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Projects. Life and Earth Sciences

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

Lead Researcher:
Rosario Perona Abellón

Research Centre: 
Instituto de Investigaciones Biomédicas de Madrid. CSIC-UAM.  (Spain).

Synopsis:
Rosario PeronaThe 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.


SCIENTIFIC PRODUCTION   

3 articles published in Journals
3 papers at national conferences  
________________________________________

Rosario Perona Abellón
Research Scientist CSIC.

EDUCATION

-Degree in Pharmacy, Central University of Venezuela 1978
-Degree in Pharmacy, Complutense University of Madrid, 1979
-Ph.D. in Pharmacy, Complutense University of Madrid, 1979-1982

SCIENTIFIC OR PROFESSIONANL ACTIVITIES

2002-present Member of Editorial Board of the journal Clinical and Translational Oncology
2003-2004, President of the FESEO (Spanish Federation of Oncology Societies) 2005-2006, President of the ASEICA (Spanish Association for Cancer Research) 2006-2010, Member of the Board of EACR (European Association for Cancer Research).
2007- Member of CIBER Rare Diseases (CIBERER).

Publications: 
I have published over 60 articles in international journals. More than 20 book chapters
Projects:
I have been awarded 27 projects as principal investigator.
Patents:
Nucleotide and GSE 24.2 peptide sequence of dyskerin telomerase-inducing activity, the procedure for obtaining it, therapeutic compositions and their applications. Current Status:
In process of licensing.
Use of GSE 24.2 inducing agents for the preparation of pharmaceutical compositions for the treatment of diseases involving cellular senescence.
Doctoral Theses.
I have supervised 8 Doctoral Theses
Teaching:
I have taught Ph.D. classes from 1986 to the present in courses related to signal transduction, Transcription, Cancer and Rare Diseases.
International Conferences:
I have been invited to give 45 papers at international conferences



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