DMD gene therapy

An MDA-supported research team has found in animal experiments that plasmapheresis can reduce the number of unwanted antibodies to gene therapy delivery vehicles

posted on December 6, 2013 - 2:45pm
A research group supported in part by MDA has found that a blood-filtering strategy known as plasmapheresis looks promising as a way to help overcome one type of unwanted immune response to gene transfer therapy. Gene transfer therapy is the addition of therapeutic genes to treat disease, and it is often administered via transport vehicles made from viruses. Louis Chicoine at Nationwide Children'...
posted on March 1, 2008 - 10:21am
QUEST Vol. 15, No. 2
A European group of researchers successfully used a combination of genetic correction and stem cells to treat Duchenne muscular dystrophy (DMD) in mice with the disease. The compound they used for the genetic correction was developed by Judith van Deutekom, then at Leiden (Netherlands) University Medical Center, with MDA support. DMD results from the loss of the muscle protein dystrophin because...
posted on March 1, 2005 - 4:13pm
In this issue: An MDA-supported gene therapy safety trial gets federal approval to move forward ** Sudden cardiac death found in people with MMD2, and those with MMD1 often require a pacemaker ** Etanercept shows promise in mysasthenia gravis
posted on June 1, 2001 - 2:36pm
In this issue: research news on Pompe disease, Becker and Duchenne MDs, Charcot-Marie-Tooth disease, limb-girdle and myotonic MDs, spinal bulbar muscular atrophy, myasthenia gravis and nemaline myopathy