Wolf Prize Laureate in Medicine 2021
Affiliation at the time of the award:
Cold Spring Harbor Laboratory, USA
“for his fundamental mechanistic discoveries on RNA splicing leading to a world’s first treatment for spinal muscular atrophy (SMA)”.
“for fundamental discoveries in RNA biology that have the potential to better human lives. They have made ground-breaking discoveries in RNA regulatory mechanisms demonstrating that RNA is not a passive template between DNA and protein, but rather plays a dominant role in regulating and diversifying gene expression”.
Adrian Krainer is a professor of biochemistry and molecular genetics. Krainer’s research focuses on how splicing normally works, how it is altered in genetic diseases and cancer, and how we can correct these defects for therapy. Krainer and his team focused on finding a way to treat SMA, a neuromuscular disease that is the leading genetic cause of death in infants, by RNA therapy.
Adrian Krainer is awarded the Wolf Prize for his major contributions have advanced our understanding of the molecular mechanisms and regulation of RNA splicing. He identified and purified the first human protein splicing factor and demonstrated its roles in constitutive and alternative splicing. Krainer used this knowledge to study two genes, SMN1 and SMN2, associated with spinal muscular atrophy. Krainer devised an ingenious strategy to rescue the protein deficit caused by SMN1 mutations by promoting the appropriate splicing of the sister gene SMN2. This treatment received accelerated approval for use in humans and has dramatically improved the lives of thousands of children born with SMA.