The Ross Prize in Molecular Medicine was established in conjunction with the Feinstein Institutes for Medical Research and Molecular Medicine to recognize biomedical scientists whose discoveries have transformed the way medicine is practiced. The awardees are midcareer researchers who have made a significant impact in the understanding of human disease pathogenesis and/or treatment. Moreover, it is anticipated that they will continue to make profound advances in the general field of molecular medicine.
The 2020 Ross Prize in Molecular Medicine will be awarded to Dr. Adrian R. Krainer for his pioneering work in introducing anti-sense therapy in clinical use, and for its successful application to spinal muscular atrophy (SMA).
In addition to studying the mechanisms of RNA splicing, Dr. Krainer uses multidisciplinary approaches to examine the ways in which they go awry in disease, and the means by which faulty splicing can be corrected. He co-developed the first FDA-approved therapy for the genetic disorder SMA — an illness that has been the leading genetic cause of infant death — based on the biological process of RNA splicing. This life-saving drug is also the first approved splicing-corrective therapy.
Cold Spring Harbor Laboratory
Rosalind Franklin University of Medicine and Science
Boston Children’s Hospital, Harvard Medical School
This symposium is made possible by the generosity of Jack and Robin Ross with support from
October 30, 2020
Welcome and Introductory Remarks
Session I: 2020 Ross Prize in Molecular Medicine Presentation and Lecture
2020 Ross Prize in Molecular Medicine Announcement and Presentation
2020 Ross Prize in Molecular Medicine Acceptance
From Base Pairs to Bedside: Antisense Modulation of RNA Splicing
Session II: Genetics of Neurological Disorders
RNA-Based Precision Medicine Approach for the Treatment of a Genetic Epilepsy
Splice-switching Antisense Oligonucleotides for the Treatment of Neurological Disorders
Patient-customized Antisense Oligonucleotides: Early Forays into Individualized Genomic Medicine