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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

FACT

1

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

FACT

2

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

FACT

3

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

FACT

4

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

FACT

5

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?