In a 1st, doctors in U.S. use CRISPR tool to treat patient with genetic disorder

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Victoria Gray, 34, of Forest, Miss., volunteered for one of the most anticipated medical experiments in decades: the first attempt to use the gene-editing technique CRISPR to treat a genetic disorder in the U.S.

For the first time, doctors in the U.S. have used the powerful gene-editing technique CRISPR to try to treat a patient with a genetic disorder.

“It is just amazing how far things have come,” says Victoria Gray, 34, of Forest, Miss. “It is wonderful,” she told NPR in an exclusive interview after undergoing the landmark treatment for sickle cell disease.

Gray is the first patient ever to be publicly identified as being involved in a study testing the use of CRISPR for a genetic disease.

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Wait, What? The first human-monkey hybrid embryo was just created in China

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Last week, news broke that a prominent stem cell researcher is making human-monkey chimeras in a secretive lab in China.

The story, first reported by the Spanish newspaper El País, has all the ingredients of a bombshell. First, its protagonist is the highly-respected Dr. Juan Carlos Izpisúa Belmonte, a Spanish-born stem cell biologist at the Salk Institute in California known for his breakthroughs in anti-aging research. His other fascination? Human-animal chimeras, in which animal embryos are injected with human cells and further developed inside a surrogate animal’s body. Second, according to El País, Izpisúa Belmonte may have collaborated with monkey researchers in China to circumvent legal issues in the US and Spain, where research with primates is heavily regulated.

The news did not sit well with Chinese scientists, who are still recovering from the CRISPR baby scandal. “It makes you wonder, if their reason for choosing to do this in a Chinese laboratory is because of our high-tech experimental setups, or because of loopholes in our laws?” lamented one anonymous commentator on China’s popular social media app, WeChat.

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How scientists built a ‘living drug’ to beat cancer

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IN 2010, EMILY Whitehead was diagnosed with Acute Lymphoblastic Leukemia, a cancer of certain cells in the immune system.

THIS IS THE most common form of childhood cancer, her parents were told, and Emily had a good chance to beat it with chemotherapy. Remission rates for the most common variety were around 85 percent.

It would be 20 months before they’d understand the shadow behind that sunny statistic, and the chilling prospect of volunteering their daughter as patient zero for the world’s first living drug.

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Using CRISPR to resurrect the dead

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Gene-editing breakthroughs could allow us to bring extinct species, like the woolly mammoth, back from the dead. But should we?

It really is worse than you think.

We’ve gorged ourselves on fossil fuels, vacuumed up the Earth’s forests and spewed toxic gases into the atmosphere for years on end. The planet is getting warmer, we’re poisoning insect populations with reckless abandon and pulling fish out of the ocean at an alarming rate. The most recent prognosis for a biodiverse Earth is incredibly grim, with 1 million species threatened with extinction in the coming decades.

The havoc we’ve generated has kickstarted Earth’s sixth great extinction event, the first by human hands. This rapid decrease in biodiversity due to human activity is unprecedented.

But we may be able to reverse it.

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His DNA solved a century-old jailhouse rape. The victim: His Grandmother

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Commercial DNA tests showed that Hiram and Bruce were related. But their link proved to be much deeper — and darker — than either could have imagined.

As a black teenager in Compton, California, in the 1970s, Hiram Johnson began to wonder about his father’s fine curly hair, and the light-brown skin that strangers sometimes thought was white.

Hiram knew only a few things about his father’s childhood. Fred Johnson was raised in Jackson, Mississippi, by his mother, Bernice. Fred said that Bernice was a “beautiful black woman,” but he never said a word about his father. All Hiram knew was that his grandfather probably wasn’t black.

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Scientists created bacteria with a synthetic genome. Is this artificial life?

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A colored scanning electron micrograph of the bacteria E. coli. Scientists in Britain created bacteria with “recoded” DNA.

In a milestone for synthetic biology, colonies of E. coli thrive with DNA constructed from scratch by humans, not nature.

Scientists have created a living organism whose DNA is entirely human-made — perhaps a new form of life, experts said, and a milestone in the field of synthetic biology.

Researchers at the Medical Research Council Laboratory of Molecular Biology in Britain reported on Wednesday that they had rewritten the DNA of the bacteria Escherichia coli, fashioning a synthetic genome four times larger and far more complex than any previously created.

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This DIY biohacker is under investigation

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Josiah Zayner rose to internet fame after performing various biohacking stunts on himself — including a livestreamed attempt to edit his own genes using CRISPR.

Those antics are coming to haunt Zayner. Now, the California Department of Consumer Affairs (DCA) is investigating a “complaint of unlicensed practice of medicine” filed against him — a strange development that could have implications for the future of biohacking.

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CRISPR gene editing is coming for the womb

 

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When an unborn baby is diagnosed with a life-threatening defect, it can be devastating. So some scientists hope to treat the fetus in the uterus, using gene editing.

WILLIAM PERANTEAU IS the guy parents call when they’ve received the kind of bad news that sinks stomachs and wrenches hearts. Sometimes it’s a shadow on an ultrasound or a few base pairs out of place on a prenatal genetic test, revealing that an unborn child has a life-threatening developmental defect. Pediatric surgeons like Peranteau, who works at Children’s Hospital of Philadelphia, usually can’t try to fix these abnormalities until their patients leave their mother’s bodies behind. And by then it might be too late.

It’s with the memory of the families he couldn’t help in the back of his mind that Peranteau has joined a small group of scientists trying to bring the fast-moving field of gene editing to the womb. Such editing in humans is a long way off, but a spate of recent advances in mouse studies highlight its potential advantages over other methods of using Crispr to snip away diseases. Parents confronted with an in utero diagnosis are often faced with only two options: terminate the pregnancy or prepare to care for a child who may require multiple invasive surgeries over the course of their lifetime just to survive. Prenatal gene editing may offer a third potential path. “What we see as the future is a minimally invasive way of treating these abnormalities at their genetic origin instead,” says Peranteau.

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CRISPR is now being used on humans in the U.S.

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The gene editing trial has two patients so far.

CRISPR therapies are entering the mainstream.

The first U.S. trial of CRISPR in humans has begun, NPR reported Tuesday. Two patients are currently being treated as part of a University of Pennsylvania study. Per NPR, both have difficult-to-treat forms of cancer and both have relapsed after regular treatments. As part of the trial, researchers are taking immune cells from the patients’ own bodies and editing them with CRISPR before putting them back in. The hope is that these edited cells will be better at identifying and attacking the cancer than their unaltered counterparts. According to the U.S. government clinical trial registry, the researchers are hoping to enroll 18 people in their study. But it’s not certain yet whether they’ll be approved for that many subjects, reports Jon Fingas for Engadget.

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Should athletes be allowed to enhance their genes?

 

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So-called gene doping is banned in sports, but some philosophers argue that it’s the way of the future

Scientists first developed gene therapy techniques in the 1990s, exploring ways to treat disease by modifying malfunctioning cells. In 1997, a team at John Hopkins University edited genes to create what the media called “Schwarzenegger mice,” which had twice the normal amount of muscle.

The researchers’ goal was to develop treatments for muscle-wasting conditions, including old age, but the same technique could theoretically be used to add muscle bulk to athletes, a concept called gene doping. Doctors could, theoretically, inject cells with enhanced genes into the relevant body part or use a benign virus to deliver modified cells. These superhumans could be the elite athletes of the future — athletes who perform faster, higher, and stronger than any “natural” human ever could.

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Advance in CAR T-cell therapy eliminates severe side effects

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An advance in the breakthrough cancer treatment known as CAR T-cell therapy appears to eliminate its severe side effects, making the treatment safer and potentially available in outpatient settings, a new USC study shows.

“This is a major improvement,” said Si-Yi Chen of the USC Norris Comprehensive Cancer Center, professor in the Department of Molecular Microbiology and Immunology at the Keck School of Medicine of USC, and senior author of the study appearing online April 22 in Nature Medicine. “We’ve made a new CAR molecule that’s just as efficient at killing cancer cells, but it works more slowly and with less toxicity.”

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Device tests thousands of stem cells super fast

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Institute for Molecular Engineering researchers have developed a “lab-on-a-chip” that could help us understand how single stem cells react to different molecules and environments. (Credit: Zhang et al.)

A new “lab-on-a-chip” can examine thousands of individual live cells over a weeklong period, performing experiments that would take more than 1 million steps in a laboratory.

The credit-card-sized, microfluidic device not only saves time and money, but also offers a new glimpse into how single stem cells react to different molecules and environments.

When researchers examined neural stem cells on the device and analyzed the data, they found several new rules that determine the timing and signaling sequences necessary for the cells to differentiate or renew themselves. The finding could have implications in understanding brain development or in treating patients with immunotherapy.

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