CORDIS Project
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This project focuses on developing a new therapeutic approach for Duchenne muscular dystrophy (DMD) by using morpholino antisense oligonucleotides to skip exon 53. The aim is to restore dystrophin protein expression in affected muscles, enhancing treatment efficacy and safety through a clinical trial involving DMD boys…
Duchenne muscular dystrophy (DMD) is a progressive, lethal muscle degenerative condition arising from the absence of dystrophin in skeletal and cardiac muscles. 65% of DMD boys have out-of-frame deletions.
Modulation of pre-mRNA splicing by exon skipping is the most promising molecular intervention in DMD. 2 Phase Ib and 2 Phase IIa clinical trials (MDEX Consortium in collaboration with Sarepta Therapeutics; a Dutch Consortium) demonstrated that delivery of antisense oligonucleotides (AOs) to me…
Partner organizations (coordinator is shown above), with normalized type and CORDIS activity type. Guests see up to 4 partners.
Belgium, TOURNAI
Type: SME
Activity type: Private for-profit entities (excluding Higher or Secondary Education Establishments)
SME: Yes
United Kingdom, Newcastle Upon Tyne
Type: University / higher education
Activity type: Higher or Secondary Education Establishments
SME: No
Italy, Milano
Type: University / higher education
Activity type: Higher or Secondary Education Establishments
SME: No
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