Adrian Krainer

Biography
Adrian's lab at CSHL uses multidisciplinary approaches to elucidate mRNA splicing mechanisms and alternative splicing regulation. An important goal is to apply this knowledge to uncover the roles of defective splicing in genetic diseases and cancer, and to use antisense technology to develop novel therapeutics that target pre-mRNA or mRNA to modulate gene expression post-transcriptionally. One notable application—in collaboration with Ionis Pharmaceuticals and Biogen—was the development of the antisense oligonucleotide nusinersen (Spinraza), which became the first FDA-approved drug for spinal muscular atrophy. His lab is currently pursuing antisense-therapeutic approaches in the context of various malignancies. Adrian served as President of the RNA Society and was elected to the National Academy of Sciences and the National Academy of Medicine. Among other awards, he received the Life Sciences Breakthrough Prize, the Wolf Prize in Medicine, the Albany Prize in Medicine & Biomedical Research, the RNA Society’s Lifetime Achievement Award, and the Ross Prize in Molecular Medicine.

---

Talk
Antisense Modulation of mRNA Splicing for Neurological Disorders
This talk will describe therapeutic antisense oligonucleotides and their mechanisms of action, focusing on the development of Spinraza for spinal muscular atrophy, and subsequent applications of this platform for other neurological disorders.