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Genetics and inheritance

Antisense oligonucleotide

A treatment approach that works by reducing how much of a particular protein the body makes.

What it means

A short piece of engineered genetic material binds to the cell's message for making one specific protein and changes how much is produced. In ALS/MND the aim is usually to make less. Because it targets one gene's message, it is only relevant to people with a change in that gene.

Why you might hear it

  • It is the approach behind several gene-targeted treatments and trials.
  • It usually has to be given into the fluid around the spinal cord.

People who may help

Where Compass goes into this

About this explanation

These are plain explanations of words you may come across, not advice about your own situation. Where a word matters to a decision, the question pages go into it properly, and your own team is the place to take it.