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Systemic AAV8-mediated gene therapy drives whole-body correction of myotubular myopathy in dogs

Started by alc , Feb 17 2017 09:55 PM
raav8 systemic gene therapy

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#1 alc

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Posted 17 February 2017 - 09:55 PM


"We report here a gene therapy dose-finding study in a large animal model of a severe muscle disease where a single treatment resulted in dramatic rescue"

 

 

http://www.cell.com/...0016(17)30056-4

 

http://www.cell.com/...(17)30056-4.pdf


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