CORDIS Project
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This project studies the role of RNA processing in Myotonic Dystrophy type 1, a disorder affecting brain function. By using advanced techniques, it aims to identify affected brain cell types and pathways, ultimately seeking to develop effective molecular therapies.
RNA biology is critical for the molecular orchestration of the interplay between specialized brain cell types, and disrupted RNA processing, can cause human disease. A prime example is Myotonic Dystrophy type 1(DM1), a multisystemic disorder caused by the expansion of a non-coding trinucleotide DNA repeat and that involves cognitive impairment and behavioral changes.
Molecular pathogenesis is driven by the nuclear accumulation of RNA foci, which sequester RNA-binding proteins that regulate splic…
INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE
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France, Paris
Type: University / higher education
Activity type: Higher or Secondary Education Establishments
SME: No
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