In a cellular model of CF, ASOs were designed to target the inhibitory secondary mRNA structures in the uORF of the 5 UTR on the CFTR mRNA transcripteffectively increasing both the expression and function of CFTR [88]
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While our speculations on secondary effects for the short side were relatively spot on, as we will discuss later, it seems out of 3 of the more speculative and slightly contrarian secondary beneficiaries (I.e
But concern remains if they cant get back to break even in a reasonably quick timeframe. Jessica Bartlett can be reached at [email protected]
