Gene therapy is a set of techniques based on the editing or modification of genetic material with the aim of preventing and/or curing diseases. Gene therapy techniques make it possible to act directly on the origin of the problem by transferring the correct version of the defective gene, which is responsible for the expression of the disease.

What is the aim of gene therapy and what are its types and the fundamentals of its methodology?

The aim of gene therapy is therefore to restore the function of the gene involved in the development of a disease. To do this, a normal copy of the gene is introduced into the cells or the function of the genes involved in the development of the disease is inhibited or blocked.

There are different types or ways of carrying out gene therapy:

• Ex vivo gene therapy: cells are removed from the patient, the repair gene is introduced in the laboratory and reimplanted back into the individual’s organism.
• In vivo gene therapy: consists of administering the repair gene directly to the patient so that it reaches the target organ.
• In situ gene therapy: consists of introducing the repair gene directly into the affected organ.

It is important to know how genes function in individuals and to find genetic patterns common to different diseases. By identifying and understanding the different genetic patterns and mechanisms, it is possible to develop more specific and effective treatments through the use of gene therapy.
On the other hand, it is also essential to know what the target tissue or organ is, to study whether it is possible to treat it in situ, as well as to know which is the appropriate vector to introduce the gene into the tissue or organ and prevent it from being destroyed by the individual’s organism before it reaches the target tissue or organ.

which diseases can be treated with gene therapy?

In its beginnings, gene therapy was only considered for the treatment of genetic diseases but, through years of research and advances, it has been proven that it can be used for the treatment of almost any pathology.

Both monogenetic hereditary diseases and complex pathologies are susceptible to to be treated by gene therapy.

For example, different gene therapies are being studied to combat cancer. The problem with treating some diseases is sometimes their late diagnosis. However, nowadays there are also tools such as the tellmeGen genetic test that make it possible to know the predisposition to present different diseases, with the aim of trying to prevent their expression and/or apply more effective and personalized treatments.

Gene therapy, together with other advances in genetics such as the genetic tests mentioned above, constitute a true revolution and have given rise to what is known as precision medicine. Precision medicine constitutes a new paradigm that is making possible the prevention of diseases through the previous study of susceptibilities by means of genetic tests, as well as the development of more effective and specific treatments such as those based on gene therapy, which make it possible to treat effectively even diseases for which until now there was no cure, thus opening up new horizons for curing diseases and saving lives.