Faulty DNA Linked To Fatal Heart Condition Removed From Embryo

Scientists have modified human embryos to remove genetic mutations that cause heart failure in otherwise healthy young people in a landmark demonstration of the controversial procedure. It is the first time that human embryos have had their genomes edited outside China, where researchers have performed a handful of small studies to see whether the approach could prevent inherited diseases from being passed on from one generation to the next.

While none of the research so far has created babies from modified embryos, a move that would be illegal in many countries, the work represents a milestone in scientists’ efforts to master the technique and brings the prospect of human clinical trials one step closer. The work focused on an inherited form of heart disease, but scientists believe the same approach could work for other conditions caused by single gene mutations, such as cystic fibrosis and certain kinds of breast cancer.

This embryo gene correction method, if proven safe, can potentially be used to prevent transmission of genetic disease to future generations,” said Paula Amato, a fertility specialist involved in the US-Korean study at Oregon Health and Science University.

The scientists used a powerful gene editing tool called Crispr-Cas9 to fix mutations in embryos made with the sperm of a man who inherited a heart condition known as hypertrophic cardiomyopathy, or HCM. The disease, which leads to a thickening of the heart’s muscular wall, affects one in 500 people and is a common cause of sudden cardiac arrest in young people. Humans have two copies of every gene, but some diseases are caused by a mutation in only one of the copies. For the study, the scientists recruited a man who carried a single mutant copy of a gene called MYBPC3 which causes HCM.

Source: https://www.theguardian.com/

Cancer: A Giant Step For Immunotherapy

A Food and Drug Administration (FDA) panel opened a new era in medicine, unanimously recommending that the agency approve the first-ever treatment that genetically alters a patient’s own cells to fight cancer, transforming them into what scientists call “a living drug” that powerfully bolsters the immune system to shut down the disease.

If the F.D.A. accepts the recommendation, which is likely, the treatment will be the first gene therapy ever to reach the market. Others are expected: Researchers and drug companies have been engaged in intense competition for decades to reach this milestone. Novartis is now poised to be the first. Its treatment is for a type of leukemia, and it is working on similar types of treatments in hundreds of patients for another form of the disease, as well as multiple myeloma and an aggressive brain tumor.

To use the technique, a separate treatment must be created for each patient — their cells removed at an approved medical center, frozen, shipped to a Novartis plant for thawing and processing, frozen again and shipped back to the treatment center.

A single dose of the resulting product has brought long remissions, and possibly cures, to scores of patients in studies who were facing death because every other treatment had failed. The panel recommended approving the treatment for B-cell acute lymphoblastic leukemia that has resisted treatment, or relapsed, in children and young adults aged 3 to 25.

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We believe that when this treatment is approved it will save thousands of children’s lives around the world,” Emily’s father, Tom Whitehead, told the panel. “I hope that someday all of you on the advisory committee can tell your families for generations that you were part of the process that ended the use of toxic treatments like chemotherapy and radiation as standard treatment, and turned blood cancers into a treatable disease that even after relapse most people survive.”

The main evidence that Novartis presented to the F.D.A. came from a study of 63 patients who received the treatment from April 2015 to August 2016. Fifty-two of them, or 82.5 percent, went into remission — a high rate for such a severe disease. Eleven others died.

It’s a new world, an exciting therapy,” said Dr. Gwen Nichols, the chief medical officer of the Leukemia and Lymphoma Society, which paid for some of the research that led to the treatment. The next step, she said, will be to determine “what we can combine it with and is there a way to use it in the future to treat patients with less disease, so that the immune system is in better shape and really able to fight.” She added, “This is the beginning of something big.”

Source: http://www.chop.edu/
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Car Pollution: Nanoparticles Travel Directly From The Nose To The Brain

The closer a person lives to a source of pollution, like a traffic dense highway, the more likely they are to develop Alzheimer’s or dementia, according to a study by the University of Southern California (USC) that has linked a close connection to pollution and the diseases. In a mobile lab, located just off of one of Los Angeles’ busiest freeways, USC scientists used a state-of-the-art pollution particle collector capable of gathering nano-sized particulate matter.

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We have shown that, as you would expect, the closer you get to the sources of these particles in our case the freeways, the higher the concentrations. So there is an exponential decay with distance. That means basically that, the concentration of where we are right now and if we were, let’s just say 20 or 10 or 50 yards from the freeway, those levels would be probably 10 times higher than where we are right now,” says Costas Sioutas, USC Professor of Environmental Engineering.

That means proximity to high concentrations of fossil fuel pollution, like a congested freeway, could be hazardous. Particulate matter roughly 30 times thinner than the width of a human hair, called PM2.5, is inhaled and can travel directly through the nose into the brain. Once there, the particles cause inflammatory responses and can result in the buildup of a type of plaque, which is thought to further the progression of Alzheimer’s. “Our study brought in this new evidence and I would say probably so far the most convincing evidence that the particle may increase the risk of dementia. This is really a public health problem. And I think the policy makers need to be aware of that, the public health risk associated with high level of PM2.5,” explains Jiu-Chiuan Chen, Associate Professor of Preventive Medicine.

USC researchers analyzed the data of more than 3,500 women who had the APOE4 gene, the major known risk-factor gene for Alzheimer’s disease. It showed that, over the course of a decade, the women who lived in a location with high levels of the PM2.5 pollution were 92 percent more likely to develop dementia.

Source: https://news.usc.edu/
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Personality prediction: ‘Person of Interest’ TV Show Becomes Real

Is Faception an ingenious way to increase public safety or an incursion into our civil liberties? The former, say its makers. The Israeli start-up says it can isolate human character traits in faces captured by photograph or video. It says it can distinguish about 20 different personality groups, ranging from champion poker players to crime suspects.

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What we do, we know, with high level of accuracy, your personality ingredients, behaviour, potential and so we can have a profile about someone, as an individual and the same we can do about a crowd…let’s say gate number eight there are too much people that potentially can be terrorists or violent audience so this is something that is very crucial for public safety“, says Shai Gilboa, CEO of Faception company.
Faception won’t say how the algorithm works, except that it somehow gleans genetic information that lies within facial expressions. The firm insists it has no interest in retaining collected data and that the system disregards racial profiling.

Security experts are not convinced that’s enough. “Certainly advancement in technologies that enable to monitor an individual and actually to assess certain traits or certain attributes about individuals in the open space opens surveillance and monitoring capabilities which kind of like put in risk private freedoms that we used to enjoy, like the freedom of privacy, like the freedom of communication that we used to enjoy and now the technoligy certainly changes the balance“, comments Dr. Nimrod Kozlovski, security expert. Counter-terror experts say the firm must improve its 86 percent successful detection rate for it to be useful in airports. Civil liberties campaigners might say it shouldn’t be used at all.

Source: http://www.faception.com/

The Gene That Causes Grey Hair Is Now Identified

No matter who you are; for most of us grey hair is an inevitable part of getting older. But what if you could switch off the gene that causes it? For the first time, scientists have identified a gene called IRF4 as the culprit behind grey hair. DNA samples from over 6,000 volunteers were collected in Latin America; chosen for the diverse ancestry of its inhabitants. And it turns out if you have your roots in Europe, grey hair is much more likely.

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This genetic variant of IRF4 has two forms; one form is present world-wide and the other form is present only in Europeans. And we saw that this particular European specific form gives you almost double the chance of hair greying,” says Dr Kaustubh Adhikari from University College London (UCL), department of cell and developmental biology.

The gene IRF4 helps regulate melanin in the body, which determines – among other things – hair colour. Age and environmental factors will, of course, influence how quickly IRF4 triggers hair greying. But the researchers say their discovery could lead to a treatment that could stop it in its tracks.

Switching off a gene is of course feasible, the issue is whether it will have the desired effect and whether it’s the right thing to do… But in terms of trying to develop a therapy to delay or prevent hair greying, that is something that is potentially feasible; yes“, comments Professor Andres Ruiz-Linares, UCL (department of BioSciences).

Scientists think that a simple cosmetic treatment for switching off the grey gene would take many more years of research. But for those keen to banish the grey forever, your prayers might one day be answered.

The study has been published in the journal  Nature Communications.

Source: https://www.ucl.ac.uk/

Nanodiamonds For Breast Cancer Treatment

UCLA researchers led by Professor Dean Ho from the Jane and Jerry Weintraub Center for Reconstructive Biotechnology, have developed a potentially more effective treatment for breast cancer. Doctors have begun to categorize breast cancers into four main groups according to the genetic makeup of the cancer cells. Which category a cancer falls into generally determines the best method of treatment. But cancers in one of the four groups — called “basal-like” or “triple-negative” breast cancer (TNBC) — have been particularly tricky to treat because they usually don’t respond to the “receptor-targeted” treatments that are often effective in treating other types of breast cancer. TNBC tends to be more aggressive than the other types and more likely to recur, and can also have a higher mortality rate. Using nanodiamonds between 4 and 6 nanometers in diameter and shaped like tiny soccer balls, the researchers form clusters following drug binding that have the ability to precisely deliver cancer drugs to tumors, significantly improving the drugs’ desired effect. In the UCLA study, the nanodiamond delivery system has been able to home in on tumor masses in mice with triple negative breast cancer.

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This study demonstrates the versatility of the nanodiamond as a targeted drug-delivery agent to a tumor site,” said Ho, who is also a member of the California NanoSystems Institute at UCLA, UCLA’s Jonsson Comprehensive Cancer Center and the UCLA Department of Bioengineering. “The agent we’ve developed reduces the toxic side effects that are associated with treatment and mediates significant reductions in tumor size.”
Findings from the study are published online April 15 in the peer-reviewed journal Advanced Materials.
Source: http://newsroom.ucla.edu/