Antisense Therapies for Spinal Muscular Atrophy and Diffuse Midline Glioma
The Richard M. Furlaud Distinguished Lecture
Event Details
- Type
- Friday Lecture Series
- Speaker(s)
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Adrian R. Krainer, Ph.D., St. Giles Foundation Professor, cancer center deputy director of research, Cold Spring Harbor Laboratory
- Speaker bio(s)
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Dr. Adrian R. Krainer will describe the design, development, and therapeutic applications of antisense oligonucleotides (ASOs) that alter the splicing patterns of, and/or degrade, target transcripts. He 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 his lab's ongoing preclinical development of ASOs for a lethal pediatric brain cancer, H3K27-altered diffuse midline glioma.
Dr. Adrian R. Krainer received a Ph.D. in Biochemistry from Harvard University in 1986, working with Professor Tom Maniatis on pre-mRNA splicing mechanisms. He continued his research on splicing as a Cold Spring Harbor Fellow, mentored by Dr. Richard J. Roberts, and joined the faculty at Cold Spring Harbor in 1989. In addition to studying RNA splicing mechanisms, regulation, and dysfunction in disease, his laboratory is engaged in the development of mechanism-based targeted therapies to correct or modulate alternative splicing in genetic diseases and cancer. This work has resulted to date in 236 publications and 13 issued patents. In collaboration with Ionis Pharmaceuticals and Biogen, Dr. Krainer’s laboratory developed nusinersen (Spinraza), which corrects the splicing defect in the SMN2 pre-mRNA and became the first approved therapy for spinal muscular atrophy, a genetic motor-neuron disease. Dr. Krainer is a co-founder, Director, and Chair of the SAB of Stoke Therapeutics. He was elected to the National Academy of Sciences, the National Academy of Medicine, the National Academy of Inventors, and the American Academy of Arts & Sciences. Recent awards include the 2019 Life Sciences Breakthrough Prize (shared with Dr. Frank Bennett), the 2019 Lifetime Achievement Award of the RNA Society, the 2019 International Prize for Translational Neuroscience (Max Planck Institute, shared with Dr. Richard Finkel), the 2019 Peter Speiser Award in Pharmaceutical Sciences (ETH-Zürich), the 2020 Takeda Pharmaceuticals Innovators in Science Senior Scientist Award in Rare Diseases, the 2020 Ross Prize in Molecular Medicine (Feinstein Institute), the 2021 Wolf Prize in Medicine (shared with Drs. Joan Steitz and Lynne Maquat), the 2021 Gabbay Award in Biotechnology & Medicine (Brandeis University, shared with Dr. Frank Bennett), and the 2022 August M. Watanabe Prize in Translational Research (Indiana University School of Medicine).
FLS lectures will take place in Caspary Auditorium and virtually via Zoom. We recommend virtual participants log out of VPN prior to logging in to Zoom. Please do not share the link or post on social media. This talk will be recorded for the RU community.
- Open to
- Tri-Institutional