Abstract The last decade (2013–2023) has seen unprecedented successes in the clinical
translation of therapeutic antisense oligonucleotides (ASOs). Eight such molecules have …

Antisense oligonucleotides (ASOs) can modulate pre-mRNA splicing. This offers therapeutic
opportunities for numerous genetic diseases, often in a mutation-specific and sometimes …

Antisense oligonucleotide (AON) therapies involve short strands of modified nucleotides that
target RNA in a sequence‐specific manner, inducing targeted protein knockdown or …

In the past few years, research in targeted mutation therapies has experienced significant
advances, especially in the field of rare diseases. In particular, the efficacy of antisense …

Although around 6% of the world's population is affected by rare diseases, only a small
number of disease‐modifying therapies are available. In recent years, antisense …

Antisense oligonucleotides (ASOs) are incredibly versatile molecules that can be designed
to specifically target and modify RNA transcripts to slow down or halt rare genetic disease …

The number of mutations identified deep in introns which activate or create novel splice sites
resulting in pathogenic pseudoexon inclusion in mRNA continues to grow for inherited …

Antisense oligonucleotides (ASOs) are single-stranded nucleic acid strings that can be used
to selectively modify protein synthesis by binding complementary (pre-) mRNA sequences …

Synthetic oligonucleotide technologies are DNA or RNA based molecular compounds that
are utilized to disrupt gene transcription or translation in target tissues or cells. Optimally …

Antisense oligonucleotides (ASOs) bind sequence specifically to the target RNA and
modulate protein expression through several different mechanisms. The ASO field is an …