Antonio Alberto RodrÃguez Sousa
Complutense University of Madrid, Spain
Title: Virology in the biomedical field: Biotechnological tool for the treatment of cystic fibrosis
Biography
Biography: Antonio Alberto RodrÃguez Sousa
Abstract
Statement of the Problem: Cystic fibrosis is the autosomal recessive genetic disorder with the highest incidence in the Caucasian population, being caused by mutations of the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Although the current treatments focus on increasing the quality of life of the patient, a gene therapy of viral origin that could involve the regeneration of damaged alveolar parenchyma is located in the experimental phase. The purpose of the present work is to establish a theoretical framework of the disease from which clinical work can be carried out aimed at the development of new therapeutic techniques.
Methodology & Theoretical Orientation: A dynamic simulation model was developed on patients' hope and quality of life, taking the number of pulmonary alveoli as an estimator of respiratory function. The model was calibrated in face of different clinical situations: healthy individual; untreated sick individual; sick individual with conventional treatments; and sick individual treated with gene therapy.
Findings: The study showed that the life expectancy of sick individuals was significantly reduced when compared with healthy individuals. On the other hand, while the application of conventional treatments reflected an improvement in the quality of life of the simulated patients, the administration of a viral-type gene therapy was consolidated as an option in which the treated individual’s hope of life was not affected due to the disease.
Conclusion & Significance: By using viruses as a biotechnological tool, being transmission vectors of functional copies of the CFTR gene that would be inserted into the host's DNA, it would be possible to correctly resume the cellular processes altered by the disease. Therefore, the research in virology and its application in gene therapies are essential to develop curative treatments that suppose a new clinical horizon.