The approach is to develop small-molecule drugs that can correct RNA mis-splicing, leading to loss of RNA expression by exon-skipping. It is another way to go after hard-to-target disease ...
Biogen meanwhile is also pursuing other avenues of RNA drug discovery and development, and in 2018 expanded the partnership with Ionis Pharma which delivered Spinraza to the market, paying $350 ...
RNA in cells performs an astounding number of functions. Messenger RNA carries the sequences of active genes to the cellular machinery, which turns... | Cell And Molecular Biology ...