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Nanomedicine for the treatment of Lafora disease: use of mRNA-loaded polymeric nanoparticles as an innovative replacement therapy

21st national competition for scientific and technical research

Rare diseases

Senior Researcher : Jordi Duran Castells

Research Centre or Institution : Universidad Ramón Llull


Lafora disease (LD) is a severe neurodegenerative condition that affects children. The first symptoms appear in the form of epileptic seizures, but the pathology progresses rapidly with a general deterioration of neurological functions and ends up causing death a few years after its onset. It is a rare disease for which there is currently no treatment. This urgent therapeutic need is the objective of this project. LD is an hereditary disease, caused by mutations in two genes: malin and laforin. Patients with mutations in one of the two genes present the same disease, indicating that both participate in the same physiological process. Being a hereditary disease, a possible treatment for LD would be gene therapy based on the re-introduction of a functional copy of the mutated gene. In this sense, our recent results with animal models indicate that this could be an effective strategy. 

The objective of this project is to validate the use of nanoparticles loaded with mRNA encoding the mutated gene (malin or laforin) to restore its expression as replacement therapy for LD. For this we have a multidisciplinary team with recognized experience both in the study of EL and in the generation of nanoparticles as therapeutic vehicles. This project is in an excellent position to signify an important step forward in the treatment of LD.

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