Adrian Krainer

Cold Spring Harbor Laboratory Professor Adrian Krainer is a pioneering scientist in RNA splicing and its therapeutic applications for genetic diseases and cancer. As the St. Giles Foundation Professor and Gene Regulation & Inheritance Co-Leader at CSHL, he has developed groundbreaking treatments including Spinraza (nusinersen) for spinal muscular atrophy (SMA). His work bridges fundamental biology and translational medicine, impacting fields like pancreatic oncogenesis, hepatocellular carcinoma, and neurodegenerative disorders.

• Education: Ph.D. in Biochemistry (Harvard, 1986), B.A. in Biochemistry (Columbia, 1981).

Dr. Krainer’s research focuses on RNA splicing mechanisms, antisense oligonucleotide therapies, and splicing factors in cancer. His team discovered how faulty splicing contributes to SMA, familial dysautonomia, and cancers like diffuse midline glioma and pancreatic malignancies. Recent work explores TGF-β signaling disruptions in colon cancer and PKM isoform switching in liver cancer.

The 15 most recent articles highlight his lab’s leadership in splice-modifying drugs, oncogenic RNA circuits, and metabolite-driven carcinogenesis. Key themes include SRSF1-AURKA-MYC interactions, antisense delivery systems, and voltage-gated ion channels as therapeutic targets.

• Scientific Recognition:
  • 2025 Heinrich Wieland Prize
  • 2024 Albany Prize in Medicine
  • 2023 IADR Honorary Membership
  • Breakthrough Prize (2018, 2019)
  • Wolf Prize in Medicine (2021)
  • National Academy of Sciences (2020) and Medicine (2019)
• Research Collaborations: Partnered with institutions in Chile, Bermuda, and global pharmaceutical leaders like Ionis Pharmaceuticals.