Trial of Gene Therapy for Duchenne Muscular Dystrophy Put on Hold
Trial of Gene Therapy for Duchenne Muscular Dystrophy Put on Hold
The US Food and Drug Administration halts a study by Solid Biosciences after a patient experiences severe side effects following treatment.
Trial of Gene Therapy for Duchenne Muscular Dystrophy Put on Hold
Trial of Gene Therapy for Duchenne Muscular Dystrophy Put on Hold

The US Food and Drug Administration halts a study by Solid Biosciences after a patient experiences severe side effects following treatment.

The US Food and Drug Administration halts a study by Solid Biosciences after a patient experiences severe side effects following treatment.

Duchenne muscular dystrophy
Image of the Day: Dystrophin Restored
Image of the Day: Dystrophin Restored
Carolyn Wilke | Mar 21, 2019
CRISPRed heart muscle cells from humans gain the ability to make a protein missing in Duchenne muscular dystrophy.
Putting Exosomes to Work
Putting Exosomes to Work
Ruth Williams | Sep 1, 2018
Researchers identify a handy tool for tinkering with the versatile vesicles.
Infographic: Directing Exosome Traffic
Infographic: Directing Exosome Traffic
Ruth Williams | Sep 1, 2018
Researchers harness a protein to send cargo-carrying exosomes to mouse muscles.
CRISPR Treatment for Duchenne Muscular Dystrophy Helps Dogs
CRISPR Treatment for Duchenne Muscular Dystrophy Helps Dogs
Catherine Offord | Aug 31, 2018
Researchers boosted levels of the dystrophin protein to nearly normal levels in a canine model of the disease.
Infographic: Treating Duchenne Muscular Dystrophy with CRISPR
Infographic: Treating Duchenne Muscular Dystrophy with CRISPR
Sandeep Ravindran | Aug 30, 2018
The disease is caused by mutations in a single gene. Can gene editing fix the problem?
Gene Editing Could One Day Treat Muscle Disorders
Gene Editing Could One Day Treat Muscle Disorders
Sandeep Ravindran | Aug 30, 2018
Scientists race to develop CRISPR therapies that could save the lives of kids with muscle-wasting conditions.
Positive Trial Results for Experimental DMD Gene Therapy
Positive Trial Results for Experimental DMD Gene Therapy
Catherine Offord | Jun 20, 2018
Preliminary data from Sarepta Therapeutics has exceeded expectations, causing a surge in company stock prices this week.
Nonviral CRISPR Delivery a Success
Nonviral CRISPR Delivery a Success
Abby Olena | Oct 2, 2017
Researchers use gold nanoparticles to deliver CRISPR-Cas9 and correct a point mutation in a mouse model of Duchenne muscular dystrophy. 
Dogs with Duchenne Treated with Gene Therapy
Dogs with Duchenne Treated with Gene Therapy
Diana Kwon | Jul 25, 2017
Researchers restored muscle function in animals with muscular dystrophy.