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Adrian R. Krainer

Adrian R. Krainer

Professor
St. Giles Foundation Professor
Cancer Center Program Co-Leader

Ph.D., Harvard University, 1986

krainer@cshl.edu | 516-367-8417

Krainer Lab Website   Faculty Profile

Our DNA carries the instructions to manufacture all the proteins needed by a cell. After each gene is copied from DNA into RNA, the RNA message is "spliced" - an editing process involving precise cutting and pasting. I am interested in how splicing normally works, how it is altered in genetic diseases and cancer, and how we can correct these defects for therapy.

Adrian Krainer’s lab studies the mechanisms of RNA splicing, ways in which they go awry in cancer and genetic diseases, and the means by which faulty splicing can be corrected. For example, they have studied splicing in spinal muscular atrophy (SMA), a neuromuscular disease that is the leading genetic cause of death in infants. In SMA, a gene called SMN2 is spliced incorrectly, making it only partially functional. The Krainer lab found a way to correct this defect using a powerful therapeutic approach. It is possible to stimulate SMN protein production by altering mRNA splicing through the introduction into cells of chemically modified pieces of RNA called antisense oligonucleotides (ASOs). Following extensive work with ASOs in mouse models of SMA, one such molecule, known as nusinersen or Spinraza, was taken to the clinic, and at the end of 2016 it became the first FDA-approved drug to treat SMA, by injection into the fluid surrounding the spinal cord. The Krainer lab is currently using antisense technology to develop therapies for other diseases caused by splicing defects, including familial dysautonomia, and to target a cancer-specific alternative-splicing event that controls the Warburg effect. In addition, they are applying antisense technology downregulate activated oncogenes, e.g., a mutant oncohistone that causes diffuse midline glioma, a lethal pediatric brain cancer. The Krainer lab has also worked to shed light on how splicing factors and alternative splicing promote cancer progression in the context of breast, liver, brain, pancreatic, and blood malignancies. Finally, the lab continues to study fundamental mechanisms of splicing and its regulation, focusing on the precise recognition of highly diverse intronic and exonic pre-mRNA features by various spliceosome components.

2024 The Albany Prize in Medicine & Biomedical Research
2022 August M. Watanabe Prize in Translational Research, Indiana University School of Medicine
2021 Jacob & Louise Gabbay Award in Biotechnology and Medicine, Brandeis University
2021 Wolf Prize in Medicine from the state of Israel
2020 Takeda Pharmaceuticals & NY Academy of Sciences Innovators in Science Senior Scientist Award in Rare Diseases
2020 Ross Prize in Molecular Medicine, Feinstein Institute
2020 Gregor Johann Mendel Medal for Outstanding Achievements in Science, Brno, Czech Republic
2020 Member, National Academy of Sciences
2019 Member, National Academy of Medicine
2019 Adrian Krainer wins 2019 Peter Speiser Award
2019 Life Sciences Breakthrough Prize
2019 Doctorate Honoris Causa, Tel Aviv University
2019 International Prize for Translational Neuroscience, Gertrud Reemtsma Foundation and Max Planck Society
2019 RNA Society Lifetime Achievement Award
2019 Bermuda Principles Award
2019 Peter Speiser Award in Pharmaceutical Sciences, ETH Zurich
2018 National Academy of Inventors Fellow, Adrian Krainer
2018 Breakthrough Prize to CSHL professor for SMA research
2017 Inventor of the Year, New York Intellectual Property Law Association
2016 Fellow of the American Academy of Arts and Sciences
2012 National Institutes of Health MERIT Award

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Selected Publications

SRSF1 interactome determined by proximity labeling reveals direct interaction with spliceosomal RNA helicase DDX23

21 May 2024 | Proceedings of the National Academy of Sciences of the United States of America | 121(21):e2322974121
Segovia, Danilo;  Adams, Dexter;  Hoffman, Nickolas;  Safaric Tepes, Polona;  Wee, Tse-Luen;  Cifani, Paolo;  Joshua-Tor, Leemor;  Krainer, Adrian;  

Specificity, synergy, and mechanisms of splice-modifying drugs

29 Feb 2024 | Nature Communications | 15(1):1880
Ishigami, Yuma;  Wong, Mandy;  Martí-Gómez, Carlos;  Ayaz, Andalus;  Kooshkbaghi, Mahdi;  Hanson, Sonya;  McCandlish, David;  Krainer, Adrian;  Kinney, Justin;  

Splicing Factor SRSF1 Promotes Pancreatitis and KRASG12D-Mediated Pancreatic Cancer

7 Jul 2023 | Cancer Discovery | 13(7):1678-1695
Wan, Ledong;  Lin, Kuan-Ting;  Rahman, Mohammad;  Ishigami, Yuma;  Wang, Zhikai;  Jensen, Mads;  Wilkinson, John;  Park, Youngkyu;  Tuveson, David;  Krainer, Adrian;  

Antisense oligonucleotide therapy for H3.3K27M diffuse midline glioma

12 Apr 2023 | Science Translational Medicine | 15(691):eadd8280
Zhang, Qian;  Yang, Lucia;  Liu, Ying;  Wilkinson, John;  Krainer, Adrian;  

Counteracting chromatin effects of a splicing-correcting antisense oligonucleotide improves its therapeutic efficacy in spinal muscular atrophy

9 Jun 2022 | Cell | 185(12):2057-2070.e15
Marasco, Luciano;  Dujardin, Gwendal;  Sousa-Luís, Rui;  Liu, Ying;  Stigliano, Jose;  Nomakuchi, Tomoki;  Proudfoot, Nick;  Krainer, Adrian;  Kornblihtt, Alberto;  

All Publications

Voltage-gated sodium channels in excitable cells as drug targets

3 Feb 2025 | Nature Reviews Drug Discovery
Alsaloum, Matthew;  Dib-Hajj, Sulayman;  Page, Dana;  Ruben, Peter;  Krainer, Adrian;  Waxman, Stephen;  

Aldehydes alter TGF-β signaling and induce obesity and cancer

31 Aug 2024 | Cell Reports | 43(9):114676
Yang, Xiaochun;  Bhowmick, Krishanu;  Rao, Shuyun;  Xiang, Xiyan;  Ohshiro, Kazufumi;  Amdur, Richard;  Hassan, Md;  Mohammad, Taj;  Crandall, Keith;  Cifani, Paolo;  Shetty, Kirti;  Lyons, Scott;  Merrill, Joseph;  Vegesna, Anil;  John, Sahara;  Latham, Patricia;  Crawford, James;  Mishra, Bibhuti;  Dasarathy, Srinivasan;  Wang, Xin;  Yu, Herbert;  Wang, Zhanwei;  Huang, Hai;  Krainer, Adrian;  Mishra, Lopa;  

SRSF1 interactome determined by proximity labeling reveals direct interaction with spliceosomal RNA helicase DDX23 (correction)

2024 | Proceedings of the National Academy of Sciences of the United States of America | 121(30)
Segovia, Danilo;  Adams, Dexter;  Hoffman, Nickolas;  Tepes, Polona;  Wee, Tse-Luen;  Cifani, Paolo;  Joshua-Tor, Leemor;  Krainer, Adrian;  

Interaction between MED12 and ΔNp63 activates basal identity in pancreatic ductal adenocarcinoma

17 Jun 2024 | Nature Genetics
Maia-Silva, Diogo;  Cunniff, Patrick;  Schier, Allison;  Skopelitis, Damianos;  Trousdell, Marygrace;  Moresco, Philip;  Gao, Yuan;  Kechejian, Vahag;  He, Xue-Yan;  Sahin, Yunus;  Wan, Ledong;  Alpsoy, Aktan;  Liverpool, Jynelle;  Krainer, Adrian;  Egeblad, Mikala;  Spector, David;  Fearon, Douglas;  Dos Santos, Camila;  Taatjes, Dylan;  Vakoc, Christopher;  

Screening Splice-Switching Antisense Oligonucleotides in Pancreas-Cancer Organoids

8 May 2024 | Nucleic Acid Therapeutics
Wan, Ledong;  Kral, Alexander;  Voss, Dillon;  Schäfer, Balázs;  Sudheendran, Kavitha;  Danielsen, Mathias;  Caruthers, Marvin;  Krainer, Adrian;  

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