The Institute of Bio-pathology and Regenerative Medicine, constituted one month by the University of Granada, has already started its path. The centre, which will be located in the Campus de la Salud, has just published it first work in one of the most prestigious journals of the world in the field of gene therapy, Gene Therapy. It consists of the development of a protocol that could substantially decrease the harmful effects provoked by gene therapy in patients with immunodeficiencies.
In this sense, Professor Ignacio Molina explains that, although gene therapy (that consists of introducing a gene into certain cell to correct its defects or modify its properties) has treated successfully certain immunodeficiencies (diseases produced due to the mutation of a gene giving raise to deficient immunological systems), this type of treatment involves certain risks for the patient as there is “chance that undesired effects appear as it not possible to discriminate the cells in which the sound gene blends with to make therapeutic proteins or the quantity they must produce”.
To get over this handicap, the research team has developed a strategy of gene therapy with which they have managed to overcome two problems: on the one hand, the gene inserted in the patient causes a protein level equivalent to that of normal cells, and therefore there is no risk of toxicity, and on the other hand, they have achieved that the gene only expresses itself in the cells where it has to.
Although this study, developed in laboratory with patients´ cells, has focused on the Wiskott-Aldrich syndrome, a congenital pathology that is characterized by a serious immunodeficiency accompanied by alterations in platelets and eczemas and affects one of every 200,000 men, the gene therapy strategy designed during the research work could also be applied to other primary immunodeficiencies, which provoke more than 150 different congenital diseases.
New method
But this is not the only novelty contributed by the team, leaded by the Institute López Neyra (CISC) and the Institute of Bio-pathology and Regenerative Medicine (Centre for Biomedical Research of the University of Granada). The instruments used to achieve these results are also revolutionizing the field of gene therapy. They are lentiviral vectors, based on viral sequences useful to efficiently introduce the theraputical genes into cells of special interest for gene therapy (such as homopoietic stem cells, able to reconstitute all the patient’s immune system). The researchers modified such vectors to obtain a tissue-specific and physiological expression, as well as its introduction in hematopoietic stem cells obtained from the umbilical cord thanks to the collaboration of Hospital Materno-Infantil of Granada.
Besides immunodeficiencies, Molina asserts that other diseases caused by gene mutations could also be treated with this protocol designed with elements that guarantee the security and efficiency of the treatments. Likewise, and due to that these vectors are specific of the hematopoietic tissue, they could be potentially applied in the treatment of autoimmune diseases and cancer immunotherapy.
The Professor of the University of Granada clarifies that, even though the name of the Institute of Bio-pathology and Regenerative Medicine already appears in this work, the study was “obviously” being developed long before the centre was set up, and he says that this project is an example of the lines that will be developed in the Institute in the future –cancer pathology and immunology and transplant- and of that “besides embryonic stem cells there are other types of treatments and approaches to regenerative medicine that can also tackle diseases that can not be treated a priori, such as gene therapy”.
Reference: Prof Ignacio J. Molina Pineda de las Infantas
Institute of Bio-Pathology and Regenerative Medicine. Phone numbers: 958 24 35 22/ 24 07 31. E-mail. imolina@ugr.es.
