The Future of Medicine: Gene Therapy and IMDs

The future of medicine: Gene therapy and IMDs

Overview

Gene therapy involves altering the genes inside your body's cells in an effort to treat or stop disease.

Genes contain your DNA — the code that controls much of your body ' s form and function, and in most Inherited Metabolic Disorders, faulty genes results in an enzyme deficiency.

How does gene therapy work?

Gene therapy is the transfer or editing of a genetic material to cure a disease. Depending on the delivery strategy chosen, gene therapy can be performed in vivo (inside the body) or ex vivo (outside the body) with integrating (i.e. permanent modification of the host genome) or non-integrating (e.g. through viral vectors which are modified versions of a virus that is different from the virus being targeted to deliver important instructions to our cells.

What do you mean...In vivo?

In vivo gene therapy refers to the injection of a vector encoding the gene of interest or molecular tools for gene editing, directly into a tissue or into the bloodstream to generate therapeutic outcomes in specific or multiple organs.

What do you mean...Ex vivo?

Ex vivo gene therapy involves manipulation of a target cell population outside of the body, often as part of stem cell therapies, in which a patient’s own cells are genetically modified with gene editing or gene supplementation and then inserted back into the patient

What are the benefits?

Gene therapy aims to address the non-functioning gene and provide a long-term treatment benefit with potentially just one dose.

This means people living with an IMD could receive gene therapy once and have all symptoms removed permanently*, allowing for a better quality of life, whilst also ensuring people can live longer.

Okay...sounds good but what are the potential negative effects?

Unwanted immune system reaction: This may cause inflammation and, in severe cases, organ failure.

Targeting the wrong cells: If this happens, healthy cells may be damaged, causing other illness or diseases, such as cancer.

Infection caused by the virus: It's possible that once introduced into the body, the viruses may recover their original ability to cause disease.

Possibility of causing a tumour: If the new genes get inserted in the wrong spot in your DNA, there is a chance that the insertion might lead to tumour formation.

*Despite these potential negative outcomes, gene therapy undergoes rigorous testing before becoming available, meaning any concerns for safety are monitored and addressed accordingly.