Project: Exploring new therapeutic strategies in Hutchinson-Gilford progeria syndrome preclinical models Hutchinson-Gilford progeria syndrome (HGPS)
| Acronym | TREAT-HGPS |
| Project Topic | 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 14.6 years mainly from myocardial infarction or stroke. It is caused by a heterozygous de novo point mutation in the LMNA gene leading to the synthesis of progerin, a permanently farnesylated prelamin A mutant protein. HGPS has no cure and clinical trials targeting progerin farnesylation showed increased mean survival of only ~1.6 years in treated patients. It is therefore urgent to develop new strategies to treat or cure HGPS. |
| Network | E-Rare-3 |
| Call | 9th JOINT CALL FOR EUROPEAN RESEARCH PROJECTS ON RARE DISEASES (JTC 2017) |
Project partner
| Number | Name | Role | Country |
|---|---|---|---|
| 1 | Fundación Centro nacional de Investigaciones Cardiovasculares Carlos III (CNIC) | Coordinator | Spain |
| 2 | Fundación Centro nacional de Investigaciones Cardiovasculares Carlos III (CNIC) | Partner | Spain |
| 3 | University of Wroclaw | Partner | Poland |
| 4 | CNR Institute of Molecular Genetics | Partner | Italy |
| 5 | School of Medicine, Technical University of Munich | Partner | Germany |