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2024 Max Perutz Lecture: Antisense Modulation of RNA Splicing for Rare Disease Therapy - In Person Only

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I will describe the design, development, and therapeutic applications of antisense oligonucleotides (ASOs) that alter the splicing patterns of, and/or degrade, target transcripts. I will focus on CNS indications, including the development and clinical impact of nusinersen (Spinraza)—the first approved drug for spinal muscular atrophy—as well as our ongoing preclinical development of ASOs for a lethal pediatric brain cancer, H3K27 -altered diffuse midline glioma.

This talk is part of the MRC LMB Seminar Series series.

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