{"entity":{"id":"adrian-krainer","kind":"person","name":"Adrian R. Krainer","aka":[],"tldr":"Adrian Krainer invented the antisense drug nusinersen and studies how splicing errors drive cancer.","summary":"Adrian R. Krainer is St. Giles Foundation Professor at Cold Spring Harbor Laboratory and an RNA biologist who studies how splicing works and how it goes wrong. He is best known for the splice-switching antisense oligonucleotide work that produced nusinersen for spinal muscular atrophy, recognised with a Breakthrough Prize. His selected paper shows antisense correction of SMN2 splicing in the central nervous system in a mouse model of the disease. His laboratory now applies oligonucleotide therapeutics to the splicing factors that are mutated in leukaemia and solid tumours, asking whether splicing errors in cancer can be corrected the same way.","asOf":"2026-09-08","links":[{"label":"PubMed","url":"https://pubmed.ncbi.nlm.nih.gov/?term=Krainer%20AR%5BAuthor%5D"}],"tags":[],"related":[],"cancers":[],"sections":[],"technologies":["antisense-sirna"],"targets":[],"drugs":[],"companies":[],"institutions":["cold-spring-harbor"],"pathways":[],"terms":[],"trials":[],"people":[],"bottlenecks":[],"keyPapers":[],"journals":["genes-and-development"],"dependsOn":[],"notes":[],"role":"St. Giles Foundation Professor","institutionId":"cold-spring-harbor","specialisms":["RNA splicing","Antisense oligonucleotides","Splicing in cancer"],"profiles":[{"label":"PubMed","url":"https://pubmed.ncbi.nlm.nih.gov/?term=Krainer%20AR%5BAuthor%5D"}],"papers":[{"title":"Antisense correction of SMN2 splicing in the CNS rescues necrosis in a type III SMA mouse model","journal":"Genes & Development","year":2010,"url":"https://doi.org/10.1101/gad.1941310","doi":"10.1101/gad.1941310"}],"orcid":"0000-0001-9024-9501"},"route":"/people/adrian-krainer/","neighbours":{"technology":[{"id":"antisense-sirna","kind":"technology","name":"Oligonucleotide therapeutics","route":"/technologies/antisense-sirna/"}],"institution":[{"id":"cold-spring-harbor","kind":"institution","name":"Cold Spring Harbor Laboratory","route":"/institutions/cold-spring-harbor/"}],"journal":[{"id":"genes-and-development","kind":"journal","name":"Genes & Development","route":"/journals/genes-and-development/"}]}}