Advancements in Translational Medicine: From Basic Research to Clinical Applications

Dr. Scott Younger is the Director of Disease Gene Engineering within the Genomic Medicine Center at Children's Mercy Hospital. His research focuses on producing patient-derived cellular models to develop functional precision medicine. He talks about using personalized antisense oligonucleotides to reverse disease phenotypes in organoid models of Duchenne muscular dystrophy. He also discusses his lab's personal connections to the rare disease community and the opportunities for collaborations with clinicians at Children's Mercy.