Adrian R. Krainer

Adrian R. Krainer is the St. Giles Foundation Professor at Cold Spring Harbor Laboratory and Co-Leader of the Cancer Center's Gene Regulation & Inheritance Program. His research focuses on RNA splicing mechanisms and developing antisense oligonucleotide therapies for genetic diseases and cancer.

His seminal work led to the development of Spinraza (nusinersen), the first FDA-approved treatment for spinal muscular atrophy. Current research targets splicing defects in familial dysautonomia and cancer-specific splicing events, including therapies for diffuse midline glioma. The laboratory combines biochemistry, mouse genetics, and clinical translation approaches.

Honors include the Wolf Prize, Breakthrough Prize in Life Sciences, and memberships in the National Academy of Sciences and National Academy of Medicine.