Interdisciplinary doctoral training in oligonucleotide-based therapies for Myotonic Dystrophy

Rare diseases pose significant diagnostic and therapeutic challenges, and Myotonic Dystrophy (DM), the most common hereditary muscular dystrophy in adults, remains without effective treatments due to its genetic complexity and the limited efficacy of antisense oligonucleotides evaluated. ENTRY-DM brings together an interdisciplinary and intersectoral consortium with 9 academic and 2 non-academic partners to train 14 high-potential doctoral candidates in ASO design and delivery, disease mechanism research, advanced experimental models, and clinical trial preparation, creating a framework transferable to other RNA-mediated diseases and aligned with European and international priorities in rare diseases. By integrating cutting-edge science, international mobility, and interdisciplinary training to overcome barriers, from genotype-phenotype correlations to ASO biodistribution, the program strengthens European leadership in ASO-based therapies and lays the foundation for future clinical trials.

Project information

Current

Acronym

ENTRY-DM

Start

01/03/2025

End

28/02/2029

Project call

HORIZON-MSCA-2023-DN-01-01

Project reference

101169266

Coordinated by IQAC

Yes

Innovation action

Yes

Total budget

3.693326 €

IQAC budget

251.971 €

Principal investigator

Other staff involved