making muscle wasting history

Systemic administration of avi-4658, a Phosphorodiamiate morpholino oligomer(pmo) to induce exon 51 skipping, is well tolerated and restores dystrophin expression in duchenne muscular dystrophin (dmd) boys in a dose dependent manner

• Last Edited: 22 Nov 2010
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1) UCL institute of child health, neorology, London,UK2) Royal Victor Infirmary,Newcastle3) 3avi biopharma Bothell USAObjective : we previsiously showed that a single im dose of avi-4658 pmo skips...

Tags: exon skipping, AVI, muscle biopsy, T cells, Phosphorodiamiate morpholino oligomerpmo.

Dystrophin immunity in duchenne muscular dystrophy

• Last Edited: 22 Nov 2010
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we report on delivery of a functional dystrophin transgene to skeletal muscule in six patients with duchenne muscular dystrophy. Dystrophin-specfic T-cellswere detected after treatment, providing ...

Tags: T cells, dystrophin.