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#1
02-17-2009, 05:47 PM
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 Morpholinos for Duchenne muscular dystrophy: clinical trial AVI BioPharma has started a clinical trial of their AVI-4658 Morpholino oligo targeted to block splicing of exon 51 in the human Dystrophin transcript. This is an experimental therapeutic for some mutations causing Duchenne muscular dystrophy, designed to restore the reading frame and ameliorate the effects of a frame-shifting mutation. This is a systemic study involving intravenous injection of an unmodified Morpholino (note: Morpholinos are also called PMO, an acronym for phosphorodiamidate Morpholino oligomer). [Only registered users see links. ] ClinicalTrials.gov identifier: NCT00844597 Update 19 Feb 09: First patient dosed. [Only registered users see links. ] Last edited by Jon Moulton; 02-19-2009 at 02:51 PM. Reason: update  |
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| clinical , dmd , duchenne , dystrophy , morpholino , morpholinos , muscular , trial |
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