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Pharmacological exon skipping in dogs

Pharmacological exon skipping in dogs - Gene Therapy

Pharmacological exon skipping in dogs - Discuss gene therapy issues, methods and protocols for viral gene therapy, problems and solutions.


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Old 03-16-2009, 05:06 PM
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Default Pharmacological exon skipping in dogs



Is modifying RNA splicing considered gene therapy?

Yokota T, Lu QL, Partridge T, Kobayashi M, Nakamura A, Takeda S, Hoffman E. [Only registered users see links. ] Annals of Neurology 2009 [epub ahead of print] doi 10.1002/ana.21627

Videos of treated and control dystrophic dogs:

[Only registered users see links. ], Non-treated Dystrophic Dog

[Only registered users see links. ], Non-treated Dystrophic Dog

[Only registered users see links. ], Treated Dystrophic Dog After 5x Weekly 120 mg/Kg Cocktail PMO Injections (7 months old)

[Only registered users see links. ], Treated Dystrophic Dog After 7 200 mg/Kg Cocktail PMO Injections (4 months old)

[Only registered users see links. ], Treated Dystrophic Dog After 11x 120 mg/Kg Cocktail PMO Injections (7 months old)

[Only registered users see links. ] on Morpholinos for Duchenne muscular dystrophy in a dog model of DMD.
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Old 03-17-2009, 10:41 AM
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Thumbs up Re: Pharmacological exon skipping in dogs

Thanks for posting this Jon. I am very interested in research into muscular dystrophies. Especially treatments like these.

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Originally Posted by Jon Moulton View Post
Is modifying RNA splicing considered gene therapy?
INteresting question. If you define gene therapy as the insertion of a foreign gene into one's genome then no.
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Old 03-17-2009, 05:04 PM
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Default Re: Pharmacological exon skipping in dogs

Then perhaps I have posted the information about the exon-skipping therapeutic in an inappropriate forum. RNA-level drugs have no established niche of their own. If we consider "gene therapy" to be the same as "nucleic acid sequence-based therapy" then the exon-skipping oligos are gene therapy. However, I lean toward agreeing with you that gene therapy should be a term reserved for DNA-level changes. Perhaps the term "transcript therapy" would capture the class of RNA-modifying drugs clearly.
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Old 12-07-2012, 11:00 AM
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Default Re: Pharmacological exon skipping in dogs

hiiii
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dmd , dog , dogs , exon , morpholino , pharmacological , skipping , splicing , systemic


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