CORDIS Project
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This project focuses on using exon skipping technology to develop mouse models for Duchenne muscular dystrophy. It aims to identify stable and functional dystrophin proteins by assessing their performance in muscle tissues, which is crucial for patient treatment.
Duchenne muscular dystrophy (DMD) stems from loss of dystrophin in skeletal and cardiac muscles, which leads to loss of ambulation and cardio-respiratory failure.
The most promising treatment that could be applicable to 83% of DMD patients is exon skipping, a technology where the EU is a world leader.
Antisense oligonucleotide mediated exon skipping targets DMD pre-mRNA to induce skipping of specific exons and restore the open reading frame.
This allows expression of shorter dystrophin proteins…
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