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What is Gene Therapy?

Gene therapy

Gene therapy is the use of DNA to treat or prevent diseases. Instead of merely treating the symptoms of a genetic disorder like cystic fibrosis, gene therapy aims to get to the root of the problem by modifying the patient's DNA.

The idea of replacing disease-causing DNA with healthy DNA has been around since at least 1970, and experimental treatments have been going on for more than thirty years. There have been some very encouraging results in that time, and effective treatments have been developed for an array of disorders ranging from HIV to vision loss.

That said, gene therapy is still a relatively new field of study, and we are not yet living in a world where gene modification treatments are commonplace and widely available. Who knows what solutions might be engineered in the future, and how many lives they might improve?

How does gene therapy work?

Gene therapy may be used to combat disease in a number of different ways. For example:

  • A gene that isn't functioning properly may be deactivated (or 'knocked out') to prevent health problems.

  • A mutated gene may be replaced with a healthy copy of the same gene.

  • A new or modified gene may be introduced to help the patient's body fight a disease.

Delivery methods vary - scientists will sometimes engineer a retrovirus that changes the patient's genome to achieve the desired result, but in other cases, 'naked' DNA may be delivered via injection or other techniques.


What are some examples of gene therapy?

Here are some examples of gene therapy treatments that are already in use:

  • Talimogene laherparepvec, better known by the brand name Imlygic, can be used to treat melanoma - a particularly deadly type of skin cancer. This gene therapy was approved by the European Medicines Agency (EMA) and the United States Food and Drug Administration (FDA) in 2015.

  • Tecartus is a gene therapy that targets mantle cell lymphoma and acute lymphoblastic leukaemia. It is manufactured by Kite Pharma and received EMA and FDA approval in 2020.

  • Zolgensma, manufactured by American biotech company Novartis Gene Therapies, is used to treat spinal muscular atrophy (SMA). It was used in the UK for the first time in 2021.

What jobs are involved in gene therapy?

Successful gene therapies are dependent on the hard work and expertise of many people in many different roles. Here are just some of the careers available in this field:

  • Research and development
  • Upstream and downstream processing
  • Project management
  • Quality assurance
  • Technology transfer
  • Equipment and facilities validation
  • Clinical trial management

Browse Cell & Gene Therapy Jobs

Last updated 6 October 2021. Photo from Pixabay.