What is gene therapy?

What is gene therapy?

Gene therapy offers the possibility of correcting genetic errors. It is a technique that has already shown great potential. UCL is acknowledged as a leader in this exciting and dynamic field.

In this interview, we meet Dr Simon Waddington, who works at UCL’s Institute for Women’s Health, leading a team of scientists who use stem cell transfer to correct diseases of early childhood. Simon explains the nature of the work he does and shows examples of what he and his team do at UCL.

How is gene therapy used?

Technically speaking, gene therapy is the use of a biological product that is administered to an individual to correct a faulty genetic sequence. It aims to alter the genetic sequence at a specific known location in an individual’s DNA.

Gene therapy can repair or replace parts of a specific gene sequence and therefore treat the defect that is causing the condition by providing a healthy copy of the faulty gene.

The steps of gene therapy (adenovirus)

Unlike a lot of medicine that focuses on symptom-limitation, gene therapy offers something unique because it treats the underlying cause, the genetic defect.

The kinds of diseases that have been the target of gene therapy are heritable blood disorders such as haemophilia, certain types of cancer, and debilitating genetic disorders of childhood, like cystic fibrosis.

The promise of gene therapy is especially significant where current medical treatments are inadequate or unavailable.

Advantages of gene therapy

There are several reasons why fetal or neonatal gene therapy can be advantageous. Prevention is better than a cure in conditions where irreversible organ damage has already begun when the baby is born.

Therefore, if this condition is anticipated and treated with gene therapy before birth this can improve their prognosis and potentially prevent further disease-related decline.

Gene therapy is more effective in fetuses and newborns as they have a smaller mass and have a large population of cells that can be targeted.

In the UK, two main regulatory bodies of gene therapy exist The Gene Therapy Advisory Committee (GTAC), and the Medicines and Healthcare Products Regulatory Agency (MHRA).

If you’d like to learn more about gene therapy, check out the full online course from UCL, below.