7. Advanced therapy medicinal products

7.1. Gene-therapy medicines

These medicines contain genes that have a therapeutic effect - they act to treat the disease. They work by inserting ‘recombinant’ genes into cells.  Sometimes they use a virus (called a ‘vector’) to deliver the genes.

  • A recombinant gene is a stretch of DNA that is created in the laboratory to have a certain sequence.
  • When the gene is delivered into a cell, it causes the cell to either produce, or stop the production of a specific protein. By doing this, it can slow down or cure a disease.
  • Diseases that have so far been targeted by gene therapy include genetic disorders, cancer and chronic (long-term) conditions.

Gene therapy using adenovirus vector

gene therapy adenovirus vector

Figure 7: Gene therapy using an adenovirus vector. A new gene is inserted into a cell using an adenovirus. If the treatment is successful, the new gene will make a functional protein to treat a disease. Image created by the National Institutes of Health (accessed on https://en.wikipedia.org/wiki/Gene_therapy)