Adrian Krainer is a biochemist recognized for his basic work on RNA splicing and the development of mechanism-based therapeutic applications. Krainer was born and grew up in Montevideo, Uruguay, and came to the U.S. in 1977 to attend college. He graduated from Columbia College, New York in 1981 with a B.A. in Biochemistry, and from Harvard University, Cambridge in 1986 with a Ph.D. in Biochemistry. He was an independent postdoctoral fellow at Cold Spring Harbor Laboratory and joined the faculty there in 1989; he is currently the St Giles Professor at CSHL and Deputy Director of Research of the CSHL Cancer Center. He is a co-founder and Director of Stoke Therapeutics. Krainer is a recipient of the Life Sciences Breakthrough Prize, the RNA Society’s Lifetime Achievement Award, the Reemtsma Foundation International Prize in Translational Neuroscience, the Speiser Award in Pharmaceutical Sciences, and the Ross Prize in Molecular Medicine. He served as President of the RNA Society, and is a member of the National Academy of Sciences, the National Academy of Medicine, the National Academy of Inventors, and the American Academy of Arts & Sciences.

Research Interests

Adrian Krainer's research at Cold Spring Harbor Laboratory focuses on the mechanisms, regulation, and fidelity of human pre-mRNA splicing. His lab uses multidisciplinary approaches to elucidate splicing mechanisms, alternative splicing regulation, the underlying RNA-RNA and RNA-protein interactions, and the role of defective splicing in genetic diseases and cancer. They also utilize antisense technology to develop novel therapeutics that target pre-mRNA or mRNA to modulate gene expression post-transcriptionally. One notable application was the development of the antisense oligonucleotide nusinersen (Spinraza), which became the first approved drug for spinal muscular atrophy, a genetic disease that causes motor-neuron degeneration. Krainer's research has also implicated splicing alterations in carcinogenesis, and his lab is currently pursuing antisense-therapeutic approaches in the context of various cancers.

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Primary Section

Section 21: Biochemistry

Secondary Section

Section 41: Medical Genetics, Hematology, and Oncology