Splicing efficiency project

Enhancement of splicing efficiency between trans‐spliced DMD AAV vectors for the production of full‐length fully functional dystrophin protein.

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Genome editing surgery

Development of genome editing surgery with applicability to all living with Duchenne – Royal Holloway University

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AAV-U7

Benefit evaluation of AAV-U7 treatment combined with corticosteroids for Duchenne muscular dystrophy – Institute of Myology

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SKIP-NMD (exon 53 programme)

In late 2012, we awarded a small grant to SKIP-NMD - an EU funded collaborative project involving 10 partners from Europe and the USA, that tested exon skipping of exon 53 in a subset of boys living with Duchenne.

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PepGen

A legacy from our supporter Hilary Hoskin supported this exon skipping research project in Oxford.

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