Convidado
THIMOTHY YU
Dr. Yu is a physician-scientist in the Division of Genetics & Genomics at Boston Children’s Hospital and Harvard Medical School. He leads a research group that works at the intersection of genomics, informatics, and neurobiology to better understand, diagnose, and treat rare neurologic disease. He completed MD-PhD training at UC San Francisco and neurology residency at Massachusetts General Hospital and Brigham and Women's Hospital, and postdoctoral training at Boston Children’s Hospital. His group has been responsible for creating new models for delivering translational genomic medicine: accelerating diagnosis with genomic sequencing in newborns, and pioneering disruptive approaches to precision genomic medicine. In 2018 he led the development of a patient-customized antisense oligonucleotide to treat a young girl with Batten disease, creating a precedent that served as the basis for the first FDA guidance for individualized genomic medicine. His laboratory continues to works closely with patients, foundations, physicians, and regulators to explore and fortify this pathway, launching a series of individualized genetic interventions for three additional orphan diseases. He is founder of the N=1 Collaborative, an independent nonprofit devoted to developing practices for individualized medicine.
Atividades
13/03/2025
17:30 às 19:15 - Sessão Internacional
18:20 às 18:45 - RNAi-based therapeutics as a model of personalized medicine
Palestrante
14/03/2025
08:00 às 09:30 - MODULO I | International perspectives on neurogenetics
08:00 às 08:25 - Molecular mechanisms underlying autism
Palestrante
15/03/2025
15:50 às 17:05 - The next decade of neurogenetics: What is coming on research, diagnostics, and therapy
Debatedor(a)