gene therapy in DMD

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.
posted on July 1, 2010 - 4:03pm
QUEST Vol. 17, No. 3
SMA Research: Raising SMN Levels Three Ways Recent research in spinal muscular atrophy (SMA) has concentrated on three different ways to raise levels of the protein known as SMN that’s required for the health of nerve cells that control muscle function.
posted on May 1, 2007 - 8:47pm
QUEST Vol. 14, No. 3
Items in this article report on clinical trials in: Duchenne muscular dystrophy and Friedreich's ataxia.