Highlights•Recent human clinical trial data on RNAi approaches to gene down-regulation for
genetic diseases.•Overview of current anti-microRNA drugs with specific emphasis on
application to target Hepatitis C infection.•Update on the current state of 'exon skipping'field
for both Duchenne muscular dystrophy and spinal muscular atrophy.Despite the discovery
more than two decades ago that antisense oligonucleotides (ASOs) could be used to
modulate protein expression, there have been only two antisense drugs approved for …