ERC Proof of Concept Grant Awarded to Max Planck Team for RNA-Based Therapy Targeting Duchenne Muscular Dystrophy

July 14, 2025

A team of researchers at the Max Planck Institute for Heart and Lung Research, led by Prof. Dr. Didier Y.R. Stainier, has been awarded a prestigious ERC Proof of Concept (PoC) grant to advance a groundbreaking RNA-based therapeutic strategy for Duchenne Muscular Dystrophy (DMD).

The grant builds upon earlier ERC-funded work uncovering the mechanism of transcriptional adaptation (TA) — a natural genetic buffering response that can compensate for disease-causing mutations. This new project seeks to harness TA using antisense oligonucleotides (ASOs) to upregulate utrophin, a functional analog of dystrophin, offering a mutation-independent therapy for all DMD patients.

3 people in formal dress — Didier Stainer, director of the 'Genetics of Development' department, Christopher Dooley as co-inventor and head of translational research in the department and Lara Falcucci, who is currently writing her dissertation on the topic.

© Photographer: Andrea Katheder

Didier Stainer, director of the 'Genetics of Development' department, Christopher Dooley as co-inventor and head of translational research in the department and Lara Falcucci, who is currently writing her dissertation on the topic.

© Photographer: Andrea Katheder

The ERC PoC award will support the collaborative efforts of Prof. Stainier, Dr. Christopher Dooley (co-inventor and translational lead), and Lara Falcucci (PhD student and first author of the team’s recent Nature paper). Their published findings provided the first evidence that targeted ASOs can induce TA and upregulate utrophin in human cell culture models.

This project is an outstanding example of how fundamental discovery science can lead to therapeutic innovation. We are excited to take this next step towards translation.

Prof. Dr. Didier Stainier

The ERC Proof of Concept program funds outstanding researchers to explore the societal or commercial potential of their ERC-funded research. This award highlights the strong potential of TA-based RNA therapeutics to transform care for patients with DMD and other severe genetic conditions.