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Translational Medicine / 转化医学Cancer Antisense Oligonucleotides

Adrian Krainer

PhD

🏢Cold Spring Harbor Laboratory🌐USA

St. Giles Foundation Professor

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Key Contributions

👥Biography 个人简介

Adrian Krainer discovered the mechanism of splice-switching antisense oligonucleotides and developed nusinersen (Spinraza) for spinal muscular atrophy, the first approved splice-modulating ASO. His laboratory extends splice correction to cancer applications. He is a foundational figure in RNA splicing and ASO therapeutics.

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🧪Research Fields 研究领域

RNA splicing
splice-switching ASO
SMA nusinersen
cancer splicing correction
SR proteins

🎓Key Contributions 主要贡献

Representative Works 代表性著作

📄Data Sources 数据来源

Last updated: 2026-04-01 | All information from publicly available academic sources

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