HUMAN beings each have an estimated 24,000 different genes, but if just one goes wrong it can spell serious problems – that’s where gene therapy comes in.

By manually replacing or de-activating faulty genes, it’s possible to correct some genetic defects, helping to treat and potentially even cure genetic diseases, from cancers to Alzheimer’s.

Genes are small sections of DNA which contain a unique code. This code acts as an instruction to the cell to trigger different characteristics or processes.

If the code is faulty, the cell will be faulty too: so even tiny defects can lead to a range of life-changing genetic conditions, including cystic-fibrosis, muscular dystrophy, sickle cell disease and many more.

While we can treat many genetic conditions, it’s not straightforward to cure them. That’s because the issue is rooted right at the core of the cell’s DNA: in our genes. But what if it was possible to edit these genes? If we could get to them then we could potentially “fix” the genetic defect, thus eliminating the disease.

Gene therapy does just that. It works by inserting new genes into a patient’s DNA.

These new genes can replace or shut off existing genes that are defective, allowing the cell to function normally.

Gene therapy is still in its early stages, but it appears to be a promising new line of treatment for diseases that are otherwise incurable. And Oxfordshire’s world-leading science infrastructure is helping to support pioneering research in this field with some fantastic results.

The process of transporting therapeutic genes into a patient is central to the effectiveness of gene therapy, and many different options are being explored. Recent research in Oxfordshire’s science facilities has uncovered a potential candidate for gene transportation in an interesting place: human breast milk.

Breast milk contains a special antimicrobial protein called Lactoferrin – this protein helps to protect feeding infants from all sorts of infections. But scientists have now found that, by rearranging fragments of Lactoferrin, it’s possible to assemble a virus-like capsule capable of penetrating human cells.

Viruses survive by invading host cells and then releasing genetic information inside. The virus hijacks the cell’s infrastructure so that it is forced to replicate this genetic information again and again: that’s how viruses are able to spread through the body.

By entering human cells, the Lactoferrin capsule functions like a virus but, instead of harmful viral genes, it contains therapeutic and potentially life-saving ones. The capsule was found to successfully deliver genes, altering a target process within the cell.

This finding could mark a significant early step towards treating a range of genetic diseases with the capsules.

It’s not just capsules that scientists are investigating in the search for gene transportation candidates. Actual viruses, including herpes and HIV, have shown promise as carriers for gene therapy. Nanoparticles, electric fields and other molecular carriers are also being explored.

Gene therapy is a relatively young field of research and there’s still a long way to go before we see it widely clinically available. There are also important questions that need to be asked about the practical and ethical implications of editing human genes, particularly if those genes may be passed down to offspring.

From “designer babies” to enhanced intelligence and physical strength, the vast potential of gene editing also prompts the important question: just how far should we go?

Nevertheless, gene therapy offers real hope for people suffering with genetic diseases: in time, it may provide us with new ways of treating debilitating and previously incurable conditions.

Gene therapy has a way to go, but one thing remains certain: for both scientists and patients, even a single gene can change the world.