The Promising Future of Gene Therapy with Duchenne Muscular Dystrophy
Controlling new dystrophin genes
One of the most recent developments involves the usage of a muscle-specific promoter, which is like a control switch intended to control the new dystrophin genes. These promoters activate genes in the muscle selectively while making sure other genes in tissues remain dormant. This is done to avoid the unwanted side effect. The study is now led by Dr. Jerry Mendell but it is still in phase one and two in clinical testing.
