RT Generic T1 Characterization in a humanized context of a rare disease: dyskeratosis congenita A1 Acitores Balboa, David AB The experimental work of the final degree project was dedicated to apply the knowledge acquired of tissue engineering and regenerative medicine, which are branches of biomedical engineering, focusing on the study of rare skin diseases.The project was developed at CIEMAT (Centro de Investigaciones MedioAmbientales y Tecnológicas) organization and with support of biomedical laboratories of Universidad Carlos III de Madrid. The project was controlled by Doctor Marta García Díez.The research was focused in the characterization of Dyskeratosis Congenita, which present different symtomes, including skin defects, due to lack of dyskerin protein. To perform this task In vitro and In vivo experiments are done for the characterization of diseased skin. In order to obtain a precise result bioengineered skin, histology, antibody detection and other methods are used.The main objective of this work is the generation and validation of an In vitro skin-humanized modelof DC (Dyskeratosis Congenita) of cells patients, recreating the cutaneous phenotype of this disease and then assess therapeutic approaches based on genetically modificated DC cells by lentiviral vectors coding for GSE-24.2. YR 2015 FD 2015 LK http://hdl.handle.net/10016/23427 UL http://hdl.handle.net/10016/23427 LA eng DS e-Archivo RD 30 abr. 2024