antisense ribonucleic acid
Quick Summary
Antisense ribonucleic acid (RNA) refers to RNA molecules that are complementary to normal coding sequences found in messenger RNAs (mRNAs), also known as sense RNAs. Unlike mRNAs, antisense RNAs cannot be translated into proteins.1
In nature, antisense RNAs function by binding to their complementary mRNA, forming a double-stranded structure that blocks the translation of the mRNA sequence, thereby inactivating the gene. This process is part of RNA interference (RNAi), a natural genetic mechanism in eukaryotic cells that controls gene activity by silencing specific genes.12
Synthetically modified derivatives of RNA or DNA, including antisense oligonucleotides (ASOs), are being developed for therapeutic purposes. These can selectively inactivate genes by interfering with normal RNA metabolism or by binding to target mRNA molecules and altering their function. ASOs are short chains of nucleotides that can influence gene expression and are used in treatments for various conditions, including Duchenne muscular dystrophy and spinal muscular atrophy.13
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