Stem Cell Gene Therapy Could be Key to Treating Duchenne Muscular Dystrophy

Scientists at the UCLA Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research and Center for Duchenne Muscular Dystrophy at UCLA have developed a new approach that could eventually be used to treat Duchenne muscular dystrophy. The stem cell gene therapy could be applicable for 60 percent of people with Duchenne, which affects approximately 1 in 5,000 boys in the U.S. and is the most common fatal childhood genetic disease.

The approach uses a technology called CRISPR/Cas9 to correct genetic mutations that cause the disease. The study, which was led by co-senior authors April Pyle and Melissa Spencer and first author Courtney Young, was published in the journal Cell Stem Cell.


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Flicker of Hope for Children With Rare and Devastating Disease

LEESBURG, Ala. — Once a year, Crystal and Jonathan Bedford drive 1,000 miles from their home in Texas to rural Alabama, their three children in tow. Beside a wooded lake, they huddle with other families whose children have the same extremely rare genetic disorder that their 5-year-old daughter, Marley, has.

The disease, rhizomelic chondrodysplasia punctata, is a painful form of dwarfism, usually accompanied by severe intellectual disability and respiratory problems. There is no cure, and children with RCDP, as it is known, rarely survive into adolescence.

The families come for advice on how to care for their fragile children, and for any scrap of information about promising research. Most years they leave with little more than warm support. But this year was different.

Link to full story at New York Times

Misunderstanding the genome:

A recent Ars feature story about genetic screening generated quite a lively debate in the discussion thread. However, it also underlined just how many misconceptions people have when it comes to genetics. Public perception hasn’t been helped by scientists overhyping their findings or by inaccurate portrayals in the media (GATTACA, anyone?). So today, I’m going to try to clear some common confusions.

Before moving recently to Ars full time, I spent six years working in the policy office of the National Human Genome Research Institute, the part of the National Institutes of Health responsible for the Human Genome Project (along with the UK’s Wellcome Trust). The job gave me a front row seat to the challenge of explaining a horribly complex topic, one where common assumptions are often counterfactual.

Maria Delany’s Ars article does a great job laying out how screening at-risk individuals for mutations in a pair of genes—BRCA1 and BRCA2—can spare people from developing cancer. Delany also explains why there isn’t unanimity among clinicians about rolling out BRCA testing at the population level. At first glance, such testing seems like a no brainer, right? Testing right now is targeted to at-risk groups, like women with a family history of breast cancer, but studies have found those mutations in people with no family history of the disease. If testing people for BRCA mutations finds them before cancer does, where’s the downside?



Full story at Ars Technica

Synthetic vaccine sought to finally eradicate polio

Synthetic vaccine sought to finally eradicate polio


An international team of scientists is to try to develop a wholly artificial vaccine to combat polio.

The disease is very close to being eradicated, with only a few hundred cases now reported worldwide each year.

The hope is that the new approach can address some shortcomings in an existing vaccine, and so help eliminate polio altogether.

The World Health Organization and the Bill & Melinda Gates Foundation are providing a $674,000 (£438,000) grant.

The project was announced at the annual meeting of the American Association for the Advancement of Science in San Jose, California.

Researchers in the US and the UK will participate. In Britain, this will pull in workers from Leeds, Oxford, Reading, and the Diamond synchrotron.


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Ebola Drug Aids Some in a Study in West Africa

or the first time, a drug is showing promising signs of effectiveness in Ebola patients participating in a study. The medicine, which interferes with the virus’s ability to copy itself, seems to have halved mortality — to 15 percent, from 30 percent — in patients with low to moderate levels of Ebola in their blood, researchers have found. It had no effect in patients with more virus in their blood, who are more likely to die.

The drug, approved as an influenza treatment in Japan last year, was generally well tolerated.

“The results are encouraging in a certain phase of the disease,” Dr. Sakoba Keita, director of disease control for the Guinean Ministry of Health, said in a telephone interview. The drug is being tested in Guinea, one of the three West African countries most affected by the Ebola crisis.

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Drugs in dirt: Scientists appeal for help

US scientists are asking the public to join them in their quest to mine the Earth’s soil for compounds that could be turned into vital new drugs.

Spurred on by the recent discovery of a potential new antibiotic in soil, the Rockefeller University team want to check dirt from every country in the world.

They have already begun analysing samples from beaches, forests and deserts across five continents.

But they need help getting samples.

Which is where we all come in.

Citizen science

On their Drugs From Dirt website, they say: “The world is a big place and we can’t get get to all of the various corners of it.

“We would like some assistance in sampling soil from around the world. If this sounds interesting to you – sign up.”

They want to hear from people from all countries and are particularly keen to receive samples from unique, unexplored environments such as caves, islands, and hot springs.

Such places, they say, could house the holy grail – compounds produced by soil bacteria that are entirely new to science.

Researcher Dr Sean Brady told the BBC: “We are not after hundreds of thousands of samples. What we really want is a couple of thousand from some really unique places that could contain some really interesting stuff. So it’s not really your garden soil we are after, although that will have plenty of bacteria in it too.”


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Most cancer types ‘just bad luck’

Most types of cancer can be put down to bad luck rather than risk factors such as smoking, a study has suggested.

A US team were trying to explain why some tissues were millions of times more vulnerable to cancer than others.

The results, in the journal Science, showed two thirds of the cancer types analysed were caused just by chance mutations rather than lifestyle.

However some of the most common and deadly cancers are still heavily influenced by lifestyle.

And Cancer Research UK said a healthy lifestyle would still heavily stack the odds in a person’s favour.


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Lurking in the Shadows

Bats harbor diverse pathogens, including Ebola, Marburg, SARS, and MERS viruses. Understanding why could help researchers stymie deadly emerging diseases.


In a dusty, subterranean room beneath a crumbling sand building a few miles north of Naqi, Saudi Arabia, University of Pennsylvania veterinary epidemiologist Jon Epstein finally found what he was looking for: bats. It was early October 2012; he’d travelled to the country with a team of scientists at the request of the Saudi Ministry of Health. A researcher in Jedda had isolated RNA from a strange virus found in the mucus coughed up by a 60-year-old Saudi businessman who’d recently suffered acute pneumonia and renal failure. The man, whose main place of business was located in Naqi, died 11 days after being admitted into the hospital in mid-June.

Epstein and Columbia University epidemiologist Ian Lipkin had been scouring the man’s homes and businesses around the desert town of Bishah in search of the source of the deadly virus. They were intent on sampling bats because a Saudi scientist had determined the new virus to be a novel type of coronavirus, a family of rapidly evolving viruses that includes the severe acute respiratory syndrome coronavirus (SARS-CoV). Both Lipkin and Epstein had worked extensively on SARS when it ripped through southern China and Hong Kong in 2002 and 2003. That disease eventually spread to some 33 countries, infected more than 8,000 people, and killed more than 800. The disease detectives had been investigating bats as a possible natural reservoir for SARS-CoV, and last year, after more than a decade of work, Epstein and colleagues announced the discovery of a nearly identical coronavirus, with the crucial ability to infect human cells, in the Chinese horseshoe bat (Rhinolophus sinicus). They speculated that the bats could have been directly infecting humans, without the need for an intermediate host, throughout the course of the SARS epidemic.1


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