With CRISPR, geneticists have a powerful new weapon in the battle against ALS

crispr als treatment gettyimages 713768585
Andrew Brookes/Getty Images
For many people today, amyotrophic lateral sclerosis, aka ALS or Lou Gehrig’s disease, is most commonly linked with both the fundraising Ice Bucket Challenge and one its most famous patients, the physicist Stephen Hawking. However, it could soon have a brand-new distinction — the next disease to be treatable using CRISPR-Cas9 gene-editing technology.

In work carried out by researchers at University of California, Berkeley, scientists have been able to disable the defective gene that triggers ALS in mice. While they didn’t get rid of the disease permanently, the treatment did extend the mice’s life span by 25 percent. The therapy delayed the onset of the muscle-wasting symptoms that characterize ALS, which ultimately become fatal when they spread to the muscles which control breathing.

“Some diseases, like Lou Gehrig’s disease, are caused by gene mutations that lead a protein in our cells to malfunction,” David Schaffer, a professor of chemical and biomolecular engineering and director of the Berkeley Stem Cell Center, told Digital Trends. “A very promising approach is to disable or delete that mutated gene. CRISPR/Cas9 is a highly promising technology to do so, but this capability needs to be delivered to the target cells. We put together CRISPR-Cas9 with a highly promising gene delivery, based on a virus, in order to disable the disease causing gene SOD1 in an animal model of ALS.”

The mice in the study were genetically engineered to exhibit a mutated human gene that is responsible for around 20 percent of all inherited forms of ALS. The team then used a specially engineered virus that delivers a gene encoding the Cas9 protein, which in turn disabled the mutant gene responsible for ALS. The treated mice lived one month longer than the typical four-month life span of mice with ALS. An average healthy mouse lives for around two years.

Hopefully, were this to be carried over to humans, those time spans would be extended. “There are challenges that remain before extending into human studies, such as using an improved virus optimized for humans, but we think there is a clear path to doing so,” Schaffer said.

A paper describing the work was recently published in the journal Science Advances.

Emerging Tech

Inflating smart pills could be a painless alternative to injections

Could an inflating pill containing hidden microneedles replace painful injections? The creators of the RaniPill robotic capsule think so — and they have the human trials to prove it.
Gaming

Your PlayStation 4 game library isn't complete without these games

Looking for the best PS4 games out there? Out of the massive crop of titles available, we selected the best you should buy. No matter what your genre of choice may be, there's something here for you.
Gaming

These are the must-have games that every Xbox One owner needs

More than four years into its life span, Microsoft's latest console is finally coming into its own. From Cuphead to Halo 5, the best Xbox One games offer something for players of every type.
Home Theater

The best Dolby Atmos movies for your home theater sound as good as they look

If you've got your hands on some sweet Dolby Atmos gear, the next step is to find films that take advantage of it. These are our picks in several genres for the best Dolby Atmos movies currently available on Blu-ray and streaming services.
Emerging Tech

NASA’s Mars 2020 rover passes its tests with flying colors

The Mars 2020 rover team has been undertaking a series of tests to see if the craft will be able to launch, navigate, and land on the Red Planet. Called Systems Test 1, or ST1, these tests represent the first test drive of the new rover.
Outdoors

Light up the night! Here are the five best headlamps money can buy

Headlamps make all the difference when camping or walking the dog at night, especially when you're in need of both hands. From Petzl to Tikkid, here are some of the best headlamps on the market.
Emerging Tech

Awesome Tech You Can’t Buy Yet: Robotic companions and computer-aided karaoke

Check out our roundup of the best new crowdfunding projects and product announcements that hit the web this week. You may not be able to buy this stuff yet, but it's fun to gawk!
Emerging Tech

A hive of activity: Using honeybees to measure urban pollution

According to a new study from Vancouver, bees could help us understand urban pollution. Scientists have found an innovative way to measure the level of source of pollution in urban environments: by analyzing honey.
Emerging Tech

Spacewalk a success as astronauts upgrade batteries on the ISS

The International Space Station was treated to some new batteries on Friday, thanks to two NASA astronauts who took a spacewalk for nearly seven hours in order to complete the upgrades.
Emerging Tech

Asteroid Ryugu is porous, shaped like a spinning top, and is formed of rubble

The Japanese Space Agency has been exploring a distant asteroid named Ryugu with its probe, Hayabusa 2. Now the first results from study of the asteroid are in, with three new papers published.
Emerging Tech

Is it a bird? Is it a plane? No, it’s a super-speedy pulsar

A super-speedy pulsar has been spotted dashing across the sky, discovered using NASA’s Fermi Gamma-ray Space Telescope and the Very Large Array. The pulsar is traveling at a breathtaking 2.5 million miles an hour.
Emerging Tech

Chilean telescope uncovers one of the oldest star clusters in the galaxy

An ultra-high definition image captured by the Gemini South telescope in Chile has uncovered one of the oldest star clusters in the Milky Way. The cluster, called HP 1, could give clues to how our galaxy was formed billions of years ago.
Emerging Tech

Astronomers discover giant chimneys spewing energy from the center of the galaxy

Astronomers have discovered two exhaust channels which are funneling matter and energy away from the supermassive black hole at the heart of our galaxy and out towards the edges of the galaxy, dubbed galactic center chimneys.
Emerging Tech

A milestone in the history of particle physics: Why does matter exist?

If matter and antimatter were both produced in equal amounts by the Big Bang, why is there so much matter around us and so little antimatter? A new experiment from CERN may hold the answer to this decades-long puzzle.