Gene transfer therapy

What is gene transfer therapy?

Gene transfer therapy or gene replacement therapy is type of gene therapy where new genetic material is introduced into specific cells to help treat genetic conditions.

A common way to deliver the therapy to these cells is via a viral vector.

How gene transfer therapy works

The risks and benefits of gene transfer therapy

Checklists

FAQs

Do viral vectors make people sick?

Scientists engineer the vector by changing it from a vehicle for a virus, to a shell that delivers what they want instead.

The viruses used as vectors are not usually linked with disease in humans, however it can trigger an immune response. Your doctor will discuss these with you in relation to specific gene therapies.

Further, each vector goes through rigorous tests before it is ever considered for human trials.

If someone received gene transfer therapy, can they receive more later?

Once a person received viral mediated gene therapy, the immune system responds to the virus that delivers the gene.

This means that further doses of the same gene therapy will not be effective.

Is there a difference between the viral vector in gene therapy and the AstraZeneca COVID-19 vaccine?

Gene therapy and the AstraZeneca COVID-19 vaccine both use packages, called vectors, to deliver genetic messages.

However, the packages and messages are different and therefore the side effects are also different. It is very unlikely that the two (given with time between) will have problems.

Further, the genetic material delivered in the COVID-19 vaccine is different to the genetic material delivered in gene therapy.

Latest news

Baby smiling
Babies diagnosed with Spinal Muscular Atrophy at birth who received life-changing gene therapy are now living longer and healthier, celebrating their first birthday symptom-free or with minimal symptoms.