GENE THERAPY has been a revolution in the way to address the treatment of genetic diseases, since it has opened a new horizon to cure diseases for which until now there were only treatments aimed at alleviating their symptoms.
Gene therapy is the set of techniques that use the transfer of genetic material (or any other method that allows to edit or modify the genetic information of the patient) to prevent or cure genetic diseases. Without a doubt it is the best alternative of all possible, but probably also the most complex. Goes directly to the root of the problemby transferring the correct version of a defective gene, which is the one that is causing the disease. Among the main obstacles of this approach is the difficulty of directing the genetic material specifically to those cells or tissues where the gene needs to perform its function, or that the regulation of the introduced gene approximates the way in which the gene is regulated in healthy people.
Through gene therapy , the function of the mutated gene can be restored , and the most common strategy is the introduction of a normal copy of it into the cells. It can also inhibit or block the functioning of those genes that contribute to the development of the disease (for example, the oncogenes involved in cancer or the virus genes that are necessary for these to multiply in the cells).
Gene therapy can be applied through different strategies.
Ex vivo strategy
It consists in extracting the cells that we must repair from a patient, repair them in the laboratory and reimplant them in the organism of the individual in question.
It consists of introducing the repair genedirectly into the individual’s own defective organ.
In vivo strategy
It consists of administering directly to the patient the correcting gene so that it reaches the point to be treated.
Advantages of gene therapy
- Gene-based treatments can fight an existing cancer and other diseases on a molecular scale, eliminating the need for drugs, radiotherapy or surgery.
- The identification of cancer- prone genes in individuals or families can play an important role in preventing the disease before it occurs.\
- From the beneficial side it is observed that the gene therapy can correct immunodeficiencies and that the gene transfer of immunomodulatory molecules to the right cell targets can empower responses therapeutic immunity or stop the response to autoantigens.
- It is available to all people;
- Allows to cure certain diseases that previously had no cure possible, (?)
- It repairs the immune system;
- Maintains existing immunity;
- The hospital stay only lasts about 1 month.
- There is great potential and promise from gene therapy as more research is been done.
Disadvantages of gene therapy
- The use
of viral vectors complicates gene therapy since the
anti-transgene or anti-vector response can extinguish their
- Gene therapy is a complicated field of research and many questions remain to be answered. Some cancers are caused by more than one gene and some vectors, if used incorrectly, can actually cause cancer or other diseases.
- The substitution of defective genes for functional copies also raises ethical questions that must be addressed before accepting these treatments to prevent cancer .
- It has a short duration, which may be due to the short life of the vector, the dysfunction of the DNA inserted in the target cells and / or the speed of cell division;Before gene therapy can become a permanent cure for any condition, the therapeutic DNA introduced into the target cells must stay working and the cells containing therapeutic DNA must be long-lived and stable. Problems with the integration of therapeutic DNA into the genome and the rapid natural division of many cells prevents gene therapy from completing its beneficial terms. Patients will have to undergo genetic therapy countless times.
- There is a need for the use of gene therapy several times;
- It can induce a tumor if the DNA is inserted into the wrong gene.
- Conditions or disorders that arise from mutations in a single gene are the best candidates for gene therapy. Unfortunately, some of the most commonly occurring disorders, such as heart disease, high blood pressure, Alzheimer’s disease, arthritis, and diabetes, are caused by variations in the combined effects of many genes. Disorders of several genes like these would be especially difficult to treat effectively using gene therapy.