Question

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”]
<BB>

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Summary

2023 BHSU @ Berkeley03/18/2023Y316.67100%67%0%
2023 BHSU @ Maryland03/11/2023Y310.00100%0%0%
2023 BHSU @ Northwestern02/25/2023Y610.00100%0%0%
2023 BHSU @ Waterloo04/15/2023Y310.00100%0%0%
2023 BHSU @ Yale04/08/2023Y316.67100%67%0%

Data

Berkeley BStanford B0101020
Stanford AIt's Joever0101020
Berkeley A[Insert Lawyer Joke Here]010010
Maryland AFan-tastic Negs and Where to Find Them010010
The Perfection of Wisdom in 8,000 NegsGlass Onyen: A Knives Out Mystery010010
Rutgers DiasporaMaryland B+010010
Chicago B+Amartya Senpai Notice Me010010
Chicago AEpic Games010010
Maryland B-Purdue010010
Maryland B-Purdue010010
Maryland B-Illinois A+010010
Maryland B-Illinois A+010010
Mojo ShojoNorthwestern010010
OSUWUSTL010010
Maryland B-Purdue010010
Maryland B-Purdue010010
Maryland B-Illinois A+010010
Maryland B-Illinois A+010010
Milan et al.Cameron et al.010010
The Vulture's Guide to BuzzParth et al.010010
Neg 5! Playing Quizbowl, Liddy StyleThe Passion According to C.H.010010
Columbia BUG Championship Players (and Mazin)0101020
nats is supposed to mean you all have gone softOlmo (Bonus) Bagelry010010
meet the new weird, same as the old weirdWatching Arthur Delot-Vilain at Chicago Open Made Me Like French People Again0101020