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Episode 58

Biotech Hangout

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RNA Eyes for Spinal Muscular Atrophy

Is it plausible to advance an RNA I for spinal muscular atrophy in the hopes of coming up with an even less frequent inter-theacle delivery solution. Yeah, unfortunately not, because it first of all that requires nuclear delivery. SoRNA eyes is harnessing a endogenous and a nuclei pathway with a go to binding. It's really not the right approach you have to use a single stranded oligo in that case.

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