The head of CIEMAT / CIBERER / ISFJD's Division of Innovative Therapies, Juan A. Bueren, described the current state and recent perspectives on gene therapy in rare diseases. I have compared the difference between pharmacological and gene therapies when looking at a bioreactor, when the latter introduced a vector, usually of the viral type, into the patient's organism.

The speaker then distinguished the non-integrative, simpler and targeted gene therapy for certain types of hemophilia, of which it is integrative. In the latter case and within lymphohematopoietic diseases, he detailed how children's stem cells are obtained for purification. Subsequently, said cells are brought into contact with the vectors (retrovirus materials) and said integration takes place. Once these cells are incorporated into the body, rejection is avoided and good immune response is achieved. These experiences, in bubble children, however, developed leukemias within two years of treatment, because the vectors activated oncogenesis, through the phenomenon known as the transactivation.