[NFS-01] Neuroscience Frontier Symposium 01「Exploring Early Clinical Development through Innovative Drug Modalities」

[NFS-01-1] Progress towards interventional neurogenetics

Timothy Yu^1,2 (1.Division of Genetics and Genomics, Boston Children's Hospital, 2.Harvard Medical School)

Dr. Yu leads a multidisciplinary research group in the Division of Genetics & Genomics and Department of Neurology at Boston Children’s Hospital that works at the intersection of genomics, informatics, and neurobiology to better understand, diagnose, and treat rare neurologic disease. A graduate of Harvard College, he completed MD-PhD training at UC San Francisco and neurology residency at Massachusetts General Hospital and Brigham and Women's Hospital, and a postdoctoral fellowship at Boston Children’s Hospital. His group has been responsible for creating new models for delivering translational genomic medicine: accelerating diagnosis and treatment with genomic sequencing in newborns, and pioneering disruptive approaches to individualized genomic medicine using antisense oligonucleotides (ASOs). He created the world's first fully individualized medicine in 2018, using a patient-customized ASO to treat a young girl with fatal neurogenetic disease, and has worked closely with patients, foundations, physicians, and regulators to create paths for individualized genetic medicine and interventional genomics.