A technique known as exon skipping, in which a molecular patch is inserted in a defective gene in patients with Duchenne muscular dystrophy, may safely halt the muscle degeneration associated with this lethal X-linked disorder, investigators reported. Administration of a splice-switching oligomer that can “skip over” the damaged exon and preserve the genetic ability of the muscle to produce dystrophin resulted in biochemical responses in seven of 19 patients, according to Francesco Muntoni, MD, of the UCL Institute of Child Health in London, and colleagues.
