[Application of gene therapy in the treatment of hematological diseases: achievements, and economic and ethical aspects of the topic].

The discovery of the structure of DNA and the mechanisms that regulate gene expression during the first half of the last century established the theoretical and methodological bases for the development of technology that makes it possible to modify a gene or a genome. The clinical application of techniques that modify the expression of a gene is called gene therapy (GT). These techniques include platforms (viral and non-viral) that bring genetic modification systems or healthy genes to the target cells. ClinicalTrial.gov is a repository of clinical trials that incorporates protocols using TG. In this work, a systematic review of the works registered in ClinicalTrial.gov on the use of TG in hematological diseases was carried out. A total of 41 GT-related clinical trials were found most of which were registered in the United States of America (56.1%). On the other hand, 41.46% of the protocols obtained private funding. Of 50% of all pathologies, only diseases of hematological origin of monogenic origin (hemophilia A, hemophilia B, and Fanconi anemia) received TG. On the other hand, the number of clinical protocols registered by country was positively correlated with economic development, scientific development, investment in health per capita, and quality of life. Finally, there are still many bioethical, social, political, and economic controversies that must be solved.