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Deciphering the role of protein S-palmitoylation in skeletal muscle physiopathology

Project description

Novel targets for Duchenne muscular dystrophy

Duchenne muscular dystrophy (DMD) is a muscle disorder characterised by progressive muscle degeneration, loss of mobility and premature death. DMD is caused by mutations in the gene encoding dystrophin, a protein that maintains the integrity of muscle cells. Funded by the Marie Skłodowska-Curie Actions, the MyoPALM project focuses on protein lipid modifications, aiming to identify new pathogenetic mechanisms in DMD. Researchers will use a mouse model of the disease to uncover new targets of potential therapeutic value and pave the way for novel treatments to improve the quality of life of DMD patients.

Objective

Duchenne muscular dystrophy (DMD) is the most prevalent form of childhood muscular dystrophy, affecting approximately 1 in 3500 newborn boys. DMD patients experience early-onset and rapidly progressive muscle weakness, leading to the loss of mobility and premature death. The burden of muscular dystrophies extends beyond the physical challenges faced by patients, indeed it poses significant social and economic implications, impacting patients’ quality of life, necessitating social welfare support and incurring substantial healthcare costs. So far there is no cure for DMD, emphasizing the urgent need to intensify research efforts aimed at uncovering new pathomolecular mechanisms and developing treatment strategies. Protein lipidation, especially protein S-palmitoylation, has gained attention in several diseases, but its role in skeletal muscle function and dystrophic conditions remains completely unexplored. MyoPALM is an innovative project that aims to bridge this knowledge gap by investigating the contribution of protein S-palmitoylation in adult skeletal muscle function. This will be accomplished through in vivo manipulation of the enzymes involved with this pathway and using cutting-edge palmitoyl-proteomic approaches. Furthermore, by exploiting the mdx mouse model, we aim to explore the potential role of protein S-palmitoylation in the pathogenesis of DMD. By unravelling this unexplored perspective, MyoPALM aims to lay the foundation for identifying new druggable targets, paving the way for future therapeutic strategies to benefit DMD patients. Through collaboration between the École Polytechnique Fédérale de Lausanne (CH) and the University of Padova (IT), the applicant seeks to reinforce his technical skills and scientific knowledge, aspiring to become an independent researcher within the European Union.

Fields of science (EuroSciVoc)

Keywords

Programme(s)

Topic(s)

Funding Scheme

HORIZON-TMA-MSCA-PF-GF - HORIZON TMA MSCA Postdoctoral Fellowships - Global Fellowships

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Call for proposal

(opens in new window) HORIZON-MSCA-2023-PF-01

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Coordinator

UNIVERSITA DEGLI STUDI DI PADOVA
Net EU contribution
€ 320 924,16
Address
VIA 8 FEBBRAIO 2
35122 Padova
Italy
Region
Nord-Est Veneto Padova
Activity type
Higher or Secondary Education Establishments
Links
Total cost
No data

Partners (1)