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Antisense technology

Antisense therapies are designed to seek out, bind to and destroy a mRNA in a highly specific manner, so that the amount of disease-causing protein is dramatically decreased. Antisense therapies can also treat diseases caused by too little protein by increasing the production of the protein, thereby restoring the protein to normal levels

5 Facts you should know



In nature, antisense RNAs play a critical role in regulating gene expression at multiple levels, including replication, transcription, and translation



Antisense RNAs are unique DNA transcripts - they are small, noncoding, and diffusible molecules, containing 19–23 nucleotides that complement mRNA



After observing antisense-mediated RNA regulation in nature, researchers tested the feasibility of using synthetic antisense oligonucleotides to reduce levels of specific mRNA transcripts



Antisense oligonucleotides (ASOs) can bind to abnormal mRNA, preventing its translation into a harmful protein



By targeting the source of the pathogenesis, ASO-mediated therapies theoretically have a higher chance of success than therapies targeting downstream pathways

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Antisense technology is also known as...

Antisense technology is also known as:

  • Antisense oligonucleotides
  • ASOs

What’s your Rare IQ?

Which of these rare diseases has several antisense therapies approved for treatment?