Antisense oligonucleotide-mediated splicing modulation is an attractive strategy for treating
genetic disorders. In 2016, two splice-switching oligonucleotides (SSOs) were approved by …

Splice-switching oligonucleotides (SSOs) are short, synthetic, antisense, modified nucleic
acids that base-pair with a pre-mRNA and disrupt the normal splicing repertoire of the …

Alternative splicing enables a single pre-messenger RNA transcript to yield multiple protein
isoforms, making it a major contributor to the diversity of the proteome. While this process is …

Antisense-mediated modulation of pre-mRNA splicing is an attractive therapeutic strategy for
genetic diseases. Currently, there are few examples of modulation of pre-mRNA splicing …

Antisense oligonucleotide (AON)-based splice modulation has been proven to hold great
promise as a therapeutic strategy for a number of hereditary conditions. AONs are small …

Today, there are emerging numbers of oligonucleotide therapies being approved by the
governmental authorities. These types of therapies present a different mode of action when …

Synthetic antisense oligonucleotides (ASOs) have emerged as one of the most promising
therapeutic approaches. So far, nine ASO drugs have received approval for clinical use, and …

The splicing of precursor messenger RNA (pre-mRNA) requires the precise cleavage and
formation of multiple phosphodiester bonds in nascent pre-mRNA polymers in order to …

Splice-switching antisense oligonucleotides (ASOs), which bind specific RNA-target
sequences and modulate pre-mRNA splicing by sterically blocking the binding of splicing …

Antisense oligonucleotides (AO) or antisense RNA can complementarily bind to a target site
in pre-mRNA and regulate gene splicing, either to restore gene function by reprogramming …