"This technique is proposed to treat hereditary genetic diseases, at present it is proposed for almost any disease, being a most promising mechanism".
DR. ANA PATIÑO GARCÍA DIRECTOR. GENOMIC MEDICINE UNIT
Gene therapy can be defined as a therapeutic technique by which a functional gene is inserted into a patient's cells to correct a genetic defect causing a pathology.
In addition to genetic diseases themselves, gene therapy is being applied experimentally to the treatment of cancer (in those tumors that have no effective treatment or in which conventional treatment has failed), AIDS (associated with antiviral treatment) and, more recently, other diseases such as coronary disease, peripheral arterial disease or rheumatoid arthritis.
However, the therapeutic possibilities of gene therapy are not limited to these diseases, and over time, once its efficacy, safety and improvement of vectors are proven, it is not unreasonable to use it in many other situations where the patient's life is not at stake.
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When is gene therapy indicated?
Despite the fact that it was originally a technique proposed to treat hereditary genetic diseases, it is currently proposed for almost any disease, being a most promising mechanism.
Among the objectives pursued by gene therapy we can mention:
- To try to complement or replace a defective gene by introducing another normal copy of it into the cells.
- To inhibit or block the functioning of those genes whose intervention contributes to the development of the disease (for example, oncogenes).
- To introduce genetic material that allows the cell to synthesize a protein that has a novel therapeutic effect (e.g. to introduce into the cells a copy of a gene that hinders the replication of a virus).
Most frequent indications for this treatment:
- Diseases of genetic origin.
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Gene therapy may be possible
Learn more about gene therapy
Gene therapy works both with the genetic material (the active ingredient) and with the vehicles that make it possible for that genetic material to enter the cells in sufficient quantity and, furthermore, maintaining its integrity.
With respect to the genetic material, the aim is to develop more effective genes, whose functioning can be predicted or controlled once inside the organism.
As for the vehicles, also called vectors, they constitute at this time the Achilles' heel of gene therapy and must be substantially improved if it is to be successful in treating patients.
At present, the most effective vectors available are the viral ones. They are based on the fact that viruses are, in reality, nothing more than genetic material that has been equipped with the necessary mechanisms to be able to enter cells effectively and produce its own proteins to multiply. To build a viral vector for gene therapy, the genes of the virus that are fundamental for it to multiply must be eliminated and replaced by the therapeutic gene, thus taking advantage of the efficiency of the virus to enter the cells, avoiding the risks of its dissemination.
CIMA UNIVERSIDAD DE NAVARRA
Research Program in Gene Therapy and Regulation of Gene Expression
We have a research line dedicated exclusively to Gene Therapy and Regulation of Gene Expression to develop new therapeutic strategies for diseases that currently lack a treatment and can benefit from therapies based on gene transfer.
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