One of these drugs named milasen (MEE-luh-sin) went from idea to FDA approval in 10 months and was personalized for a specific mutation in Mila Makovec, a 6-year old girl with Batten's disease. For 10 points each:
[10h] Name this type of drug exemplified by nu·sin·er·sen, which was approved to treat spinal muscular atrophy in 2016.
ANSWER: antisense oligonucleotide [accept antisense therapy or antisense RNA or ASOs; prompt on oligonucleotides or RNA; prompt on complementary RNA or complementary oligonucleotides; prompt on descriptive answers indicating an RNA molecule that binds to a target mRNA; prompt on gene therapy; reject “morpholinos” or “siRNA” or “miRNA” or “microRNA”]
[10e] Nusinersen works by binding to a silencer site to alter the “alternative” form of this process, in which introns are removed from a pre-mRNA.
ANSWER: alternative splicing
[10m] Most splicing events in humans follow a “rule” named for two dinucleotides, found at the 5-prime end of the intron and the 3-prime end of the intron. Name either dinucleotide.
ANSWER: GU or AG [accept the GU-AG rule; reject “UG” or “GA” or “AG-GU rule”]
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