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Dog model of DMD, treatment with Morpholinos


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#1 Jon Moulton

Jon Moulton

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Posted 16 March 2009 - 08:57 AM

A study using unmodified Morpholino oligos from Gene Tools in a dog model of Duchenne muscular dystrophy has been published along with videos of treated and control dogs. Exon-skipping Morpholino oligos from AVI BioPharma are in clinical trials for DMD.

Yokota T, Lu QL, Partridge T, Kobayashi M, Nakamura A, Takeda S, Hoffman E. Efficacy of systemic morpholino exon-skipping in duchenne dystrophy dogs. Annals of Neurology 2009 [epub ahead of print] doi 10.1002/ana.21627


Videos (Quicktime format) of treated and control dystrophic dogs:

* Video 1, Non-treated Dystrophic Dog (7 months old)
* Video 2, Non-treated Dystrophic Dog (7 months old)":
* Video 3, Treated Dystrophic Dog (7 months old): After 5x Weekly 120 mg/Kg Cocktail Morpholino Injections
* Video 4, Treated Dystrophic Dog (4 months old): After 7 x 200 mg/Kg Cocktail Morpholino Injections
* Video 5, Treated Dystrophic Dog (7 months old): After 11x 120 mg/Kg Cocktail Morpholino Injections

NIH press release on Morpholinos for Duchenne muscular dystrophy in a dog model of DMD.

Children's National Medical Center press release

Edited by Jon Moulton, 16 March 2009 - 03:46 PM.

Jon D. Moulton
Gene Tools, LLC
www.gene-tools.com




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