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SUMMARY:2024 Max Perutz Lecture: Antisense Modulation of RNA Splicing for 
 Rare Disease Therapy - In Person Only - Adrian R. Krainer\, Ph.D.\, St. Gi
 les Foundation Professor\, Cold Spring Harbor Laboratory
DTSTART:20241111T110000Z
DTEND:20241111T120000Z
UID:TALK206377@talks.cam.ac.uk
CONTACT:Scientific Meetings Co-ordinator
DESCRIPTION:I will describe the design\, development\, and therapeutic app
 lications of antisense oligonucleotides (ASOs) that alter the splicing pat
 terns of\, and/or degrade\, target transcripts. I will focus on CNS indica
 tions\, including the development and clinical impact of nusinersen (Spinr
 aza)—the first approved drug for spinal muscular atrophy—as well as ou
 r ongoing preclinical development of ASOs for a lethal pediatric brain can
 cer\, H3K27-altered diffuse midline glioma.
LOCATION:In person in the Max Perutz Lecture Theatre (CB2 0QH) 
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