Rustbelt RNA Meeting 2019

Download Poster [.pdf]

Contact

For more information, please contact the conference chairs:

• Hua Lou, hxl47@case.edu
• Peng Yao, Peng_Yao@URMC.rochester.edu
• Jo Ann Wise, jaw17@case.edu

Professor Adrian R. Krainer

Cold Spring Harbor Laboratory

 

Dr. Adrian R. Krainer is the St. Giles Foundation Professor at the Cold Spring Harbor Laboratory.  Although currently best known as a pioneer in the use of anti-sense oligonucleotides (ASOs) as therapeutic agents, Professor Krainer has made many key contributions to our understanding of basic mechanisms of alternative pre-messenger RNA splicing.  He began his independent career as the first Cold Spring Harbor Junior Fellow and was soon thereafter named a Pew Scholar in the Biomedical Sciences. The many other honors Professor Krainer has received over the years include the 2019 RNA Society Lifetime achievement award and the 2019 Life Sciences Breakthrough Prize.  The discovery that led to the latter award emerged from studies of the faulty splicing that leads to spinal muscular atrophy (SMA), a neuromuscular disease that is the leading genetic cause of death in infants.  The Krainer laboratory devised a way to correct this defect by altering pre-mRNA splicing through the introduction into cells of chemically modified ASOs, first in mouse models of SMA and subsequently in human clinical trials.  In 2016, the ASO known as nusinersen or Spinraza became the first FDA-approved drug to treat SMA.  Professor Krainer also serves as an advocate for increased funding for SMA research, often alongside families with children who have benefitted from the drug.  His laboratory’s current research is focused on developing therapies for other diseases caused by splicing defects such as familial dysautonomia.