Duchenne muscular dystrophy (DMD) and spinal muscular atrophy (SMA) are two of the
most common inherited neuromuscular diseases in humans. Both conditions are fatal and
no clinically available treatments are able to significantly alter disease course in either case.
However, by manipulation of pre-mRNA splicing using antisense oligonucleotides, defective
transcripts from the DMD gene and from the SMN2 gene in SMA can be modified to once
again produce protein and restore function. A large number of in vitro and in vivo studies …