Gene therapy is still in development to test its safety and effectiveness, but it may be a promising new treatment for rheumatoid arthritis (RA).

Gene therapy alters certain genes to help treat certain disorders. It may involve replacing an atypical gene with a new copy, deactivating a gene that is causing disease, or delivering a new or adapted gene into the body.

This article explains the procedure, effectiveness, and possible risks of gene therapy for RA and who it may suit.

A doctor examining a person's palm and wrist.-1Share on Pinterest
Igor Alecsander/Getty Images

According to a 2021 articleTrusted Source, the goal of gene therapy for RA is:

  • achieving remission or low level disease activity rather than just relieving pain
  • preventing irreversible damage to the affected joints
  • avoiding some of the side effects and limitations of current RA treatments and yielding long-term results

Most current treatments for RA only provide short-term pain relief, and some cases of RA may not respond to antirheumatic drugs. Drugs that target the whole body to treat RA may cause significant side effects, such as suppressing the immune system.

RA causes inflammation of the affected joint. The earlier 2021 article suggests gene therapy for RA would target this inflammation. The therapy would deliver a gene with anti-inflammatory effects to relieve the joint inflammation.

There are various methods of delivering a gene into the body, but researchers found that viral vectors are the only suitable option for RA gene therapy.

Viral vectors use a virus to deliver genes into cells in the body. A laboratory alters the virus first so it is unable to cause an infection.

The 2021 article suggests adeno-associated virus (AAV) as the best viral vector to use, as it is safe, effective, and less likely to cause an immune response within the body.

Gene therapy is still in the experimental stages as a potential treatment for RA. Clinical trials are necessary to ensure gene therapy with viral vectors for treating RA is safe and effective.

This technique may have risksTrusted Source, although research suggests AAV does not cause disease.

If people want to find out more about gene therapy for RA, they can talk with a doctor about participating in clinical trials.

There is currently no guidance as to who is a suitable clinical trial candidate for RA gene therapy. However, the stage of disease and pain severity may be the most important factors.

Research to investigate the safety and effectiveness of gene therapy for RA in clinical trials is ongoing.

In theory, the procedure may involveTrusted Source intra-articular injections of the viral vector and therapeutic gene. Intra-articular means the injection goes into the affected joint.

Doctors may use an ultrasound to guide them during targeted joint injections. They may also use a type of imaging called fluoroscopy in an X-ray suite. However, ultrasound may be preferable as a doctor can use it at the point of care, and the method involves no radiation exposure.

Researchers are still investigating which joint is best to inject into. Although RA affects the wrist joint in up to 75% of cases, injections into the wrist may cause complications, which may be harder to treat than in other joints.

For this reason, some researchers suggest using the knuckle joint in trials, as any complications may be easier to treat.

According to a 2020 articleTrusted Source, animal studies found that gene therapy involving the IL-4 and IL-10 genes reversed cartilage degradation and protected the joints, even with high levels of inflammation.

Clinical trials have not shown these genes to be very effective on their own, but the delivery of both genes may be a promising treatment for RA.

In some cases, gene therapy with certain genes may have unpredictable results and may potentially worsen RA. Researchers require further studies in humans to better understand the safety and effectiveness of gene therapy for RA.

A potential benefitTrusted Source of gene therapy for RA is that it is long lasting, so people may not need repeat injections or daily medication.

Further studies and clinical trials are necessary to understand the possible risks of gene therapy for RA. Potential risks or complications may includeTrusted Source:

  • injections into the synovial tissue, which lines the inside of joints, may increase the risk of infections, while the synovial tissue has a reduced ability to fight infections
  • septic arthritis due to bacteria from the surface of the skin entering a joint as the needle goes into the body
  • ruptured tendon or nerve damage due to misplacement of an injection
  • side effects from the viral vector, such as a hyperinflammatory response

Risks may depend on which joint the injection is going into. In certain joints, complications may be easier to treat, such as removing synovial tissue with an infection or replacing a damaged joint.

Gene therapy for RA may be a potential treatment option in the future, but current treatment options for RA includeTrusted Source:

If people want to learn more about gene therapy for RA, they can talk with a doctor about the possibility of participating in RA gene therapy clinical trials.

It is important to discuss the potential risks and complications of taking part in gene therapy trials and to weigh these up with potential benefits.

If people experience severe pain or disability with RA, a doctor may be able to suggest different treatment options or newer treatments in other clinical trials.

In theory, gene therapy could deliver anti-inflammatory genes into the affected joint to target inflammation, reduce disease activity, or achieve remission.

However, researchers are still testing gene therapy for rheumatoid arthritis (RA) to find out if it could be a safe and effective treatment for the condition in humans.

People can speak with a healthcare professional to find out more about gene therapy for RA and whether they may be suitable for a clinical trial.