TrialLineage Concept
RNA biology
RNA is the molecular intermediary between DNA and protein. Beyond its role as a messenger, RNA participates in gene regulation, catalysis, and cellular signaling. Understanding RNA biology is foundational to the development of RNA-targeted therapies — including antisense oligonucleotides, siRNAs, and mRNA medicines.
In plain language
What is RNA?
DNA stores genetic instructions, but those instructions must be converted into action. RNA performs this conversion: messenger RNA (mRNA) carries gene sequences from the nucleus to ribosomes, where proteins are built. Other RNA types — transfer RNA, ribosomal RNA, and regulatory non-coding RNAs — perform structural, catalytic, and regulatory roles.
Non-coding RNAs are particularly relevant to disease: they can silence genes, regulate protein production, and control developmental programs. When these regulatory RNAs malfunction or can be therapeutically manipulated, they become potential drug targets.
Why RNA biology matters for medicine
RNA-targeted therapies work by intervening at the RNA level — before a protein is ever made. This allows treatments to address diseases caused by toxic proteins, missing proteins, or dysregulated gene expression. The discovery that antisense oligonucleotides and siRNAs can selectively degrade or block specific RNA transcripts opened a new class of medicines.
In Angelman syndrome, the therapeutic target is a long non-coding antisense RNA (UBE3A-ATS) that silences the paternal UBE3A gene in neurons. Reducing this transcript is the mechanism by which ION582 aims to restore UBE3A expression.
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