AVI-4658
National Institutes of Health
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Can manipulation of splicing offer gene therapy possibilities to those with tumour-prone disorders?
We previously conducted a proof of principle; dose escalation study in Duchenne muscular dystrophy (DMD) patients using the…
AVI-4658 is a phosphorodiamidate morpholino oligomer (PMO) drug designed to restore dystrophin expression in a subset of patients…
AVI-4658 is a phosphorodiamidate morpholino oligomer (PMO) designed to induce skipping of dystrophin exon 51 and restore its…
Duchenne muscular dystrophy (DMD) is caused by dystrophin gene mutations. Restoration of dystrophin by exon skipping was…
Purpose of reviewDuchenne muscular dystrophy is a progressive muscle degenerative disease caused by dystrophin mutations. The…