Speakers: Scott Younger, PhD

Scott Younger, PhD

Director of Disease Gene Engineering, Genomic Medicine Center
Children’s Mercy Kansas City

 

Dr. Scott Younger is the Director of Disease Gene Engineering within the Genomic Medicine Center at Children’s Mercy Kansas City. Since joining Children’s Mercy his laboratory has pioneered methods for the rapid, robust, and scalable production of patient-derived cellular models. The laboratory is currently focused on the development of platform-based approaches for functional precision medicine with an emphasis on reduction to practice and clinical implementation. Dr. Younger came to Children’s Mercy from the Broad Institute of MIT and Harvard where his group worked on the development of new methodologies to expand the utility of CRISPR-based genetic screens. Prior to working at the Broad Institute he completed his postdoctoral studies at Harvard University as an American Cancer Society Fellow. He holds a Ph.D. in cell and molecular biology from UT Southwestern Medical Center. He also received an M.S. in biotechnology from the University of Texas at San Antonio and a B.S.I. in bioinformatics from Baylor University.

 

Individualized Therapeutic Screening in Patient-Derived Organoid Models of Genetic Disease

A critical barrier in translational sciences is the lack of practical laboratory technologies with the capacity to inform evidence-based treatments in a timely manner. For clinical indications with multiple therapeutic options that exhibit varied efficacy across individuals in the impacted patient population, the process of identifying the optimal therapeutic approach for any given individual can be a major bottleneck. To address this challenge, we’re aiming to build a high-throughput ex vivo screening platform to facilitate the integration of individualized cellular technologies into clinical decision making for children impacted by rare disease. I will discuss the status of these efforts as well as the informatics challenges ahead.