Preclinical development of personalized ASO therapeutics using patient-derived organoid systems
- N1C Moderator
- Apr 22
- 1 min read
Updated: Dec 1
October 6, 2025 at 12:30 pm US EDT
This presentation will cover our laboratory's ongoing efforts in the areas of personalized and precision medicine. Active research programs focused on the identification of clinically actionable rare genetic variation will also be discussed.
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.
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Good morning, my name is Gustavo from Argentina, and my 15-month-old daughter Emilia has a genetic variation called GNAO! variant c607G<A (G203R). We wanted to find out if you are researching any treatments for this condition or if you currently have any active treatments for it. We are truly distressed and desperate to provide our little Emilia with a better quality of life. I would greatly appreciate any information you can provide.
Here's my personal contact:
grdconsultores@gmail.com
Thank you very much.