El descubrimiento de la estructura del ADN y los mecanismos que regulan la expresión de los genes durante la primera mitad del siglo pasado establecieron las bases teóricas y metodológicas para el desarrollo de tecnología que permite modificar un gen o genoma. La aplicación clínica de las técnicas que modifican la expresión de un gen se denomina terapía génica (TG). Estás técnicas incluyen plataformas (virales y no virales) que acarrean sistemas de modificación genética o genes sanos hasta las células blanco. La ClinicalTrial.gov es un repositorio sobre ensayos clínicos que incluye protocolos con uso de TG. En este trabajo se realizó una revisión sistemática de los trabajos registrados en ClinicalTrial.gov sobre el uso de TG en enfermedades hematológicas. Se encontraron un total de 41 ensayos clínicos relacionados con TG, de los cuales la mayoría estaba registrada en Estados Unidos de América del Norte (56.1%). El 41.46% de los protocolos obtuvieron financiamiento privado. Del 50% de todas las patologías solo las enfermedades de origen hematológico de origen monogénico (hemofilia A, hemofilia B y la anemia de Fanconi) recibieron TG. Por otro lado, el número de protocolos clínicos registrados por país se correlacionó de forma positiva con el desarrollo económico, el desarrollo científico, con la inversión en salud per cápita y con la calidad de vida. Finalmente, aún existen controversias bioéticas, sociales, políticas y económicas, que aún deben resolverse.
Abstract
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.
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