Future treatment
for alpha-1 antitrypsin deficiency
Several possible treatments for AATD liver disease are being tested. They target the genetic problem that causes AATD. Or the process that makes the faulty AAT protein.
The information on this page is for:
- Adults and children with A1ATD or suspected A1ATD.
- Family, friends, carers, and healthcare professionals.
New types of genetic treatment mean there is a lot of research going on at the moment. We will update this information when new trials or results are published.
If you might be interested in taking part in research, talk to your AATD specialist. They should be able to tell you about any research that might be suitable for you.
On this page:
DNA editing
The AAT gene is a section of DNA. It carries the instructions for making the AAT protein. The Z version of the AAT gene has a mistake in it. This is like a spelling mistake. It means that the instructions are wrong, so the AAT protein that is made in the wrong shape.
Researchers are testing a DNA treatment that travels to the liver cells. Once in the cells, it snips out and corrects the mistake in the AAT gene.
Researchers hope that this will mean:
- Less of the misshaped protein will be made so it will not build up in the liver and cause damage.
- More of the normal protein will be made, so it will be able to protect the lungs.
Early trials have gone well. The treatment has been tested in a small number of people who do not have liver disease. Based on these early tests, the treatment seems to be safe and to work.
A lot more testing is needed. Including tests in people who already have AATD liver disease.
RNA editing
When a liver cell needs to make a protein, it makes a copy of the instructions using a molecule called RNA. If there is a mistake in the gene, the RNA carries this mistake.
Several studies are testing treatments that target the RNA carrying instructions from the Z allele.
The treatments aim to correct the mistake in the RNA. This should mean that the correct version of the AAT protein is made.
As with the DNA treatment, this could prevent liver damage. It could also increase the amount of AAT protein protecting the lungs.
These treatments are at an early stage of testing. But results so far have been good.
siRNA
Instead of editing a mistake. Another option could be to stop the RNA message with the mistake from being delivered.
Researchers are testing a molecule called siRNA. This stands for “small interfering RNA”.
The siRNA targets RNA in the liver that is carrying faulty AAT instructions.
Results so far have been good. People on the trial were found to have less of the faulty protein in their blood. They also had less of the faulty protein building up in the liver.
This type of treatment does not help the lungs because it does not lead to more of the normal protein.
But this new treatment could be helpful for people who don’t have lung problems. Or people with lung conditions that are well controlled with other treatments.
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This content was last reviewed: June 2026
Our expert reviewers:
We would like to thank everyone who helped with creating and reviewing this page. Including Dr Bill Griffiths, consultant hepatologist, Cambridge university hospitals, Professor Dino Hadzic, consultant paediatric hepatologist, King’s college hospital, Dr Girish Gupte, consultant paediatric hepatologist, Birmingham children’s hospital. And all our patient and family reviewers.
Liver UK thanks Takeda UK for their kind donation to support the development of this information. Takeda UK has had no influence in the initiation, development or content of this project.
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Everyone’s experience of liver disease will be different. Always talk to your specialist medical team for personal advice.
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