OVERVIEW 

The Krainer laboratory uses multidisciplinary approaches to investigate the fundamental mechanisms and regulation of human pre-mRNA splicing, and the role of defective splicing in cancer and genetic diseases. We also study the relationship between splicing and other steps in post-transcriptional gene regulation, such as nonsense-mediated mRNA decay (NMD). In addition to these basic studies, the Krainer lab has pioneered novel methods for modulating alternative splicing and for correcting splicing defects in cells and mouse models for therapeutic applications, including in neurodegenerative diseases and cancer. In particular, we were involved in the invention and preclinical development of nusinersen (Spinraza), a splice-switching antisense oligonucleotide that became the first FDA-approved drug for spinal muscular atrophy and the first approved nucleic-acid therapy in neurology.