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Terfiqec

Comprehensive research in Effective Therapies for the treatment of Cystic Fibrosis and Related Diseases (TERapias efectivas para el tratamiento de la FIbrosis Quística y Enfermedades Conexas), by correcting the genetic defect that causes the disease, in order to obtain a cure.


Cystic Fibrosis (CF) is a hereditary disease caused by the absence or malfunction of the Cystic Fibrosis Transmembrane Conductase Regulator (CFTR) gene. The first symptoms appear in early childhood, generally in the respiratory tract, then extending to other organs. CF is an eventually fatal degenerative disease, which can be considered as the leading cause of death due to genetic disease in Caucasian children. The lethality is basically associated with the pulmonary pathology.

terfiqec

The identification and isolation of the gene responsible for CF aroused high hopes of finding an effective treatment in a short period of time. However, various attempts at developing effective gene therapy protocols have yielded unsatisfactory results.

 

In these circumstances, BIOPRAXIS is conducting, in collaboration with the CAUBET-CIMERA Foundation, the University of the Basque Country and the University of Barcelona, an R&D project to obtain an effective treatment for CF patients undergoing autologous transplant of pulmonary epithelial cells in which the congenital CFTR gene defect has been corrected.

 

The method proposed for achieving this is based on: (i) production of induced pluripotent stem cells (iPS) obtained from differentiated cells from patients with the ΔF508 mutation and mice that carry the same mutation. (ii) correction of the CFTR mutation in the iPS cells by homologous recombination induced with a functional allele of the gene. (iii) in vitro redifferentiation of the iPS carriers of the repaired gene to bring them to a state of differentiation compatible with their integration into the pulmonary epithelium. (iv) transplant of the differentiated cells into suitable recipients.

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