Scott V. Dindot

Company: Ultragenyx Pharmaceuticals

Job title: Executive Director of Molecular Genetics

Seminars:

Harnessing a Large Animal Model to Overcome Translational Barriers in Angelman Syndrome 9:00 am

Unpack why mouse models fail to recapitulate Angelman syndrome phenotypes and challenges associated with rodent models in preclinical development Rationale and characterization of the Angelman pig model, including phenotypic fidelity, regulatory implications, and translational value Target discovery in a complex genomic landscape: Exploring how integrated approaches in genetics and biology helped identify functionally relevant regions…Read more

day: Day 2