Pharmacological exon skipping in dogs

[Jon Moulton]

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.

[Frank]

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

Originally Posted by Jon Moulton

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.

[Jon Moulton]

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.

[CarolynDwitt]

hiiii