A complex molecular machine, the spliceosome, ensures that the genetic information from the genome, after being transcribed into mRNA precursors, is correctly assembled into mature mRNA. Splicing is a ...
RNA splicing involves the removal of non-coding introns and the joining of coding exons in precursor mRNA (pre-mRNA) to produce mature messenger RNA (mRNA). Splicing changes can lead to diseases, ...
The FDA approved a risdiplam tablet for SMA patients aged 2+ and over 44 lbs, matching the oral solution's efficacy and safety. Risdiplam, an SMN2 pre-mRNA splicing modifier, enhances SMN protein ...
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