Respected Medical Geneticist Sir Peter Harper Dies at 81
Respected Medical Geneticist Sir Peter Harper Dies at 81
The Cardiff University researcher was famous both for his work on genetic disorders and for his documentation of the history of his field.
The Cardiff University researcher was famous both for his work on genetic disorders and for his documentation of the history of his field.
Muscular Dystrophy
Trial of Gene Therapy for Duchenne Muscular Dystrophy Put on Hold
Catherine Offord | Nov 12, 2019 | 2 min read
The US Food and Drug Administration halts a study by Solid Biosciences after a patient experiences severe side effects following treatment.
Image of the Day: Dystrophin Restored
Carolyn Wilke | Mar 21, 2019 | 1 min read
CRISPRed heart muscle cells from humans gain the ability to make a protein missing in Duchenne muscular dystrophy.
Infographic: Directing Exosome Traffic
Ruth Williams | Sep 1, 2018 | 1 min read
Researchers harness a protein to send cargo-carrying exosomes to mouse muscles.
CRISPR Treatment for Duchenne Muscular Dystrophy Helps Dogs
Catherine Offord | Aug 31, 2018 | 2 min read
Researchers boosted levels of the dystrophin protein to nearly normal levels in a canine model of the disease.
Positive Trial Results for Experimental DMD Gene Therapy
Catherine Offord | Jun 20, 2018 | 2 min read
Preliminary data from Sarepta Therapeutics has exceeded expectations, causing a surge in company stock prices this week.
Nonviral CRISPR Delivery a Success
Abby Olena, PhD | Oct 2, 2017 | 3 min read
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
Diana Kwon | Jul 25, 2017 | 1 min read
Researchers restored muscle function in animals with muscular dystrophy.
CRISPR Restores Muscle Function in Mice
Diana Kwon | Jul 17, 2017 | 2 min read
Scientists use the gene-editing tool to treat animals with a rare form of congenital muscular dystrophy.