How Do Antisense Oligonucleotides Work?

Antisense oligonucleotides are artificially created pieces of DNA used for the treatment of disorders involving gene mutation.

Generally, to produce a protein, a gene is first copied into a molecule called messenger RNA. The messenger RNA serves as a template for the cells to make the corresponding protein.

Antisense oligonucleotides bind to this messenger RNA and prevent its ability to make protein. In this way, the production of faulty protein is obstructed. In short, antisense oligonucleotides may be used to block the production of proteins needed for cell growth.

Antisense oligonucleotides are used for the treatment of:

• Duchenne muscular dystrophy
• Spinal muscular dystrophy
• Huntington’s disease

Antisense oligonucleotides are being studied in the treatment of several types of cancer.

Antisense oligonucleotides, when injected, can cause the following side effects:

• Upper respiratory tract infection
• Injection site reaction
• Cough
• Fever
• Joint pain
• Diarrhea
• Vomiting
• Abdominal pain
• Ejection fraction decrease
• Rash
• Headache
• Ear pain
• Nausea
• Ear infection
• Post-traumatic pain
• Dizziness
• Lightheadedness

The information contained herein is not intended to cover all possible side effects, precautions, warnings, drug interactions, allergic reactions, or adverse effects. Check with your doctor or pharmacist to make sure these drugs do not cause any harm when you take them along with other medicines. Never stop taking your medication and never change your dose or frequency without consulting your doctor.

Generic and brand names of antisense oligonucleotides include:

• Amondys 45
• Casimersen
• Eteplirsen
• Exondys 51
• Golodirsen
• Nusinersen
• Spinraza
• Viltepso
• Viltolarsen
• Vyondys 53