A Conversation with Adrian Krainer
- Senior Editor, Nature Structural & Molecular Biology
This extract was created in the absence of an abstract.
Adrian Krainer is the St. Giles Foundation Professor at Cold Spring Harbor Laboratory.
Anke Sparmann:You were awarded the 2019 Breakthrough Prize in Life Sciences together with Dr. Frank Bennett of Ionis Pharmaceuticals for the development of antisense oligonucleotide drugs to target RNA splicing and the incredible success story of SPINRAZA, the first drug approved for spinal muscular atrophy. Can you start by telling us about this devastating disease and the molecular mechanism underlying it that you discovered?
Dr. Krainer:SMA, or spinal muscular atrophy, is a motor neuron disease. It's very severe, and it mainly affects infants and young children. There are milder forms of the disease, with delayed onset, which affect older patients, including adults. Depending on the type of SMA, it leads to progressive muscle weakness and paralysis, and it can be lethal. It's inherited as an autosomal recessive, Mendelian kind of disorder. The disease was well-characterized, …
