Gene therapy: The breakthrough that inspired Spider-Man | Euronews Tech Talks

Gene Therapy Brings Superhero Science Closer to Real Medicine

Poinews.com – Gene therapy features in Spider-Man: Brand New Day, where Peter Parker faces increasingly difficult changes as his DNA mutates and his spider-like abilities intensify. The film is fictional, but its reference to RNA interference draws on real medical research that can reduce the activity of selected genes.

Modern genetic medicine is not designed to give people superhero powers. Its purpose is to treat disease by acting on biological processes inside cells, often at the level of DNA or RNA. Initially associated mainly with inherited conditions caused by a single altered gene, the field now also has applications in research and treatment involving cancer and HIV.

Treating the Underlying Cause of Genetic Disease

Alberto Auricchio, director of the Telethon Institute of Genetics and Medicine in Naples, professor at the University of Naples and president of the European Society of Gene & Cell Therapy, says the central aim is to tackle disease at its source.

“Gene therapy basically targets the root cause of a genetic disease,”

Treatment can involve introducing nucleic acids, including DNA or RNA, into targeted cells or tissues. Depending on the condition, these genetic instructions may restore a missing function, correct harmful activity or reduce the effect of a gene that is contributing to illness.

There are two principal approaches. In vivo treatment delivers therapeutic material directly into the patient’s body. Ex vivo treatment involves removing cells, modifying them outside the body and returning them through transplantation. In either case, the genetic material must be transported into the relevant cells.

Viral vectors are one delivery option because viruses naturally enter cells; for medical use, they are adapted to carry therapeutic material rather than cause infection. Non-viral systems can also transport genetic instructions. The appropriate approach depends on the disease, the target tissue and the intended genetic effect.

Gene Addition, Correction and Silencing

This area of medicine includes several distinct strategies. Gene addition supplies a gene that is missing or not working properly. Gene correction seeks to repair a dysfunctional gene. Gene silencing reduces or switches off the activity of a particular gene, making it the approach most closely related to the RNA interference mentioned in the Spider-Man story.

These methods reflect the fact that inherited diseases have different causes. Some result from insufficient gene function, while others may be linked to genetic activity that needs to be reduced. Researchers can select techniques according to the biological problem they are trying to address.

The technology has moved beyond experimental research alone, with approved products now available for some patients.

“We are beyond clinical trials now. There are approved [gene therapy] products that are available to patients,”

Europe’s first approved in vivo treatment was Glybera. The European Commission granted it marketing authorisation in 2012 for people with lipoprotein lipase deficiency who experience severe or repeated pancreatitis attacks.

In 2016, Europe approved its first ex vivo treatment, Strimvelis, for severe combined immunodeficiency caused by adenosine deaminase deficiency. Developed at Hospital San Raffaele in Milan, it offered a treatment option for a rare and serious immune disorder.

Why Long-Term Effects Matter

A major potential benefit is durability. Conventional medicines may need to be taken regularly to manage symptoms or maintain their effect. A successful genetic treatment may be designed to deliver a longer-lasting result, potentially after a single administration.

“There are approved gene therapy drugs, for which we have decades of follow-up in patients where we know that the therapeutic effect is stable over decades, and in theory for the lifetime of the individual,”

This possibility is particularly important for conditions that begin in childhood or require ongoing care. A lasting therapeutic effect could reduce the need for repeated interventions, although outcomes, risks and eligibility differ between diseases and treatments.

Frequently Asked Questions

Is gene therapy the same as gene editing?

No. Genetic treatments can add, replace or silence genetic material without necessarily editing a patient’s DNA. Gene editing refers more specifically to techniques intended to alter DNA sequences.

Can gene therapy cure every inherited disease?

No. It is available only for certain conditions, and its suitability depends on the disease, the affected cells and the treatment approach. Research continues for many other inherited disorders.

Is RNA interference a real medical technique?

Yes. RNA interference is a real approach used to reduce the activity of selected genes. Its use in superhero fiction is exaggerated, but the underlying scientific principle is genuine.

Are approved treatments always permanent?

Not necessarily. Some therapies are intended to have long-lasting effects, but results vary according to the treatment, the disease and the individual patient. Long-term follow-up remains important.