Adrian R. Krainer
Cold Spring Harbor Laboratory
Molecular geneticist; Research institution scientist
Biochemistry, Biophysics, and Molecular Biology
Led the RNA-processing field in efforts to understand the biochemical mechanisms and regulation of pre-mRNA splicing, the distinguishing features of exons and introns, and the fundamental principles of their recognition by RNA-binding proteins and snRNAs. His team made important contributions towards understanding various splicing defects associated with genetic diseases and cancer, and towards the development of powerful mechanism-based therapeutic approaches involving correction of defective splicing or modulation of alternative splicing. He invented an antisense-based drug to correct splicing of a defective gene associated with spinal muscular atrophy, which became the first approved treatment for this disease.