E-RARE17-093 “TREAT-HGPS” (program E-Rare-3), budget 800 000 Euro.

Participants:
coordinator – prof. Vicente Andres (Laboratory of Molecular and Genetic Cardiovascular Pathophysiology, Centro Nacional de Investigaciones Cardiovasculares Carlos III (CNIC).
dr Ryszard Rzepecki, (Laboratory of Nuclear Proteins, Faculty of Biotechnology, Wrocław University).
dr Karima Djabali (School of Medicine, Technical University of Munich, Department Dermatology).
dr David Fulgueiras (Fundación Investigación Biomédica Hospital Clínico San Carlos, Madrid, Spain).
dr Giovanna Lattanzi (Institute of Molecular Genetic, Italian Research Center, Bologna Unit).

Abstract:

Hutchinson-Gilford progeria syndrome (HGPS) is an extremely rare genetic disease (prevalence: 1 in 20 million) characterized by multiorgan defects, accelerated aging, and death at an average age of 14 yrs mainly from myocardial infarction or stroke. It is caused by progerin, a permanently farnesylated prelamin A mutant protein (LMNA gene). HGPS has no cure and clinical trials targeting progerin farnesylation showed increased mean survival of only ~1.6 yrs in treated patients. It is therefore urgent to develop new strategies to treat or cure HGPS.

TREAT-HGPS is a transnational 3-yr program involving 4 European nations which will exploit existing HGPS cell and mouse models to a) discover novel combinations of therapeutic drugs to fight HGPS, and b) provide essential new knowledge about reversibility of progerin-induced damage.

All interested in work in this project are welcomed to contact us.