’re very big on freedoms, equality, equal rights. What’s more of an equal right than being able to control what genes we have? I think people should be able to choose that. I’m not saying anything I can do can help treat people, but treating things genetically is the ultimate medicine.

I grew up in the 90s with the computer hacker movement, the development of the internet – the whole open-source movement was amazing. Who created Linux, the most used operating system ever? Not students from Harvard or Cambridge, but Linus Torvalds, a student in Finland working in his apartment.

I don’t think for a second I’m going to be the mastermind behind a great biotech revolution, but I think there’s some brilliant person waiting to be discovered out there that could be. Continue Reading →

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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.

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.” Continue Reading →

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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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