How Do Rheumatologic Antisense Oligonucleotides Work?

Reviewed on 11/3/2021

How Do Antisense Oligonucleotides Work?

Antisense oligonucleotides are medications to treat transthyretin (TTR) amyloidosis, a condition caused by hereditary mutation in TTR gene that encodes the TTR protein. Transthyretin amyloidosis affects multiple organs including peripheral nerves, heart, eyes, kidneys, thyroid, bone marrow, gastrointestinal and central nervous systems.

Transthyretin is a soluble protein synthesized by the liver that helps transport vitamin A (retinol) and thyroid hormone (thyroxine) in the body. In people with transthyretin amyloidosis, TTR proteins become insoluble fibers (fibrils) resistant to degradation and build up in tissues of multiple organs, interfering with their normal functioning.

Antisense oligonucleotides bind to the TTR protein’s messenger RNA (mRNA), which is vital for the protein’s synthesis, preventing the synthesis of the mutant protein in the liver, which results in the reduction of TTR protein in blood and TTR deposits in tissues.

How Are Antisense Oligonucleotides Used?

Antisense oligonucleotides are administered as subcutaneous injections into the tissue under the skin, approved by the FDA for the treatment of adults with polyneuropathy (damage to multiple peripheral nerves) associated with hereditary transthyretin amyloidosis (hATTR).

What Are Side Effects of Antisense Oligonucleotides?

Side effects of antisense oligonucleotides may include the following:

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.


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What Are Names of Antisense Oligonucleotide Drugs?

Generic and brand names of the FDA-approved antisense oligonucleotide drug are:


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