Adrian R. Krainer
St. Giles Foundation Professorship
Ph.D., Harvard University, 1986
email@example.com | (516) 367-8417
Our DNA carries the instructions to manufacture all the molecules 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 disease, and the means by which faulty splicing can be corrected. In particular, they study 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 this approach for the study of other diseases caused by splicing defects, including familial dysautonomia. In addition, they are applying antisense technology to stabilize mRNAs that are destroyed by a process called nonsense-mediated mRNA decay (NMD), both to learn about the underlying mechanisms and to develop new therapies. The Krainer lab has also worked to shed light on the role of splicing proteins in cancer. They found that the splicing factor SRSF1 functions as an oncogene, and they characterized the splicing changes it elicits when overexpressed in the context of breast cancer; several of these changes contribute to various aspects of cancer progression. Finally, the lab continues to study fundamental mechanisms of splicing and its regulation, and they identified novel ways in which the U1 snRNA can recognize natural 5’ splice sites that deviate from the consensus.
2019 Life Sciences Breakthrough Prize
2019 Fellow of the National Academy of Inventors
2019 Doctorate Honoris Causa, Tel Aviv University
2019 Klaus Joachim Zülch Prize for Basic Neurological Research, Gertrud Reemtsma Foundation and and Max Planck Society
2019 RNA Society Lifetime Achievement Award
2019 Bermuda Principles Award
2019 Peter Speiser Award in Pharmaceutical Sciences, ETH Zurich
Mutation’s role in blood cancers revealed by ideal team-up
January 31, 2020
Researchers from CSHL and MSKCC have determined how a mutation implicated in many blood cancers reduces the proteins that make healthy blood cells.
Researchers identify gene behind spread of deadly breast cancer
December 9, 2019
Jackson Laboratory and Cold Spring Harbor Laboratory researchers uncovered novel targets for therapies related to deadly triple negative breast cancers.
Adrian Krainer wins 2019 Peter Speiser Award
November 27, 2019
Professor Adrian Krainer was awarded the Peter Speiser Award for advances in the pharmaceutical sciences.
High school senior wins big in Breakthrough Junior Challenge
November 5, 2019
Winning the 2019 Breakthrough Junior Challenge, student Jeffery Chen earned his high school a $100,000 science lab designed by the DNA Learning Center.
Adrian Krainer elected to National Academy of Medicine
October 23, 2019
Professor Adrian Krainer joins the National Academy of Medicine in honor of his groundbreaking work on SMA.
Adrian Krainer wins Zülch Prize for lifesaving SMA Treatment
October 8, 2019
Professor Adrian Krainer is awarded the K-J. Zülch Prize for the development and testing of a first-of-its-kind treatment for spinal muscular atrophy.
Mutant cells team up to make an even deadlier blood cancer
October 2, 2019
Two very different mutations have been identified as unexpected partners-in-crime for causing the deadly blood cancer acute myeloid leukemia.
How one family secured the future of a laboratory
September 12, 2019
The Robertson family gifts to CSHL in the early 1970s were of extraordinary importance, for without them the Laboratory might not have survived.
Adrian Krainer wins RNA Society’s Lifetime Achievement Award
March 8, 2019
Professor Adrian Krainer was awarded the RNA Society’s Lifetime Achievement Award for his life-saving research.
National Academy of Inventors Fellow, Adrian Krainer
December 11, 2018
Professor Adrian Krainer has been named a NAI 2018 Fellow for his work on the first FDA-approved treatment for spinal muscular atrophy.