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Development of a therapy for the treatment of congenital dyskeratosis, X-linked Werner syndrome and aplastic anaemia based on the reactivation of telomerase activity by an internal fragment of dyskerin

15th National Programme for the allocation of Research Grants for Life and Matter Sciences

Rare diseases

Senior Researcher : Rosario Perona Abellón

Research Centre or Institution : Instituto de Investigaciones Biomédicas de Madrid. CSIC-UAM.


The overall objective of this project is to further the validation and development of tools for the therapeutic application of the GSE24-2 fragment for the treatment of various diseases, including dyskeratosis congenita, Werner syndrome, idiopathic pulmonary fibrosis, aplastic anaemia, ulcerative colitis and skin ageing as well as obtaining immortal human cell lines. Following the original plan, in 2010 lentiviral vectors were derived, using hPGK and CMV promoters, with which primary human cells, both lymphoblasts and fibroblasts, were infected and an increase in telomerase activity was observed. These vectors will begin to be used to infect bone marrow cells in mice. Vectors have also been obtained for the 24 2 peptide production in E. coli and the peptide from bacterial extracts has been purified. This peptide is capable of activating the promoter of c myc and reactivating telomerase activity in a proportion similar to that obtained with the retroviral plasmid vector.

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