Max Perutz Lecture 2024: 11:00 am Monday 11th November. Adrian R. Krainer, Ph.D., St. Giles Foundation Professor, Cold Spring Harbor Laboratory
Abstract: 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.
Date:
Monday, 11 November, 2024 - 11:00 to 12:45
Subject:
Event location:
mrc