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Possibilities and limitations of antisense oligonucleotide therapies for the treatment of monogenic disorders

5 Jan 2024

Abstract excerpt

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 progression. They offer the potential to target groups of patients or can be tailored for individual cases. Nonetheless, not all genetic variants and disorders are amenable to ASO-based treatments, and hence, it is important to...

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PMID: 38182878
DOI: 10.1038/s43856-023-00419-1

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