revertant fibers

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.

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