Muscular Dystrophy

A new gene therapy for Duchenne muscular dystrophy (DMD) has shown promise in not only slowing the progression of the disease but potentially even reversing the muscle damage, with human trials set to begin within two years.

In a new study, researchers have accidentally discovered that an existing cancer drug shows promise in slowing the progress of Duchenne Muscular Dystrophy, by changing the type of muscle fibers to be more resilient.

By tweaking the CRISPR gene-editing technique to target RNA instead of DNA, researchers have been able to destroy toxic accumulations of this molecule, which could pave the way for new treatments for muscular dystrophy and related conditions.

“Use it or lose it” applies to muscles, but unfortunately so does “don’t use it too much or lose it.” Now, researchers at Temple University have tested a drug that appears to reverse muscle damage from overuse injuries in rats.

Duchenne muscular dystrophy (DMD) is one of the most common and most devastating muscular diseases. Now researchers have managed to use the CRISPR gene-editing tool to correct the condition in pigs, bringing the treatment ever closer to human trials.

In recent years scientists have been able to correct genetic diseases by removing stem cells, editing their genomes and implanting them back into patients, but that adds complications. Now new research led by Harvard scientists has successfully edited the genes of stem cells while still in the body.

Georgia Tech researchers have developed a hydrogel that holds muscle stem cells, and by injecting this near the site of a muscle injury they can get to work repairing it. The team says the technique could be effective at treating injuries in the elderly and people with muscular dystrophy.

Researchers have developed a CRISPR technique to efficiently correct the function of heart cells in​ patients with Duchenne muscular dystrophy (DMD). It involves making a single cut at strategic points along patient's DNA and has the potential to correct most of the 3,000 mutations that cause DMD.

CRISPR-Cas9 shows incredible promise, but the long term effects of cutting DNA in living organisms isn’t known. Now scientists from the Salk Institute have modified it to work without cutting, switching targeted genes on and off instead, and used it to treat diabetes and muscular dystrophy in mice.

Duchenne muscular dystrophy (DMD) is the most severe form of the debilitating genetic disorder. Researchers have used a new gene therapy technique to restore muscle strength and stabilize the symptoms of the disorder in dogs, in an important step towards the treatment being applied to children.

Duchenne muscular dystrophy causes the victim's muscles to shrink throughout their lifetime, often to the point that the arms and legs can't be used at all. That's why the A-Gear project is designing two arm-worn exoskeletons, intended to help Duchenne patients retain the use of their arms.

Scientists have developed a new procedure to increase the length of human telomeres. This increases the number of times cells are able to divide, which may point the way to treating various age-related disorders – or even muscular dystrophy.