The concept of using antisense oligonucleotides to interfere with gene expression offers a
new therapeutic strategy for the treatment of diseases resulting from overexpression or
dysfunction of certain genes. Phosphorodiamidate morpholino oligomers (PMOs) represent
a neutral class of antisense agents that interfere with target gene expression either by
binding and sterically blocking the assembly of translation machinery, resulting in inhibition
of translation, or by altering splicing of pre-mRNA. Studies in animal models and human …