Future gene therapy trials in Duchenne muscular dystrophy may need to assess participants' immunity to dystrophin, recent findings show
posted on October 6, 2010 - 2:00pm
Editor's note: This story was updated April 27, 2012.
Immune-system rejection of newly synthesized dystrophin protein occurred in at least some of the boys with Duchenne muscular dystrophy (DMD) who participated in a safety trial of dystrophin gene therapy.
The finding was a partial surprise to researchers and demonstrates the value of small, phase 1 clinical trials.