research / researchCancer RNA Biology

Adrian R. Krainer

阿德里安·克雷纳

PhD

🏢Cold Spring Harbor Laboratory(冷泉港实验室)🌐USA

St. Giles Foundation Professor; Head, Pre-mRNA Processing Group圣贾尔斯基金会教授；pre-mRNA加工研究组负责人

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Key Papers

Awards

Key Contributions

👥Biography 个人简介

World authority on pre-mRNA splicing mechanisms and pioneer of antisense oligonucleotide (ASO) splicing correction therapy. Developed the science behind nusinersen (Spinraza), and applies splicing therapeutic principles to cancer aberrant splicing correction.

🧪Research Fields 研究领域

Pre-mRNA Splicing Regulationpre-mRNA剪接调控

Antisense Oligonucleotides反义寡核苷酸

Splicing in Cancer癌症中的剪接

SMN2 Splicing CorrectionSMN2剪接矫正

RNA Therapeutic DevelopmentRNA治疗开发

🎓Key Contributions 主要贡献

ASO Splicing Therapy Development

Developed antisense oligonucleotide strategies to correct aberrant splicing, culminating in nusinersen for SMA and establishing the platform for RNA splicing therapeutics in cancer.

SR Protein Splicing Regulation

Defined the molecular mechanisms by which SR splicing factors regulate constitutive and alternative splicing, revealing how cancer mutations in SR proteins dysregulate the transcriptome.

Representative Works 代表性著作

[1]

Bridging of exons by SR proteins via an exon enhancer

Nature (1993)

Defined SR protein exonic splicing enhancer mechanism fundamental to splicing regulation in cancer and normal cells.

[2]

Antisense correction of SMN2 splicing in the CNS rescues necrosis in a type III SMA mouse model

Genes & Development (2010)

Proof-of-concept for ASO splicing correction in vivo, establishing the clinical basis for nusinersen.

[3]

A therapeutic strategy for a genetic disease by skipping the mutation-containing exon

Nature Biotechnology (2018)

Generalizable ASO exon-skipping strategy applicable to cancer splicing correction.

🏆Awards & Recognition 奖项与荣誉

🏆Breakthrough Prize in Life Sciences

🏆Ross Prize in Molecular Medicine

🏆CSHL Double Helix Medal

🏆National Academy of Sciences Member

📄Data Sources 数据来源

Institution

Last updated: 2026-01-15 | All information from publicly available academic sources

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