Gene therapy reverses muscular dystrophy symptoms in dogs

dog
Damedeeso/123RF
As difficult as it is when our beloved pet dogs get old, it’s a whole lot worse if their decline is the result of a horrible disease like Duchenne muscular dystrophy, a genetic disorder characterized by progressive muscle degeneration and weakness. Fortunately, a team of gene-editing researchers from France and the U.K. have been working to develop gene therapy as an answer — and it’s one that could help humans, too.

Their solution involves using gene therapy to restore muscle strength and stabilize clinical symptoms. This is achieved by way of a shortened version of the dystrophin gene, containing just 4,000 base pairs, which is combined with a viral vector and injected into patients.

“Duchenne muscular dystrophy is a debilitating muscle-wasting disease affecting young boys and male animals, which is caused by inheritance of a damaged gene,” George Dickson, professor of molecular cell biology at University of London, told Digital Trends. “There is currently no very effective cure. Our work has involved producing a healthy functioning copy of the damaged gene in the lab, and then using a harmless virus to carry the gene into the affected muscles, a so-called gene therapy.”

The gene treatment has so far been tested on twelve golden retrievers affected by Duchenne muscular dystrophy. After a single dose, the dogs’ dystrophin production — the protein responsible for maintaining the integrity and strength of muscles — returned to its normal levels. Best of all, the trial was carried out a few years back, and all signs point to this being a lasting treatment. A paper describing the work was recently published in the journal Nature Communications.

“We now have treated a number of dogs affected by this condition with a single round of gene therapy,” Dickson continued. “At the correct dose, the results have been very encouraging with dogs looking very active and healthy over 3 years after the treatment. The treatment involves a simple intravenous infusion, a one-off treatment, and we have been very pleased and quite surprised at how effective the gene therapy has been given that muscle is a major tissue spread all over the body.”

Next up, the researchers hope to extrapolate the findings to human children, since they they are roughly the same weight and display similar clinical symptoms to canines.

“The goal is to expand this gene therapy treatment into human patients, boys suffering from the Duchenne muscular dystrophy disease,” Dickson said. “We have to scale up production of the gene therapy medicine for human use, complete more safety tests, and then embark on recruiting patients into full-scale clinical trials.”

Should all go according to plan, it’ll be another reminder of why gene therapy is the future of medicine as we know it.

Emerging Tech

Meet the gene-edited bacteria that could make cannabis plants obsolete

Ever wanted to brew cannabis like you brew craft beer? At UC Berkeley, biologists have managed to engineer brewer’s yeast so that it produces the main cannabinoids found in marijuana.
Gaming

These awesome free-to-play games might be even better than the ones you paid for

Believe it or not, free-to-play games have evolved into engaging, enjoyable experiences. Here are a few of our favorites that you can play right now, including Warframe and the perennially popular League of Legends.
Movies & TV

Skip the flowers and sunshine this spring and watch the best shows on Hulu

It's often overwhelming to navigate Hulu's robust library of TV shows. To help, we put together a list of the best shows on Hulu, whether you're into frenetic cartoons, intelligent dramas, or anything in between.
Emerging Tech

Scientists manage to 3D print an actual heart using human cells

Scientists at Tel Aviv University have achieved a world-first by 3D printing a small-scale heart, complete with blood vessels, ventricles, and chambers. Here's why that's so exciting.
Emerging Tech

Awesome Tech You Can’t Buy Yet: Robots that eat landmines and clean your floors

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

SpaceX experiences problem during test, Crew Dragon capsule may have exploded

SpaceX has experienced a problem during the testing of its Crew Dragon capsule. During the engine test firing at Cape Canaveral yesterday afternoon, an unspecified anomaly occurred which lead to plumes of smoke rising from the test site.
Emerging Tech

The grid of the future will be powered by … giant subterranean bagpipes?

In order to transition to a more renewable-focused energy system, we need to scale up our grid storage capacity --- and our existing methods aren't going to cut it. Could compressed air be the key?
Emerging Tech

Beresheet crash caused by manual command, but reflector device may have survived

Details are emerging about what may have gone wrong with spacecraft Beresheet's failed moon landing. A manual command was entered which led to a chain reaction. But NASA still hopes to salvage use of its Laser Retroreflector Array device.
Emerging Tech

The oldest type of molecule in the universe has been located at last

A milestone in the development of the early universe was the combination of helium and hydrogen atoms into a molecule called helium hydride. But strangely enough, this ancient molecule has never been detected in space before now.
Emerging Tech

Mercury’s wobble as it spins reveals that it has an inner solid core

Scientists have long wondered what the inside of Mercury looks like, and they now have strong evidence that the planet has a large and solid metallic core. The data for the new findings was collected by the now-defunct MESSENGER mission.
Emerging Tech

Gravitational forces at heart of Milky Way shaped this star cluster like a comet

Hubble has captured the stunning Messier 62 cluster. The cluster is warped, with a long tail which stretches out to form a shape like a comet. It is thought this distortion is due to Messier 62's proximity to the center of the galaxy.
Emerging Tech

Burgers are just the beginning: Embracing the future of lab-grown everything

You’ve almost certainly heard of the 'farm to fork' movement, but what about 'lab to table'? Welcome to the fast-evolving world of lab-grown meat. Is this the future of food as we know it?
Emerging Tech

Troubleshooting Earth

It’s no secret that humans are killing the planet. Some say it’s actually so bad that we’re hurtling toward a sixth major extinction event -- one which we ourselves are causing. But can technology help us undo the damage we’ve…
Emerging Tech

Inside the Ocean Cleanup’s ambitious plan to rid the ocean of plastic waste

In 2013, Boyan Slat crowdfunded $2.2 million to fund the Ocean Cleanup, a nonprofit organization that builds big, floating trash collectors and sets them out to sea, where they’re designed to autonomously gobble up garbage.